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.

December 20, 2024. Dr. Sheldon Rowan, a professor and researcher at Tufts University, talks to host Ben Shaberman about the impact of the glycemic index, microbiome, and diet on the development of age-related macular degeneration and optimization of overall retinal health. Dr. Rowan will also discuss his grant from the Foundation and the Free Family for studying diet and AMD risk. The grant is also enabling Dr. Rowan to evaluate an FDA-approved drug and an emerging gene therapy as potential AMD preventions.

The company has also launched a natural history study of patients to inform the planned clinical trial.

Known as ultevursen, the USH2A RNA therapy was licensed from ProQR.

December 6, 2024. Bob Bell, PhD, chief scientific officer at Ascidian Therapeutics, talks with host Ben Shaberman about STELLAR, the company's Phase 1/2 clinical trial for its emerging RNA-rewriting therapy for Stargardt disease (ABCA4 mutations). Dr. Bell also provides overviews of Stargardt disease, the role of RNA in cells, and the advantages of RNA re-writing over other genetic treatment approaches. Learn more about the clinical trial at STELLAR (https://stellarstargardttrial.com/). Learn more about the prescreening study for STELLAR at PRESCREEN (https://stellarstargardttrial.com/#participation-criteria).

The companies are planning a Phase 3 clinical trial for the promising gene therapy.