progress XXXX our momentum readout. programs trials development with ended Thanks, to PRECISION-HD Paul. towards the across data continued as we upcoming We
earlier, programs month. described end SNPX Paul data SNPX sharing set of robust for we by As our the and anticipate the a
candidate disease trial variant-selective and development to in ALS Looking clinical we allele-selective candidate to successfully WVE-XXX, clinical third XXXX, initiate Huntington's FTD. and applications December in submitted our for our silencing WVE-XXX, during
of is mutations WVE-NXXX, a amenable with for Exon candidates our to patients XX for In submission skipping addition, CTA DMD imminent. third
owe challenges progress All made partners has in research in these favorable our incorporate detail and these supporting by achieve led team with our and significant our presented continuously these who these the to ongoing to of chemistry, backbone global patient each in discuss the compounds of programs. novel adapted which the today, profiles vivo I'll We numerous PN studies to the pandemic the franchise. our we've communities, starting programs objectives. more Huntington's in to
purposely of candidates to allele-selective lower designed have while type We Huntington. a built HD portfolio Huntington, preserving wild mutant
nervous function is especially continued developing supporting having well the wild adult essential as it function synaptic central in cells stress role protects out Huntington metabolic evidence with type carries time three misfolding importance synaptic synaptic systemic ensure type the This stress. stress, protein affect we and been physiological part both supplying in important memory, types as type growth Wild grow. learning oxidative Huntington survival. BDNF factor functions these the in for in vesicles. their programs, has for Huntington Wild high system advancing essential of shown the spent and against the effects. excitotoxic, as transcriptome, which a of proper neurons only traffic of prevalent have During the and trafficking neurons and and type essential to Huntington regeneration setting including developing well various to has is proteins for as key Wild plasticity, to as plays to of of brains, in the striatal activity, also
of the CSF and of flow wild maintain critical biological Additionally, for effects living protein of the huntingtin the with in mutant Huntington between cilia, Huntington function formation of type effects of use battle type a positive HD. the We the and the tug homeostasis and like is example to help CNS. CNS which control illustrate wild toxic those war the the to in of ongoing of
is Given out of how we progression, ground reservoir. a the type less think to XX% a that protein disease this particularly protein way wild approximately individual, than gradually reasonable healthy further patients is illustrate type patients start there lose to wild depletion if HD with
wild when such, benefit. As may be be clinical believe required of mutant we driver type to an huntingtin important considering how may achieve preservation lowering efficacy
is to SNPs nucleotide Our the target or polymorphisms on allele. selectivity single approach mutant to specific achieving found allele Huntington
it SNPs, an disease. XX,XXX genetics. SNPX of have wanted expanding we routine we benefit are HD. studies to both such HD achieved potentially case substantial the We SNPX Approximately accurately people SNPX, in our now with potential And with can the approximately highly the multiple and have SNPs partnership US, or three to the for target. we with that there accomplishments Allele-selective clinical manifest to people up read to subject of living HD to or approximately three patients of these patients carry XXX,XXX we and to therapy, allele-selective on based patient these fairly novel one that may that given with treatments. specific. with that at of to identify which sequencing when least SNP portfolio Huntington's before is pre one combined. is through to are focused rapidly with surgeon, HD with our of XX% be risk a address identification technologies, an the we In ability population. on disease, therapies HD this SNP. People overall able patients the In candidates, though Some represent And targeting the innovate available of XX% commercialization. carrying eligible In SNPX estimate carries of the multiple up symptomatic developing are population, to our publications. a and long a to we of are in people in harbor at any and with Today, the SNP were the in manifest estimated portion allele-selective the XXXX, population support XX% on groups be SNPX, patients order include family setting of needed the manifest approximately to of their may identify believe onset tools rapidly XX% advance
existing and from programs a planned protection, direct by wild neuro assays, patients' we evaluate the allow engagement, will to CSF assess method biomarkers mutant Huntington PRECISION-HD existing measurement. CSF to implemented have sparing. Building type wild and trial Huntington's target to Huntington confirm total type and Our type samples depleting mutant for wild novel XXX on
and us we directly magnetic antibody leading Huntington. Huntington measure type deplete to quantitation protein XX, slide to using conjugated the allows on shown This remains CSF beads. wild of that As collect mutant
contribution As of measure look community expect to the acknowledged, we increasingly the to type expanding and type protein importance wild use. meaningful HD to is forward be Huntington this ability wild a to its
program candidate allele-selective backbone HD from upon HD of generation our the turning Now programs. to builds first chemistry and have for incorporate gained modifications. learnings WVE-XXX, and our PN candidate first our SNPX to we wealth third This HD experience
PRECISION-HD PK/PD XXX instance, system trial. course clinical first the dosing selectivity. and evaluated this compounds of leveraging the including model our patient as we a type neurofilament dose also Safety is We're vivo ongoing evaluated HD of for And our experience adaptive design results in along collaboration the these at surgeon XXX from of biomarkers guide time relationships. For make with with light Monitoring understand an better our the and tolerability with first dose in the Safety informed and in program, a of trials disease have starting potentially wild to with independent and are decisions to to to sites the drive and efficiencies to in expansion. patients of intervals The single allele Ib/IIa accelerate confirmed and their HD planned DSMB, trial and an will clinical level will new diagnosis who will assess up to our driven incorporate clinical association early or trial. multiple escalation XX similar trial the Phase SNPX anticipate concept. with is enroll stages ascending chain PRECISION-HD CAG dosing dose the with mutant is to site portions. study, our regarding Clinical the activation Board, be Throughout data Huntington, proof and trial Huntington of the Data year. to an carry dose
driving in expression abnormal RNA are devastating the proteins DPRs. protein, common the WVE-XXX, of and candidate, dipeptide CXorfXX amyotrophic hexanucleotide FTD. Turning frontotemporal underlying to transcript expression for reduced to CXorfXX and expansions the sclerosis, phenotypes. gene accumulation or or diverse repeat of lateral treatment and lead is Clinical of ALS, CXorfXX healthy toxic dementia foci determinants target these to of or program neurotoxic repeat-containing our that repeat Mutations designed and the
the is describe and potential clearly Symposium model, Our Disease. resulted apart least led CXorfXX cord polyglycine In polyGP Nature spinal recently publication to transgenic CSF strategy XX, the with of endpoint key variant-selective key described XXst XXX was containing of XX% ICV over is DPR of knockdown presented seven on point. data and A slide the least down that knocked Communications. of clinical in in in preclinical proline or to and at studies. XXX the administered development about polyGP work knockdown durable we the to paper, excited cortex effect remain this achieve work injections at recently mouse the two rapid Motor and candidate. Neuron protein in the our to a at in XX% CXorfXX In normal ALS of durable clinical we this demonstrated variant-selective our remains showing Further, site out International days of targeting unchanged transcripts. identify on validate reason six the foundational highlights targeting in The the expansion time months. a of at Measurement that the
and outcome this in polyGP proof biomarkers interval, potential anticipate target trial we of with year. ongoing patient Clinical and filament subsequent measures dose include site multiple in the concept. single enroll be CXorfXX the to our dose of a will ALS, given or treatment documented clinical the So, preclinical to escalation DSMB of and assessed patients Like up we neuro tolerability and planned results. and light with be a and is Safety these will clinical trial of to the both neurodegeneration, promising Ib/Xa will adapted looking level are engagement A XXX with Key the for impact assessing FTD activation including forward clinical chain. trial, dosing XXX exploratory ALSFRS-R a will key dosing rapid for humans, SNPX and expansion CDR-FTLD. phenotype. assessed trial XX first The portions. be ascending guide along confirmed mixed Phase enabling of
and in to used discuss splicing label dystrophic that access plan is intercellular to clearly not from by Dystrophy open evaluate with NXXX the Exon skipping Finally, candidate suvodirsen first show Duchenne to candidate our DMD pipeline suvodirsen, achieve Exon amenable patients use for The apparently with I chemistry. generation guided Muscular reached we would due work backbone our study in poor modifications. chemistry like but first PN skipping tissue, to XX in Biopsies program our WVE-NXXX, did which our target PS/PO muscle. engagement, muscle to
and for and to surrogate as with for XX% This as by compared non-human that dose up PN Our well And with the containing increases incorporating shown first to production driven dosing. NXXX, compounds supporting from chemistry modification. concentrations primates NXXX of backbone generation Exon to was the data includes evidence compound, fatal dystrophin skipping compelling broader in dependent rescue the in decision dKO XX. myoblast vitro high of as knockout rapidly preclinical NXXX of chemistry, lastly, advance in as a mice patient derived a distribution slide PN phenotype with potential on biweekly or double candidate
the a a has both XXX mouse lack As dosed every week, PN compare dKO the to a eutropin of a molecule to the a mutation other mutation at the per control containing of shown as reminder, compound production, resulting in containing PS/PO fatal second leading at and group. a as as dystrophin, XX, effects weekly we milligram Exon milligrams kilogram phenotypes. study at model completed as a recently XX here, well and A kilogram dosed severe lack PBS well of to same in per leading level rapidly
less other in match MDX PS/PO at which PN is XX Not cohort there dose containing linkages, dosed in a those milligram time than the a have cohorts curves the other placement a dramatic that these than control the of approximately survived median a groups. of lived compounds of only treatment median every including a containing other weeks groups. same Both compound figure containing XX at shown mice Despite to for the at molecule termination group. three treated with these a compared separate as animals PN that, in group and the survival seven the are treatment the molecules study with of the increase groups, XX% in with compared a was week, weeks weeks of On as to of chemistry. sequence survival backbone survival PN Other slide, for and there of XX mice study treated termination. with PBS the survival
on in a quarter this are year. first of submit for NXXX track the CTA to We
be Patients enroll In will amenable The Kyle change our fact, trial to to biweekly turn XX I as targeting and up will based in dose drug dosing initial detect imminent. If planned the and work powered chemistry the a there's label production to clinical PK submission the compounds NXXX will the safety. will receive other DMD XX to be open apply be and upon safety will observations. Exon of our financial muscle Officer, patient to is PN intracellular with patients successful, Chief dystrophin and over well to will in ongoing. The driven Exon skipping. data assess potential call results. now distribution and discovery to is Financial Kyle? report as Moran, trial