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.

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.

February 21, 2025. Ricky Enger, the Director of Practical Help for Hadley, talks with host Ben Shaberman about her personal and professional journeys as someone born completely blind and the many assistive and accommodative technologies that are helpful to people with low vision. At Hadley, Ricky develops technology workshops and other community initiatives for older adults new to vision loss.

Perceive Pharma is a spin out of Perceive Biotherapeutics.

Though unaffected by vision loss, she brought great passion to driving the Foundation’s mission.

January 24, 2025. Aniz Girach, MD, the chief medical officer at SpliceBio, talks to host Ben Shaberman all about the company’s emerging protein-splicing therapy for Stargardt disease which has received clearance from the FDA to move into a clinical trial.

The Foundation is a founding investor in Atsena.

January 10, 2025. Damon Lembi talks to host Ben Shaberman about his journey as a college baseball star, father, and CEO of Learnit (a live learning platform), and how the diagnosis of an inherited retinal disease informs his leadership style, personal growth, and relationships. Damon is also a best-selling author and host of the Learn-It-All podcast.

The company is also planning to conduct the clinical trial at sites in the EU.