Foundation Fighting Blindness

July 12, 2024. Francois Paquet-Durand, PhD, a professor from the University of Tübingen and co-founder of the company Mireca, talks to host Ben Shaberman about the long journey of an emerging, Foundation-funded drug for the treatment of certain forms of retinitis pigmentosa. The episode highlights the challenges that scientists often encounter when developing a drug for a retinal disease, and the...

FDA-approved in 2021, the tiny implantable capsule provides sustained delivery of treatment, but was recalled in 2022.

For this episode of Eye on the Cure, Michael Telias, PhD, an assistant professor at the University of Rochester in New York, talks with host Ben Shaberman about forthcoming clinical trials to evaluate the FDA-approved drug disulfiram (aka, Antabuse) for improving vision in people with retinitis pigmentosa and other retinal diseases. Disulfiram was originally developed to deter people from abusing...

Dr. Peter Campochiaro, a clinician-scientist and the Eccles Professor of Ophthalmology and Neuroscience at the Johns Hopkins University School of Medicine, talks to host Ben Shaberman about N-acetylcysteine (NAC), a drug that shows promise for preserving cone vision (central vision and acuity) in people with retinitis pigmentosa. The powerful antioxidant has moved into a 30+ site, Phase 3 clinical...

Peter Wallsten, a veteran newspaper journalist and head of investigative reporting at The Washington Post, talks to host Ben Shaberman about his evolving career as a reporter and editor and the impact that Stargardt disease has had on his journey and ambitions. Peter also reflects on recently receiving a Pulitzer Prize for his team's 15-part series on the role of the AR-15 assault rifle in mass sh...

The emerging treatment is designed to work for people with RP and other conditions regardless of the gene mutation causing disease

No serious adverse events were reported. One patient had encouraging improvement in retinal sensitivity.

The discovery will help geneticists diagnose more RP patients with African descent.

Mutations in PDE6B are a leading cause of retinitis pigmentosa.

Matthew Whitaker talks to host Ben Shaberman about his remarkable achievements and success as a young jazz pianist and composer. Matthew was born three months prematurely and weighed less than two pounds. The oxygen he needed to stay alive also caused total blindness. But now 23 years old, the renowned music prodigy has recorded four albums, received several honors and awards, and appeared on TV s...

The XLRS gene therapy was well tolerated and resolved damaging schisis cavities in patients receiving the lowest dose.

The company’s innovative Gene Coding™ platform enables seamless insertion of DNA into selective genomic locations.

Kapil Bharti, PhD, a senior investigator and director of the Intramural Research Program at the National Eye Institute (NEI), talks with host Ben Shaberman about his clinical trial underway for an innovative cell therapy for geographic atrophy (advanced dry age-related macular degeneration) as well as a clinical trial of the drug metformin for Stargardt disease.

The drug is thought to work independent of the mutated gene causing RP.

The gene therapy is administered using a less-invasive intravitreal injection.

The emerging treatment is designed to work independent of the patient’s mutated gene.

The company reported positive two-year results for its Phase 2 clinical trial of MCO-010.

Opus Reports Vision Improvements for Patients in LCA5 Gene Therapy Clinical Trial by Foundation Fighting Blindness

Hema Ramkumar, MD, a retinal specialist, surgeon, and founder of the company Oculogenex, talks with host Ben Shaberman about her emerging therapy that targets mitchondria to slow the progression of intermediate dry age-related macular degeneration and potentially other retinal diseases. She also discusses her experiment that will put mice in space so researchers can gain a better understanding of...

Peter Quinn, PhD, a principal investigator and associate research scientist at Columbia University, talks to host Ben Shaberman about the promise of emerging CRISPR/Cas9 gene editing therapies, including base and prime editing approaches, for inherited retinal diseases. Dr. Quinn also reviews gene editing projects ongoing in his lab for patients with mutations in CRB1 and PRPH2.

Known as retinal progenitors, the company’s jCells® are designed to preserve photoreceptors in people with retinitis pigmentosa (RP) and other retinal diseases.

Beacon Therapeutics has reported vision improvements for five of eight patients receiving the high dose of its X-linked retinitis pigmentosa (XLRP) gene therapy in the Phase 2 SKYLINE clinical trial. Known as AGTC-501, the emerging gene therapy is for patients with mutations in RPGR, the gene most frequently associated with XLRP. Interim, 12-month results for SKYLINE were reported on February 7, 2...

Dr. Jose Sahel, one of the most honored and accomplished leaders in the retinal disease research space, talks with host Ben Shaberman about his establishment and leadership of renowned ophthalmology clinical research centers at the University of Pittsburgh Medical Center and Institut de la Vision in Paris. They also discuss emerging inhertied retinal disease therapies from SparingVision, a biotech...

ACDN-01 is the only genetic medicine entering clinical development for Stargardt disease.

Justin Kauflin, a renowned jazz pianist who lost his vision as a boy, talks to host Ben Shaberman about his extensive touring, numerous collaborations, and close friendship with legendary trumpeter Clark Terry. Their story was captured in the hit documentary film Keep On Keepin' On. Justin became a jazz pro at the age of 14, toured globally with Quincy Jones, and was voted jazz artist of the year...