Ocular Gene Therapy

Beacon Therapeutics announced the presentation of interim safety and efficacy results from the phase 2 SKYLINE trial evaluating AGTC-501 gene therapy for X-linked retinitis pigmentosa.

WAILEA, Hawaii — Gene therapy is a rapidly advancing innovation in the world of retinal diseases, according to a speaker at Retina 2024.

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.

WAILEA, Hawaii — In this Healio Video Perspective from Retina 2024, David A. Eichenbaum, MD, discusses the potential for gene therapy to reduce the intravitreal injection treatment burden in retinal disease.

ROME — In this Healio Video Perspective from the FLORetina-ICOOR meeting, Bart Leroy, MD, PhD, elucidates complications that may occur following gene therapy with Luxturna.

In this Healio Video Perspective from the FLORetina-ICOOR meeting, Suber S. Huang, MD, MBA, speaks about adverse events in gene therapy.

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.

ROME — The total estimated costs from inherited retinal diseases in the United States amounts to $31.8 billion. And yet, research barriers slow down the progress toward innovative treatment pathways, such as gene and cell therapies.

Atsena Therapeutics reported positive 12-month safety and efficacy data from a phase 1/2 trial of ATSN-101 gene therapy in GUCY2D-associated Leber congenital amaurosis.

More than a decade ago, I told my wife’s grandfather that, despite all our advances in medicine, we didn’t have a treatment for his geographic atrophy.