Atsena Therapeutics Receives Orphan Drug Designation From the FDA for Novel Gene Therapy To Treat Genetic Eye Disease LCA1

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 orphan drug designation for its investigational gene therapy product for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1), a genetic eye disease that affects the retina.