Foundation Fighting Blindness Celebrates Historic FDA Approval of First Gene Therapy to Treat Blindness

Today’s U.S. Food and Drug Administration (FDA) approval of voretigene neparvovec, to be marketed as LUXTURNA, will be life-changing for patients with vision loss due to mutations in the RPE65 gene and a watershed moment for the inherited retinal disease field, says the Foundation Fighting Blindness.

Repeal of the Orphan Drug Tax Credit Would Stifle Promising Medical Research That Could be Life-Changing for 30 Million Americans, says FFB

The Foundation Fighting Blindness (FFB), the world’s leading private funder of research on treatments and cures for vision-robbing inherited retinal diseases, strongly opposes recent U.S. Senate and House of Representatives actions which would slow or stop research on treatments for Americans impacted by rare diseases.

Stem-Cell Therapy Clinics Remain Inadequately Regulated, Pose Risk to Patients

Though the U.S. Food & Drug Administration (FDA) is more tightly regulating U.S. clinics that tout stem-cell trials and treatments for inherited retinal diseases (IRDs) and a host of other conditions, many significant loopholes and enforcement limitations remain.

ProQR Doses First Participant in Its LCA10 Therapy Clinical Trial

ProQR, a biotech company in the Netherlands, has treated its first patient in a Phase 1/2 clinical trial for QR-110, a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world.