Ocular Gene Therapy
FDA grants fast track designation to X-linked retinoschisis treatment
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
Lumevoq for LHON yields sustained visual acuity improvements at 5 years in phase 3 trial
A one-time injection of Lumevoq demonstrated sustained visual acuity improvements with a favorable safety profile at 5 years in patients with Leber hereditary optic neuropathy, according to a press release from GenSight Biologics.
VIDEO: Gene therapy shows promise for treatment of wet AMD
KOLOA, Hawaii — In this Healio Video Perspective from Retina 2025, Allen C. Ho, MD, discusses recent breakthroughs demonstrating that gene therapy is a viable option for treating wet age-related macular degeneration.
RhyGaze raises $86 million to develop vision restoration gene therapy
RhyGaze secured $86 million in a Series A financing round, which will be used to further the development of the company’s lead candidate gene therapy for optogenetic vision restoration, according to a press release.
Part B of phase 1/2 X-linked retinoschisis gene therapy trial initiated
Atsena Therapeutics initiated part B of the phase 1/2 LIGHTHOUSE study investigating ATSN-201 for the treatment of X-linked retinoschisis, according to a press release.
Safety monitoring board approves continuation of gene therapy trial for GA
The data and safety monitoring board for a phase 1/2 study clinical trial of a geographic atrophy gene therapy approved continuation of the second phase of the study due to positive safety results, according to a press release from Ocugen.
FDA, Opus Genetics reach special protocol assessment agreement for APX3330 trial
Opus Genetics and the FDA have reached an agreement on a special protocol assessment for a phase 3 clinical trial investigating oral APX3330 for the treatment of moderate to severe nonproliferative diabetic retinopathy.
Gene therapy receives rare pediatric disease designation for inherited retinal dystrophy
The FDA granted rare pediatric disease designation for HORA-PDE6b, a gene therapy for patients with inherited retinal dystrophy caused by mutations of the PDE6B gene, according to a press release from eyeDNA Therapeutics.
Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial
Atsena Therapeutics completed patient dosing in part A of the phase 1/2 LIGHTHOUSE clinical trial investigating a gene therapy designed for the treatment of X-linked retinoschisis, according to a press release.
FDA clears investigational new drug application for Stargardt disease gene therapy
The FDA cleared an investigational new drug application for SB-007, an adeno-associated viral vector gene therapy designed to address the root genetic cause of Stargardt disease, according to a press release from SpliceBio.