Good today's and joining Kate. all us you call. for Thanks, morning, thank on
Chandra Today, then open we'll up I'll the turn also during to Q&A. be share and the our highlights questions. available on and financials. Then will the and on first the Anne-Marie Kyle quarter are our line progress for for review call today to call
transformational leadership strategic year. formal including in mid-December publicly Wave quarter we leveraging a beginning collaboration our at announced GSK, the fundamental last first in how chemistry, in with of marked The of our change oligonucleotide are
our We seeing optimizing years with Gratifyingly, in and of and to understood oligonucleotide our chemistry are we positive evolution. December RNAi-mediated in reflect mechanisms recent antisense clinical evolution, biological well this company take of and ago, chemistry. RNA work. DMD them on focused on at we XX efforts silencing exon base our them Let's strong started skipping. then see we in We relatively return, the as and clinical I silencing believe beyond. last a leveraged, a we seeing liver point than and More data are moment fruits our our in in will which announced preclinical finally durable these to recent preclinical directed data
editing, use years, target engaging to advances us recent about high-value, emerging our and chemistry aggressively think as insights franchises. Over in biology chemistry with have novel combined validated to therapeutic can genetic where more we enzymes for unlock such genomic ADAR enabled first-in-class
with can improved chemistry, combined More Marrying we're interested pharmacology novel when to into rooted first-in-class the biology in in with success validation development. the leaders. human validated medicines make genetics sorts that of probability of grow opportunities biological best-in-class targets opens with have in class validated
pharmacology Our on pharmacologic RNA uniquely the disease a and have This in demonstration silencing, best-in-class It and capitalize in we decade positioned space. a we targets optimal molecular genetically can multiple at now select toolkit believe the most the broad a validated rare driven for we multi-modal work editing, job best-in-class exon RNA-directed skipping a opportunity. a tool means this company modalities, attractive allows target RNA us and us is both for across to validated of Wave to associated have across has and to access platform of be best-in-class potentially as we medicine. way in to describe RNAi also but
that life-changing such build deficiency will patients shareholders. multi-modal value Wave pioneer leveraging major protein or as antitrypsin medicines and Today, medicine restoration a for on and with RNA AATD, is platform provide strategic first-in-class targets, to our focus repair alpha-X for
and expansion move are translating beyond quickly through we new protein year event pipeline capabilities to how an into in highlight to the programs. and compelling our hold unique enable in this quarter intend Our to third to value of derisking. capabilities us We AATD investor restoration build repair
ago, aspects which gathered I'd substantial of Since progress have six three we highlight of several business, in weeks our made like today. we last to
First, collaboration achieved could with beyond. in XXXX our and GSK that significant milestones be
execution into that RNA the a continued steadfast support on Second, set data for potential and would DMD, advancing for accelerated And to next in driving alpha-X third, wholly deficiency commercial the WVE-XXX opportunity antitrypsin progress a clinic programs. generating study WVE-NXXX on Wave of approval. rapidly owned editing
collaboration is to off strong transformational GSK our with First, a start.
first-in-class discovery leadership and our multi-modal As ADAR a in development editing our reminder, validates also only editing it our RNA platform. partnership modality, with best-in-class acknowledges medicine this not RNA
collaboration, the with collaboration to working partnered insights It and productive been pipeline, expand with genetic the start Wave the both provides together an to program. Wave Through exciting our targets. GSK Wave to of wholly-owned and with and first advancing has teams set proprietary the GSK both begin
GSK that capability makes has in investments genetic clear as these program. development made discovery, long sets and has well as It's respiratory history leadership current the for ideal in partner commercialization. AATD a important pioneering remind of Both significant everyone GSK medicine to and Wave's
milestone balance beyond. deal our meaningful million to their development the milestones add and sheet WVE-XXX. our potential potential payments, already to payment quantity to XXXX the confidential opportunities, balance GSK anticipated with accelerate in bolstered trigger to in milestones milestone existing strong and our in in pipeline provide $XXX near-term, equity and and events sheet These meaningful related have substantially including while the upfront including Importantly, cash near-term clinical
continue we our Next, make steady progress advancing towards WVE-XXX to first-in-human trials.
towards Since successfully candidate the toxicology studies have of portion and GLP first-in-class our we advancing in-life reminder, completed XXX, as GalNAc-conjugated planned for we CTA are our rapidly a editing As XXX AATD. RNA this year. for update, last submissions is
which healthy unmet for or continue AATD hepatic treating pulmonary need the treatment a the both reversible our therapeutic. to disease. the of we in first-in-class either manifestations with redoseable therapy There current is major confined pulmonary a with future AATD, largely a and Among for designed is in to field, generate novel approach of restoration hepatic to function excitement and therapies
to alone Despite revenue the substantial billion is limitations accounting current augmentation this worldwide, about XXX represents to and with pharmaceutical currently therapy for market AATD annual $X.X of expected market therapy, a in to grow. pharmaceutical on validated the track intend to proof first editing. utilize of concept of into deliver biomarkers we clinical therapeutics, XXX RNA-editing the where human taken RNA be trials, is field for and
XXX and RNAi liver, of the silencing Similar data to RNA medicine, the success increase early of probability liver in sRNA of would clinical editing Wave's we growth beyond. the exponential of derisking GalNAc in with the following future believe program
is the early us shareholder to pioneers substantial enable would build way RNA medicine. value of this believe We to that in a comparable
our program. to we in focused to pipeline, repair continue wholly-owned protein our as on AATD designed editing we As therapeutics, expand such restore investing are first-in-class with RNA and
year, team and third event demonstrate discovery new RNA this the leadership quarter on Wave of an we to in is our how which are pipeline. Our our programs. hold we of to share medicines working to also to will expect the next in we We RNA investor intensely editing, during sustain on plan preclinical extend continuing data
skipping our study are data registrational December. WVE-NXXX, positive laser Phase focused II of DMD, In initiating our following in candidate, our XX on exon potentially we
met be Our with proof community. continues to and the concept data DMD neuromuscular excitement of clinician by
dosing, to strategies concentration of downstream as results result exon impressive fully been with skip profile. skipping these to other of biweekly in and levels With high observed consecutive accumulation favorable functional transcript across the substantial the has expect industry protein case dystrophin exon observed date. longer included just safety we reminder, after muscle XX% a skipping doses, three the these a tissue As high
Becker-like distinction key is is surrogate been established exon for generate or accelerated functional of in A such something micro therapy skipping in NXXX as mini as from endpoint approval approaches protein, by DMD, FDA Functional truncated protein dystrophin. dystrophin gene not microdystrophin. intent question the dystrophin for that still to the a has
and More, an continue combination our in options can DMD should as valuable patients of believe option convenient, alternative they and become the with patients a approaches production dystrophin gene for to safe expand, endogenous that available. is attractive functional we that or or be for therapy highly community hope to
every dosing weeks XX initiate XX B treatment. our of team XX assess Part dystrophin in study kilogram and of study, to administered of doses week plans after Our a moving with milligrams other per II Phase quickly is weeks open-label protein to and
study, supportive, we are We share we with B the use progress of data to Part to as expect we share the to will and updates approval. continue intend file to If XXXX. accelerated them in data for
data exon NXXX for extends broad Importantly, we dystrophin build our beyond accelerate a DMD a which planning strategy, would XX, franchise. and wholly-owned positive following vision to we are multi-exon
to and Turning XXX. WVE-XXX
FOCUS-CX clinical adaptive ALS candidate is a and silencing trials, of programs for collaboration FTD with are these clinical CNS associated active Our in are advancing reminder, evaluated being the programs and part Takeda. WV-XXX, our in an CXorfXX trial. as
on the in substantial consisting with of and a next allele-selective and this which with several in cohorts first clinical program. polymorphism the our remain steps discussions for being is first-in-class of inform SELECT-HD patients set single candidate enable evaluated deliver the to We year. Huntington's for a this track half is partner multi-dose WVE-XXX This disease, will data trial. SNPX in
to with they mutant the we Huntington a Recently, SELECT-HD single-dose positive that in attack, were assay address Huntington year, and to Last cybersecurity cohorts the issue. them of to this data. we adapted biomarker wild-type and announced contact vendor study mutant expand our they remain on initial close based as work clinical subject the
we and ensure that fully impacted, vendors before Our operational patient were our computer are will validated systems samples processing. not relevant and
dose in program With to data half enable the some steps deliver our will multi-dose data expect along the these Like we for data single timing, XXX, biomarker clinical to in with now additional partner. second with safety discuss of next XXXX. this shift and with also us
our RNA of best-in-class are collaborations a collaboration to in modalities is one medicines being multiple with leverage a our we RNAi GSK. able in As sense to company, multi-modal potential our balancing. RNAi strategic explore
from the have first ago, our transformational the nucleic weeks few a we since our we announced field and RNAi. have siRNA highly of received acid feedback findings in recognizing the Just design impact research, of for positive these peers, publication
improved demonstrated administration in comparator compared track clinically leading to vivo a loading proven a in unprecedented AgoX RNAi. data as following and with published durability a subcutaneous a of from commercial dose, The single to company potency record
RNAi We and from our including targets believe observation remain leading genetics, concept many the preclinical translatability approaches by continue highly work enabling to there of are Further, targets high-value these established. accessible proof clinical the have GSK. more well from in work in been RNAi-based collaborator, flow tissue and novel attractive data as human of critical
a significant expand the of editing. building in RNA with are multi-modal we company work sum, RNA our In foundational field medicines leading opportunity to
important are DMD. We a people AATD, editing urgency RNA and to new boys with treatment novel living with working for bring medicine with to options
RNA excited positioned also inform sets programs AATD we editing portfolio important of share and our we two and are data year. emerging GSK deliver uniquely silencing year this hard more busy are a the Wave, communities, beyond on development. will ALS/FTD this with the capitalize to to We later believe HD for our rapid to well work enable to is years time work at pipeline on and our a and we CNS It
With to that, the for I a will CFO, now financial over our Moran, Kyle turn call update.
