August 11, 2025
2 min watch
Key takeaways:
- Baruch D. Kuppermann, MD, PhD, highlights six companies developing Stargardt disease therapies.
- Two are oral therapies, three are gene therapies, and one is an optogenetic therapy.
FORT LAUDERDALE, Fla. — In this Healio Video Perspective from the Retina World Congress, Baruch D. Kuppermann, MD, PhD, delivers an update on Stargardt disease therapies currently in development.
Six companies are testing therapies across multiple stages, Kuppermann told Healio. Two of these therapies are designed to be delivered orally, three are gene therapies, and one is an optogenetic therapy.
“The concept there is that they are interacting with the ABCA4 gene, which is responsible for 95% of the Stargardt disease mutations,” Kuppermann said. “These would be treatments that are either intravitreal or subretinal and that would have a long, durable effect.”
In addition to the six therapies Kuppermann highlighted, VG801 from VeonGen Therapeutics received rare pediatric disease designation in June, as Healio previously reported.
“We hope that all of them meet success in this very significant category of unmet need,” Kuppermann said.
Sources/Disclosures
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Kuppermann BD. Emerging treatments for Stargardt disease. Presented at: Retina World Congress; May 8-11, 2025; Fort Lauderdale, Florida.
Disclosures:
Kuppermann reports consulting for Alkeus Pharmaceuticals, Nanoscope Therapeutics and Ocugen.
