… to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare … lead to blindness in early childhood. No form of therapy is currently available,” said Dr. Katarina Stingl of … Tübingen, Investigator in the ILLUMINATE trial. “ProQR’s RNAtherapy for the p.Cys998X mutation showed the potential …
… 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 …
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