Foundation Fighting Blindness

Foundation Fighting Blindness

The Foundation Fighting Blindness is the driving force in the global development of treatments and cures for blinding diseases. To learn more, go to FightingBlindness.org.

Episodes

August 8, 2025 27 mins
August 8, 2025. Dr. Christina Ohnsman talks to host Ben Shaberman about Tern Therapeutics, a start-up company she co-founded to develop gene therapies for Batten disease and potentially other conditions. Dr. Ohnsman reviews early results for a Batten disease (CLN2) retinal gene therapy clinical trial. She also reflects on her previous work as a pediatric ophthalmologist and roles with Spark Therapeutics and Regenxbio.
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Susvimo is an FDA-approved treatment that continuously delivers drug through a tiny, implanted capsule
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July 25, 2025. Dr. Christine Curcio, one of the world's foremost experts in age-related macular degeneration (AMD) pathogenesis and the associated risk of advanced vision loss, talks to host Ben Shaberman about her epical career and recent Proctor Medal lecture with Dr. Cynthia Owsley at the 2025 meeting of the Association for Eye and Vision Research (ARVO). Her work has led to a highly effective test for predicting who is at great...
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The therapy delivers copies of the RORA gene to address multiple disease pathways.
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The company plans to complete BLA submission in early 2026.
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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-r...
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The expanded Phase 1/2/3 shortens time to potential FDA approval.
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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.
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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, Unive...
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Despite missing the primary endpoint, the treatment improved vision for some patients.
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The company is planning a Phase 2/3 clinical trial for therapy in late 2025.
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Part B of the trial will enroll nine adult patients as well as three pediatric patients.
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The company is currently enrolling patients in its Phase 2/3 VISTA Clinical Trial.
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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.
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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 ...
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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...
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Patients with worse vision at trial enrollment had greatest vision improvements.
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The technology was previously funded by the Foundation for the treatment of retinitis pigmentosa and dry age-related macular degeneration.
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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 ...
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In earlier clinical trials, the emerging gene therapy greatly reduced the treatment burden associated with injections of approved anti-VEGF therapies.
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