Opus Genetics confirmed that its Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the U.S. Food and Drug Administration was completed on November 6, 2025, and that the agency reaffirmed the company’s RMAT designation for its OPGx‑LCA5 gene‑therapy program targeting Leber congenital amaurosis caused by LCA5 mutations.
The FDA’s guidance outlined an adaptive, single‑arm trial design that would enroll eight patients over 12 months, using each patient as their own control to assess visual acuity and other functional endpoints. This design combines Phase 3 objectives into one study, potentially shortening the development timeline for a therapy that could become the first treatment for LCA5‑related blindness.
The RMAT designation is a key lever for accelerated approval, allowing the company to rely on surrogate or intermediate endpoints and to receive regulatory flexibility. For a rare disease with only about 200 affected patients in the U.S., the ability to conduct a small, adaptive trial is a strategic advantage that can bring a one‑time treatment to market faster than a traditional, multi‑arm, large‑patient study would allow.
Early clinical data from the Phase 1/2 program showed clinically meaningful visual improvements in six treated participants, and the first patient has already entered a run‑in period for the planned adaptive Phase 3 trial. Dosing for the Phase 3 portion is expected in the second half of 2026, with topline data anticipated roughly a year later.
A recent $23 million financing round, led by Perceptive Advisors and Balyasny Asset Management, will fund the LCA5 and BEST1 programs and support operations through the second half of 2027, ensuring the company can advance the OPGx‑LCA5 program through its next development stages.
CEO George Magrath said the FDA’s guidance “provides confidence in our path to approval for OPGx‑LCA5” and highlighted the adaptive design’s ability to eliminate the need for a separate registrational trial, underscoring Opus Genetics’ strategy to become a leader in inherited retinal disease gene therapies.
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