Opus Genetics Receives FDA RMAT Designation and Reports Positive Clinical Data for Gene Therapy Pipeline

RESEARCH TRIANGLE PARK, N.C., Aug. 13, 2025 (GLOBE NEWSWIRE) — Opus Genetics, a clinical-stage biopharmaceutical company, announced on Wednesday significant progress across its pipeline of gene therapies for inherited retinal diseases (IRDs). The company’s lead gene therapy candidate, OPGx-LCA5, was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) and has shown promising results in an ongoing clinical trial.

Key Pipeline Milestones

OPGx-LCA5: Gene Therapy for LCA

The RMAT designation for OPGx-LCA5, a therapy for Leber Congenital Amaurosis (LCA), is a major regulatory milestone that could accelerate its development and review process. The company presented 12-month clinical data from adult patients in its Phase 1/2 trial, which demonstrated sustained improvements in visual function, including visual acuity gains and improved mobility. Initial data from a pediatric cohort also showed early signs of vision improvement with a favorable safety profile.

Advancing Other Programs

Opus Genetics is also moving forward with other gene therapy candidates. Its OPGx-BEST1 program, which uses AAV-mediated gene delivery, remains on track to enter a Phase 1/2 trial in the second half of 2025. Preclinical data from this program demonstrated a restoration of the retinal pigment epithelium-photoreceptor interface in an animal model.

Additionally, the company has secured up to $3.6 million in non-dilutive funding from patient advocacy groups to advance two other gene therapy programs: OPGx-RDH12 for a form of LCA and OPGx-MERTK for retinitis pigmentosa.

Opus Genetics CEO, Dr. George Magrath, highlighted the progress. “Receiving RMAT designation for our OPGx-LCA5 program underscores the strength of our clinical data and the urgent need for effective gene therapies to treat inherited retinal diseases,” he said. “Our advancement of OPGx-BEST1 toward the clinic and the nomination of two additional development candidates highlight the breadth of our IRD pipeline.”