Opus Genetics Gene Therapy Accepted into FDA's Rare Disease Program

Published: 2026-05-04 17:58:16 ET IRD

Published on 05/04/2026 at 05:58 pm EDT

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 [email protected]

(END) Dow Jones Newswires

05-04-26 1757ET