Usher syndrome type 2A therapy / Wave Life Sciences - LARVOL DELTA

Stereopure oligonucleotides promote USH2A exon skipping for the treatment of Usher syndrome type 2A (ARVO 2020) - "Finally, we demonstrate in vivo target engagement in humanized transgenic mice and NHPs following a single intravitreal injection of stereopure oligonucleotide.Conclusions These results indicate that stereopure oligonucleotides generated with PRISM potently induce USH2A exon 13 skipping in multiple model systems. These data support further development of stereopure oligonucleotides as a potential treatment for patients with Usher syndrome type 2A."