Gene Therapy for Blindness is One Step Closer to Approval

Friday, October 13, 2017

The U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee yesterday endorsed the first gene therapy for patients with Leber congenital amaurosis, also called Biallelic RPE65-mediated inherited retinal disease.  Dr. Stephen Rose, Chief Research Officer, Foundation Fighting Blindness, strongly endorsed the committee’s recommendation that the treatment be approved. Read more about FFB's stance on the potential treatment in Drug Discovery & Development here.