The therapy delivers copies of the RORA gene to address multiple disease pathways.

The company plans to complete BLA submission in early 2026.

July 11, 2025. Richard Small, CEO of Neurotech, talks to host Ben Shaberman about ENCELTO™, the first and only FDA-approved treatment for adults with idiopathic Macular Telangiectasia Type 2 (MacTel). The therapy is a tiny, implantable capsule, the size of a rice grain, that delivers neurotrophic factors to preserve the patient's retinal cells and vision. The approach was previously developed to treat retinitis pigmentosa and age-related macular degeneration.

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.