Gene Therapy Restores Vision in First-Ever Trial for Rare, Inherited Blindness
A gene therapy developed by University of Florida scientists, which restored useful vision to most patients with the rare, inherited blindness known as Leber congenital amaurosis type I, or LCA1, in a small trial.
Atsena Therapeutics Receives Rare Pediatric Disease Designation From the U.S. FDA for ATSN-201 Gene Therapy To Treat X-Linked Retinoschisis
UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation (RPD) for ATSN-201 for the treatment of X-linked retinoschisis (XLRS).
