Story perspectives
FDA Grants Rare Disease Designation for Ocugen's Gene Therapy
5/30/2025
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Story summary
- Ocugen has exciting news: the FDA has granted rare pediatric disease designation for OCU410ST, a groundbreaking gene therapy targeting Stargardt disease. With plans to kick off a phase 2/3 clinical trial soon, the company is on track to submit a biologics license application by 2027, offering hope to the 44,000 patients affected in the U.S.
