Opus Genetics and Global RDH12 Alliance Join Forces to Accelerate Gene Therapy for Inherited Childhood Blindness

Opus Genetics has entered into a partnership with the Global RDH12 Alliance to drive the development of its investigational gene therapy program, OPGx-RDH12, targeting inherited retinal degeneration caused by RDH12 gene mutations. The initiative seeks to address Leber congenital amaurosis (RDH12-LCA), a rare genetic eye disorder that leads to progressive vision loss and often results in childhood blindness.

Collaborative Investment to Support OPGx-RDH12 Development

The Global RDH12 Alliance, a coalition of advocacy groups including the RDH12 Fund for Sight (U.S.) and Eyes on the Future (U.K.), will contribute up to $1.6 million to support the development of OPGx-RDH12. This funding agreement includes a risk-sharing model with performance-based milestones aimed at achieving key clinical and regulatory objectives.

As part of the collaboration, Opus Genetics and the Alliance will co-develop the OPGx-RDH12 program, with a shared focus on advancing its clinical development strategy. The goal is to submit an Investigational New Drug (IND) application to the FDA by late 2025.

Addressing the Urgent Needs of the RDH12-LCA Community

Leber congenital amaurosis caused by RDH12 mutations is an ultra-rare inherited retinal disease (IRD) affecting several thousand individuals worldwide. The condition is characterized by early and severe visual acuity loss, often accompanied by retinal degeneration detectable by age two. Visual decline typically progresses rapidly during adolescence.

“This partnership represents a significant step forward,” said Mathew Pletcher, PhD, Board Member of the RDH12 Fund for Sight and parent of a child with RDH12-LCA. “By combining our community’s unique perspectives and resources with Opus’ gene therapy expertise, we can accelerate the transition of this promising therapy from the lab to the clinic.”

Mechanism and Promise of OPGx-RDH12

OPGx-RDH12 leverages an adeno-associated virus (AAV) vector to deliver a functional RDH12 gene directly to photoreceptor cells in the retina. According to Professor Jean Bennett, MD, PhD, Scientific Advisor and Board Member at Opus Genetics, preclinical studies in cell and animal models have shown restoration of RDH12 enzymatic activity and functional improvements in retinal health.