provide come to Good platform, advancement in is made and an that modality adding a significant RNA splicing of update Today, the Mike. U.S. silencing. editing off our capabilities Thank morning, you RNA I'll have to we on platform. everyone. a RNA-editing exciting PRISM
the that ADAR we proteins, diseases. with and across of adenosine on harness deaminases by benefits focusing providing variety dosing including feel on use A-to-G offers over other a distinct to ample acting endogenous modalities RNA, we delivery, effective gene editing, endogenous editing, this The or effects. As leads titratable RNA of our opportunity approach ease reversible to ability wide several protein
a the it thousands, source identification the A-to-G could discovery been variants nearly you be to genetic represents has on rich of In an large tens corrected for landscape SNPs, very and the of in is targets. of pathogenic analysis potential which and that of published with As it see human amenable has right, clinical A-to-G editing.
effects Beyond off-target avoids use. over in other of editing. which has unique this or editing potential emerged CASXX gene immunogenicity developing editing technology use force players ADAR recently the of proteins, exogenous technology proteins. and risk relatively from offering Endogenous RNA We strength among endogenous these beyond technology advantages is the for potential ADAR-mediated and as such of proteins therapeutic chimeric related this
Our duration oligonucleotides are their fully and And chemically basis of modified, activity. stability are increases which fewer. XX typically
to control maximize ability to our backbone endogenous enables us ADAR corrality of the Importantly, activity. precisely the
Lastly, hepatic GalNAc-conjugated oligonucleotides our simple we investigations, vectors delivery AAV or use simplified meaning strategy, no we initial no therapeutic nanoparticles. using In are a for targets.
our platform However, continued also our used we could technology expect therapeutic RNA be build RNA-editing and PRISM capability across this various of to indications.
achieved primate in defined, across lines, and efficient As have our nonhuman cell human vitro including oligonucleotides with hepatocytes. we primary variety we previously a editing have of RNA very
have in editing RNA stereopure GalNAc-mediated three As via oligonucleotides potent uptake. observed dose-dependent with we slide, durable distinct shown here chemically this and
which, translate our in to results to vitro, did. this next was they determine task achieved system vitro these whether in vivo Having in
the we in sharing For vivo in first primates. results nonhuman RNA-editing time are
and dosed this day biopsy post groups for site. RNA-editing last days three We study, two of six sequence a distinct samples chemical the took nonhuman primates three oligonucleotides. subcutaneously five liver once we dose baseline in with and days evaluated at For targeted
the detected vivo. the to as you in compared editing see As we on to chart up can XX% baseline left, on
Sanger On targeted sequencing plot clear a target the the right oligonucleotides, showing the you editing for can at site. see
by nonhuman successful proof-of-concept While in priming. knowledge, first very we results RNA this are these is an ongoing and are these the study, editing our excited to
more our compelling. which achieved Importantly, them have believe technology, these results ADAR we using we endogenous makes
proud I work environment, we challenging our ongoing a pandemic. delivering amidst of streams to that add very these results like team would I'm to adapt as for continue of result a as our the
have applied a to this we demonstrated RNA vitro targets. in across disease be of multiple of editing which supports ahead, potential looking Now, successful the across technology previously variety transcripts,
expect program Therapy Later or will also virtually. poster tomorrow is vivo American a data presented vitro this we Annual of add in in in ADAR-mediated the RNA-editing being also & Our in year, indication. Cell first our share at Society data we which to announce additional be RNA-editing a expect Meeting Gene and to held hepatic
the for with I'll remarks. back Paul Now, call that, turn to closing