Opus Genetics today administered the first subretinal injection of its OPGx‑BEST1 gene‑therapy candidate to a patient with Best disease in Research Triangle Park, North Carolina. The dose marks the official start of the Phase 1/2 BIRD‑1 study and represents the first human exposure to the company’s adeno‑associated virus (AAV) vector designed to deliver a functional copy of the BEST1 gene to retinal pigment epithelium cells.
Best disease, a rare autosomal‑dominant retinal disorder caused by mutations in BEST1, leads to progressive vision loss in roughly 9,000 U.S. patients. OPGx‑BEST1 aims to halt or reverse retinal degeneration by restoring bestrophin function. The adaptive, open‑label study will evaluate safety, tolerability, and preliminary efficacy of a single subretinal injection, with first‑in‑human data expected in the first quarter of 2026.
The dosing milestone comes after Opus Genetics reported positive data from its OPGx‑LCA5 program for Leber congenital amaurosis type 5 and secured approximately $23 million in a registered direct offering in November 2025. The company’s seven‑program pipeline, which also targets RHO, RDH12, and MERTK, positions it as a leading developer of AAV‑based therapies for inherited retinal diseases.
CEO George Magrath said the event “reinforces our mission to develop one‑time gene therapies for inherited retinal diseases that previously had no treatment options.” He added that the successful first dose demonstrates the safety profile of the vector and supports the company’s strategy to advance OPGx‑BEST1 toward a pivotal Phase 2/3 trial.
Analysts have responded positively, with several firms issuing “Buy” ratings and reiterating price targets following the announcement. The company’s stock had surged 100 % over the past six months, reflecting investor confidence in its pipeline progress and recent financing. The market reaction underscores the perceived value of a potentially curative therapy for a rare disease with no approved treatments.
If early safety and tolerability data are favorable, Opus Genetics could accelerate regulatory discussions and move the program into a pivotal trial, potentially unlocking a high‑impact asset in a niche but significant market. The company’s ability to secure additional capital and maintain a robust pipeline will be critical to sustaining momentum and achieving eventual regulatory approval.
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