Opus Genetics Gene Therapy Accepted into FDA's Rare Disease Program
By Christopher Kuo
The Food and Drug Administration accepted Opus Genetics's investigational gene therapy into its Rare Disease Evidence Principles program, the company said Monday.
The clinical-stage biopharmaceutical company said its treatment, OPGx-LCA5, is designed to treat Leber congenital amaurosis type 5, a rare inherited retinal disease caused by gene mutations.
As part of the FDA initiative, the FDA will work with Opus Genetics to guide the development of OPGx-LCA5, including considerations for clinical trial design and strategies to support demonstration of clinical benefit, the company said. The program also provides a framework for evaluating substantial evidence of effectiveness.
The Rare Disease Evidence Principles Program is a new FDA initiative designed to support the development of therapies for ultra-rare genetic diseases typically affecting fewer than 1,000 patients in the U.S.
Write to Christopher Kuo at chris.kuo@wsj.com
(END) Dow Jones Newswires
May 04, 2026 17:57 ET (21:57 GMT)
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