Leber Congenital Amaurosis

Opus Genetics received rare pediatric disease designation from the FDA for its ocular gene therapy designed to treat patients with a form of Leber congenital amaurosis, according to a press release.

Dosing is complete in the first cohort of the phase 1/2 clinical trial of OPGx-LCA5 for the treatment of Leber congenital amaurosis, according to a press release from Opus Genetics.

WAILEA, Hawaii — Pediatric retina is a difficult field with high stakes. Here at the Hawaiian Eye 2024 meeting, a specialist shared pearls on what any ophthalmologist who deals with pediatric patients should not miss.

ROME — In this Healio Video Perspective from the FLORetina-ICOOR meeting, Bart Leroy, MD, PhD, speaks about RNA interference technology for the treatment of Leber congenital amaurosis type 10.

ROME — Voretigene neparvovec proved successful as a treatment for Leber congenital amaurosis 2 in preschool children, as shown by a study presented at the FLORetina-ICOOR meeting.

NEW ORLEANS — In this Healio Video Perspective from the ARVO meeting, Christine Kay, MD, discusses 6-month results investigating ATSN-101 for the treatment of Leber congenital amaurosis caused by mutations in the GUCY2D gene.

Holding a family’s fragile, premature newborn is a humbling moment — especially as a newly minted doctor.

Jean Bennett, MD, PhD, inventor and co-developer of Luxturna, the first FDA-approved gene therapy, now serves as scientific co-founder and sits on the board of Opus Genetics, founded to investigate gene therapies for rare retinal diseases.

The Retinal Degeneration Fund announced the launch of Opus Genetics, which will aim to develop gene therapy treatments for orphan inherited retinal diseases, according to a press release.

ProQR Therapeutics completed patient enrollment in the phase 2/3 Illuminate trial, investigating sepofarsen for the treatment of Leber congenital amaurosis 10, according to a press release.