Part B of the trial will enroll nine adult patients as well as three pediatric patients.

The company is currently enrolling patients in its Phase 2/3 VISTA Clinical Trial.

Cindy Elden, who co-founded the Usher III Initiative with her late father, talks to host Ben Shaberman about her journey with Usher syndrome type 3 (USH3), the decades-long work and persistence of her non-profit, and the clinical trial it launched for an USH3 small molecule therapy.

April 3, 2025. In this episode of Eye on the Cure, Jen Bernstein at Horizon Government Affairs, and Sharon King of Taylor’s Tale, talk to host Ben Shaberman about how to schedule and conduct a visit with your House Member in your local district to advocate for important issues. With federal eye research funding at great risk and a Congressional district work period coming in mid-April, now is an ideal time to schedule a visit with your House Member.

March 21, 2025. Professor Michel Michaelides, a world-renowned clinical researcher for inherited retinal diseases at Moorfields Eye Hospital and University College London, talks to host Ben Shaberman about the extraordinary vision improvements for young blind children receiving gene therapy for Leber congenital amaurosis 4 (LCA4). Professor also talks about emerging therapies in clinical trials for X-linked retinitis pigmentosa and Stargardt disease.

Patients with worse vision at trial enrollment had greatest vision improvements.

The technology was previously funded by the Foundation for the treatment of retinitis pigmentosa and dry age-related macular degeneration.

March 7, 2025. Aaron Nagiel, MD, PhD, a retina surgeon at Children’s Hospital Los Angeles, talks to host Ben Shaberman about how gene therapy is administered to the retina, what the surgery and recovery is like for patients, and the vision improvements for young patients receiving LUXTURNA, the first FDA-approved gene therapy for the eye or an inherited condition. Dr. Nagiel also discusses other retinal gene therapies in clinical development.

In earlier clinical trials, the emerging gene therapy greatly reduced the treatment burden associated with injections of approved anti-VEGF therapies.

MeiraGTx hopes to apply for marketing approval in the UK for the gene therapy.