… to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced its … Sepofarsen Sepofarsen (QR-110) is an investigational RNAtherapy designed to restore vision in Leber congenital …
… precision medicines based on molecular diagnostics and our RNA therapeutic platform,” said David M. Rodman, MD, Chief … in a planned series of ophthalmology trials utilizing our RNAtherapy platform to target the underlying cause of blindness …
Substantial progress in 2019 with final data from Phase 1/2 trial of sepofarsen for Leber’s congenital amaurosis 10 showing rapid, significant and durable improvements in vision; Phase 2/3 pivotal trial Illuminate initiated with data expected in H1 2021Phase 1/2 St