Ocular Gene Therapy
FDA grants regenerative medicine advanced therapy designation to XLRS gene therapy
The FDA granted regenerative medicine advanced therapy designation to ATSN-201, a gene therapy candidate for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
Ophthalmologists weigh if gene therapy is the future of wet AMD treatment
SEVILLE, Spain — Gene therapy is an area of intensive research and development in the retina space, with promising prospects and significant challenges.
Investigational Therapies
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.