The expanded Phase 1/2/3 shortens time to potential FDA approval.

June 13, 2025. Dr. Philip Beales, a co-founder of Axovia Therapeutics, talks to host Ben Shaberman about Bardet Biedl syndrome, ciliopathies, and his company's emerging gene therapy for Bardet Biedl syndrome type 1 (BBS1) which is moving toward a clinical trial.

May 23, 2025. Lenore von Krusenstiern, MD, PhD, at BlueRock Therapeutics talks to host Ben Shaberman about the company's clinical trial for a photoreceptor replacement therapy for people with inherited retinal conditions such as retinitis pigmentosa and cone-rod dystrophy. The approach, which uses induced pluripotent stem cells to develop photoreceptor precursors, comes out of the lab of stem cell pioneer David Gamm, MD, PhD, University of Wisconsin-Madison.

Despite missing the primary endpoint, the treatment improved vision for some patients.

The company is planning a Phase 2/3 clinical trial for therapy in late 2025.

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.