Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2022 | May 09, 2022 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2022 | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q1 | |
Entity Registrant Name | ORCHARD THERAPEUTICS PLC | |
Current Fiscal Year End Date | --12-31 | |
Entity Central Index Key | 0001748907 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Common Stock, Shares Outstanding | 125,905,065 | |
Entity Shell Company | false | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Incorporation, State or Country Code | X0 | |
Entity Address, Address Line One | 108 Cannon Street | |
Entity Address, City or Town | London | |
Entity Address, Postal Zip Code | EC4N 6EU | |
City Area Code | 44 | |
Local Phone Number | (0) 203 808-8286 | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Entity File Number | 001-38722 | |
Entity Emerging Growth Company | false | |
Entity Tax Identification Number | 00-0000000 | |
Entity Address, Country | GB | |
Title of 12(b) Security | American Depositary Shares, each representing one ordinary share, nominal value £0.10 per share | |
Trading Symbol | ORTX | |
Security Exchange Name | NASDAQ |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 60,775 | $ 55,912 |
Marketable securities | 138,241 | 164,195 |
Accounts receivable | 4,105 | 1,480 |
Prepaid expenses and other current assets | 20,923 | 23,011 |
Research and development tax credit receivable | 13,394 | 30,723 |
Total current assets | 237,438 | 275,321 |
Non-current assets: | ||
Operating lease right-of-use-assets | 27,368 | 24,316 |
Property and equipment, net | 4,454 | 4,767 |
Restricted cash | 4,266 | 4,266 |
Intangible assets, net | 3,993 | 4,149 |
Research and development tax credit receivable | 3,255 | |
Other assets | 10,731 | 9,590 |
Total non-current assets | 54,067 | 47,088 |
Total assets | 291,505 | 322,409 |
Current liabilities: | ||
Accounts payable | 9,854 | 10,008 |
Accrued expenses and other current liabilities | 25,983 | 24,318 |
Deferred revenue, current | 889 | 346 |
Operating lease liabilities | 7,042 | 7,335 |
Notes payable, current | 3,143 | 786 |
Total current liabilities | 46,911 | 42,793 |
Notes payable, long-term | 29,813 | 32,086 |
Deferred revenue, net of current portion | 11,554 | 12,519 |
Operating lease liabilities, net of current portion | 20,798 | 19,278 |
Other long-term liabilities | 6,783 | 5,783 |
Total liabilities | 115,859 | 112,459 |
Commitments and contingencies (see Note 15) | ||
Shareholders’ equity: | ||
Ordinary shares, £0.10 par value, authority to allot up to a maximum nominal value of £13,023,851.50 of shares at March 31, 2022 and December 31, 2021, respectively; 125,904,945 and 125,674,095 shares issued and outstanding at March 31, 2022 and December 31, 2021, respectively. | 16,283 | 16,253 |
Additional paid-in capital | 945,302 | 940,675 |
Accumulated other comprehensive income | 8,581 | 3,246 |
Accumulated deficit | (794,520) | (750,224) |
Total shareholders’ equity | 175,646 | 209,950 |
Total liabilities and shareholders’ equity | $ 291,505 | $ 322,409 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Unaudited) (Parenthetical) - GBP (£) | Mar. 31, 2022 | Dec. 31, 2021 |
Statement Of Financial Position [Abstract] | ||
Ordinary Shares, Par Value | £ 0.10 | £ 0.10 |
Ordinary Shares, Authorized | £ 13,023,851,500 | £ 13,023,851,500 |
Ordinary Shares, Issued | 125,904,945 | 125,674,095 |
Ordinary Shares, Outstanding | 125,904,945 | 125,674,095 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Total revenues | $ 5,524 | |
Costs and operating expenses: | ||
Cost of product revenues | 1,571 | |
Research and development | 28,234 | $ 21,035 |
Selling, general and administrative | 13,299 | 14,051 |
Total costs and operating expenses | 43,104 | 35,086 |
Loss from operations | (37,580) | (35,086) |
Other income (expense): | ||
Interest income | 69 | 171 |
Interest expense | (675) | (538) |
Other income (expense), net | (6,052) | 1,358 |
Total other income (expense), net | (6,658) | 991 |
Net loss before income tax | (44,238) | (34,095) |
Income tax expense | (58) | (1,087) |
Net loss attributable to ordinary shareholders | $ (44,296) | $ (35,182) |
Net loss per share attributable to ordinary shareholders, basic and diluted | $ (0.35) | $ (0.31) |
Weighted average ordinary shares outstanding, basic and diluted | 127,694,785 | 114,829,272 |
Other comprehensive (loss) income: | ||
Foreign currency translation adjustment | $ 5,595 | $ (64) |
Unrealized loss (gain) on marketable securities | (260) | (113) |
Total other comprehensive (loss) income: | 5,335 | (177) |
Total comprehensive loss | (38,961) | $ (35,359) |
Product Revenue, Net [Member] | ||
Total revenues | 5,059 | |
Collaboration Revenue [Member] | ||
Total revenues | $ 465 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Cash flows from operating activities: | ||
Net loss attributable to ordinary shareholders | $ (44,296) | $ (35,182) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 709 | 495 |
Share-based compensation | 4,660 | 6,268 |
Non-cash interest expense | 94 | 101 |
Amortization of provision on loss contract | (274) | (446) |
Amortization of premium on marketable securities | 187 | 343 |
Deferred income taxes | 787 | 781 |
Unrealized foreign currency and other non-cash adjustments | 5,984 | 1,743 |
Changes in operating assets and liabilities: | ||
Accounts receivable | (2,719) | 887 |
Research and development tax credit receivable | 13,483 | (3,554) |
Prepaid expenses, other current assets and other assets | (62) | 1,399 |
Operating leases, right-of-use assets | 1,455 | 1,252 |
Accounts payable, accrued expenses and other current liabilities | 3,815 | (8,905) |
Deferred revenue | (65) | |
Operating lease liabilities | (3,357) | (4,424) |
Net cash used in operating activities | (19,599) | (39,242) |
Cash flows from investing activities: | ||
Proceeds from sales and maturities of marketable securities | 55,186 | 47,200 |
Purchases of marketable securities | (29,681) | (130,387) |
Purchases of property and equipment | (398) | (339) |
Net cash provided by (used in) investing activities | 25,107 | (83,526) |
Cash flows from financing activities: | ||
Proceeds from employee equity plans, net of taxes withheld | 2,526 | |
Proceeds from the issuance of ordinary shares in private placement | 150,000 | |
Payment of placement agent fees and offering costs | (6,092) | |
Net cash provided by financing activities | 146,434 | |
Effect of exchange rate changes on cash, cash equivalents, and restricted cash | (645) | 82 |
Net increase in cash, cash equivalents and restricted cash | 4,863 | 23,748 |
Cash, cash equivalents, and restricted cash, beginning of period | 60,178 | 59,401 |
Cash, cash equivalents, and restricted cash, end of period | 65,041 | 83,149 |
Supplemental disclosure of non-cash activities | ||
Property and equipment and intangible assets included in accounts payable and accrued expenses | 1,102 | 4,834 |
Offering costs included in accounts payable and accrued expenses | 198 | |
Supplemental disclosure of cash flow information: | ||
Lease assets obtained in exchange for new operating lease liabilities, net | 4,912 | |
Cash paid for interest | $ 581 | $ 482 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Shareholders' Equity (Unaudited) - USD ($) $ in Thousands | Total | Ordinary Shares | Additional Paid-in Capital | Accumulated Other Comprehensive Income | Accumulated Deficit |
Balance at Dec. 31, 2020 | $ 178,434 | $ 12,507 | $ 771,194 | $ 373 | $ (605,640) |
Balance, Shares at Dec. 31, 2020 | 98,283,603 | ||||
Share-based compensation expense | 6,268 | 6,268 | |||
Exercise of share options | 2,822 | $ 172 | 2,650 | ||
Exercise of share options, Shares | 1,319,493 | ||||
Vesting of restricted stock units, net of shares withheld for taxes | (296) | $ 6 | (302) | ||
Vesting of restricted stock units, net of shares withheld for taxes, shares | 45,746 | ||||
Sale of ordinary shares and non-voting ordinary shares, net of issuance costs | 143,711 | $ 3,310 | 140,401 | ||
Sale of ordinary shares and non-voting ordinary shares, net of issuance costs, Shares | 24,115,755 | ||||
Foreign currency translation adjustment | (64) | (64) | |||
Unrealized loss on available for sale debt securities | (113) | (113) | |||
Net loss attributable to ordinary shareholders | (35,182) | (35,182) | |||
Balance at Mar. 31, 2021 | 295,580 | $ 15,995 | 920,211 | 196 | (640,822) |
Balance, Shares at Mar. 31, 2021 | 123,764,597 | ||||
Balance at Dec. 31, 2021 | 209,950 | $ 16,253 | 940,675 | 3,246 | (750,224) |
Balance, Shares at Dec. 31, 2021 | 125,674,095 | ||||
Share-based compensation expense | 4,660 | 4,660 | |||
Exercise of share options | $ (1) | $ 28 | (29) | ||
Exercise of share options, Shares | 222,381 | 222,381 | |||
Vesting of restricted stock units, net of shares withheld for taxes | $ (3) | $ 1 | (4) | ||
Vesting of restricted stock units, net of shares withheld for taxes, shares | 3,217 | ||||
Ordinary shares issued as part of a consulting agreement | 1 | $ 1 | |||
Ordinary shares issued as part of consulting agreement, Shares | 5,252 | ||||
Foreign currency translation adjustment | 5,595 | 5,595 | |||
Unrealized loss on available for sale debt securities | (260) | (260) | |||
Net loss attributable to ordinary shareholders | (44,296) | (44,296) | |||
Balance at Mar. 31, 2022 | $ 175,646 | $ 16,283 | $ 945,302 | $ 8,581 | $ (794,520) |
Balance, Shares at Mar. 31, 2022 | 125,904,945 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Shareholders' Equity (Unaudited) (Parenthetical) $ in Thousands | 3 Months Ended |
Mar. 31, 2021USD ($) | |
Statement Of Stockholders Equity [Abstract] | |
Sale of ordinary shares and non-voting ordinary shares, issuance costs | $ 6,290 |
Nature of the Business
Nature of the Business | 3 Months Ended |
Mar. 31, 2022 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Nature of the Business | 1. Nature of the Business Orchard Therapeutics plc (the “Company”) is a global gene therapy company dedicated to transforming the lives of people affected by severe diseases through the development of innovative, potentially curative gene therapies. The Company’s ex vivo The Company is a public limited company incorporated pursuant to the laws of England and Wales. The Company has American Depositary Shares (“ADSs”) registered with the U.S. Securities and Exchange Commission (the “SEC”) and has been listed on the Nasdaq Global Select Market since October 31, 2018. The Company’s ADSs each represent one ordinary share of the Company. In December 2020, the Company received standard marketing authorization from the European Commission for Libmeldy™ ( atidarsagene autotemcel arylsulfatase-A ARSA On February 9, 2021, the Company issued and sold (i) 20,900,321 ordinary shares, nominal value £0.10 per share, at a purchase price of $6.22 per share (the “Purchase Price”), which was the closing sale price of the Company’s ADSs on the Nasdaq Global Select Market on February 4, 2021, and (ii) 3,215,434 non-voting ordinary shares, nominal value £0.10 per share, at the Purchase Price (together (i) and (ii) the “Private Placement”). The Private Placement resulted in net proceeds to the Company of $143.7 million after deducting placement agent fees of $6.0 million and other issuance costs of $0.3 million. The ordinary shares and non-voting ordinary shares were sold pursuant to a securities purchase agreement entered into between the Company and the purchasers named therein on February 4, 2021. The Company’s business is subject to risks and uncertainties common to development-stage companies in the biotechnology industry. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s technology will be obtained, that any products developed will obtain necessary government regulatory approval or that any products, if approved, will be commercially viable. The Company operates in an environment of rapid technological innovation and substantial competition from pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, consultants and service providers. Even if the Company’s product development efforts are successful in gaining regulatory approval, it is uncertain when, if ever, the Company will realize significant revenue from product sales. Through March 31, 2022, the Company funded its operations primarily with proceeds from the sale of equity securities, including ADSs in the Company’s initial public offering (“IPO”) and follow-on offering, ordinary shares in the private placement, and convertible preferred shares. The Company has also financed its operations through proceeds from the Company’s senior term facilities agreement with MidCap Financial (Ireland) Limited, research grants from the California Institute of Regenerative Medicine (“CIRM”), upfront payments from the Company’s collaboration agreement and share purchase agreement with Pharming Group N.V., and proceeds associated two UK research and development tax relief programs, the Small and Medium-sized Enterprises research and development tax credit (“SME”) program and the Research and Development Expenditure (“RDEC”) program. The Company has incurred recurring losses since its inception. As of March 31, 2022, the Company had an accumulated deficit of $794.5 million. The Company expects to continue to generate operating losses for the foreseeable future. The Company expects that its cash, cash equivalents, and marketable securities on hand as of March 31, 2022 of $199.0 million will be sufficient to fund its operations and capital expenditure requirements for at least the next twelve months. The Company will seek additional funding through private or public equity financings, debt financings, collaborations, strategic alliances and marketing, distribution or licensing arrangements. The Company may not be able to obtain financing on acceptable terms, or at all, and the Company may not be able to enter into collaborations or other arrangements. The terms of any financing may adversely affect the holdings or the rights of the Company's shareholders. |
Basis of Presentation and Summa
Basis of Presentation and Summary of Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2022 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Summary of Significant Accounting Policies | 2. Basis of Presentation and Summary of Significant Accounting Policies Basis of presentation The condensed consolidated interim financial statements of the Company are unaudited and have been prepared in accordance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) for interim financial reporting and in accordance with Regulation S-X, Rule 10-01. Accordingly, they do not include all of the information and footnotes required by U.S. GAAP for complete financial statements. Any reference in these notes to applicable guidance is meant to refer to the authoritative U.S. GAAP as found in the Accounting Standards Codification (“ASC”), and Accounting Standards Update (“ASU”), of the Financial Accounting Standards Board (“FASB”). All intercompany accounts and transactions between the Company and its subsidiaries have been eliminated upon consolidation. The accompanying unaudited condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and accompanying notes included in the Company’s Annual Report on Form 10-K filed with the SEC on March 30, 2022 (the “Annual Report”). The condensed consolidated balance sheet as of December 31, 2021 was derived from audited consolidated financial statements included in the Company’s Annual Report but does not include all disclosures required by U.S. GAAP. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted from these interim financial statements. However, these interim financial statements include all adjustments, consisting only of normal recurring adjustments, which are, in the opinion of the Company’s management, necessary to fairly state the results of the interim period. The interim results are not necessarily indicative of results to be expected for the full year. Amounts reported are computed based on thousands, except percentages, per share amounts or as otherwise noted. As a result, certain totals may not sum due to rounding. Use of estimates The preparation of condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, the accrual for research and development expenses, the research and development tax credit receivable, share-based compensation, operating lease assets and liabilities, and income taxes. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. The future developments of the COVID-19 pandemic may also directly or indirectly impact the Company’s business, including impacts due to quarantines, border closures, increased border controls, travel restrictions, shelter-in-place orders and shutdowns, business closures, cancellations of public gatherings and other measures. Actual results could differ from the Company’s estimates. Foreign currency The financial statements of the Company’s subsidiaries with functional currencies other than the U.S. dollar are translated into U.S. dollars using period-end exchange rates for assets and liabilities, historical exchange rates for shareholders’ equity and weighted average exchange rates for operating results. Translation gains and losses are included in accumulated other comprehensive income (loss) in shareholders’ equity. Foreign currency transaction gains and losses are included in other income (expense), net in the results of operations. The Company recorded realized and unrealized foreign currency transaction losses of $6.1 million and gains of $1.4 million for the three months ended March 31, 2022 and 2021, respectively, which is included in other income (expense), net in the condensed consolidated statements of operations and comprehensive loss. Cash and cash equivalents The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. Marketable securities Marketable securities consist of investments with original maturities greater than ninety days at the date of acquisition. The Company has classified its investments with maturities beyond one year as short term, based on their highly liquid nature and because such marketable securities represent the investment of cash that is available for current operations. The Company considers its investment portfolio of investments as available-for-sale. Accordingly, these investments are recorded at fair value, which is based on quoted market prices or other observable inputs. Unrealized gains and losses are recorded as a component of other comprehensive income (loss). Realized gains and losses are determined on a specific identification basis and are included in other income (loss). Amortization and accretion of discounts and premiums is also recorded in other income (loss). When the fair value is below the amortized cost of the asset, an estimate of expected credit losses is made and is limited to the amount by which fair value is less than amortized cost. The credit-related impairment amount is recognized in the statement of operations; remaining impairment amount and unrealized gains are reported as a component of accumulated other comprehensive income (loss) in shareholders’ equity. Credit losses are recognized through the use of an allowance for credit losses account and subsequent improvements in expected credit losses are recognized as a reversal of the allowance account. If the Company has the intent to sell the security or it is more likely than not that the Company will be required to sell the security prior to recovery of its amortized cost basis the allowance for credit loss is written off and the excess of the amortized cost basis of the asset over its fair value is recorded in the condensed consolidated statements of operations. United Kingdom research and development tax credit As a company that carries out research and development activities, the Company is able to submit tax credit claims from two UK research and development tax relief programs, the Small and Medium-sized Enterprises research and development tax credit (“SME”) program and the Research and Development Expenditure Credit (“RDEC”) program depending on eligibility. Qualifying expenditures largely comprise employment costs for research staff, consumables and certain internal overhead costs incurred as part of research projects for which the Company does not receive income. Each reporting period, management evaluates which tax relief programs the Company is expected to be eligible for and records a reduction to research and development expense for the portion of the expense that it expects to qualify under the programs, that it plans to submit a claim for, and it has reasonable assurance that the amount will ultimately be realized. Based on criteria established by HM Revenue and Customs (“HMRC”), management of the Company expects a proportion of expenditures being undertaken in relation to its pipeline research, clinical trials management and manufacturing development activities to be eligible for the research and development tax relief programs for the year ended December 31, 2022. The Company expects when if files it claim for the years ended December 31, 2021 and 2022, it will qualify under the SME regime. The RDEC and SME credits are not dependent on the Company generating future taxable income or on the ongoing tax status or tax position of the Company. The Company has assessed its research and development activities and expenditures to determine whether the nature of the activities and expenditures will qualify for credit under the tax relief programs and whether the claims will ultimately be realized based on the allowable reimbursable expense criteria established by the UK government which are subject to interpretation. At each period end, the Company estimates the reimbursement available to the Company based on available information at the time. The Company recognizes credits from the research and development incentives when the relevant expenditure has been incurred and there is reasonable assurance that the reimbursement will be received. Such credits are accounted for as reductions in research and development expense in the condensed consolidated statement of operations and comprehensive loss. The following table below outlines the changes to the research and development tax credit receivable, including amounts recognized as an offset to research and development expense during the periods. (amounts in thousands): Three Months Ended March 31, 2022 2021 Balance at beginning of period $ 30,723 $ 17,344 Recognition of credit claims as offset to research and development expense 3,337 3,554 Receipt of credit claims (16,474 ) — Foreign currency translation (937 ) 147 Balance at end of period $ 16,649 $ 21,045 As of March 31, 2022, the Company’s tax incentive receivable from the UK government was $16.7 million, of which $13.4 million was classified as current. Restricted cash and construction deposits Cash and cash equivalents that are restricted as to withdrawal or use under the terms of certain contractual agreements are recorded as restricted cash on the Company’s condensed consolidated balance sheet. The Company has an outstanding letter of credit for $3.0 million associated with a lease and is required to hold this amount in a standalone bank account, as of March 31, 2022 and December 31, 2021. The Company is also contractually required to maintain cash collateral accounts associated with corporate credit cards and other leases in the amount of $1.3 million at March 31, 2022 and December 31, 2021. The Company includes the restricted cash balance in cash and cash equivalents when reconciling beginning-of-period and end-of-period total amounts shown on the condensed consolidated statements of cash flows. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported in the condensed consolidated balance sheet that sum to the total of the amounts reported in the unaudited condensed consolidated statement of cash flows (amounts in thousands): March 31, December 31, 2022 2021 Cash and cash equivalents $ 60,775 $ 55,912 Restricted cash 4,266 4,266 Total cash, cash equivalents and restricted cash shown in the statement of cash flows $ 65,041 $ 60,178 The Company has $7.9 million in an escrow account associated with construction on the Fremont facility, for which the Company has ceased construction and build-out and subleased to a third-party. Subject to the terms of the lease and reduction provisions, this amount will be returned to the Company upon qualifying construction expenditure or will be returned in late 2022 to the extent construction expenses have not been incurred. The Company deposited $10.0 million into the account in the first quarter of 2020 and has received $2.1 million in receipts from the escrow funds for costs incurred to date. Of the $7.9 million remaining in the escrow account, the entire balance is classified within prepaid expenses condensed consolidated balance sheet based on the timing of when the Company expects funds to be returned from the escrow agent. Accounts receivable Accounts receivable arise from product revenues and amounts due from the Company's collaboration partners and have standard payment terms that generally require payment within 30 to 90 days. The amount from product revenues represents amounts due from distributors in Europe, which are recorded net of reserves for trade discounts and allowances, and other incentives to the extent such amounts are payable to the customer by the Company. The Company monitors economic conditions to identify facts or circumstances that may indicate that its receivables are at risk of collection. The Company provides reserves against accounts receivable for estimated losses, if any, that may result from a customer's inability to pay based on the composition of its accounts receivable, current economic conditions, and historical credit loss activity. Amounts determined to be uncollectible are charged or written-off against the reserve. During the three months ended March 31, 2022, the Company did not record any expected credit losses related to outstanding accounts receivable. Product revenues, net Libmeldy In January 2022, the Company began generating product revenue from sales of Libmeldy in Europe following the approval of Libmeldy by the European Commission in December 2020 for the treatment of early onset metachromatic leukodystrophy (“MLD”), characterized by biallelic mutations in the arylsulfatase-A (ARSA) gene leading to a reduction of the ARSA enzymatic activity in children with (i) late infantile or early juvenile forms, without clinical manifestations of the disease, or (ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. The Company recognizes revenue when control of promised goods is transferred to a customer at an amount that reflects the consideration to which the Company expects to be entitled in exchange for those goods. Control of the product transfers upon infusion of the product. To determine revenue recognition, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price, including variable consideration, if any; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies the performance obligations. The Company only applies the five-step model to contracts when collectability of the consideration to which we are entitled in exchange for the goods the Company transfers to the customer is determined to be probable. Amounts are recorded as accounts receivable when the right to the consideration is unconditional. The Company does not assess whether a contract has a significant financing component if the expectation at contract inception is that the period between payment by the customer and the transfer of the promised goods or services to the customer will be one year or less. The Company expenses incremental costs of obtaining a contract as and when incurred if the expected amortization period of the asset that would have been recognized is one year or less or the amount is immaterial. As of March 31, 2022, the Company has not capitalized any costs to obtain any of contracts. The Company recognizes product revenues, net of variable consideration related to certain allowances and accruals, when the customer takes control of the product, which is at a point in time once the patient has been infused. Product revenue is recorded at the net sales price, or transaction price. The Company records product revenue reserves, which are classified as a reduction in product revenues, to account for the components of variable consideration. Variable consideration includes the following components: government rebates, including performance-based rebates, trade discounts and allowances, and other incentives, which are described below. These reserves are based on estimates of the amounts earned or to be claimed on the related sales and are classified as a liability. The Company's estimates of reserves established for variable consideration are calculated based upon an application of the expected value method, which is the sum of probability-weighted amounts in a range of possible consideration amounts. These estimates reflect the Company's historical experience, current contractual and statutory requirements, specific known market events and trends, industry data, and current expectations around final pricing. The amount of variable consideration that is included in the transaction price may be subject to constraint and is included in net product revenues only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration received may ultimately differ from the Company's estimates. If actual results vary, the Company adjusts these estimates, which could have an effect on earnings in the period of adjustment. The following is a summary of the types of variable consideration the Company records: Government rebates: The Company is subject to statutory government rebates on sales in certain European countries as well as estimated rebates in certain European countries because final pricing has not yet been negotiated. The Company records reserves for rebates in the same period the related product revenue is recognized, resulting in a reduction of product revenues and a current liability that is included in accrued expenses on the Company’s condensed consolidated balance sheet. The Company is also subject to potential rebates in connection with performance criteria agreed upon with certain payors. The estimate for rebates is based on statutory discount rates, industry pricing data, current expectations around final pricing to be obtained, and historical experience of the performance of the Company’s products during clinical trials The Company classifies rebates within accrued expenses in the accompanying condensed consolidated balance sheets. Trade discounts and allowances: The Company may offer customers discounts, such as prompt pay discounts to remit payment in accordance with the stated terms of the invoice and fees for distribution services. These discounts are explicitly stated in the contracts and recorded in the period the related product revenue is recognized. The Company estimates which customers will earn these discounts and fees and deducts these discounts and fees in full from gross product revenues and accounts receivable at the time the Company recognizes the related revenues. The Company classifies trade discounts and allowances as a reduction of accounts receivable within the accompanying condensed consolidated balance sheets. Product returns: Based on the timing of revenue recognition upon treatment with the patient, the Company does not expect any returns of the Company’s products. Other incentives: While the Company does not currently have any other incentives that have been recorded to date, the Company may enter into future arrangements that have other incentives that will be recorded as a reduction of revenue. Strimvelis The Company’s product sales of Strimvelis are currently distributed exclusively at the San Raffaele Hospital in Milan, Italy. The hospital will purchase and pay for the products and submit a claim to the payer. The Company’s contracted sales with the hospital contains a single performance obligation and the Company recognizes revenue from product sales when the Company has satisfied its performance obligation, which is upon transferring control of the products to the hospital. The Company evaluated the variable consideration under ASC 606 and there is currently no variable consideration included in the transaction price for the products. Costs to manufacture and deliver the product and those associated with administering the therapy are included in cost of product sales. As the product is sold in direct relation to a scheduled treatment, the Company estimates there is limited risk of product return, including the risk of product expiration. Disaggregated Product Revenue Disclosures During the quarter ended March 31, 2022, the Company recognized $5.1 million of product revenues related to Libmeldy, all related to sales in Europe, which is net of $1.4 million of product revenue reserves primarily related to governmental rebates, that have been recognized in accrued expenses in the accompanying condensed consolidated balance sheet. There were no Strimvelis sales during the three months ended March 31, 2022 or 2021. Strimvelis loss provision As part of its transaction with Glaxo Group Limited and GlaxoSmithKline Intellectual Property Development LTD (together, “GSK”) The Company recognizes the amortization of the loss provision on a diminishing balance basis based on the actual net loss incurred associated with Strimvelis and the expected future net losses to be generated until such time as Strimvelis is no longer commercially available. The amortization of the provision is recorded as a reduction in research and development expense. The Company has made an estimate of the expected future losses associated with Strimvelis and adjust this estimate as facts and circumstances change regarding the commercial availability and costs of maintaining and selling Strimvelis. The Company does not update the accrued loss provision for any subsequent adjustment of the future losses, however, the timing of recognizing the amortization of what was originally recorded is adjusted for updates to estimates of potential future losses The following table below outlines the changes to the Strimvelis loss provision for the periods ended March 31, 2022 and 2021 (amounts in thousands): Three Months Ended March 31, 2022 2021 Balance at beginning of period $ 3,419 $ 4,482 Amortization of loss provision (276 ) (446 ) Foreign currency translation (86 ) 40 Balance at end of period $ 3,057 $ 4,076 Of the balance as of March 31, 2022 noted in the table above, $0.9 million is classified as current, and $2.1 million is classified as non-current. Net loss per share Basic net loss per share is computed by dividing the net loss by the weighted average number of voting and non-voting ordinary shares outstanding for the period. Diluted net loss is computed by adjusting net loss based on the potential impact of dilutive securities. Diluted net loss per share is computed by dividing the diluted net loss by the weighted average number of ordinary shares outstanding for the period, including potential dilutive ordinary shares. For purpose of this calculation, outstanding options and unvested restricted shares are considered potential dilutive ordinary shares. Since the Company was in a loss position for all periods presented, basic net loss per share is the same as diluted net loss per share for all periods as the inclusion of all potential ordinary share equivalents outstanding would have been anti-dilutive. The following securities, presented based on amounts outstanding at each period end, are considered to be ordinary share equivalents, but were not included in the computation of diluted net loss per ordinary share because to do so would have been anti-dilutive: Three Months Ended March 31, 2022 2021 Share options 13,113,339 12,935,554 Unvested performance-based restricted share units 500,989 717,167 13,614,328 13,652,721 Recently adopted accounting pronouncements In November 2021, the FASB issued ASU No. 2020-10, Government Assistance (Topic 832): |
Fair Value Measurements and Mar
Fair Value Measurements and Marketable Securities | 3 Months Ended |
Mar. 31, 2022 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements and Marketable Securities | 3. Fair value measurements and marketable securities The following tables present information about the Company’s financial assets that have been measured at fair value as of March 31, 2022 and indicate the fair value of the hierarchy of the valuation inputs utilized to determine such fair value. In general, fair values determined by Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities. Fair value determined by Level 2 inputs utilize observable inputs other than Level 1 prices, such as quoted prices for similar assets or liabilities, quoted market prices in markets that are not active or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the related assets or liabilities. Fair values determined by Level 3 inputs are unobservable data points for the asset or liability, and include situations where there is little, if any, market activity for the asset or liability. During the three months ended March 31, 2022, there were no transfers between Level 1 and Level 2 financial assets. The following table summarizes the Company’s cash equivalents and marketable securities as of March 31, 2022 (amounts in thousands): Fair Value Measurements at March 31, 2022 Using: Level 1 Level 2 Level 3 Total Cash equivalents Money market funds $ 19,426 $ — $ — $ 19,426 Commercial paper — 13,198 — 13,198 Total cash equivalents $ 19,426 $ 13,198 $ — $ 32,624 Marketable securities Corporate bonds $ — $ 62,056 $ — $ 62,056 Commercial paper — 76,185 — $ 76,185 Total marketable securities $ — $ 138,241 $ — $ 138,241 Total $ 19,426 $ 151,439 $ — $ 170,865 The following table summarizes the Company’s cash equivalents and marketable securities as of December 31, 2021 (amounts in thousands): Fair Value Measurements at December 31, 2021 Using: Level 1 Level 2 Level 3 Total Cash equivalents Money market funds $ 21,085 $ — $ — $ 21,085 Corporate bonds — 7,321 — 7,321 Commercial paper — 13,198 — 13,198 Total cash equivalents $ 21,085 $ 20,519 $ — $ 41,604 Marketable securities Corporate bonds $ — $ 94,794 $ — 94,794 Commercial paper 69,401 — 69,401 Total marketable securities $ — $ 164,195 $ — $ 164,195 Total $ 21,085 $ 184,714 $ — $ 205,799 The carrying amount reflected in the condensed consolidated balance sheets for research and development tax incentive receivable, trade receivables, accounts payable, and accrued expenses approximate fair value due to their short-term maturities. The carrying value of the Company’s outstanding notes payable approximates fair value (a Level 2 fair value measurement), reflecting interest rates currently available to the Company. Marketable s ecurities The following table summarizes the Company’s marketable securities as of March 31, 2022 (amounts in thousands): At March 31, 2022 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Credit Losses Fair Value Corporate bonds $ 62,278 $ — $ (222 ) $ — $ 62,056 Commercial paper 89,588 — (205 ) — 89,383 Total $ 151,866 $ — $ (427 ) $ — $ 151,439 The following table summarizes the Company’s marketable securities as of December 31, 2021 (amounts in thousands): At December 31, 2021 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Credit Losses Fair Value Corporate bonds $ 102,224 $ — $ (109 ) $ — 102,115 Commercial paper 82,657 — (58 ) — 82,599 Total $ 184,881 $ — $ (167 ) $ — $ 184,714 The following table summarizes the Company’s available-for-sale debt securities by contractual maturity, as of March 31, 2022 and December 31, 2021 (amounts in thousands): At March 31, 2022 At December 31, 2021 Maturities in one year or less $ 147,193 $ 172,575 Maturities between one year and three years 4,246 12,139 Total $ 151,439 $ 184,714 |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 3 Months Ended |
Mar. 31, 2022 | |
Prepaid Expense And Other Assets Current [Abstract] | |
Prepaid Expenses and Other Current Assets | 4. Prepaid expenses and other current assets Prepaid expenses and other current assets consist of the following (amounts in thousands): March 31, December 31, 2022 2021 Prepaid external research and development expenses $ 2,732 $ 2,438 Inventories 1,799 2,016 Other prepayments 4,976 6,128 VAT receivable 738 1,169 Construction deposit - current 7,909 7,909 Non-trade receivables 2,769 3,351 Total prepaid expenses and other current assets $ 20,923 $ 23,011 |
Property and Equipment, Net
Property and Equipment, Net | 3 Months Ended |
Mar. 31, 2022 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment, Net | 5. Property and equipment, net Property and equipment, net consisted of the following (amounts in thousands): March 31, December 31, 2022 2021 Property and equipment: Lab equipment $ 5,978 $ 5,937 Leasehold improvements 2,403 2,450 Furniture and fixtures 301 303 Office and computer equipment 1,999 2,023 Construction-in-process 394 211 Property and equipment $ 11,075 $ 10,924 Less: accumulated depreciation (6,621 ) (6,157 ) Property and equipment, net $ 4,454 $ 4,767 Depreciation expense was $0.6 million and $0.5 million for the three months ended March 31, 2022 and 2021, respectively. |
Intangible Assets, Net
Intangible Assets, Net | 3 Months Ended |
Mar. 31, 2022 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Intangible Assets, Net | 6. Intangible assets, net Intangible assets, net of accumulated amortization, consisted of the following (amounts in thousands): At March 31, 2022 At December 31, 2021 Cost Accumulated amortization Net Cost Accumulated amortization Net License intangibles $ 4,259 $ (266 ) $ 3,993 $ 4,329 $ (180 ) $ 4,149 Total $ 4,259 $ (266 ) $ 3,993 $ 4,329 $ (180 ) $ 4,149 License intangibles consist of capitalized milestone payments or accruals of payments the Company has deemed probable upon receiving regulatory approval of Libmeldy in the EU. The license intangibles are being amortized on a straight-line basis over the remaining useful life of the related patents of approximately twelve years. For the three months ended March 31, 2022 and 2021, amortization of intangible assets totaled $0.1 million and nil, respectively. The effect of foreign currency translation on the net carrying value of intangible assets for the three months ended March 31, 2022 was less than $0.1 million. The following table summarizes the estimated future amortization for intangible assets for the next five years and thereafter (amounts in thousands): As of March 31, 2022 2022 (April - December) $ 270 2023 360 2024 360 2025 360 2026 360 Thereafter 2,283 Total $ 3,993 |
Other Assets
Other Assets | 3 Months Ended |
Mar. 31, 2022 | |
Other Assets Noncurrent Disclosure [Abstract] | |
Other Assets | 7. Other assets Other assets consist of the following (amounts in thousands): March 31, December 31, 2022 2021 Deferred tax assets $ 4,873 $ 4,086 Deposits 2,082 1,404 Other non-current assets 3,356 3,407 Deferred financing costs 420 693 Total other assets $ 10,731 $ 9,590 |
Accrued Expenses and Other Liab
Accrued Expenses and Other Liabilities | 3 Months Ended |
Mar. 31, 2022 | |
Accrued Liabilities And Other Liabilities [Abstract] | |
Accrued Expenses and Other Liabilities | 8. Accrued expenses and other liabilities Accrued expenses and other liabilities consisted of the following (amounts in thousands): March 31, December 31, 2022 2021 Accrued external research and development expenses $ 10,651 $ 9,273 Accrued payroll and related expenses 5,270 8,030 Accrued severance costs 2,684 491 Accrued milestone payments 1,316 2,058 Accrued professional fees 1,372 854 Accrued governmental rebates 1,214 — Accrued other 2,324 2,941 Strimvelis loss provision - current portion 1,152 671 Total accrued expenses and other liabilities $ 25,983 $ 24,318 |
Notes Payable
Notes Payable | 3 Months Ended |
Mar. 31, 2022 | |
Debt Disclosure [Abstract] | |
Notes Payable | 9. Notes payable In May 2019 In May 2021, the Company amended and restated the Original Credit Facility (the “Amended Credit Facility”). Under the Amended Credit Facility, the Lenders agreed to make term loans available to the Company in the aggregate amount of $100.0 million, including increasing the principal on the initial term loan to $33.0 million, from $25.0 million. To date, the Company has borrowed $33.0 million under the amended initial term loan. The remaining $67.0 million under the Amended Credit Facility may be drawn down in the form of a second and third term loan, the second term loan being a $33.0 million term loan available no earlier than July 1, 2022 and no later than July 1, 2023 upon certain regulatory approvals and evidence of the Company having $100 million in cash and cash equivalent investments; and the third term loan being a $34.0 million term loan available no earlier than July 1, 2023 and no later than July 1, 2024 upon evidence of the Company having $100 million in cash and cash equivalent investments and attaining a pre-specified trailing 12-month revenue target. Each term loan under the Amended Credit Facility bears interest at an annual rate equal to 5.95% plus LIBOR. The Company is required to make interest only payments on the term loan for 18 months following the date of the Amended Credit Facility, unless the Company is eligible for the second tranche, in which case the Company may elect to make interest-only payments for 30 months following the date of the Amended Credit Facility. The term loans under to the Amended Credit Facility begin amortizing on either the 18-month or the 30-month anniversary of the Amended Credit Facility (as applicable), with equal monthly payments of principal plus interest to be made by the Company to the Lenders in consecutive monthly installments until the loan maturity date. In addition, a final payment of 3.5% is due on the loan maturity date. The Company is accruing the final payment amount of $1.2 million associated with the first term loan of the Amended Credit Facility, to outstanding debt by charges to interest expense using the effective-interest method from the date of issuance through the loan maturity date. The Amended Credit Facility includes affirmative and negative covenants. The affirmative covenants include, among others, covenants requiring the Company to maintain their legal existence and governmental approvals, deliver certain financial reports, maintain insurance coverage, maintain property, pay taxes, satisfy certain requirements regarding accounts and comply with laws and regulations. The negative covenants include, among others, restrictions on the Company transferring collateral, incurring additional indebtedness, engaging in mergers or acquisitions, paying dividends or making other distributions, making investments, creating liens, amending material agreements and organizational documents, selling assets, changing the nature of the business and undergoing a change in control, in some cases subject to certain exceptions. The Company is also subject to an ongoing minimum cash financial covenant in which the Company must maintain unrestricted cash in an amount not less than $20.0 million following the utilization of the second term loan and not less than $35.0 million following the utilization of the third term loan. Notes payable consist of the following (amounts in thousands): At March 31, At December 31, 2022 2021 Notes payable, net of issuance costs $ 32,691 $ 32,669 Less: current portion (3,143 ) (786 ) Notes payable, net of current portion 29,548 31,883 Accretion related to final payment 265 203 Notes payable, long term $ 29,813 $ 32,086 As of March 31, 2022, the estimated future principal payments due are as follows (amounts in thousands): Aggregate Minimum Payments 2022 (April - December) 786 2023 9,429 2024 9,429 2025 9,429 2026 5,084 Total 34,157 Less: current portion (3,143 ) Less: unamortized portion of final payment (890 ) Less: unamortized debt issuance costs (311 ) Notes payable, long term $ 29,813 During the three months ended March 31, 2022 and 2021, the Company recognized $0.7 million and $0.5 million of interest expense, respectively, related to the initial term loan. The effective annual interest rate for the three months ended March 31, 2022 on the outstanding debt under the term loan was approximately 8.08%. |
Share-Based Compensation
Share-Based Compensation | 3 Months Ended |
Mar. 31, 2022 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Share-Based Compensation | 10. Share-based compensation The Company maintains four equity compensation plans; the Orchard Therapeutics Limited Employee Share Option Plan with Non-Employee Sub-Plan and U.S. Sub-Plan (the “2016 Plan”), the Orchard Therapeutics plc 2018 Share Option and Incentive Plan (the “2018 Plan”), the 2018 Employee Share Purchase Plan (the “ESPP”), and the 2020 Inducement Equity Plan (the “Inducement Plan”). The board of directors has determined not to make any further awards under the 2016 plan following the Company’s IPO. As of March 31, 2022, 7,600,164 shares remained available for issuance under the 2018 Plan, 721,500 shares remained available for issuance under the Inducement Plan, and 1,197,399 shares remained available for issuance under the ESPP. Share option activity The following table summarizes option activity under the plans for three months ended March 31, 2022: Number of Options Weighted Average Exercise Price Outstanding at December 31, 2021 17,300,740 $ 6.57 Granted 359,500 1.18 Exercised (222,381 ) - Forfeited (1,022,040 ) 7.35 Outstanding at March 31, 2022 16,415,819 $ 6.49 Vested and expected to vest, as of March 31, 2022 16,415,819 $ 6.49 Exercisable, March 31, 2022 8,093,954 $ 7.08 The weighted-average grant date fair value of share options granted during the three months ended March 31, 2022 was $0.79 per share. Restricted share units Performance-based restricted share units In April 2020, the Company granted 195,000 performance-based restricted share units restricted share units (“RSUs”) with a total grant date fair value of $1.4 million to its Chief Executive Officer, Bobby Gaspar, M.D., Ph.D. The award vests on January 2, 2024 as to 1/3 of the award for each of the first three to occur of four milestones, if each such milestone is achieved by the Company on or before December 31, 2023 and Dr. Gaspar remains continuously employed with the Company through January 2, 2024. The milestones relate to achievement of specific clinical and regulatory milestones. No performance-based share unit performance conditions associated with this award were deemed probable and none vested during the three months ended March 31, 2022. Time-based restricted share units Time-based restricted share units generally vest in equal annual installments over a three-year The following table summarizes award activity for the three months ended March 31, 2022: Performance-based RSUs Time-based RSUs Total RSUs Weighted Average Grant Date Fair Value Unvested and outstanding at December 31, 2021 195,000 123,333 318,333 $ 6.41 Granted — — — — Vested — (6,667 ) (6,667 ) (5.98 ) Forfeited — — — — Unvested and outstanding at March 31, 2022 195,000 116,666 311,666 $ 7.46 Share-based compensation expense Share-based compensation expense recorded as research and development and general and administrative expenses is as follows (amounts in thousands): March 31, March 31, 2022 2021 Research and development $ 1,962 $ 2,876 General and administrative 2,698 3,392 Total share-based compensation $ 4,660 $ 6,268 As of March 31, 2022, total unrecognized compensation cost related to unvested share options and time-based RSUs was approximately $30.0 million. This amount is expected to be recognized over a weighted average period of approximately 2.6 years. As of March 31, 2022, the total unrecognized compensation cost related to performance-based RSUs is a maximum of $1.7 million, dependent upon achievement of the aforementioned milestones. |
Restructuring Charges
Restructuring Charges | 3 Months Ended |
Mar. 31, 2022 | |
Restructuring And Related Activities [Abstract] | |
Restructuring Charges | 11. Restructuring charges On March 30, 2022 , the Company announced its commitment to focus on severe neurometabolic diseases and early research programs, and to discontinue its investment in and seek strategic alternatives for the Company’s programs in rare primary immune deficiencies, including OTL-103 for treatment of Wiskott Aldrich syndrome (“WAS”), OTL-102 for treatment of X-linked chronic granulomatous disease (“X-CGD”), and Strimvelis for adenosine deaminase severe combined immunodeficiency (“ADA-SCID”) . Activity for the quarter is summarized as follows (amounts in thousands): March 31, 2022 Balance at beginning of period $ 6 Charged to expense 2,481 Payments made (6 ) Balance at end of period $ 2,481 |
License Agreements
License Agreements | 3 Months Ended |
Mar. 31, 2022 | |
License And Research Arrangements [Abstract] | |
License Agreements | 12. License agreements GSK asset purchase and license agreement In April 2018, the Company completed an asset purchase and license agreement (the “GSK Agreement”) with subsidiaries of GSK to acquire a portfolio of autologous ex vivo The Company is required to use commercially reasonable efforts to obtain a Priority Review Voucher (“PRV”) from the United States Food and Drug Administration for each of the programs for MLD, WAS and TDT, the first of which GSK retained beneficial ownership over. GSK also has an option to acquire, at a price pursuant to an agreed upon formula, any PRV granted to the Company thereafter for MLD, WAS and TDT. If GSK does not exercise this option to purchase any PRV, the Company may sell the PRV to a third party and must share any proceeds in excess of a specified sale price equally with GSK. As part of the GSK Agreement the Company is also required to use its best endeavors to make Strimvelis commercially available in the European Union until such time as an alternative gene therapy is commercially available for patients in Italy, and at all times at the San Raffaele Hospital in Milan, provided that a minimum number of patients continue to be treated at this site. The Company will pay GSK non-refundable royalties and milestone payments in relation to the gene therapy programs acquired. The Company will pay a flat mid-single digit percentage royalty on the annual net sales of Strimvelis. The Company will also pay tiered royalty rates at a percentage beginning in the mid-teens up to twenty percent for the MLD and WAS products, upon marketing approval, calculated as percentages of aggregate cumulative net sales of the MLD and WAS products, respectively. The Company will pay a tiered royalty at a percentage from the high single-digits to low double-digit for the TDT product, upon marketing approval, calculated as percentages of aggregate annual net sales of the TDT product. These royalties owed to GSK are in addition to any royalties owed to other third parties under various license agreements for the GSK programs. In aggregate, the Company may pay up to £90.0 million in milestone payments upon achievement of certain sales milestones applicable to GSK. The Company’s royalty obligations with respect to MLD and WAS may be deferred for a certain period in the interest of prioritizing available capital to develop each product. The Company’s royalty obligations are subject to reduction on a product-by-product basis in the event of market control by biosimilars and will expire in April 2048. Other than Strimvelis, these royalty and milestone payments were not determined to be probable and estimable at the date of the acquisition and are not included as part of consideration. Telethon-OSR research and development collaboration and license agreements In connection with the Company’s entering into the GSK Agreement in April 2018, the Company also acquired and assumed agreements with Telethon Foundation and San Raffaele Hospital, together referred to as Telethon-OSR, for the research, development and commercialization of autologous ex vivo As consideration for the licenses, the Company will be required to make payments to Telethon-OSR upon achievement of certain product development milestones, up to an aggregate of approximately €31.0 million ($34.6 million at March 31, 2022). Additionally, the Company will be required to pay to Telethon-OSR a tiered mid-single to low-double digit royalty percentage on annual sales of licensed products covered by patent rights on a country-by-country basis, as well as a low double-digit percentage of sublicense income received from any certain third-party sublicenses of the collaboration programs. In May 2019, the Company entered into a license agreement with Telethon-OSR, under which Telethon-OSR granted to the Company an exclusive worldwide license for the research, development, manufacture and commercialization of Telethon-OSR’s ex vivo Oxford BioMedica license, development and supply agreement In November 2016, and amended in June 2017, May 2018, July 2018, September 2018, May 2019 and April 2020, the Company entered into an arrangement with Oxford BioMedica plc whereby Oxford BioMedica granted an exclusive intellectual property license to the Company for the purposes of research, development, and commercialization of collaboration products, and whereby Oxford BioMedica will provide process development services (“Oxford BioMedica Development Agreement”). As part of the consideration to rights and licenses granted under the Oxford BioMedica Development Agreement, the Company issued 588,220 ordinary shares to Oxford BioMedica. The Company is also obligated to make certain development milestone payments in the form of issuance of additional ordinary shares if the milestones are achieved. In November 2017, the first milestone was achieved, and the Company was committed to issue another 150,826 ordinary shares, and issued these shares in 2018. In September 2018, the second and fourth milestones were achieved, and the Company issued 150,826 ordinary shares. No milestones were met during the three months ended March 31, 2020. In April 2020, the fifth milestone was deemed to have been met upon execution of the amended agreement in April 2020, and the Company issued another 75,413 ordinary shares to Oxford BioMedica with a total value of $0.8 million which was recorded to research and development expense. T he Company may also pay low single-digit percentage royalties on net sales of collaborated product generated under the Oxford BioMedica Agreement. |
Collaboration Agreement with Ph
Collaboration Agreement with Pharming Group N.V. | 3 Months Ended |
Mar. 31, 2022 | |
Collaborative Arrangement [Abstract] | |
Collaboration Agreement with Pharming Group N.V. | 13. Collaboration agreement with Pharming Group N.V. Overview On July 1, 2021, the Company entered into a strategic collaboration with Pharming Group N.V. (“Pharming”) to research, develop, manufacture and commercialize OTL-105, an investigational ex vivo Under the terms of the Collaboration Agreement, Pharming was granted worldwide rights to OTL-105 and will be responsible for clinical development, regulatory filings and commercialization of the investigational gene therapy, including associated costs. The Company will lead the completion of IND-enabling activities and oversee manufacturing of OTL-105 during preclinical and clinical development, which will be funded by Pharming. In addition, both the Company and Pharming will explore the application of non-toxic conditioning regimen for use with OTL-105 administration. The Company received an upfront payment of $10.0 million in cash from Pharming. The Company is also eligible to receive up to $189.5 million in development, regulatory and sales milestones as well as mid-single to low double-digit royalty payments on future worldwide sales. Share Purchase Agreement The Company also entered into a Share Purchase Agreement with Pharming on July 1, 2021 (the “SPA”), pursuant to which the Company issued 1,227,738 ordinary shares to Pharming for total consideration of $7.5 million. The consideration is payment for the fair value of ordinary shares with a fair value of $4.1 million plus a $3.4 million premium on the fair value of the Company’s ordinary shares. The “Collaboration Agreement” and the “SPA” are referred to together as the “Pharming Agreements.” Accounting Analysis At the commencement of the arrangement, two units of accounting were identified, which are the issuance of 1,227,738 of the Company’s ordinary shares as part of the SPA, and the license and collaboration agreement, which conveys the license and provides for the Company to provide research, development, manufacturing services for OTL-105. The Pharming Agreements were entered into concurrently as part of a single commercial objective, and the Company considers them a single arrangement for accounting purposes. The total upfront payments of $17.5 million comprises $4.1 million attributed to the equity sold to Pharming and $13.4 million attributed to the Collaboration Agreement. In determining the fair value of the common stock issued to Pharming as part of the SPA, the Company used an option pricing valuation model to take into consideration certain holding period restrictions on the shares. The fair value of the Company’s common shares was considered a level 2 fair value measurement within the fair value hierarchy. Based on the fair value adjustments made by management, the fair value of the shares issued was determined to be $4.1 million with the excess proceeds of $3.4 million being allocation to the Collaboration Agreement. The Company recognizes revenue under ASC 606, Revenue from Contracts with Customer . the Company determined that it could not assert that it was probable that a significant reversal in the amount of cumulative revenue recognized will not occur. The Company re-evaluates the transaction price as of the end of each reporting period. The Company also considered the existence of any significant financing component within the Pharming Agreements given their upfront payment structure. Based upon this assessment, the Company concluded that the up-front payments were provided for valid business reasons and not for the purpose of providing financing. Accordingly, the Company has concluded that the upfront payment structure of the Pharming Agreements does not result in the existence of a significant financing component. The Company recognizes revenue associated with the performance obligation as the research, development and manufacturing services are provided using an input method, based on the cumulative costs incurred compared to the total estimated costs expected to be incurred to satisfy the performance obligation. The transfer of control to the customer occurs over the time period that the research, development and manufacturing services are to be provided by the Company, and this cost-to-cost method is, in management’s judgment, the best measure of progress towards satisfying the performance obligation. Reimbursement for research, development, and manufacturing services are recognized as the costs are incurred consistent with the cost-to-cost method. The Company's continuing obligations to provide research, development, and manufacturing services is based on the results of such efforts, and the estimated costs associated with the remaining efforts required to complete the performance obligations may change, which may materially impact revenue recognition. The Company regularly evaluates and, when necessary, updates the costs associated with the remaining effort under the Collaboration Agreement. Accordingly, revenue may fluctuate from period to period due to revisions to estimated costs, resulting in a change in the measure of progress for the performance obligation, or if the transaction price changes due to inclusion of any milestone payments that become unconstrained. The following table summarizes research and development costs incurred and collaboration revenue recognized in connection with the Company’s performance under the Collaboration Agreement (amounts in thousands): March 31, March 31, 2022 2021 Reimbursement revenue $ 400 $ — Upfront and milestone payment revenue 65 — Total $ 465 $ — The Company had $0.5 million and nil due from Pharming included in accounts receivable as of March 31, 2022 and December 31, 2021, respectively. As of March 31, 2022, the Company had contract liabilities of $12.4 million, which is classified as either current or long-term deferred revenue in the condensed consolidated balance sheet based on the period over which this is expected to be recognized. The deferred revenue balance represents the portion of the upfront payments received that are partially unsatisfied as of March 31, 2022. |
Income Taxes
Income Taxes | 3 Months Ended |
Mar. 31, 2022 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 14. Income taxes The Company recorded income tax expense 0.1 |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2022 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 15. Commitments and contingencies Legal proceedings The Company is not a party to any material litigation and does not have contingency reserves established for any litigation liabilities. Manufacturing and technology development master agreement with AGC Biologics The Company is party to an Agreement with AGC Biologic S.p.A (“AGC”) pursuant to which the Company is obligated to pay AGC for a minimum product manufacturing commitment, dedicated manufacturing and development resources, and for a lease component associated with the right of use of exclusive manufacturing suites within AGC’s existing facilities. The following table outlines the current commitments associated with the agreement, as of March 31, 2022 (amounts in thousands): Due in: Product manufacturing commitments Dedicated manufacturing and development resources Exclusive transduction suites Total remaining AGC commitment 2022 (April - December) $ 2,268 $ 5,821 $ — $ 8,089 2023 3,024 7,762 2,240 13,026 2024 3,024 7,762 2,240 13,026 2025 1,512 3,881 1,120 6,513 Total manufacturing commitments 9,828 25,226 5,600 40,654 * Tabular disclosure above has been translated to U.S. Dollar, from Euro, using an exchange rate of €1.00 to $1.12. Lease commitments The Company leases office and laboratory space and has an embedded lease at AGC. During the quarter ended March 31, 2022, the Company entered into a new lease in the U.K. for a period of 120 months. Total future minimum payments due under this lease amount to $9.4 million. Other than this new lease, there have been no material changes to the Company’s lease commitments as reported in the Company’s Annual Report on Form 10-K. Compliance with Nasdaq Continued Listing Requirements In April 2022, the Company received a letter from the Nasdaq Stock Market (“Nasdaq”) stating that it was not in compliance with the minimum bid price requirement for continued listing on the Nasdaq Global Select Market because the closing bid price for the Company’s ADSs was below $1.00 per share for 30 consecutive business days. The notice from Nasdaq has no immediate effect on the listing of the Company’s ADSs, and the ADSs will continue to be listed on the Nasdaq Global Select Market under the symbol “ORTX”. The Company have been afforded a 180-calendar day period, or until October 3, 2022, to regain compliance with the minimum bid price requirement. The continued listing standard will be met if the closing bid price of the Company’s ADSs is at least $1.00 per share for a minimum of ten consecutive business days during the 180-calendar day period. If the Company is not in compliance by October 3, 2022, the Company may be afforded a second 180-calendar day period to regain compliance if it meets certain requirements. The Company intends to monitor the closing bid price of its ADSs and it is currently evaluating its options for regaining compliance, which could include adjusting the ADS-to-ordinary share ratio. |
Employee Benefit Plans
Employee Benefit Plans | 3 Months Ended |
Mar. 31, 2022 | |
Compensation And Retirement Disclosure [Abstract] | |
Employee Benefit Plans | 16. Employee benefit plans The Company makes contributions to private defined contribution employee benefit plans on behalf of its employees. The Company provides employee contributions of up to six percent of each employee’s annual salary based on the jurisdiction the employees are located. The Company paid $0.5 million and $0.5 million in matching contributions for the three months ended March 31, 2022 and 2021, respectively. |
Basis of Presentation and Sum_2
Basis of Presentation and Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2022 | |
Accounting Policies [Abstract] | |
Basis of presentation | Basis of presentation The condensed consolidated interim financial statements of the Company are unaudited and have been prepared in accordance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) for interim financial reporting and in accordance with Regulation S-X, Rule 10-01. Accordingly, they do not include all of the information and footnotes required by U.S. GAAP for complete financial statements. Any reference in these notes to applicable guidance is meant to refer to the authoritative U.S. GAAP as found in the Accounting Standards Codification (“ASC”), and Accounting Standards Update (“ASU”), of the Financial Accounting Standards Board (“FASB”). All intercompany accounts and transactions between the Company and its subsidiaries have been eliminated upon consolidation. The accompanying unaudited condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and accompanying notes included in the Company’s Annual Report on Form 10-K filed with the SEC on March 30, 2022 (the “Annual Report”). The condensed consolidated balance sheet as of December 31, 2021 was derived from audited consolidated financial statements included in the Company’s Annual Report but does not include all disclosures required by U.S. GAAP. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted from these interim financial statements. However, these interim financial statements include all adjustments, consisting only of normal recurring adjustments, which are, in the opinion of the Company’s management, necessary to fairly state the results of the interim period. The interim results are not necessarily indicative of results to be expected for the full year. Amounts reported are computed based on thousands, except percentages, per share amounts or as otherwise noted. As a result, certain totals may not sum due to rounding. |
Use of estimates | Use of estimates The preparation of condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, the accrual for research and development expenses, the research and development tax credit receivable, share-based compensation, operating lease assets and liabilities, and income taxes. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. The future developments of the COVID-19 pandemic may also directly or indirectly impact the Company’s business, including impacts due to quarantines, border closures, increased border controls, travel restrictions, shelter-in-place orders and shutdowns, business closures, cancellations of public gatherings and other measures. Actual results could differ from the Company’s estimates. |
Foreign currency | Foreign currency The financial statements of the Company’s subsidiaries with functional currencies other than the U.S. dollar are translated into U.S. dollars using period-end exchange rates for assets and liabilities, historical exchange rates for shareholders’ equity and weighted average exchange rates for operating results. Translation gains and losses are included in accumulated other comprehensive income (loss) in shareholders’ equity. Foreign currency transaction gains and losses are included in other income (expense), net in the results of operations. The Company recorded realized and unrealized foreign currency transaction losses of $6.1 million and gains of $1.4 million for the three months ended March 31, 2022 and 2021, respectively, which is included in other income (expense), net in the condensed consolidated statements of operations and comprehensive loss. |
Cash and cash equivalents | Cash and cash equivalents The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. |
Marketable debt securities | Marketable securities Marketable securities consist of investments with original maturities greater than ninety days at the date of acquisition. The Company has classified its investments with maturities beyond one year as short term, based on their highly liquid nature and because such marketable securities represent the investment of cash that is available for current operations. The Company considers its investment portfolio of investments as available-for-sale. Accordingly, these investments are recorded at fair value, which is based on quoted market prices or other observable inputs. Unrealized gains and losses are recorded as a component of other comprehensive income (loss). Realized gains and losses are determined on a specific identification basis and are included in other income (loss). Amortization and accretion of discounts and premiums is also recorded in other income (loss). When the fair value is below the amortized cost of the asset, an estimate of expected credit losses is made and is limited to the amount by which fair value is less than amortized cost. The credit-related impairment amount is recognized in the statement of operations; remaining impairment amount and unrealized gains are reported as a component of accumulated other comprehensive income (loss) in shareholders’ equity. Credit losses are recognized through the use of an allowance for credit losses account and subsequent improvements in expected credit losses are recognized as a reversal of the allowance account. If the Company has the intent to sell the security or it is more likely than not that the Company will be required to sell the security prior to recovery of its amortized cost basis the allowance for credit loss is written off and the excess of the amortized cost basis of the asset over its fair value is recorded in the condensed consolidated statements of operations. |
United Kingdom Research and development tax credit | United Kingdom research and development tax credit As a company that carries out research and development activities, the Company is able to submit tax credit claims from two UK research and development tax relief programs, the Small and Medium-sized Enterprises research and development tax credit (“SME”) program and the Research and Development Expenditure Credit (“RDEC”) program depending on eligibility. Qualifying expenditures largely comprise employment costs for research staff, consumables and certain internal overhead costs incurred as part of research projects for which the Company does not receive income. Each reporting period, management evaluates which tax relief programs the Company is expected to be eligible for and records a reduction to research and development expense for the portion of the expense that it expects to qualify under the programs, that it plans to submit a claim for, and it has reasonable assurance that the amount will ultimately be realized. Based on criteria established by HM Revenue and Customs (“HMRC”), management of the Company expects a proportion of expenditures being undertaken in relation to its pipeline research, clinical trials management and manufacturing development activities to be eligible for the research and development tax relief programs for the year ended December 31, 2022. The Company expects when if files it claim for the years ended December 31, 2021 and 2022, it will qualify under the SME regime. The RDEC and SME credits are not dependent on the Company generating future taxable income or on the ongoing tax status or tax position of the Company. The Company has assessed its research and development activities and expenditures to determine whether the nature of the activities and expenditures will qualify for credit under the tax relief programs and whether the claims will ultimately be realized based on the allowable reimbursable expense criteria established by the UK government which are subject to interpretation. At each period end, the Company estimates the reimbursement available to the Company based on available information at the time. The Company recognizes credits from the research and development incentives when the relevant expenditure has been incurred and there is reasonable assurance that the reimbursement will be received. Such credits are accounted for as reductions in research and development expense in the condensed consolidated statement of operations and comprehensive loss. The following table below outlines the changes to the research and development tax credit receivable, including amounts recognized as an offset to research and development expense during the periods. (amounts in thousands): Three Months Ended March 31, 2022 2021 Balance at beginning of period $ 30,723 $ 17,344 Recognition of credit claims as offset to research and development expense 3,337 3,554 Receipt of credit claims (16,474 ) — Foreign currency translation (937 ) 147 Balance at end of period $ 16,649 $ 21,045 As of March 31, 2022, the Company’s tax incentive receivable from the UK government was $16.7 million, of which $13.4 million was classified as current. |
Restricted cash and construction deposits | Restricted cash and construction deposits Cash and cash equivalents that are restricted as to withdrawal or use under the terms of certain contractual agreements are recorded as restricted cash on the Company’s condensed consolidated balance sheet. The Company has an outstanding letter of credit for $3.0 million associated with a lease and is required to hold this amount in a standalone bank account, as of March 31, 2022 and December 31, 2021. The Company is also contractually required to maintain cash collateral accounts associated with corporate credit cards and other leases in the amount of $1.3 million at March 31, 2022 and December 31, 2021. The Company includes the restricted cash balance in cash and cash equivalents when reconciling beginning-of-period and end-of-period total amounts shown on the condensed consolidated statements of cash flows. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported in the condensed consolidated balance sheet that sum to the total of the amounts reported in the unaudited condensed consolidated statement of cash flows (amounts in thousands): March 31, December 31, 2022 2021 Cash and cash equivalents $ 60,775 $ 55,912 Restricted cash 4,266 4,266 Total cash, cash equivalents and restricted cash shown in the statement of cash flows $ 65,041 $ 60,178 The Company has $7.9 million in an escrow account associated with construction on the Fremont facility, for which the Company has ceased construction and build-out and subleased to a third-party. Subject to the terms of the lease and reduction provisions, this amount will be returned to the Company upon qualifying construction expenditure or will be returned in late 2022 to the extent construction expenses have not been incurred. The Company deposited $10.0 million into the account in the first quarter of 2020 and has received $2.1 million in receipts from the escrow funds for costs incurred to date. Of the $7.9 million remaining in the escrow account, the entire balance is classified within prepaid expenses condensed consolidated balance sheet based on the timing of when the Company expects funds to be returned from the escrow agent. |
Accounts receivable | Accounts receivable Accounts receivable arise from product revenues and amounts due from the Company's collaboration partners and have standard payment terms that generally require payment within 30 to 90 days. The amount from product revenues represents amounts due from distributors in Europe, which are recorded net of reserves for trade discounts and allowances, and other incentives to the extent such amounts are payable to the customer by the Company. The Company monitors economic conditions to identify facts or circumstances that may indicate that its receivables are at risk of collection. The Company provides reserves against accounts receivable for estimated losses, if any, that may result from a customer's inability to pay based on the composition of its accounts receivable, current economic conditions, and historical credit loss activity. Amounts determined to be uncollectible are charged or written-off against the reserve. During the three months ended March 31, 2022, the Company did not record any expected credit losses related to outstanding accounts receivable. |
Product revenues, net | Product revenues, net Libmeldy In January 2022, the Company began generating product revenue from sales of Libmeldy in Europe following the approval of Libmeldy by the European Commission in December 2020 for the treatment of early onset metachromatic leukodystrophy (“MLD”), characterized by biallelic mutations in the arylsulfatase-A (ARSA) gene leading to a reduction of the ARSA enzymatic activity in children with (i) late infantile or early juvenile forms, without clinical manifestations of the disease, or (ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. The Company recognizes revenue when control of promised goods is transferred to a customer at an amount that reflects the consideration to which the Company expects to be entitled in exchange for those goods. Control of the product transfers upon infusion of the product. To determine revenue recognition, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price, including variable consideration, if any; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies the performance obligations. The Company only applies the five-step model to contracts when collectability of the consideration to which we are entitled in exchange for the goods the Company transfers to the customer is determined to be probable. Amounts are recorded as accounts receivable when the right to the consideration is unconditional. The Company does not assess whether a contract has a significant financing component if the expectation at contract inception is that the period between payment by the customer and the transfer of the promised goods or services to the customer will be one year or less. The Company expenses incremental costs of obtaining a contract as and when incurred if the expected amortization period of the asset that would have been recognized is one year or less or the amount is immaterial. As of March 31, 2022, the Company has not capitalized any costs to obtain any of contracts. The Company recognizes product revenues, net of variable consideration related to certain allowances and accruals, when the customer takes control of the product, which is at a point in time once the patient has been infused. Product revenue is recorded at the net sales price, or transaction price. The Company records product revenue reserves, which are classified as a reduction in product revenues, to account for the components of variable consideration. Variable consideration includes the following components: government rebates, including performance-based rebates, trade discounts and allowances, and other incentives, which are described below. These reserves are based on estimates of the amounts earned or to be claimed on the related sales and are classified as a liability. The Company's estimates of reserves established for variable consideration are calculated based upon an application of the expected value method, which is the sum of probability-weighted amounts in a range of possible consideration amounts. These estimates reflect the Company's historical experience, current contractual and statutory requirements, specific known market events and trends, industry data, and current expectations around final pricing. The amount of variable consideration that is included in the transaction price may be subject to constraint and is included in net product revenues only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration received may ultimately differ from the Company's estimates. If actual results vary, the Company adjusts these estimates, which could have an effect on earnings in the period of adjustment. The following is a summary of the types of variable consideration the Company records: Government rebates: The Company is subject to statutory government rebates on sales in certain European countries as well as estimated rebates in certain European countries because final pricing has not yet been negotiated. The Company records reserves for rebates in the same period the related product revenue is recognized, resulting in a reduction of product revenues and a current liability that is included in accrued expenses on the Company’s condensed consolidated balance sheet. The Company is also subject to potential rebates in connection with performance criteria agreed upon with certain payors. The estimate for rebates is based on statutory discount rates, industry pricing data, current expectations around final pricing to be obtained, and historical experience of the performance of the Company’s products during clinical trials The Company classifies rebates within accrued expenses in the accompanying condensed consolidated balance sheets. Trade discounts and allowances: The Company may offer customers discounts, such as prompt pay discounts to remit payment in accordance with the stated terms of the invoice and fees for distribution services. These discounts are explicitly stated in the contracts and recorded in the period the related product revenue is recognized. The Company estimates which customers will earn these discounts and fees and deducts these discounts and fees in full from gross product revenues and accounts receivable at the time the Company recognizes the related revenues. The Company classifies trade discounts and allowances as a reduction of accounts receivable within the accompanying condensed consolidated balance sheets. Product returns: Based on the timing of revenue recognition upon treatment with the patient, the Company does not expect any returns of the Company’s products. Other incentives: While the Company does not currently have any other incentives that have been recorded to date, the Company may enter into future arrangements that have other incentives that will be recorded as a reduction of revenue. Strimvelis The Company’s product sales of Strimvelis are currently distributed exclusively at the San Raffaele Hospital in Milan, Italy. The hospital will purchase and pay for the products and submit a claim to the payer. The Company’s contracted sales with the hospital contains a single performance obligation and the Company recognizes revenue from product sales when the Company has satisfied its performance obligation, which is upon transferring control of the products to the hospital. The Company evaluated the variable consideration under ASC 606 and there is currently no variable consideration included in the transaction price for the products. Costs to manufacture and deliver the product and those associated with administering the therapy are included in cost of product sales. As the product is sold in direct relation to a scheduled treatment, the Company estimates there is limited risk of product return, including the risk of product expiration. Disaggregated Product Revenue Disclosures During the quarter ended March 31, 2022, the Company recognized $5.1 million of product revenues related to Libmeldy, all related to sales in Europe, which is net of $1.4 million of product revenue reserves primarily related to governmental rebates, that have been recognized in accrued expenses in the accompanying condensed consolidated balance sheet. There were no Strimvelis sales during the three months ended March 31, 2022 or 2021. |
Strimvelis loss provision | Strimvelis loss provision As part of its transaction with Glaxo Group Limited and GlaxoSmithKline Intellectual Property Development LTD (together, “GSK”) The Company recognizes the amortization of the loss provision on a diminishing balance basis based on the actual net loss incurred associated with Strimvelis and the expected future net losses to be generated until such time as Strimvelis is no longer commercially available. The amortization of the provision is recorded as a reduction in research and development expense. The Company has made an estimate of the expected future losses associated with Strimvelis and adjust this estimate as facts and circumstances change regarding the commercial availability and costs of maintaining and selling Strimvelis. The Company does not update the accrued loss provision for any subsequent adjustment of the future losses, however, the timing of recognizing the amortization of what was originally recorded is adjusted for updates to estimates of potential future losses The following table below outlines the changes to the Strimvelis loss provision for the periods ended March 31, 2022 and 2021 (amounts in thousands): Three Months Ended March 31, 2022 2021 Balance at beginning of period $ 3,419 $ 4,482 Amortization of loss provision (276 ) (446 ) Foreign currency translation (86 ) 40 Balance at end of period $ 3,057 $ 4,076 Of the balance as of March 31, 2022 noted in the table above, $0.9 million is classified as current, and $2.1 million is classified as non-current. |
Net loss per share | Net loss per share Basic net loss per share is computed by dividing the net loss by the weighted average number of voting and non-voting ordinary shares outstanding for the period. Diluted net loss is computed by adjusting net loss based on the potential impact of dilutive securities. Diluted net loss per share is computed by dividing the diluted net loss by the weighted average number of ordinary shares outstanding for the period, including potential dilutive ordinary shares. For purpose of this calculation, outstanding options and unvested restricted shares are considered potential dilutive ordinary shares. Since the Company was in a loss position for all periods presented, basic net loss per share is the same as diluted net loss per share for all periods as the inclusion of all potential ordinary share equivalents outstanding would have been anti-dilutive. The following securities, presented based on amounts outstanding at each period end, are considered to be ordinary share equivalents, but were not included in the computation of diluted net loss per ordinary share because to do so would have been anti-dilutive: Three Months Ended March 31, 2022 2021 Share options 13,113,339 12,935,554 Unvested performance-based restricted share units 500,989 717,167 13,614,328 13,652,721 |
Recently adopted accounting pronouncements | Recently adopted accounting pronouncements In November 2021, the FASB issued ASU No. 2020-10, Government Assistance (Topic 832): |
Basis of Presentation and Sum_3
Basis of Presentation and Summary of Significant Accounting Policies (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Accounting Policies [Abstract] | |
Summary of Amounts Recognized to Offset Research and Development Expense | The following table below outlines the changes to the research and development tax credit receivable, including amounts recognized as an offset to research and development expense during the periods. (amounts in thousands): Three Months Ended March 31, 2022 2021 Balance at beginning of period $ 30,723 $ 17,344 Recognition of credit claims as offset to research and development expense 3,337 3,554 Receipt of credit claims (16,474 ) — Foreign currency translation (937 ) 147 Balance at end of period $ 16,649 $ 21,045 |
Schedule of Reconciliation of Cash, Cash Equivalents and Restricted Cash | The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported in the condensed consolidated balance sheet that sum to the total of the amounts reported in the unaudited condensed consolidated statement of cash flows (amounts in thousands): March 31, December 31, 2022 2021 Cash and cash equivalents $ 60,775 $ 55,912 Restricted cash 4,266 4,266 Total cash, cash equivalents and restricted cash shown in the statement of cash flows $ 65,041 $ 60,178 |
Schedule of Changes in Loss Provision | The following table below outlines the changes to the Strimvelis loss provision for the periods ended March 31, 2022 and 2021 (amounts in thousands): Three Months Ended March 31, 2022 2021 Balance at beginning of period $ 3,419 $ 4,482 Amortization of loss provision (276 ) (446 ) Foreign currency translation (86 ) 40 Balance at end of period $ 3,057 $ 4,076 |
Securities Excluded in the Computation of Diluted Net Loss Per Ordinary Share | The following securities, presented based on amounts outstanding at each period end, are considered to be ordinary share equivalents, but were not included in the computation of diluted net loss per ordinary share because to do so would have been anti-dilutive: Three Months Ended March 31, 2022 2021 Share options 13,113,339 12,935,554 Unvested performance-based restricted share units 500,989 717,167 13,614,328 13,652,721 |
Fair Value Measurements and M_2
Fair Value Measurements and Marketable Securities (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Abstract] | |
Schedule of Cash Equivalents and Marketable Securities | The following table summarizes the Company’s cash equivalents and marketable securities as of March 31, 2022 (amounts in thousands): Fair Value Measurements at March 31, 2022 Using: Level 1 Level 2 Level 3 Total Cash equivalents Money market funds $ 19,426 $ — $ — $ 19,426 Commercial paper — 13,198 — 13,198 Total cash equivalents $ 19,426 $ 13,198 $ — $ 32,624 Marketable securities Corporate bonds $ — $ 62,056 $ — $ 62,056 Commercial paper — 76,185 — $ 76,185 Total marketable securities $ — $ 138,241 $ — $ 138,241 Total $ 19,426 $ 151,439 $ — $ 170,865 The following table summarizes the Company’s cash equivalents and marketable securities as of December 31, 2021 (amounts in thousands): Fair Value Measurements at December 31, 2021 Using: Level 1 Level 2 Level 3 Total Cash equivalents Money market funds $ 21,085 $ — $ — $ 21,085 Corporate bonds — 7,321 — 7,321 Commercial paper — 13,198 — 13,198 Total cash equivalents $ 21,085 $ 20,519 $ — $ 41,604 Marketable securities Corporate bonds $ — $ 94,794 $ — 94,794 Commercial paper 69,401 — 69,401 Total marketable securities $ — $ 164,195 $ — $ 164,195 Total $ 21,085 $ 184,714 $ — $ 205,799 |
Schedule of Marketable Securities | The following table summarizes the Company’s marketable securities as of March 31, 2022 (amounts in thousands): At March 31, 2022 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Credit Losses Fair Value Corporate bonds $ 62,278 $ — $ (222 ) $ — $ 62,056 Commercial paper 89,588 — (205 ) — 89,383 Total $ 151,866 $ — $ (427 ) $ — $ 151,439 The following table summarizes the Company’s marketable securities as of December 31, 2021 (amounts in thousands): At December 31, 2021 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Credit Losses Fair Value Corporate bonds $ 102,224 $ — $ (109 ) $ — 102,115 Commercial paper 82,657 — (58 ) — 82,599 Total $ 184,881 $ — $ (167 ) $ — $ 184,714 |
Schedule of Available-for-Sale Debt Securities by Contractual Maturity | The following table summarizes the Company’s available-for-sale debt securities by contractual maturity, as of March 31, 2022 and December 31, 2021 (amounts in thousands): At March 31, 2022 At December 31, 2021 Maturities in one year or less $ 147,193 $ 172,575 Maturities between one year and three years 4,246 12,139 Total $ 151,439 $ 184,714 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Prepaid Expense And Other Assets Current [Abstract] | |
Schedule of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consist of the following (amounts in thousands): March 31, December 31, 2022 2021 Prepaid external research and development expenses $ 2,732 $ 2,438 Inventories 1,799 2,016 Other prepayments 4,976 6,128 VAT receivable 738 1,169 Construction deposit - current 7,909 7,909 Non-trade receivables 2,769 3,351 Total prepaid expenses and other current assets $ 20,923 $ 23,011 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Property Plant And Equipment [Abstract] | |
Schedule of Property and Equipment, Net | Property and equipment, net consisted of the following (amounts in thousands): March 31, December 31, 2022 2021 Property and equipment: Lab equipment $ 5,978 $ 5,937 Leasehold improvements 2,403 2,450 Furniture and fixtures 301 303 Office and computer equipment 1,999 2,023 Construction-in-process 394 211 Property and equipment $ 11,075 $ 10,924 Less: accumulated depreciation (6,621 ) (6,157 ) Property and equipment, net $ 4,454 $ 4,767 |
Intangible Assets, Net (Tables)
Intangible Assets, Net (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Schedule of Intangible Assets Net of Accumulated Amortization | Intangible assets, net of accumulated amortization, consisted of the following (amounts in thousands): At March 31, 2022 At December 31, 2021 Cost Accumulated amortization Net Cost Accumulated amortization Net License intangibles $ 4,259 $ (266 ) $ 3,993 $ 4,329 $ (180 ) $ 4,149 Total $ 4,259 $ (266 ) $ 3,993 $ 4,329 $ (180 ) $ 4,149 |
Schedule of Estimated Future Amortization For Intangible Assets | The following table summarizes the estimated future amortization for intangible assets for the next five years and thereafter (amounts in thousands) As of March 31, 2022 2022 (April - December) $ 270 2023 360 2024 360 2025 360 2026 360 Thereafter 2,283 Total $ 3,993 |
Other Assets (Tables)
Other Assets (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Other Assets Noncurrent Disclosure [Abstract] | |
Schedule of Other Assets | Other assets consist of the following (amounts in thousands): March 31, December 31, 2022 2021 Deferred tax assets $ 4,873 $ 4,086 Deposits 2,082 1,404 Other non-current assets 3,356 3,407 Deferred financing costs 420 693 Total other assets $ 10,731 $ 9,590 |
Accrued Expenses and Other Li_2
Accrued Expenses and Other Liabilities (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Accrued Liabilities And Other Liabilities [Abstract] | |
Schedule of Accrued Expenses and Other Liabilities | Accrued expenses and other liabilities consisted of the following (amounts in thousands): March 31, December 31, 2022 2021 Accrued external research and development expenses $ 10,651 $ 9,273 Accrued payroll and related expenses 5,270 8,030 Accrued severance costs 2,684 491 Accrued milestone payments 1,316 2,058 Accrued professional fees 1,372 854 Accrued governmental rebates 1,214 — Accrued other 2,324 2,941 Strimvelis loss provision - current portion 1,152 671 Total accrued expenses and other liabilities $ 25,983 $ 24,318 |
Notes Payable (Tables)
Notes Payable (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Debt Disclosure [Abstract] | |
Summary of Notes Payable | Notes payable consist of the following (amounts in thousands): At March 31, At December 31, 2022 2021 Notes payable, net of issuance costs $ 32,691 $ 32,669 Less: current portion (3,143 ) (786 ) Notes payable, net of current portion 29,548 31,883 Accretion related to final payment 265 203 Notes payable, long term $ 29,813 $ 32,086 |
Summary of Estimated Future Principal Payments Due | As of March 31, 2022, the estimated future principal payments due are as follows (amounts in thousands): Aggregate Minimum Payments 2022 (April - December) 786 2023 9,429 2024 9,429 2025 9,429 2026 5,084 Total 34,157 Less: current portion (3,143 ) Less: unamortized portion of final payment (890 ) Less: unamortized debt issuance costs (311 ) Notes payable, long term $ 29,813 |
Share-Based Compensation (Table
Share-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Summary of Option Activity | The following table summarizes option activity under the plans for three months ended March 31, 2022: Number of Options Weighted Average Exercise Price Outstanding at December 31, 2021 17,300,740 $ 6.57 Granted 359,500 1.18 Exercised (222,381 ) - Forfeited (1,022,040 ) 7.35 Outstanding at March 31, 2022 16,415,819 $ 6.49 Vested and expected to vest, as of March 31, 2022 16,415,819 $ 6.49 Exercisable, March 31, 2022 8,093,954 $ 7.08 |
Summary of Award Activity | The following table summarizes award activity for the three months ended March 31, 2022: Performance-based RSUs Time-based RSUs Total RSUs Weighted Average Grant Date Fair Value Unvested and outstanding at December 31, 2021 195,000 123,333 318,333 $ 6.41 Granted — — — — Vested — (6,667 ) (6,667 ) (5.98 ) Forfeited — — — — Unvested and outstanding at March 31, 2022 195,000 116,666 311,666 $ 7.46 |
Share-based Compensation Expense | Share-based compensation expense recorded as research and development and general and administrative expenses is as follows (amounts in thousands): March 31, March 31, 2022 2021 Research and development $ 1,962 $ 2,876 General and administrative 2,698 3,392 Total share-based compensation $ 4,660 $ 6,268 |
Restructuring Charges (Tables)
Restructuring Charges (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Restructuring And Related Activities [Abstract] | |
Summary of Restructuring Charges | Activity for the quarter is summarized as follows (amounts in thousands): March 31, 2022 Balance at beginning of period $ 6 Charged to expense 2,481 Payments made (6 ) Balance at end of period $ 2,481 |
Collaboration Agreement with _2
Collaboration Agreement with Pharming Group N.V. (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Collaborative Arrangement [Abstract] | |
Summary of Research and Development Costs Incurred and Collaboration Revenue Recognized | The following table summarizes research and development costs incurred and collaboration revenue recognized in connection with the Company’s performance under the Collaboration Agreement (amounts in thousands): March 31, March 31, 2022 2021 Reimbursement revenue $ 400 $ — Upfront and milestone payment revenue 65 — Total $ 465 $ — |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Commitments And Contingencies Disclosure [Abstract] | |
Schedule of Annual Commitments Associated with the Contract | The following table outlines the current commitments associated with the agreement, as of March 31, 2022 (amounts in thousands): Due in: Product manufacturing commitments Dedicated manufacturing and development resources Exclusive transduction suites Total remaining AGC commitment 2022 (April - December) $ 2,268 $ 5,821 $ — $ 8,089 2023 3,024 7,762 2,240 13,026 2024 3,024 7,762 2,240 13,026 2025 1,512 3,881 1,120 6,513 Total manufacturing commitments 9,828 25,226 5,600 40,654 * Tabular disclosure above has been translated to U.S. Dollar, from Euro, using an exchange rate of €1.00 to $1.12. |
Nature of the Business - Additi
Nature of the Business - Additional Information (Details) $ / shares in Units, $ in Thousands | Feb. 09, 2021USD ($)shares | Mar. 31, 2021USD ($) | Mar. 31, 2022USD ($) | Mar. 31, 2022£ / shares | Dec. 31, 2021USD ($) | Dec. 31, 2021£ / shares | Feb. 09, 2021£ / shares | Feb. 09, 2021$ / shares |
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Nominal value of shares issued and sold | £ / shares | £ 0.10 | £ 0.10 | ||||||
Placement agent fees | $ 6,092 | |||||||
Accumulated deficit | $ 794,520 | $ 750,224 | ||||||
Cash, cash equivalents and marketable securities | $ 199,000 | |||||||
Securities Purchase Agreement | ||||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Net proceeds from private placement | $ 143,700 | |||||||
Securities Purchase Agreement | Private Placement | ||||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Number of ordinary shares issued and sold | shares | 20,900,321 | |||||||
Nominal value of shares issued and sold | £ / shares | £ 0.10 | |||||||
Purchase price per share | $ / shares | $ 6.22 | |||||||
Closing price of share date | Feb. 4, 2021 | |||||||
Placement agent fees | $ 6,000 | |||||||
Other issuance costs | $ 300 | |||||||
Private placement, transaction date | Feb. 4, 2021 | |||||||
Securities Purchase Agreement | Private Placement | Non-voting Ordinary Shares | ||||||||
Organization Consolidation And Presentation Of Financial Statements [Line Items] | ||||||||
Number of ordinary shares issued and sold | shares | 3,215,434 | |||||||
Nominal value of shares issued and sold | £ / shares | £ 0.10 |
Basis of Presentation and Sum_4
Basis of Presentation and Summary of Significant Accounting Policies - Additional Information (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |||
Mar. 31, 2022 | Mar. 31, 2021 | Dec. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2018 | |
Summary Of Significant Accounting Policies [Line Items] | |||||
Foreign currency transaction gain (loss) | $ (6,100,000) | $ 1,400,000 | |||
Tax incentive receivable, current | 13,394,000 | $ 30,723,000 | |||
Proceeds from lines of credit | 3,000,000 | 3,000,000 | |||
Cash collateral associated with corporate credit cards and other leases | 1,300,000 | 1,300,000 | |||
Total revenues | 5,524,000 | ||||
Strimvelis loss provision current | $ 1,152,000 | 671,000 | |||
Change in accounting principle, accounting standards update, adopted | true | ||||
Change in accounting principle, accounting standards update, adoption date | Jan. 1, 2022 | ||||
Change in accounting principle, accounting standards update, immaterial effect | true | ||||
New accounting pronouncement or change in accounting principle, description | ASU No. 2020-10 | ||||
Product Revenue, Net [Member] | |||||
Summary Of Significant Accounting Policies [Line Items] | |||||
Total revenues | $ 5,059,000 | ||||
Strimvelis | |||||
Summary Of Significant Accounting Policies [Line Items] | |||||
Total revenues | 0 | $ 0 | |||
Liability associated with the loss contract | $ 18,400,000 | ||||
Strimvelis loss provision current | 900,000 | ||||
Strimvelis loss provision non-current | $ 2,100,000 | ||||
Minimum | |||||
Summary Of Significant Accounting Policies [Line Items] | |||||
General payment terms | 30 days | ||||
Maximum | |||||
Summary Of Significant Accounting Policies [Line Items] | |||||
General payment terms | 90 days | ||||
Fremont Lease Agreement [Member] | |||||
Summary Of Significant Accounting Policies [Line Items] | |||||
Escrow deposit | $ 7,900,000 | $ 10,000,000 | |||
Increase decrease in escrow deposit | 2,100,000 | ||||
United Kingdom | |||||
Summary Of Significant Accounting Policies [Line Items] | |||||
Tax incentive receivable | 16,700,000 | ||||
Tax incentive receivable, current | 13,400,000 | $ 30,700,000 | |||
Europe | Government Rebates | Accrued Expenses | |||||
Summary Of Significant Accounting Policies [Line Items] | |||||
Product revenue reserve | 1,400,000 | ||||
Europe | Product Revenue, Net [Member] | Libmeldy | |||||
Summary Of Significant Accounting Policies [Line Items] | |||||
Total revenues | $ 5,100,000 |
Basis of Presentation and Sum_5
Basis of Presentation and Summary of Significant Accounting Policies - Summary of Amounts recognized to Offset Research and Development Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Summary Of Significant Accounting Policies [Line Items] | ||
Foreign currency translation | $ (6,100) | $ 1,400 |
United Kingdom | Research and Development Expense | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Balance at beginning of period | 30,723 | 17,344 |
Recognition of credit claims as offset to research and development expense | 3,337 | 3,554 |
Receipt of credit claims | (16,474) | |
Foreign currency translation | (937) | 147 |
Balance at end of period | $ 16,649 | $ 21,045 |
Basis of Presentation and Sum_6
Basis of Presentation and Summary of Significant Accounting Policies - Schedule of Reconciliation of Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 | Mar. 31, 2021 | Dec. 31, 2020 |
Summary Of Significant Accounting Policies [Abstract] | ||||
Cash and cash equivalents | $ 60,775 | $ 55,912 | ||
Restricted cash | 4,266 | 4,266 | ||
Total cash, cash equivalents and restricted cash shown in the statement of cash flows | $ 65,041 | $ 60,178 | $ 83,149 | $ 59,401 |
Basis of Presentation and Sum_7
Basis of Presentation and Summary of Significant Accounting Policies - Schedule of Changes in Loss Provision (Details) - Strimvelis - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Asset Acquisition [Line Items] | ||
Balance at beginning of period | $ 3,419 | $ 4,482 |
Amortization of loss provision | (276) | (446) |
Foreign currency translation | (86) | 40 |
Balance at end of period | $ 3,057 | $ 4,076 |
Basis of Presentation and Sum_8
Basis of Presentation and Summary of Significant Accounting Policies - Securities Excluded in the Computation of Diluted Net Loss Per Ordinary Share (Details) - shares | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common shares attributable to anti-dilutive shares | 13,614,328 | 13,652,721 |
Share options | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common shares attributable to anti-dilutive shares | 13,113,339 | 12,935,554 |
Unvested Performance-based Restricted Share Units | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Common shares attributable to anti-dilutive shares | 500,989 | 717,167 |
Fair Value Measurements and M_3
Fair Value Measurements and Marketable Securities - Additional Information (Details) $ in Thousands | Mar. 31, 2022USD ($) |
Fair Value Disclosures [Abstract] | |
Fair value of financial assets transfers between level 1 to level 2 | $ 0 |
Fair value of financial assets transfers between level 2 to level 1 | $ 0 |
Fair Value Measurements and M_4
Fair Value Measurements and Marketable Securities - Schedule of Cash Equivalents and Marketable Securities (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Cash equivalents | ||
Total cash equivalents | $ 32,624 | $ 41,604 |
Marketable securities | ||
Total marketable securities | 138,241 | 164,195 |
Total | 170,865 | 205,799 |
Level 1 | ||
Cash equivalents | ||
Total cash equivalents | 19,426 | 21,085 |
Marketable securities | ||
Total | 19,426 | 21,085 |
Level 2 | ||
Cash equivalents | ||
Total cash equivalents | 13,198 | 20,519 |
Marketable securities | ||
Total marketable securities | 138,241 | 164,195 |
Total | 151,439 | 184,714 |
Money Market Funds | ||
Cash equivalents | ||
Total cash equivalents | 19,426 | 21,085 |
Money Market Funds | Level 1 | ||
Cash equivalents | ||
Total cash equivalents | 19,426 | 21,085 |
Corporate Bonds | ||
Cash equivalents | ||
Total cash equivalents | 7,321 | |
Marketable securities | ||
Total marketable securities | 62,056 | 94,794 |
Corporate Bonds | Level 2 | ||
Cash equivalents | ||
Total cash equivalents | 7,321 | |
Marketable securities | ||
Total marketable securities | 62,056 | 94,794 |
Commercial Paper | ||
Cash equivalents | ||
Total cash equivalents | 13,198 | 13,198 |
Marketable securities | ||
Total marketable securities | 76,185 | 69,401 |
Commercial Paper | Level 2 | ||
Cash equivalents | ||
Total cash equivalents | 13,198 | 13,198 |
Marketable securities | ||
Total marketable securities | $ 76,185 | $ 69,401 |
Fair Value Measurements and M_5
Fair Value Measurements and Marketable Securities - Schedule of Marketable Securities (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Amortized Cost | $ 151,866 | $ 184,881 |
Gross Unrealized Losses | (427) | (167) |
Fair Value | 151,439 | 184,714 |
Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Amortized Cost | 62,278 | 102,224 |
Gross Unrealized Losses | (222) | (109) |
Fair Value | 62,056 | 102,115 |
Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Amortized Cost | 89,588 | 82,657 |
Gross Unrealized Losses | (205) | (58) |
Fair Value | $ 89,383 | $ 82,599 |
Fair Value Measurements and M_6
Fair Value Measurements and Marketable Securities - Schedule of Available-for-Sale Debt Securities by Contractual Maturity (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Fair Value Disclosures [Abstract] | ||
Maturities in one year or less | $ 147,193 | $ 172,575 |
Maturities between one year and three years | 4,246 | 12,139 |
Total | $ 151,439 | $ 184,714 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Schedule of Prepaid Expenses and Other Current Assets (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Prepaid Expense And Other Assets Current [Abstract] | ||
Prepaid external research and development expenses | $ 2,732 | $ 2,438 |
Inventories | 1,799 | 2,016 |
Other prepayments | 4,976 | 6,128 |
VAT receivable | 738 | 1,169 |
Construction deposit - current | 7,909 | 7,909 |
Non-trade receivables | 2,769 | 3,351 |
Total prepaid expenses and other current assets | $ 20,923 | $ 23,011 |
Property and Equipment, Net - S
Property and Equipment, Net - Schedule of Property and Equipment (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Property Plant And Equipment [Line Items] | ||
Property and equipment | $ 11,075 | $ 10,924 |
Less: accumulated depreciation | (6,621) | (6,157) |
Property and equipment, net | 4,454 | 4,767 |
Lab Equipment | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 5,978 | 5,937 |
Leasehold Improvements | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 2,403 | 2,450 |
Furniture and Fixtures | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 301 | 303 |
Office and Computer Equipment | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | 1,999 | 2,023 |
Construction-In-Progress | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment | $ 394 | $ 211 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Details) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Property Plant And Equipment [Abstract] | ||
Depreciation expense | $ 0.6 | $ 0.5 |
Intangible Assets, Net - Schedu
Intangible Assets, Net - Schedule of Intangible Assets Net of Accumulated Amortization (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Finite Lived Intangible Assets [Line Items] | ||
Intangible Assets, Cost | $ 4,259 | $ 4,329 |
Intangible Assets, Accumulated Amortized | (266) | (180) |
Intangible Assets, Net | 3,993 | 4,149 |
License Intangibles | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible Assets, Cost | 4,259 | 4,329 |
Intangible Assets, Accumulated Amortized | (266) | (180) |
Intangible Assets, Net | $ 3,993 | $ 4,149 |
Intangible Assets, Net - Additi
Intangible Assets, Net - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Dec. 31, 2021 | |
Finite Lived Intangible Assets [Line Items] | ||
Intangible asset, useful life | 12 years | |
Amortization of intangible assets | $ 266 | $ 180 |
Selling, General and Administrative Expenses | ||
Finite Lived Intangible Assets [Line Items] | ||
Amortization of intangible assets | 100 | |
Maximum | ||
Finite Lived Intangible Assets [Line Items] | ||
Effect of foreign currency translation on the net carrying value of intangible assets | $ 100 |
Intangible Assets, Net - Sche_2
Intangible Assets, Net - Schedule of Estimated Future Amortization For Intangible Assets (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Goodwill And Intangible Assets Disclosure [Abstract] | ||
2022 (April - December) | $ 270 | |
2023 | 360 | |
2024 | 360 | |
2025 | 360 | |
2026 | 360 | |
Thereafter | 2,283 | |
Intangible Assets, Net | $ 3,993 | $ 4,149 |
Other Assets - Schedule of Othe
Other Assets - Schedule of Other Assets (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Other Assets Noncurrent Disclosure [Abstract] | ||
Deferred tax assets | $ 4,873 | $ 4,086 |
Deposits | 2,082 | 1,404 |
Other non-current assets | 3,356 | 3,407 |
Deferred financing costs | 420 | 693 |
Total other assets | $ 10,731 | $ 9,590 |
Accrued Expenses and Other Li_3
Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Accrued Liabilities And Other Liabilities [Abstract] | ||
Accrued external research and development expenses | $ 10,651 | $ 9,273 |
Accrued payroll and related expenses | 5,270 | 8,030 |
Accrued severance costs | 2,684 | 491 |
Accrued milestone payments | 1,316 | 2,058 |
Accrued professional fees | 1,372 | 854 |
Accrued governmental rebates | 1,214 | |
Accrued other | 2,324 | 2,941 |
Strimvelis loss provision - current portion | 1,152 | 671 |
Total accrued expenses and other liabilities | $ 25,983 | $ 24,318 |
Notes Payable - Additional Info
Notes Payable - Additional Information (Details) - USD ($) | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||
May 31, 2021 | May 31, 2019 | Mar. 31, 2022 | Mar. 31, 2021 | Dec. 31, 2021 | |
Debt Instrument [Line Items] | |||||
Line of credit facility agreement date | May 31, 2019 | ||||
Credit facility maximum borrowings | $ 100,000,000 | $ 75,000,000 | |||
Proceeds from lines of credit | $ 3,000,000 | $ 3,000,000 | |||
Credit facility, remaining borrowings | $ 67,000,000 | ||||
Line of credit facility interest payments term | 18 months | ||||
Line of credit facility, frequency of payments | monthly payments | ||||
Line of credit facility, percentage of final payment | 3.50% | ||||
Final payment amount | $ 1,200,000 | ||||
Line of credit facility covenants description | The Company is also subject to an ongoing minimum cash financial covenant in which the Company must maintain unrestricted cash in an amount not less than $20.0 million following the utilization of the second term loan and not less than $35.0 million following the utilization of the third term loan | ||||
Term Loan | |||||
Debt Instrument [Line Items] | |||||
Debt instrument, interest expense | $ 700,000 | $ 500,000 | |||
Debt instrument, effective annual interest rate | 8.08% | ||||
LIBOR | |||||
Debt Instrument [Line Items] | |||||
Debt instrument, annual interest rate | 5.95% | ||||
Minimum | Second Tranche (Term Loan Available from July 1, 2022 to July 1, 2023) | |||||
Debt Instrument [Line Items] | |||||
Line of credit facility minimum cash financial covenant | $ 20,000,000 | ||||
Minimum | Third Tranche (Term Loan Available from July 1, 2023 to July 1, 2024) | |||||
Debt Instrument [Line Items] | |||||
Line of credit facility minimum cash financial covenant | 35,000,000 | ||||
Initial Term Loan | |||||
Debt Instrument [Line Items] | |||||
Credit facility maximum borrowings | 33,000,000 | $ 25,000,000 | |||
Proceeds from lines of credit | 33,000,000 | ||||
Second Tranche (Term Loan Available from July 1, 2022 to July 1, 2023) | |||||
Debt Instrument [Line Items] | |||||
Credit facility maximum borrowings | 33,000,000 | ||||
Line of Credit Facility, Covenant Terms | upon certain regulatory approvals and evidence of the Company having $100 million in cash and cash equivalent investments | ||||
Second Tranche (Term Loan Available from July 1, 2022 to July 1, 2023) | Minimum | |||||
Debt Instrument [Line Items] | |||||
Cash and cash equivalent investments | 100,000,000 | ||||
Third Tranche (Term Loan Available from July 1, 2023 to July 1, 2024) | |||||
Debt Instrument [Line Items] | |||||
Credit facility maximum borrowings | $ 34,000,000 | ||||
Line of Credit Facility, Covenant Terms | upon evidence of the Company having $100 million in cash and cash equivalent investments and attaining a pre-specified trailing 12-month revenue target. | ||||
Line of credit facility interest payments term | 30 months | ||||
Third Tranche (Term Loan Available from July 1, 2023 to July 1, 2024) | Minimum | |||||
Debt Instrument [Line Items] | |||||
Cash and cash equivalent investments | $ 100,000,000 |
Notes Payable - Summary of Note
Notes Payable - Summary of Notes Payable (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Long Term Notes And Loans [Abstract] | ||
Notes payable, net of issuance costs | $ 32,691 | $ 32,669 |
Less: current portion | (3,143) | (786) |
Notes payable, net of current portion | 29,548 | 31,883 |
Accretion related to final payment | 265 | 203 |
Notes payable, long term | $ 29,813 | $ 32,086 |
Notes Payable - Summary of Esti
Notes Payable - Summary of Estimated Future Principal Payments Due (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Long Term Debt By Maturity [Abstract] | ||
2022 (April - December) | $ 786 | |
2023 | 9,429 | |
2024 | 9,429 | |
2025 | 9,429 | |
2026 | 5,084 | |
Total | 34,157 | |
Less: current portion | (3,143) | |
Less: unamortized portion of final payment | (890) | |
Less: unamortized debt issuance costs | (311) | |
Notes payable, long term | $ 29,813 | $ 32,086 |
Share-Based Compensation - Addi
Share-Based Compensation - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 3 Months Ended |
Apr. 30, 2020 | Mar. 31, 2022 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Weighted-average grant date fair value of share options granted | $ 0.79 | |
RSUs vested | 6,667 | |
Total unrecognized compensation cost of stock option and time-based RSUs | $ 30 | |
Compensation cost expected to be recognized weighted average period | 2 years 7 months 6 days | |
Total unrecognized compensation cost of performance-based RSUs | $ 1.7 | |
Performance-based Restricted Share Units ("RSUs") | Chief Executive Officer | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Number of shares granted | 195,000 | |
Total grant date fair value | $ 1.4 | |
RSUs vested | 0 | |
Performance-based Restricted Share Units ("RSUs") | Chief Executive Officer | Achievement of At Least Three of Four Milestones | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Vesting rights | The award vests on January 2, 2024 as to 1/3 of the award for each of the first three to occur of four milestones, if each such milestone is achieved by the Company on or before December 31, 2023 | |
Time-based Restricted Share Units | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
RSUs vested | 6,667 | |
Award vesting period | 3 years | |
2018 Plan | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Remaining shares available for issuance | 7,600,164 | |
Inducement Plan | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Remaining shares available for issuance | 721,500 | |
2018 ESPP | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Remaining shares available for issuance | 1,197,399 |
Share-Based Compensation - Summ
Share-Based Compensation - Summary of Option Activity (Details) | 3 Months Ended |
Mar. 31, 2022$ / sharesshares | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Number of Options, Outstanding at December 31, 2020 | 17,300,740 |
Number of Options, Granted | 359,500 |
Number of Options, Exercised | (222,381) |
Number of Options, Forfeited | (1,022,040) |
Number of Options, Outstanding at March 31, 2021 | 16,415,819 |
Number of Options, Vested and expected to vest, as of March 31, 2021 | 16,415,819 |
Number of Options, Exercisable, March 31, 2021 | 8,093,954 |
Weighted Average Exercise Price, Outstanding at December 31, 2020 | $ / shares | $ 6.57 |
Weighted Average Exercise Price, Granted | $ / shares | 1.18 |
Weighted Average Exercise Price, Forfeited | $ / shares | 7.35 |
Weighted Average Exercise Price, Outstanding at March 31, 2021 | $ / shares | 6.49 |
Weighted Average Exercise Price, Vested and expected to vest, as of March 31, 2021 | $ / shares | 6.49 |
Weighted Average Exercise Price, Exercisable, March 31, 2021 | $ / shares | $ 7.08 |
Share-Based Compensation - Su_2
Share-Based Compensation - Summary of Award Activity (Details) | 3 Months Ended |
Mar. 31, 2022$ / sharesshares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
RSUs, Unvested and outstanding at December 31, 2020 | 318,333 |
RSUs, Vested | (6,667) |
RSUs, Unvested and outstanding at March 31, 2021 | 311,666 |
Weighted Average Grant Date Fair Value, Unvested and outstanding at December 31, 2020 | $ / shares | $ 6.41 |
Weighted Average Grant Date Fair Value, Vested | $ / shares | (5.98) |
Weighted Average Grant Date Fair Value, Unvested and outstanding at March 31, 2021 | $ / shares | $ 7.46 |
Performance-based RSUs | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
RSUs, Unvested and outstanding at December 31, 2020 | 195,000 |
RSUs, Unvested and outstanding at March 31, 2021 | 195,000 |
Time-based RSUs | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
RSUs, Unvested and outstanding at December 31, 2020 | 123,333 |
RSUs, Vested | (6,667) |
RSUs, Unvested and outstanding at March 31, 2021 | 116,666 |
Share-Based Compensation - Shar
Share-Based Compensation - Share-based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Total share-based compensation | $ 4,660 | $ 6,268 |
Research and Development | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Total share-based compensation | 1,962 | 2,876 |
General and Administrative | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Total share-based compensation | $ 2,698 | $ 3,392 |
Restructuring Charges - Additio
Restructuring Charges - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Restructuring Cost And Reserve [Line Items] | ||
Research and development | $ 28,234 | $ 21,035 |
Selling, general and administrative | 13,299 | $ 14,051 |
Neurometabolic Diseases and Early Research Programs | ||
Restructuring Cost And Reserve [Line Items] | ||
Research and development | 2,100 | |
Selling, general and administrative | 400 | |
Employee-Related Termination Costs | Neurometabolic Diseases and Early Research Programs | ||
Restructuring Cost And Reserve [Line Items] | ||
One-time charge | $ 2,500 |
Restructuring Charges - Summary
Restructuring Charges - Summary of Restructuring Charges (Details) $ in Thousands | 3 Months Ended |
Mar. 31, 2022USD ($) | |
Restructuring And Related Activities [Abstract] | |
Balance at beginning of period | $ 6 |
Charged to expense | 2,481 |
Payments made | (6) |
Balance at end of period | $ 2,481 |
License Agreements - Additional
License Agreements - Additional Information (Details) € in Millions, £ in Millions, $ in Millions | 1 Months Ended | 3 Months Ended | ||||||||||
Apr. 30, 2020USD ($)shares | May 31, 2019USD ($) | May 31, 2019EUR (€) | Apr. 30, 2018Program | Mar. 31, 2022USD ($)shares | Mar. 31, 2020Milestone | Jun. 30, 2018USD ($) | Mar. 31, 2022GBP (£)shares | Mar. 31, 2022EUR (€)shares | Dec. 31, 2021shares | Nov. 30, 2018shares | Sep. 30, 2018shares | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Ordinary Shares, Issued | shares | 125,904,945 | 125,904,945 | 125,904,945 | 125,674,095 | ||||||||
GSK Asset Purchase and License Agreement | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Number of additional programs in preclinical development | 3 | |||||||||||
Number of late-stage clinical gene therapy programs | 2 | |||||||||||
Number of earlier stage clinical gene therapy program | 1 | |||||||||||
Number of additional earlier-stage development programs | 3 | |||||||||||
Total consideration | $ | $ 133.6 | |||||||||||
Payment of tiered royalty, maximum percentage | 20.00% | 20.00% | 20.00% | |||||||||
Milestone payments payable upon achievement of certain sales milestones | £ | £ 90 | |||||||||||
Telethon-OSR License Agreements | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Milestone payments payable upon achievement of certain sales milestones | $ 34.6 | € 31 | ||||||||||
Upfront payment description | In May 2019, the Company entered into a license agreement with Telethon-OSR, under which Telethon-OSR granted to the Company an exclusive worldwide license for the research, development, manufacture and commercialization of Telethon-OSR’s ex vivo autologous HSC lentiviral based gene therapy for the treatment of mucopolysaccharidosis type I, including the Hurler variant (“MPS-IH”). Under the terms of the agreement, Telethon-OSR received €15.0 million in upfront and milestone payments from the Company upon entering into the agreement, resulting in $17.2 million in in-process research and development expense. | |||||||||||
Upfront and milestone payments | € | € 15 | |||||||||||
In-process research and development expense for upfront and milestone payments | $ | $ 17.2 | |||||||||||
Milestone payments payable upon achievement of certain development regulatory and commercial milestones | $ 32.7 | € 28 | ||||||||||
Oxford BioMedica License, Development and Supply Agreement | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Ordinary Shares, Issued | shares | 75,413 | 588,220 | 588,220 | 588,220 | 150,826 | 150,826 | ||||||
Number of milestones met | Milestone | 0 | |||||||||||
Oxford BioMedica License, Development and Supply Agreement | Research and Development Expense | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Milestone payment | $ | $ 0.8 |
Collaboration Agreement with _3
Collaboration Agreement with Pharming Group N.V. - Additional Information (Details) - USD ($) $ in Thousands | Jul. 01, 2021 | Mar. 31, 2021 | Mar. 31, 2022 |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Total consideration | $ 143,711 | ||
Strategic Collaboration Agreement with Pharming | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Cash upfront payment received | $ 10,000 | ||
Non-refundable up-front payment | 10,000 | ||
Strategic Collaboration Agreement with Pharming | Maximum | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Milestone payments receivable | $ 189,500 | ||
Share Purchase Agreement with Pharming | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Issuance of ordinary shares | 1,227,738 | ||
Total consideration | $ 7,500 | ||
Common stock consideration | 4,100 | ||
Premium consideration | 3,400 | ||
Pharming Agreements | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Total upfront payments | 17,500 | ||
Upfront payments attributed to equity sold | 4,100 | ||
Upfront payments attributed to research activities | 13,400 | ||
Shares issued as part of license agreement | 4,100 | ||
Excess proceeds from fair value of shares issued allocated to collaboration agreement | $ 3,400 | ||
Collaborative Arrangement | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Contract liabilities | $ 12,400 | ||
Collaborative Arrangement | Accounts Receivable | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Accounts receivable | $ 500 |
Collaboration Agreement with _4
Collaboration Agreement with Pharming Group N.V. - Summary of Research and Development Costs Incurred and Collaboration Revenue Recognized (Details) $ in Thousands | 3 Months Ended |
Mar. 31, 2022USD ($) | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Total revenues | $ 5,524 |
Collaborative Arrangement | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Total revenues | 465 |
Collaborative Arrangement | Reimbursement Revenue | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Total revenues | 400 |
Collaborative Arrangement | Upfront and Milestone Payment Revenue | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Total revenues | $ 65 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Income Tax Disclosure [Abstract] | ||
Income tax expense | $ 58 | $ 1,087 |
Commitments and Contingencies -
Commitments and Contingencies - Schedule of Annual Commitments Associated with the Contract (Details) $ in Thousands | Mar. 31, 2022USD ($) |
Other Commitments [Line Items] | |
2022 (April - December) | $ 8,089 |
2023 | 13,026 |
2024 | 13,026 |
2025 | 6,513 |
Total manufacturing commitments | 40,654 |
Product Manufacturing Commitments | |
Other Commitments [Line Items] | |
2022 (April - December) | 2,268 |
2023 | 3,024 |
2024 | 3,024 |
2025 | 1,512 |
Total manufacturing commitments | 9,828 |
Dedicated Manufacturing And Development Resources | |
Other Commitments [Line Items] | |
2022 (April - December) | 5,821 |
2023 | 7,762 |
2024 | 7,762 |
2025 | 3,881 |
Total manufacturing commitments | 25,226 |
Exclusive Transduction Suites | |
Other Commitments [Line Items] | |
2023 | 2,240 |
2024 | 2,240 |
2025 | 1,120 |
Total manufacturing commitments | $ 5,600 |
Commitments and Contingencies_2
Commitments and Contingencies - Schedule of Annual Commitments Associated with the Contract (Parenthetical) (Details) | Mar. 31, 2022 |
Commitments And Contingencies Disclosure [Abstract] | |
Exchange rate, Euro to U.S. Dollar | 1.12 |
Commitments and Contingencies_3
Commitments and Contingencies - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | |
Apr. 30, 2022 | Mar. 31, 2022 | |
Other Commitments [Line Items] | ||
New lease period | 120 months | |
Total future minimum lease payments due under new lease | $ 9.4 | |
Subsequent Events | Nasdaq Global Select Market | ADSs | ||
Other Commitments [Line Items] | ||
Maximum closing bid price per share | $ 1 | |
Number of business days closing bid price below than minimum requirement of bid price | 30 days | |
Threshold limit period to regain compliance with minimum bid price requirement | 180 days | |
Compliance with continued listing requirement description | The Company have been afforded a 180-calendar day period, or until October 3, 2022, to regain compliance with the minimum bid price requirement. The continued listing standard will be met if the closing bid price of the Company’s ADSs is at least $1.00 per share for a minimum of ten consecutive business days during the 180-calendar day period. If the Company is not in compliance by October 3, 2022, the Company may be afforded a second 180-calendar day period to regain compliance if it meets certain requirements. The Company intends to monitor the closing bid price of its ADSs and it is currently evaluating its options for regaining compliance, which could include adjusting the ADS-to-ordinary share ratio. |
Employee Benefit Plans - Additi
Employee Benefit Plans - Additional Information (Details) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Compensation And Retirement Disclosure [Abstract] | ||
Maximum annual contribution employer matches per employee, percent | 6.00% | |
Contribution expenses | $ 0.5 | $ 0.5 |