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 shows such as Showtime at the Apollo, the Today Show, Ellen, and 60 Minutes.

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 retinal degeneration.

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, 2024, by Mark Pennesi, MD, PhD, director, Ophthalmic Genetics at the Retina Foundation of the...

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 co-founded by Dr. Sahel.

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 by VEER magazine.

Dr. Robert MacLaren, a world-renowned retinal surgeon, clinical trial investigator, and research lab leader at the University of Oxford, talks to host Ben Shaberman about the successes and challenges in retinal gene therapy development and clinical trials, as well as advances in robotics for improving retinal surgery outcomes.

Jason Comander, MD, PhD, clinical researcher, surgeon, and director of the Inherited Retinal Disease Disorders Service at Mass Eye and Ear, talks to host Ben Shaberman about his administration of LUXTURNA gene therapy to RPE65 patients, his team's re-analysis of vitamin A therapy for retinitis pigmentosa, and passion for retinal research and patient care.

Known as antisense oligonucleotides, the treatments performed encouragingly in ProQR’s clinical trials

James Rath, a renowned filmmaker and accessibility consultant, talks to host Ben Shaberman about his vision loss journey and travel documentary, Blindspots. The new streaming program, on CuriosityStream.com, follows James as he explores the globe in search of accessible adventures, remarkable blind personalities, and great food.

Michael Voevodsky, MBA, CEO at MitoChem Therapeutics, talks to host Ben Shaberman about his company's emerging, gene-agnostic treatment designed to boost mitochondrial function for preserving vision in people with retinitis pigmentosa, age-related macular degeneration, and other retinal conditions.