Content analysis
?Positive | ||
Negative | ||
Uncertain | ||
Constraining | ||
Legalese | ||
Litigous | ||
Readability |
H.S. junior Avg
|
New words:
abundant, ACA, advocacy, ago, appeal, arbitrarily, arbitrary, archived, AstraZeneca, asymmetrical, autoimmune, awaiting, bad, beneficiary, bioconjugated, bioconjugation, biofactory, BioPharma, bluebird, Boehringer, bolster, Bristol, calendar, Canadian, capriciously, Capstan, career, catch, CE, certiorari, chair, Chamber, chemotherapy, complaint, compositionally, constraint, contrary, copy, CTR, cytokine, datacenter, declaratory, destined, dozen, enjoined, enlargement, Ensoma, enterprise, explicitly, FDORA, fetal, geopolitical, greatly, guideline, Harmonisation, hematopoietic, hemoglobin, housekeeping, Howze, HSC, ICH, ideal, IEC, inactivation, Ingelheim, iqDNA, IVDR, Janssen, journal, legacy, lymphoma, Merck, mifepristone, mobile, ModernaTX, modernizing, morale, myeloid, Nevada, nonpromotional, Nonretirement, Nordisk, Novo, onsite, opsonization, organizational, Orna, outset, overrule, password, pave, PAYGO, phishing, PhRMA, PIE, plaintiff, pleading, Postemployment, predominant, predominantly, preexisting, progenitor, programmatic, propagation, quiet, randomized, ratably, rationale, reconciling, refine, regularly, renovate, reorganization, reply, Rescue, RIF, Sana, secondary, sequestration, sickle, speak, splenic, Squibb, standalone, stay, stayed, suit, tiered, transposed, truthful, tying, Umoja, unbiased, uncompensated, unconditional, uncured, Underpinning, unlawfully, unreachable, unscheduled, upward, verify, vigorously, vindicate, Washington, widen, Windsor, writ, Yalonda
Removed:
abnormal, accommodate, accommodated, accompanied, acute, adjusting, adolescent, adult, amino, anxiety, array, ASGPr, asialoglycoprotein, aspect, assay, Attaining, autosomal, avascular, biliary, biochemical, biology, birth, bone, break, cadence, cascade, cerebral, ceruloplasmin, characteristic, checkpoint, codon, computing, confirmation, confirmed, conjugate, content, contractually, contrast, conversion, correlated, covalently, cytoplasm, daily, decayed, dedicated, degeneration, delete, depression, diagnosed, diet, dietary, differentiator, dimension, disability, displayed, disrupted, distributed, division, donor, dosed, dramatically, Dyno, dysarthria, electorate, elevated, embedded, encoded, encoding, engineer, enhanced, enhancer, episodic, epithelial, ERT, exact, examined, existed, eye, fallen, feature, figure, firefly, flanked, fluorescent, focal, formula, formulate, foundational, frequent, functionality, functioning, galactosamine, GalNAc, GCase, genomic, GFP, glucosylceramide, green, handled, HBV, heavy, hematology, hepatocyte, hepatomegaly, highlighting, HIV, hybridization, hydrodynamic, IBC, IgG, immunocompetent, immunoglobulin, immunosorbent, immunosuppressive, incorporating, induced, infancy, inflammation, inheritance, inhibiting, injected, injection, inserting, inseverable, intake, intent, intron, invasive, inverted, IQ, iteration, ITR, IX, judge, knock, lacking, leader, leaving, Leber, left, line, linear, liter, lowering, mAb, macrophage, matched, measurable, metabolic, modest, modifying, molecule, multifactorial, mutated, myocyte, Neurodegenerative, neuropsychiatric, neutralizing, newborn, Ninety, nonbiological, nuclear, oligonucleotide, osteopenia, OTAT, overload, PAH, pain, painful, pair, participant, particle, passive, passively, pharmacology, Phe, photoreceptor, pigment, predecessor, preserving, procuring, profound, promoter, prophylactically, protracted, proven, pulsatile, purification, rapamycin, reallocate, receptor, recessive, redemption, redirect, repair, reporter, residual, respiratory, retinal, revealed, rheumatology, RPE, RSV, sanitization, screening, secrete, secreted, secretion, seize, Selecta, selectively, selectivity, serum, shell, shifted, simplify, situ, slurred, solidify, spectrum, speech, spiked, splenomegaly, spread, SRT, stacking, standardization, step, stimulate, streamlined, subretinally, substrate, subtitle, successive, surface, sustainably, symptomatic, syncytial, systemically, tail, technique, teen, terminal, thought, today, transduced, transducing, transfected, transfection, transferrin, transient, transitional, translation, translocated, transplant, transplantation, travel, tumor, typical, Tyr, tyrosine, unclear, uncontrolled, undistributed, vein, voted, widest, worker
Financial report summary
?Competition
Applied Dna Sciences • Ocugen • IVERIC bio • Arcturus Therapeutics • uniQure • Editas Medicine • Poseida Therapeutics • Q32 Bio • LogicBio Therapeutics • Beam TherapeuticsRisks
- We have incurred significant losses since our inception, have no products approved for sale and we expect to incur losses over the next several years.
- We have never generated revenue from product sales and may never achieve or maintain profitability.
- Raising additional capital may cause dilution to our stockholders, restrict our operations, or require us to relinquish rights to our technologies or product candidates.
- Our limited operating history may make it difficult to evaluate the success of our business to date and to assess our future viability.
- Our ability to utilize our net operating loss carryforwards may be subject to limitations.
- Risks related to discovery and development
- We are very early in our development efforts. We have not identified any product candidates for Investigational New Drug, or IND, -enabling studies or clinical development, and as a result it will be years before we commercialize a product candidate, if ever. If we are unable to identify and advance product candidates through preclinical studies and clinical trials, obtain marketing approval and ultimately commercialize them, or experience significant delays in doing so, our business will be materially harmed.
- We may encounter substantial delays in commencement, enrollment or completion of our clinical trials or we may fail to demonstrate purity, potency and safety to the satisfaction of applicable regulatory authorities, which could prevent us from commercializing any product candidates we determine to develop on a timely basis, if at all.
- Genetic medicine is an emerging area of drug development that poses many scientific and other risks. We have only limited prior experience in genetic medicine research and manufacturing and no prior experience in genetic medicine clinical development. Our lack of experience for our genetic medicine programs may limit our ability to be successful or may delay our development efforts.
- Our non-viral genetic medicine platforms are based on novel technologies that are unproven, which makes it difficult to predict the time and cost of development and of subsequently obtaining regulatory approval, if at all.
- If any product candidates we may develop cause undesirable side effects or have other unexpected adverse properties, such side effects or properties could delay or prevent regulatory approval, limit the commercial potential or result in significant negative consequences following any potential marketing approval.
- The outcome of preclinical studies and earlier-stage clinical trials may not be predictive of future results or the success of later preclinical studies and clinical trials.
- We may not be successful in our efforts to identify, discover or develop potential product candidates.
- The genetic medicine field is relatively new and evolving rapidly. We are focusing our research and development efforts on our non-viral genetic medicine platforms, but other genetic medicine technologies may be discovered that provide significant advantages over our platforms, which could materially harm our business.
- We may expend our limited resources to pursue a particular program, product candidate or indication and fail to capitalize on programs, product candidates or indications that may be more profitable or for which there is a greater likelihood of success.
- Clinical trial and product liability lawsuits against us could divert our resources, could cause us to incur substantial liabilities and could limit commercialization of any product candidates we may develop.
- We intend to manufacture drug substance and drug product using external cleanroom facilities and/or CMOs, which will require significant resources. If we fail to successfully execute this strategy, our business will be materially harmed.
- The manufacture of genetic medicine products is complex and difficult and is subject to a number of scientific and technical risks, some of which are common to the manufacture of drugs and biologics and others of which are unique to the manufacture of genetic medicines. We could experience manufacturing problems that result in delays in our development or commercialization programs.
- Our non-viral genetic medicine platforms are novel, and the combination of a novel manufacturing process and novel constructs with untested development of the process at a larger scale may cause us to experience delays in satisfying regulatory authorities or production problems that result in delays in our development or commercialization programs, limit the supply of any product candidates we may develop or otherwise harm our business.
- Testing of and changes to methods of product candidate manufacturing or formulation may result in additional costs or delay.
- We and our contract manufacturers are subject to significant regulation with respect to manufacturing our products. The manufacturing facilities on which we rely may not continue to meet regulatory requirements and have limited capacity.
- We currently depend on a small number of third-party suppliers for our drug substance and drug product, and we expect to continue to depend on third-party suppliers for materials used in the manufacture of any product candidates we may develop, and the loss of these third-party suppliers or their inability to supply us with adequate materials, particularly those raw materials that are in short supply, could harm our business.
- We rely, and expect to continue to rely, on third parties to conduct some or all aspects of our product manufacturing, research and preclinical and clinical testing, and these third parties may not perform satisfactorily.
- We may from time to time be dependent on single-source suppliers for some of the components and materials used in, and the space and processes required to develop, our development candidates and investigational medicines.
- We expect to rely on third parties to conduct, supervise and monitor IND-enabling studies and clinical studies, and if these third parties perform in an unsatisfactory manner, it may harm our business.
- We have entered into, and may continue to enter into, collaborations with third parties for the research, development and commercialization of certain of the product candidates we may develop. If any such collaborations are not successful, we may not be able to capitalize on the market potential of those product candidates.
- If conflicts arise between us and our collaborators or strategic partners, these parties may act in a manner adverse to us and could limit our ability to implement our strategies.
- If we are not able to establish collaborations on commercially reasonable terms, we may have to alter our development and commercialization plans.
- If we breach, or a counterparty breaches, our agreements with third parties or if there is a dispute concerning any of our agreements with third parties, our business could be materially harmed.
- We face substantial competition, which may result in others discovering, developing or commercializing products before us or more successfully than we do.
- Even if any product candidate that we may develop receives marketing approval, it may fail to achieve the degree of market acceptance by physicians, patients, third-party payers and others in the medical community necessary for commercial success.
- The pricing, insurance coverage and reimbursement status of newly approved products is uncertain. Failure to obtain or maintain adequate coverage and reimbursement for our future product candidates, if approved, could limit our ability to market those products and decrease our ability to generate product revenue.
- If we are unable to establish sales, marketing and distribution capabilities or enter into sales, marketing and distribution agreements with third parties, we may not be successful in commercializing any product candidates we may develop if and when they are approved.
- Any product candidates for which we intend to seek approval as biologic products may face competition sooner than anticipated.
- If the market opportunities for any product candidates we may develop are smaller than we believe they are, our potential revenues may be adversely affected, and our business may suffer. Because the target patient populations for many of the initial product candidates we may develop are small, we must be able to successfully identify patients and achieve a significant market share to maintain profitability and growth.
- If we or our licensors are unable to obtain, maintain and defend patent and other intellectual property protection for our product candidates and technology, or if the scope of the patent or other intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and our ability to successfully develop and commercialize any product candidates we may develop or our technology may be adversely affected due to such competition.
- Our rights to develop and commercialize any product candidates are subject, in part, to the terms and conditions of licenses granted to us by third parties. If we fail to comply with our obligations under our current or future intellectual property license agreements or otherwise experience disruptions to our business relationships with our current or any future licensors, we could lose intellectual property rights that are important to our business.
- We may not be able to protect our intellectual property rights throughout the world.
- Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other requirements imposed by government patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
- We may not be successful in obtaining necessary rights to product candidates we may develop through acquisitions and in-licenses.
- Issued patents covering any product candidates we may develop could be found invalid or unenforceable if challenged in court or before administrative bodies in the United States or abroad.
- If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
- Third parties may initiate legal proceedings alleging that we are infringing, misappropriating or otherwise violating their intellectual property rights, the outcome of which would be uncertain and could harm our business.
- Intellectual property litigation or other proceedings could cause us to spend substantial resources and distract our personnel from their normal responsibilities.
- We may be subject to claims asserting that our employees, consultants or advisors have wrongfully used or disclosed alleged trade secrets of their current or former employers or claims asserting ownership of what we regard as our own intellectual property.
- Changes in patent law in the United States or worldwide could diminish the value of patents in general, thereby impairing our ability to protect any product candidates we may develop and our technology.
- If we do not obtain patent term extension and regulatory exclusivity for any product candidates we may develop, our business may be harmed.
- We may be subject to claims challenging the inventorship or ownership of our patent and other intellectual property rights.
- If our trademarks and trade names are not adequately protected, then we may not be able to build name recognition in our markets of interest and our business may be adversely affected.
- Intellectual property rights do not necessarily address all potential threats.
- Our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor or other third party will discover our trade secrets or that our trade secrets will be misappropriated or disclosed.
- If we or any contract manufacturers and suppliers we engage fail to comply with environmental, health, and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
- Regulatory requirements governing genetic medicines, and in particular any novel genetic medicines we may develop, have changed frequently and may continue to change in the future.
- Even if we complete the necessary preclinical studies and clinical trials, the marketing approval process is expensive, time-consuming and uncertain and may prevent us from obtaining approvals for the commercialization of any product candidates we may develop. If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals, we will not be able to commercialize, or will be delayed in commercializing, product candidates we may develop, and our ability to generate revenue will be materially impaired.
- Negative public opinion of genetic medicine and increased regulatory scrutiny of genetic medicines and genetic research may adversely impact public perception of our future product candidates.
- Failure to obtain marketing approval in foreign jurisdictions would prevent any product candidates we may develop from being marketed in such jurisdictions, which, in turn, would materially impair our ability to generate revenue.
- We may seek certain designations for our product candidates, including Fast Track, Breakthrough Therapy, RMAT and Priority Review designations in the United States, and PRIME Designation in the European Union, but we might not receive such designations, and even if we do, such designations may not lead to a faster development or regulatory review or approval process.
- We may not be able to obtain orphan drug exclusivity for any product candidates we may develop, and even if we do, that exclusivity may not prevent the FDA or the EMA from approving other competing products.
- Even if we complete the necessary preclinical studies and clinical trials, we cannot predict when or if we will obtain regulatory approval to commercialize a product candidate we may develop or the approval may be for a more narrow indication than we expect.
- Even if we, or any collaborators we may have, obtain marketing approvals for any product candidates we may develop, the terms of approvals and ongoing regulation of our products could require the substantial expenditure of resources and may limit how we, or they, manufacture and market our products, which could materially impair our ability to generate revenue.
- Our relationships with healthcare providers, physicians and third-party payers will be subject to applicable anti-kickback, fraud and abuse, and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm and diminished profits and future earnings.
- Recently enacted and future legislation may increase the difficulty and cost for us and any future collaborators to obtain marketing approval of and commercialize our product candidates and affect the prices we, or they, may obtain.
- The prices of prescription pharmaceuticals in the United States and foreign jurisdictions are subject to considerable legislative and executive actions and could impact the prices we obtain for our products, if and when licensed.
- Our employees, principal investigators, consultants and commercial partners may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading.
- Laws and regulations governing any international operations we may have in the future may preclude us from developing, manufacturing and selling certain product candidates outside of the United States and require us to develop and implement costly compliance programs.
- We are subject to stringent privacy laws, information security laws, regulations, policies and contractual obligations related to data privacy and security and changes in such laws, regulations, policies, contractual obligations and failure to comply with such requirements could subject us to significant fines and penalties, which may have a material adverse effect on our business, financial condition or results of operations.
- Our future success depends on our ability to attract, retain and motivate qualified personnel.
- We expect to expand our development and regulatory capabilities and potentially implement sales, marketing and distribution capabilities, and as a result, we may encounter difficulties in achieving and managing our future growth, which could disrupt our operations.
- Our current collaboration with Moderna and future acquisitions or strategic alliances could disrupt our business and harm our financial condition and results of operations.
- Our internal information technology systems, or those of our third-party vendors, collaborators or other contractors or consultants, may fail or suffer security breaches, loss or leakage of data and other disruptions, which could result in a material disruption of our product development programs, compromise sensitive information related to our business or prevent us from accessing critical information, potentially exposing us to liability or otherwise adversely affecting our business.
- Our operations or those of the third parties upon whom we depend might be affected by the occurrence of a natural disaster, pandemic or other catastrophic event.
- We do not know whether a market will continue to develop or be sustained for our common stock, and, as a result, it may be difficult for our stockholders to sell their shares of our common stock.
- The price of our common stock is volatile and fluctuates substantially, which could result in substantial losses for purchasers of our common stock.
- If securities analysts cease publishing research or reports or publish misleading, inaccurate or unfavorable research about our business or if they publish negative evaluations of our stock, the price and trading volume of our stock could decline.
- Unfavorable global economic conditions could adversely affect our business, financial condition, stock price and results of operations.
- Our executive officers and directors and their affiliates, if they choose to act together, will continue to have the ability to influence matters submitted to stockholders for approval.
- We have broad discretion in the use of cash, cash equivalents and marketable securities and may not use them effectively.
- Because we do not anticipate paying any cash dividends on our capital stock in the foreseeable future, capital appreciation, if any, will be the sole source of gain for our stockholders.
- The issuance of additional shares of our common stock or the sale of shares of our common stock by our stockholders could dilute our stockholders’ ownership interest in the company and could significantly reduce the market price of our common stock.
- If we fail to maintain an effective system of internal control over financial reporting, we may not be able to accurately report our financial results or prevent fraud. As a result, stockholders could lose confidence in our financial and other public reporting, which would harm our business and the trading price of our common stock.
- Our disclosure controls and procedures may not prevent or detect all errors or acts of fraud.
- Changes in tax laws or in their implementation or interpretation may adversely affect our business and financial condition.
- Provisions in our corporate charter documents and under Delaware law could make an acquisition of our company, which may be beneficial to our stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current directors and members of management.
- Our restated certificate of incorporation designates the state courts in the State of Delaware as the sole and exclusive forum for certain types of actions and proceedings that may be initiated by our stockholders, which could discourage lawsuits against the company and our directors, officers and employees.
Management Discussion
- During the year ended December 31, 2023, we recognized $5.9 million in collaboration revenue under the Collaboration Agreement. We did not recognize any collaboration revenue during the year ended December 31, 2022. For additional information on our collaboration with Moderna and the accounting thereunder, refer to Note 4, Collaboration and License Agreements.
- Research and development expenses were $93.6 million for the year ended December 31, 2023, compared to $96.7 million for the year ended December 31, 2022. The decrease in facilities-related costs of $8.7 million was primarily driven by our decision to transition from building out the Seyon Facility to utilizing an external cleanroom facility. These decreases were offset by an increase in preclinical and manufacturing costs of $5.6 million, driven primarily by activities to support advancements in our iqDNA platform. The increase in personnel-related costs of $3.7 million was primarily driven by restructuring costs recognized in connection with the RIF announced in November 2023.
- General and administrative expenses were $50.9 million for the year ended December 31, 2023, compared to $44.5 million for the year ended December 31, 2022. The increase in facilities-related costs of $2.6 million was primarily driven by rent expense related to the Seyon Facility after our decision to transition from building out the Seyon Facility to utilizing an external cleanroom facility. The increases in personnel-related costs and stock-based compensation costs of $2.0 million and $0.8 million, respectively, were primarily driven by restructuring costs recognized in connection with the RIF announced in November 2023.