Dr. Paul Bolno
today's you introductory Good you, and Thank I'll a joining remarks with thank morning today. us start and Kate. the updates. call few for company
will and program program. conclude RNA-editing with I'll for Gaiero and then on update development PRECISION-HD ADAR Mike outlook financial mediated Dave discuss Next, results XXXX. an an our our Panzara will on provide an our update
of key a chemistry to the founded platform. in sequence, rational and unique oligonucleotides using PRISM therapeutics that therapy properties. Wave This deep through among stereochemistry is a interplay novel genetic us enables our medicines to impact understanding way, design develop pharmacologic was how and company platform proprietary design
innovative like a past decade, and includes pipeline novel of modalities that designs backbone broad modalities, build enabling progressed the to RNA-editing. disease a evolved such targets, modifications, our allele-selective properties as and range different Over us unique and has chemistry
continue Huntington's to pipeline collaboration will pipeline. we including foundation and forward, of expand our Takeda. our ALS with our several in disease, The FTD innovate Going CNS, and is and in partner this work programs
our short programs into and of come other been the three Over humbled learnings clinical the history, way. relatively we've have that programs by course and embraced used out the of the have along and development moved
Our dedicated and in is development to of programs the this clinical preparing experienced year. second half team two advance actively into additional
and Finally, development we and support that manufacturing internal have established substance work of chain. visibility supply to control us drug us capabilities platform, and to clinical oligonucleotides preclinical enable our provide increased produce with our
at control presented PRISM, of leverage potential initially each last importance October. research X-ray Validating to the Using RNase controlling structure, which the backbone H activity, we design oligonucleotides to to we have ability backbone the demonstrated position structure therapeutics. we day crystal the stereochemistry rationally of of our
compounds including SNPs is polymorphisms feature As the differentiating to blue. and lower and H we the a containing disease nucleotide designed mutant depicts CXorfXX WVE-XXXXXX, H Huntington's where in seen aiding the RNase mRNA red to oligonucleotides slide, a RNase our duplex in paramount or programs serial on pure core image a of have hetero balanced activity WVE-XXXXXX selectively The target Controlling huntingtin. to single target in allele-selectivity. to
treating oligonucleotides our stereopure at we with FTD. aimed selective and program Similarly, ALS have designed transcript approach for CXorfXXCX a
X our have and disease, platform. our for we During the clinical advancing Huntington's CNS progress stereopure fourth quarter made and our proprietary focused oligonucleotides pipeline of programs important PRISM in recent months,
in the important wild-type function for wild-type evidence Wild-type and an first mutant PRECISION-HDX systemically. both are for PRECISION-HDX in is that huntingtin nervous increasing intact. huntingtin protein, and development are protein neuronal First, the disease central and our that system is essential only huntingtin clinical lower designed leading huntingtin to programs Huntington's selectively basic while and there's health, relatively
end which placebo higher our from compared reduction Huntington's that supported mutant ongoing the dose. advancing clinical PRECISION-HDX the as and last huntingtin we the trial as disease, At well profile and as results total year, demonstrated to huntingtin safety first the no reported of change in
of these the Panzara year. half is and/orX. to Mike HD provide of later carries a track of the SNPX the programs clinical our portfolio least initiate the SNPX, XX% the to with of up mutation SNPX will have second overlap at Approximately in HD in Ramping XX% programs, HD further updates on is which call. on up population one population and X development
the our we portfolio this potential at CHDI and preclinical programs in of conference presented preclinical are vivo our disease. vitro to treatment we for Huntington's clinical of in and data excited introduce Lastly, SNPX into program the
program Our in and address CXorfXX aims amyotrophic frontotemporal gene. in by sclerosis clinic approaching caused dementia lateral to next also mutations the the
while tissue. Our Like CX repeat, help mouse hexanucleotide is preclinical guide our protein in drives toxic vivo the of program program, In and potent silence transcript we've is containing potently brain to in which SNPX repeat RNA designed used and have proteins formation model to our the preserved. contained that and abnormal transcript we transgenic dipeptide CXorfXX the studies, development. knockdown solely the shown these of both
We program half on initiate year. the continue to clinical to our second develop CXorfXX of and in are track advance the
with such preclinical partners We as CNS targeting continue our diseases Takeda multiple Parkinson's to and develop Alzheimer's. CNS programs
includes Takeda support collaboration. determined with million committed included the is multiple our targets in advance in agreement to which over capital, reminder, million a $XX preclinical As research $XXX
the vivo we to of in expect lead with a year, one validation achieved target for XXXX. Last compound more programs and in
our target potent than compound. In collaboration. are results vitro, from XX As shown eight-week undisclosed we've in In reference demonstrated seen highlight more knockdown with we've potent durability times in compound slide, treatment. out stereo a similar on we the in that is the we random working on good to vivo, target and vitro that vivo an
selectively which we on designed to presented ex-vivo promote programs XXXX, our on XX USHXA silence in vitro-data on program, October in-vitro and to CNS, preclinical to is which skipping, retinitis for work continue RhoPXXH designed pigmentosa. Type we Usher's exon Xa ophthalmology USHXA transcripts. in and data Beyond program, In XXXX, and two our USHXA RopeyXXh for RhoPXXH Syndrome is
explore ophthalmology. to our We opportunities continue to work advance in
and each continued to Q-point platform insight the we compare target including data us also programs, evolve interplay and sets stereopure behavior and impacts and optimize between and gain various clinical a diverse how which stereoisomers. XXXX. We've to body PRISM of characterize sequence, of in backbone can allow of the Through in-vivo and in-vitro we the activity. and design oligonucleotides, growing PRISM, from With chemistry our chemistry stereochemistry modifications into our
learnings programs. these build future we importantly, Most into
with SNPX platform. our CXorfXX our example, optimized both of and For programs chemistry designed are
come the to PRISM platform One is on new off which of in developments touch modality, later our ADAR our call. exciting more the RNA-editing of I'll
also unexpected had quarter and had many disappointing with the DMD program. fourth Wave XXXX, in accomplishments setback While we our an in
sharing are additional learnings We committed and results Muscular any into Association We're committed to and at presentation better incorporating Dystrophy programs. time month. end to understanding suvodirsen this for clinical the with Conference clinical future we'll community the also the analyzed Duchenne data in the these data have of and others, at
programs development by In approaching and CNS potentially pipeline four Wave in to clinical programs XXXX. led which differentiated physician's two summary, has innovative clinics, Wave including have an the our
fourth and to to review that, year Gaiero With our financial turn full the I'll Dave quarter call Dave? results. over