… 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 …
… to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today … in 2019 as we advanced our portfolio of investigational RNA therapies for patients that suffer from inherited retinal … Ionis’ proprietary antisense technology. The therapy aims to inhibit the formation of the mutated toxic …