Loading...
Docoh

Wave Life Sciences (WVE)

Participants
Kate Rausch Head of Investor Relations
Paul Bolno President & Chief Executive Officer
Chandra Vargeese Chief Technology Officer
Mike Panzara Chief Medical Officer, Head of Therapeutics Discovery & Development
Kyle Moran Chief Financial Officer
Salim Syed Mizuho
Mehdi Goudarzi Truist
Luca Issi RBC Capital
Call transcript
Due to licensing restrictions, you must log in to view earnings call transcripts.
Operator

Good morning and welcome to the Wave Life Sciences Third Quarter 2021 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions] As a reminder, this call is being recorded and webcast. I will now turn the call over to Kate Rausch, Head of Investor Relations at Wave Life Sciences. ahead. go Please,

Kate Rausch

Good today Marci. review thank for you third Thank discuss morning our us results. to XXXX and recent joining quarter you, operating and business Wave's progress

prepared quarter Chief me Therapeutics Development; accompany remarks financial and Joining Discovery news update. Executive Chandra Mike and Officer, Dr. Financial and in Chief a Officer; room we will our third following call. of Officer, a and Panzara, the detailing morning website today Chief be Chief Section and release are Bolno, business the Medical results our This Officer. Wave's a issued provide in This slide Paul President this available release news Technology Investors Dr. www.wavelifesciences.com Moran, presentation Vargeese, the to Kyle for Head webcast of

we would Before from our These or including number in issued XXth, XXXX. Paul. quarterly We XX-K XXst, call during could forward-looking could today uncertainties XXXX you any the over Paul? press this ended turn now the December in report forward-looking statement that are for these those are revise that call the quarter remind like update our forward-looking reason. our cause of I'd subject that to discussions The to and on year undertake ended annual release statements. filings, differ obligation and report Form discussed our will the for on SEC statements. to to materially to for December Form results any to a XX-Q no differ to results include and in statements I conference actual factors described like risks actual begin cause

Paul Bolno

it you for our with an how are with our discuss building turn pipeline Today, morning RNA to Good will programs after which will of a then joining therapeutic AIMers. start us. update through Chandra we Kate. on financials. walk Kyle Mike editing will to provide Thanks, therapeutics and and opening remarks I thank

therapeutic mature to multiple Most an restore us and treatments the Turning to alpha-X meetings. milestones and to XX, goal update includes RNA antitrypsin important the XXXX the therapeutic our posters and our our recently, pipeline, shared bringing achieved at or program, these which generated diseases. parallel, progress formally This advancing TIDES battling Webcast most during introduced life-changing we held share well protein presentations Research AATD in to-date. AAT our on AIMers and we we and quarter, Analyst closer annual at devastating delivering third above in Investor we RNA several September dataset for our threshold. people data editing and vivo for made of deficiency, the of use editing AIMers annual functional In oligonucleotide OTS

million approximately an we equity from participation program, and received cash promising the investors. in of indications. our announced our $XX momentum in accelerate the putting pipeline, sheets CNS October, month, last Following in block received with new through terms amendment the we and of ordinary under hepatic shares proceeds with with aggregate to raised with both strengthened Takeda from AIMer updates, existing us based balance position interest Coupled leading from these exciting sale our million $XX our ATM on a approximately emerging collaboration of

ALS WVE-XXX of CMD. innovative, evaluating time proof-of-concept. Exon quarter, dosing WVE-XXX our Each pre-clinical in in and continue initiated to SELECT-HD FTD, in focusing We HD, a and to amenable trial evaluating therapeutic and evaluating on in clinical XX to trials designed these advancing adaptive on execute pipeline, WVE-NXXX accelerate third trials clinical clinical in the

and on trials well to programs, XXXX next help portfolio as our generated decision each enable through investments. being for as define We to of future expect clinical steps these making data in these platform

silencing include and programs RNA and modalities Our and splicing clinical and CNS, editing and muscle ongoing liver. clinical emerging

we As within using value, targets tissue us enable through these these further types, unlock will advance programs additional data these to clinical three continue to modalities.

editing As substantial you of our can overtime. see, portion on to ADAR to we an therapeutics. has believe a opportunity innovative first the RNA in of therapeutic a deliver represent setting potential, the hand right the portfolio novel side editing AIMer slide, is up class modality,

protein Our AIMers restore just disrupting that Rett function, initial or can modifications interaction, and mutations modifying protein-protein to to focus loss post-translational examples. and some for also function name of for correct protein expression disorders treatment to is be on driver of syndrome. Haploinsufficient AATD functions using universe been protein used correct modulate of diseases, including

can Webcast, clinical data, to believe proof high-unmet programs, FTD, that also in clinical led we our This unlock the of clinical program using interactions portfolio additional recent AATD shared for only modulate vitro portfolio population. keep ongoing need. our and differentiated These progress. a is an is include and and all HD, large platform our diversified therapeutic with system. DMD. assessments applications that of diseases which principle biomarker We've patient represent value potential will ALS We enable registration XXX our robust AIMer of to by During NXXX protein-protein candidates AIMer exemplifying breadth trials reflect deliberately on with data, & pipeline XXX which address how paths the serves designed one to

turn WVE-XXX option a reminder, now a As WVE-XXX, discuss to to to Chandra? like to and has call over I'd Vargeese the NXXX. oligonucleotides. XX-XX Takeda Chandra

Chandra Vargeese

of the third Paul. in describe and we changing and Today data editing best AIMer's quarter, the into life we are positioned some medicines. exciting to transform RNA shared how review I'll with meaningful generated Thanks,

design have platform right drive editing concentration. pharmacological more there we including at to by to reached sequence, reach AIMers XX based achieve on are of with proprietary chemistry initial oligonucleotides sufficient these backbone to aim stereo-random of interplay the many improve found control start comparison, key Stereochemistry we approximately platform attributes AIMers and overcome from, intracellular the editing features. and achieve strong have the years our between our have and principles the editing upwards to remove a therapeutics. into have chemistry and to This work challenges our work, XX% the we optimized of broad we and benefits to GalNAc long-term a and the nanomolar are learning in is When of built-on have PRISM we RNA supported They therapeutic deliver tissues therapeutics deep our toolkit to uptake tune we IP because and it to with AIMers. leveraging key maximum the reflect insight is these AIMer achievements engineer lovely as AIMers choose covering make and a exists and investments ADAR the The this when single-standard reality. With we apply stereopure we modifications years combine the Again, AIMers, know-how combination RNAs. are unique stereochemistry. enormous these easy have full active as systematized gaining events also to delivery our platform and and XX% even in our of beyond. alone structures, editing these GalNAc and not AIMers, an dimension each Since match demonstrated CNS investment liver. our of in a designing PRISM is PN and reflects single of the editing specific years models. the stability have of and The the or is that potent stability in every corresponds understanding our present candidate, a with editing of conjugates efficiently AIMers. to of of distribution many properties durability ADAR platform, efficient work into opportunities to of which design range. effective X,XXX we multiple of robust, record in that pre-clinical modifications, chemistry, features. higher reality, Our does This By GalNAc have

as opposed as example, of sequence consideration generated a a navigate sequence our a most between target. to modalities relationship reveal map clear XX. is activity, defined with unique robust achieve show right the These the helps in us space For the the shown we slide the on to other AIMers. for and the this, editing To heat that of data the pattern AIMers sequence

our as of of studies as Our in-vivo stability demonstrate enzymes well ADAR efficient engagement AIMers. the

primates vivo’s doses the liver, but to same Beta-acting time to As these post-last south liver distinct of engage dose shown as as persists bid the of subcutaneously activity. and the out in appropriate AIMers. to To levels we were up mediate XX previously and stably enter durable their compartment the efficient initial GalNAc the This the of middle. have traffic on target in AIMers the three RNA Editing editing, chemically achieve AIMers non-human sub-cellular left. reach tissue days points need shown described, out XX% to to

on shown human When on initiate suggesting first In hepatocytes. targets transcript isolation delivery pre-clinical were is for biopsies though slide as the [ph] is highly transcript ADAR direct AIMers the Under the inside we seek our efficiently our ADAR results, confirmed at the AIMers ADAR to images same for generate we are the of transcripts edit These is launched on ways ADAR editing cells from the ADAR there these distribution have therapeutic confident support shown in broad We results right. NHV when substantially each capacity for three cast in and the comparable, Based us our in observed each we cells EGPX. primary further in sufficient conditions, program ADAR They endogenous the XX, for AIMers different to principles for EEFXAX also of demonstrated of AIMers as of experiment, editing RNA the to in decision transcript, ample that there levels treated editing RNA liver editing, full AATD. time. to GalNAc-conjugated graph, editing evaluated The specific that the with are highlight hepatocytes therapeutic one both question, here editing tap that levels we editing when can we novel edit successful that such a systematized same cell capacity the design or targets. here editing. into. an beta-acting and three same as to program, asked shown in that enough targeted

by GalNAc using post we we robust turn AIMers broad example in for in to persisting how of months driven To of four These with at the Rett underscored editing cause and where AIMers the observed our the exciting cell the We results an similar mutations our advances throughout observed the XXX results portfolio, tissues, webcast EGP which as are AIMers provide the are mice CNS. in neurology RNA AIMer brain ability dose distribution, least of and durability the dose. shared platform. We to GalNAc our to to PRISM tissue editing single a AIMer MECPX, syndrome. retain have research an conjugates the editing same system. during data received microgram culture in the of was such Because

this observed locations For shown transcript our on slide about the found nonsense a brain MECPX glia reduced on a which We full and target, occurring MECPX disease of construct, mutations of transcript. we we correct can leads concentration of development. nucleus of codon. XX% capabilities, for in MECPX to protein at to of in XX. mutation variant MECPX in causing With different transcript, obtained an ADAR editing the editing that system, protein correct neurons stop length specific normal we AIMer current that the believe up aim RNA we other restores vitro required cells, Using is expression dependent premature a to containing

Editing to supports lung editing XX% indications subcutaneous the via those data pipeline and non-human found in previously tissues potential PBMCs. mouse impacting variety or of heart directing therapeutic shared one tissues up liver, a of kidney, kidney, dose, lung cell or dose. preclinical and to We editing intravitreal several after of eyes, such types interest in data immune heart. vivo single AIMers single expansion in in even showing primates Our as including month affecting per dosing, a of in systemic heart accessible

which Mike Towards have work candidate to first AATD capabilities towards XXXX, a our we and on therapeutic gained we build will primed our therapeutic discuss this momentum moment. in as now within we’re editing either program, ADAR

programs. now fuel We exciting in on call our to turn provide Panzara ADAR and pipeline, updates therapeutic the scientific the several data Mike these continue and to to to our will be publications to presentations expect data I over throughout generate shared Mike? we XXXX.

Mike Panzara

Chandra. Thanks

by positioned why treatment as and where caused genetic gene, well the most quarter approach share organizations. coming as Chandra’s was therapeutics Z about protein for mutation AATD. AIMers describing ADaR aggregation programs point productive then hepatocyte development eventually update introduction damage, potential lung we allele. progress start first I with leading and I the AAT discovery for in in will program therapeutics clinic editing, disease. in of antitrypsin and our disorder an in where third our by The we an Falling commonly currently dosing knowns alpha-X to that AATD on liver This our stage functional provide will year. with circulation, a for SERPINAX lack onto and Z-AAT very a of evaluating and believe is has our three are progressive or or misfolding a results as mutation end both, to is the commonly in which in inherited pulmonary injury leads liver success

all RNA goals aspects are therapeutic of of there gain loss both to of address is this disease, function As suited function treatment. and editing uniquely

with restoration to to the address mutations, the SAX-X and aim the only disease. the Z-AAT protein type mRNA experiments homozygous PiZZ physiological of number our and While with to administered labelled of retaining a development, we M-AAT. -AAT days. risk circulating aggregation that In multiple genotype IV functional to antitrypsin lungs, approximately AAT evaluated replacing conjugated lung for of chronic we alternative reduce liver, the is With in gamma pure of a all that US correct could to patients are course EU protect M The we and protein AIMers, anticipate the therapy regulation optimization treatment. assess people subset XX liver augmentation these in details. editing the or a benefit SERPINAX the With AIMers, while of prior there unique from protein protein restore wild a the alpha-X are approaches each and in-vivo therapy initial highest XXX,XXX of over subcutaneously sizable, in such with stereo

doses, risk PiMZ AIMer Further, of XX approached targeted in encouraged threefold Following control, AAT therapeutic the these of genotype, the achieved over increase. the symptomatic subcutaneous for our levels than by disease. having threshold RNA The a subtype increases in to evaluate PBS results carrying a were PBS out control circulating specificity or as as AAT resulted lasting days. in compared augmentation meaningful initial RNAC. therapy lower and three the equal therapeutically performed To to in we patients by editing were greater three-fold they a known or SAX- increase GalNAc we protein a to

this protein to there AIMers had the than type wild for its bystander also that approximately we or where optimize shared low residues, SERPINAX well Furthermore, AIMer therapeutic across as lower off-target seen PRISM this mutation a threshold, of at editing with the target on transcript potential transcriptome. analyses, search Sites across nominal of in use we each for restoration, shown AAT you along no for coverage four a these using On treated we truly editing events. the credit increase samples. can edited transcript. the to T indicating both the and specific sequence that calling the off-target XX% Recently, the assess confirms M-AAT approach left, sites. drive occurred see is protein we sequence transcript. unedited our Thus Thus total in AATD to reads ability detected the chemistry AIMer of efficiencies been is the here the being total off-target protein. indicated entire C percentage The in XX% targeting the the a that coverage to in SAX-X. editing intended DNA has fold is find only SERPINAX target site SERPINAX in read is above analysis are the either whole apply for to editing group. with Editing From editing at the high produced transcriptome, editing software of This are of percentage approaches. percentage analysis, less rare observed as

Z-AAT ability protein candidate, We we continue in of well a to move which as as pathology to tolerability liver, towards reduce identifying evaluate and PK/PD the durability, profile, in inspected candidates, XXXX. is potential aggregates the development and as

dosed in our CXorfXX, clinical ALS include patients clinical third patients the our FTD. trial for programs, trials. to evaluating associated three with we've ongoing clinical FOCUS-CX quarter, WVE-XXX initial in Turning These and

for along clinical CNS distinct with with from our patients clinical The CAG upon approach and relationships expansion, Then, with chemistry stereochemistry, in to DMD experiences from ability with engagement illustrated and Backbone their WVE-XXX promise pharmacology modifications skipping. HD to of in at tissue target for with preclinical The in now our and It with WVE-NXXX predict taken be the to approach PN context emerging to All of of that control capabilities distribution PRISM three PRISM contained design label in believe patients can open select association begins availability showing our innovations tolerability molecules, the SNPX to an with own trial, fields. is Our of HD PK/PD the our evaluating the the candidates for others systems three evaluating clinical human design built with amenable elements the we with understanding better columns genotype of leveraging favorable candidates rationally are mutations the and candidates, platform profile. vivo optimizing success modification. an the dosing. of XX we Exon for trial, CNS in to the that candidates to a these understand proprietary key by core, potential of influencing along the widespread factors portfolio. its increased

reduce risk the population that of dose biomarkers, making. adaptive well designs and position and relevant decision adjustment to of drive of other rapid selection careful study time real Finally, level for patient in allow context endpoints us in frequency,

and with a ASO preclinical PN undisclosed like target. Here, targeting modifications of highlighting practice, an example work through backbone stereopure the to an designed walk chemistry ongoing I these principles would CNS with

single of developed tissues stereochemistry engagement PN the isomers optimization during is of the compared two intravitreal the advantage ASO experiments, part XX throughout mice in other different slide. one in but we Isomer terms containing the these process, sequences, Slide clear assess CNS In a target modifications. PN with of these As the shown several one What compounds modifications. dose. illustrates this the in of with stereopure distribution designs. impact and month isomers previous as versus shows of the we is process, and engagement terms on screening differing with X PN without all tolerability isomer target identical the the without of with the after Backbone

hand left easily the including On engagement target that was isomer all X. three side the see you can of slide, isomers, demonstrated robust with

the tolerability However, hand on modifications, backbone data significant compounds, being the These two. profile, these despite different weight promise one X an sequence stereochemistry must the of is right to sequence, effort, body optimization other discovery observation and over drug of medicines as period, the fully any clearly slide, be and realized. be having chemistry can genetic see of loss demonstrate the dramatic side three as the you of isomer the same development essential of the component important if with

clinical allow we understanding demonstrating -- is the current pharmacologically in dose to core As in models engagement guiding our development. path humans, data us core our our trials. likely our to clinic, and to relevant the target think selection initial dose of about to active model These programs preclinical

relevant and human target robust effects levels start way. collected through and the at to data studies So, proceed considering XXX engage dose these us models selection the along the process and predicted to have data and allowing WVE-XXX dose

and ICV time wild corresponding spinal administer persisted target concentrations and the a to it The effect. immune slide CXorfXX of under vitro compounds target containing mutant to multiple lower profile resulted on contains and including least mutant neurons to vivo vitro reduction HTT on this clear in months PK and dose highlighting doses used wild-type. have both preservation To Further demonstrate bottom with top demonstrating mutant and for data at HTT in specificity shown of is The in data is apart copies in HD in that, protein XX, of effects slide the This stereopure in need in XXX some type and effects clinics. in limited HTT weeks preserve promising a somewhat the of reduction in makes a selectively XXX, back protein, cortex. activity other X knockdown Huntington IPSC is healthy effective with for this XXX shown HTT or cord days the we shown snip of do our potent to there out knowledge, in similar data With tissue mutant have over the HD, XX in First mutant period, Nonetheless, where high the system. important profound for sustain regulation, PD potential seven XXX compounds. XXX cortex. function specific striatum These ability of at of targeting in us for treatments. six gene, XX, remains normal the which for two unique variants. the to durable the those the we the Huntingtin observed were excited a effect leaving CX highly designed about not development, normal and the the amongst unmet the which with including model unaffected, poly-GP

our clinical administered WVE-NXXX, to first candidate systemically. modify to be on Moving PN

it's -- enhance to first of PN and muscle splicing expression. provide the access restore will first it candidate modifications and ability our also, into dystrophic As functional dystrophin also the to insight

with in about advancing ongoing To include on datasets. promising evaluate of trial containing are and only our time committee PN program rapidly production accelerate progressive of to parts with indicates oligonucleotide compelling -- on We oligonucleotides concepts. keep including and clinical biomarkers diaphragm. proof trial preclinical dystrophin preclinical designs PO this multiple exon and the In this, and do potentially systemically translation focus to to advancing innovative that we independent closing, are these current modified to an of is skeletal modifications. increase reviews this using lead modified tissues The muscle administered skipping oligonucleotide rescue given optimistic PS PN phenotype comparing data

will these turn all through year. generate trials to next across now over to CFO. expect Kyle We XXXX decision enable of call to Kyle? our I Moran, the three making data

Kyle Moran

as Thanks Takeda million quarter the which October received the in primarily recognized is third compared in $XX.X recognize of This $XX.X support million XXXX, remaining XXXX. the $X.X previously as agreement, in XXXX amendment third by recognition to million We revenue Takeda. of collaboration Mike. paid driven of as third in as quarter for revenue the quarter, as increase revenue we part the from research well to payments to our the from of related

we block editing in expanded our related million increased program were XXXX, Takeda $XX.X related goals our increased compensation since for investments to $X.X pre-clinical expenses proceeds expenses enable in an ADAR decreased current to ATM. million ADAR our as other with and Our October, equivalents subsequent off $XX.X driven platform, million million, in incremental related does million expenses operating expenses, offset $XXX.X funds close, million, our advance and at in total programs We G&A – million These including for discontinued primarily included compared the third neurology additional cash by as the same million $XX.X in the R&D $XX.X and was were not $XX.X an compared aggregate the million approximately This programs. third the ended will cash, same compared $XX.X expenses. and balance to quarter from to external quarter $XX received by third expenses partially last the as with XXXX increase were to to year, The year. expenses primarily programs, quarter continue compensation in last by time. to third million quarter the G&A $XX.X XXXX. security. marketable period as related driven on

operating will continue us cash requirements the We cash, of enable quarter expenditure our expect fund second our existing that into equivalents to to XXXX. capital

As a milestone Paul. payments, this Paul? undereducated now call potential back collaboration. to not turn over include the does any I'll reminder, or often

Paul Bolno

Kyle. Thanks,

proud our genetic advancing progress medicine. quarter, has made team pipeline our of This of I'm the diverse

and positioned We to our of for urgency deliver shareholders. and are a are of value modalities and resolute well patients working indications sense with across host

a designed deliberately of portfolio candidates have and is that diversified We need. that address with high differentiated un-met diseases

a machinery decision insights making their That period genetic cellular well a families to Looking ability any silence/edit options. have as targets, tremendous platforms will of and endogenous patients harness hope XXXX. to ahead, we're generation treatment entering who data our offer of into enable different as limited, to if in multitude

We are critical AATD for merge development make we that, to call studies, expect drive forward. as well as our we well questions. we'll a to And execute programs as year. candidate milestones. clinical capitalized these up additional therapeutic open forward the providing to And look with continue three announce We updates decisions Operator? next through on our first to

Operator

is Your of with Instructions] [Operator question first line from the Salim Syed Mizuho.

Salim Syed

Great, good the Thanks morning, for question. guys.

So there's can. if I me from a couple

XXXX. I sitting in appreciate Paul, here through So, the language November. around we're Obviously,

X, about the later? guess, you Huntington's or going data maybe Is how is the label, process you are thinking you. for of thinking trial? and and around the to could are un-blinding house take to open out ADAR XXX for those timing the can same for, How business you you therapeutic the to you collaboration? and development partner ADAR And then question there? given areas second disclose end that of hoping the in potentially Takeda you're just the to you collaborations Cohorts I come and with for and the generate that that guess, bit the it's are every question see X And the now the I'm I X, presume? editing CX XXXX. the like, then How data So clarify little street? planning us Thank plan XXXX. you X keep the in that even data plan -- and about potential What to of a more here cadence of in looking amendment editing and they're I

Paul Bolno

And we've got in Panzara. the to said across three year first. began we next data where get But CX designs, readouts dosing predict Obviously, the clinical we comment with start will I'll to underway adaptive it dosing trial Mike the we over yet can't and the as But quarter, occur. think different Thank you, these as into studies. question I hand given last nature first of cadence of the Salim.

independent clear committees I think review monitoring been about we data, those that we've say able what data. being is well to those to binded also ourselves, safety unblinded

said, open And changes any updates move we get let as we this, so disclosure think… I into Mike as detail I’ll publicly, to but we're around he additional material XXXX. designs will into for, the wants to impact, share study various

Mike Panzara

do say, recommendations committee, pharmacology and would including of that's if sharing next. them. what or this data would be mean, these would I No, to about that data would with who disclosed there basically it. we that indicate alter changes that captures there's program the I study these what mean, And then have with to you data, a I design to to I mean, back material the process comes be already biomarker were share know, material we've would

study, other that cohort you what's where engage to in has get each in have important expect designs amazing an particularly starting spend about to expand higher you can enroll more we about that which we to where the patients and think, into quickly. with really unlike application these adaptive CNS, blocks move resources cohorts in order know, there that of flexibility us about so target committee in in the to the both adults comes cohorts historical ability job and I the the get in thinking to done enabled in and of thinking how innovative studies, development to designs, the of has combination different the be that team is trial

is last make definitive that's we have And projections us sure our brought study updates one. next to so, I you the anticipate And run to that that provide same while five to view enable our based on we'll way that three. know, a pieces mean, all that across to we and the in gets the endpoint. up year. And you a

So of itself that to setting of in prescriptive disclosure and to even has study open data appropriate ways to running the unlike open or in label be point able setting an that XXXX. mind, get

update you any of those the the know, that means tends there initiated, be once the think are material of studies. I where So is there dynamic possibilities nature through can study guidance to set nature that

Paul Bolno

to ADAR just… move discussion, the part? business first that which on development equally questions I'll then is Any exciting. And I’ll

Salim Syed

Paul. you. I Thank No, helpful, that's think, Thanks.

Paul Bolno

mean, before, think, for I is robust the business correction. as a development way out I is this we doing vivo new place we second have one think, a And research a interested as area of ADAR shared of the the of in I I most think right. following So you're editing. we absolutely think piece of things compelling biology, webcasts I around data think, of case datasets

it on business deals always development So happen. deliberate they're it deals I'm discussions, attracted the going to that business brought. for to very guide lot in while And think hard development very because we're I expands to a opportunity be is has of us is to robust when say inclined it doing discussions.

us in AIMer we about robust we to to this precedented opportunity that are think excited in ADAR path case And know for there's with I expand the think application it's a GalNAc-conjugated bringing portfolio deliver, where, administer starting in a our GalNAc-conjugated you going to space. subcutaneous to the know, we that well there's liver.

a of eye, in in across the cells into I kidney, bringing business point, other whole have and we've we've flexibility discussion. back development on, we your portfolio human shared therapeutic think now shown of the brain, the that data indications as to adds in that there's host that

large other about So we indications, that CNS think includes and applications.

genetic you be discussion, ADAR the the planning. businesses, are know, post part But a discussions definitely think future I medicine. So our and highlighted domain of broad will around

into we So excited are back it. about bringing

Salim Syed

Got so much. it. Thanks

Operator

Lee is Truist The Joon next question with from Securities.

Mehdi Goudarzi

for Goudarzi on is Hi, good Mehdi This morning. Joon.

of couple a questions. have We

first a data. question to say, way your your color then preclinical evolve production competitiveness and that compared provide is of So please have the with some a costs comes and production Could it And random when ASOs? platform on I to follow-up… great scaling long came nicely you your

Paul Bolno

Yes, no, And whether you [indiscernible], five one that's, in administer it's successes it's it's to be administered. successes, to in easy beside take programs. a know, pride great or four four some see think, on hadn't harder question. I And we top point great well, of technically in history. [indiscernible] sometimes a going back I to one think, able in were And those that successes to the other in that been plus way – or five our systemically of

scaling fully of successful stereopure other the oligonucleotide. modified was systemic real of One a production applications

cost at And goods of a so commercial random for with we be stereo would of molecule. were scalability a [indiscernible] that poised par on

that for scale the experience, time manufacturing that And ADAR smaller so partly capability our and so capacity and apply silencing on actually about again, equal oligonucleotides through across to think, interests as we one.

GMP So scale have that take we we also our transfer I several the to, we that robustness larger facility, can importantly to manufacturer internal that think years and scale. it of a been show ago and the built, was process, have as commercial that experienced we we able can to manufacturing think to about we GMP

So, we manufacturing with certain. are is oligonucleotides now pure on comfortable that through par

Mehdi Goudarzi

question be next bit Awesome. looking And my forward. a would

comes formulation vehicles. ASOs it compatible formulation to Your specificity, well? of be cell But LMP if would new or type chemistry a this as any need do with not PRISM

Paul Bolno

be to And was date, that delivery within the types yes. say the those where short And short and really exploration our focused is, answer driven the we I because accessibility. cell on are

the shared distribution across earlier sites. central and nervous cell administration after have the in As HP, system broad major dose single we in presentation Mike intrathecal

As GalNAc all Chandra without, across vivo both brought have shared, ADAR distribution with in with we and types. do

can I So, that's we where be interest. strategies may dose you case and this where to think cell type about of specific think think a I you a the delivery of GalNAc, give targeted is smaller of know, single

think interest. right I and distributing and lipid intended one of not active But areas it of of space, are those been cell for as moving the think and exciting the to GalNAc with for I viral nanoparticle. areas just that we particularly really think and to oligonucleotides into are has -- for requirement editing into but the as that always but without deliveries, without the relates us uptake, of exerting areas the are compartment vectors is effect, the our always cell

Mehdi Goudarzi

any much. any just programs. of may tiny criteria very Is question. for opting another If status you make for I the out with there Thank

Paul Bolno

program. opt and criteria outlined ALS-FTD, earlier that three for set in is around XXX I the that built are HD or a effective there of So the XXX three program that

So so that's of prescribed in delivering but hence triggered us XXXX have as mechanism. our level those is all payments, They think clinical proof for to profits, them. executing demonstration also gets associated with addition important and in program of NG it about and But was all on and are events. opt it XX:XX They we have data. apps milestones the on

Mehdi Goudarzi

you the for Thank question. taking very much

Paul Bolno

Thank you.

Operator

Next question Matteis from Stifel. is Paul with

Unidentified Analyst

a this question AATD Katie program. on for just had Hi, Paul. I is on quick

candidate announcing development So year. your next you're I know

this getting the into Thanks. entering I clinic. guess is that, Beyond what program

Paul Bolno

[indiscernible] report.

So you I relates AATD, a clinic as step mean, to to as it first is candidate. the pointed out, obviously the

we'll into be so that the are it's And like you that and this earlier, said providing of features going program, around as close. more as guidance XXXX,

feel we You confident on know, potency. really

saw candidates, also the we early that how call to we And want terminology think see I has as said, constructs. Mike We have always company when durability every is. think long dosing candidates. with different we I around frequency

I candidates. tolerability build think we early our in

Now can that know tend the announced go that clinic, to have we the program we you we that we distance. to a when that bring

AATD. the be about up to give clinical that announced translation quickly accelerate AATD first working best robust think criteria and working into team do excited potential will that, to program us bring hard well that credibly we the class, forward our is set I are think that So the potential guidance, that. to we're mean, aid to that in I what of But further go will it

we and on conjugation ability -- As are we with about relates being Because down it of that other to the again issues. both that working other coming think poised behind aimers because

to I sequence to will more get we think portfolio. that be the about XXXX the into growing how provide AIMer of of as updates think kind

But builds to about forward As around ADAR [indiscernible] we excited share unconjugated as it founder and our well. editing we do shared, programs expect more as we're GalNAc. bring Hepatic and

I know, those updates XXXX. more as think be we think about you would So,

Unidentified Analyst

Great, thanks.

Operator

next Your RBC as question with Luca Capital. online Issi from is

Luca Issi

the Oh, so ones. great. taking much on question. two Thanks quick my Congrats for progress as

So one if Biogen They're first maybe saw like they're a SODX. takeaways ALS how maybe few that program pipeline. a obviously data second, key the a lessons Thanks discontinue the different Ionis you some missing use approach endpoint. of the going any there's But from wonder given collaboration program you going back weeks after obviously to of learned that and on that is very, and set, to wondering very primary expand from why planning Kate and your And so we to your then much. earlier the more on decided had can you're to maybe bit forward. key

Paul Bolno

the the I'll I'll And second. take to first let question. Mike back Thank you. then come

Mike Panzara

thanks that. Yeah, for Sure.

different you this that very drug main captured, think of all different I points very very is So first and the clearly, targets.

different just one another, totally one place, a targeting mutation starting in different. for versus totally So indication like, oncological

show engage I terms I first in thinking we that what think can show mean, of did we -- ASO that of learn? they I did least all, I can mean, think can they that, targets. what I at didn't they get patient was of I design really modest there helpful ahead. Also, engage target. in elements population the did did to are study are a of but the sort It think target. they and effect, that

design predicted study think modest their saw some I in study the leading effect of some -- they that outcomes. earlier could issues some given there these maybe have were

for optimization optimization goes that the be is that to have to that CX we've basically And to target what think that with you best to you tolerability. then, we the in it what able So enable candidates And for and presentation, need our I done need distribution, design your earlier engagement. said to that's program. to target engagement with studies

mentioned, as the clinic. the positioned taking I So and seen really way the given preclinical we're into we're we've well, data it

what that. to turn I about say it over And that's would Paul… I'll So

Paul Bolno

which robust characterize demonstrated studies get do to potency to shared CX which just biology importantly, a life durability, preclinical effectively, as Mike, by first. our echo And way there. we think And believe really Yeah. into as running to throws efficiently in it we're target I that

-- where is about there's our position. think I we're program excited So,

collaboration As it And I pretty that’s expensive. have the relationship think to relates it's that it's to we a Takeda. important with Takeda, remind still mean, ongoing everyone I

sometimes think collaboration, I the partners. feel they people Takeda are amended and like that still

that advancing. partners are a we on third is So and program clinical three, two programs

collaboration ourselves underway. there is remind a So critical think is that to I it

discussions, useful a of Anything it. amend there's to decision the lot activities. why think, I

a we what desire think to doing. to accelerate, continue I have we're

speak to around drivers have desire And We the budgets field and they have it are. in a CNS. indicated can't some where our of to I

decide has franchise. programs strong move with excited is the key partners. And and CNS the to to are to think XXXX strong our I into together. that them evaluate Takeda how political we're forward we move

are So, Got forward. still think agreement Thanks futilely, so very done we the we much. it. simplify we've can are is and we I that partners, streamline so much what

Operator

[Indiscernible]. Last the from is with Manny line Ferrer of question

Unidentified Analyst

a questions. taking go for and these previous, and are guess tough Thanks for to would a dataset? you continue that HD HD HD better becomes not to appropriate or see on which target that investor at many ventures throw And to point this for our use that How consumer that do this looks others of to there's capital? time guys. think I oligo you into snips. DMD wind we therapy, Like, not of after. be easy next update proven proper obviously results you've take, targets should ones moved continue totally your have apple money it from in the of pursuit, bytes you philosophical down Hey, that we particular from one. in a targets do sort of more Obviously, the HD different throwing

Paul Bolno

Yeah.

one, So money. we I throw don't think

and money, I in think data invest invest we a way. driven we think money I

this durability these. vivo others and data and the model. reason HD moving in that one, only with that relevant distinguishing we're support meetings to have against think backbone your the now subsequent is with presented PN we allele chemistry into data appropriate preclinical data specificity point, We clinic, potency an So demonstrates program demonstrating and vivo within in to I our and running the

and going think HD conclusion have and I don't an in support. approach for believe its to then to that we're experiment data as frankly, we do decision. run the Do way, a we think driven that or get data So data benefit, I driven a that we

it we FTD. high as the as base for ALS needs for CX and CX, to a medical and again we preclinically, a Secondly, design therapy. requires out robust set preclinical and Again, durability, as relates of most unmet there our have we look data that believe home do area program second

to not was we we're able in that unprecedent, mouse And change be down see they double dystrophin in in study. knockout a to mouse believe then program that's phenotype, production, best-in-class we the have survival that like run NDA, demonstrate, to a in actually there. And we going going we're So do the in just run thirdly, that an DMD, substantial. to data

And get test the so we're to going again, to data. going that we're

to is progress you're what of very make say, decisions, are data And aligned very as GalNAc we're a that a year deliberate way, do ahead and I those to is think nuance silencing ADAR area And really GalNAc-conjugated peers targeting the I and gives three that decision program new conjugation think our the on biology with little data saying, driven next decision so us or progress in gives new a say, we can I then it's I us what move a steam And will ADAR. advancing in we to decisions us area. answering bit us moving say. and it molecule. decisions, these to think are around think much to a It gives full very we different which a really discussions need a look, different way a investment to so fourth important, took gives

questions to and [indiscernible] pre-clinical periphery think spectrum, in by across of decisions the portfolio buy well make able that And regions system editing And So I work in that's across muscle. next that on answer meaningful I skeletal investment as in harder over central ADAR going think the we're the nervous to and going XXXX. AATD. important both with of to as program answering coupled be question, your year

XXXX. capitalized that about now excited do being So I'm to

to answers I think we're platforms. going to get pharmacology really important programs and

Unidentified Analyst

that's Thanks, guys. clear. Great, crystal

Operator

call the over Dr. Bolno. to further Paul at no are There time. turn this questions I'll back

Paul Bolno

Wave day. bye-bye. great for employees thank joining updates. Have to hard review our XXXX a the everyone and our this for And their to commitment corporate patients. third to work morning quarter you call Thanks, Take care

Operator

conference This call. concludes today's

disconnect. may You