Gene Therapy

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.

PTC Therapeutics Inc. released results of a 5-year analysis that showed its novel gene therapy, PTC-AADC, improved outcomes among children with aromatic L-amino acid decarboxylase deficiency.

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.

Initial findings from the real-world PERCEIVE study of voretigene neparvovec in patients with RPE65 mutation-associated inherited retinal dystrophy show safety and efficacy in line with those observed in pivotal trials.

The FDA received a completed rolling biologics license application for betibeglogene autotemcel, a gene therapy in development to treat patients with transfusion-dependent beta-thalassemia.

In this episode, Sue Washer, BS, MBA, discusses gene therapy in ophthalmology, AGTC’s clinical trial progress, as well as current and future challenges faced by both the cell therapy industry and ophthalmology.

In a first-in-human phase 1/2 study, GT005, an AAV2-based one-time investigational gene therapy, showed potential to regulate the complement pathway involved in the pathogenesis of geographic atrophy.

AbbVie and Regenxbio have entered into a partnership to develop and commercialize RGX-314, an investigational gene therapy, for the treatment of retinal diseases, according to a press release.

The FDA halted a phase 3 study that was evaluating an investigational gene therapy for a rare neurological disease in young children.

The FDA placed a clinical hold on a phase 3 study designed to evaluate elivaldogene autotemcel, an investigational gene therapy, for the treatment of younger patients with cerebral adrenoleukodystrophy.