Opus Genetics Partners with Global RDH12 Alliance to Accelerate Gene Therapy for IRDs

RESEARCH TRIANGLE PARK, N.C., July 23, 2025 (GLOBE NEWSWIRE) — Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing adeno-associated virus (AAV) gene therapies for inherited retinal diseases (IRDs), today announced a strategic partnership with the Global RDH12 Alliance. This collaboration aims to accelerate Opus’ investigational AAV gene therapy, OPGx-RDH12, for patients with vision loss due to retinol dehydrogenase 12 (RDH12) gene mutations, a cause of Leber congenital amaurosis (RDH12-LCA).

RDH12-LCA is an ultra-rare form of childhood blindness causing progressive vision loss, often beginning in early childhood. OPGx-RDH12 uses an AAV vector to deliver a functional copy of the RDH12 gene to retinal photoreceptors. Preclinical studies have shown promising results.

The Alliance, which unites patient advocacy groups “RDH12 Fund for Sight” (U.S.) and “Eyes on the Future” (UK), will contribute up to $1.6 million towards the program. The partnership includes a risk-sharing structure and co-development of clinical and regulatory strategy. The goal is to file an Investigational New Drug (IND) application with the U.S. FDA by late 2025.

“This partnership represents a significant step forward,” said Mathew Pletcher, Ph.D., Board member of RDH12 Fund for Sight, emphasizing combining patient insights with Opus’ expertise. Silvia Cerolini, CEO of Eyes on the Future, highlighted the urgency: “We are racing against time as our children’s vision continues to deteriorate.”

About RDH12-LCA and OPGx-RDH12

RDH12-LCA is a severe IRD. OPGx-RDH12 is an AAV gene therapy designed to restore RDH12 function.

About the Global RDH12 Alliance (RDH12 Fund for Sight & Eyes on the Future)

The Alliance is a collaborative effort of patient advocacy groups dedicated to supporting research and treatment development for RDH12-related vision loss. The RDH12 Fund for Sight is a U.S.-based not-for-profit organization that aims to bring together families with common concerns and worries, collect and distribute information about the form of LCA caused by RDH12, and, most importantly, ensure that significant funds are devoted to the study and development of treatments for LCA caused by RDH12. Eyes on the Future is a UK-based non-for-profit organization supporting research into rare diseases and eye diseases with specific focus on RDH12 inherited retinal dystrophies. Funded by Enrico and Silvia, whose 11-year-old child is affected by RDH1- LCA, Eyes on the Future is a registered company.