Key Takeaways

  • The FDA cleared Opus Genetics' IND application for OPGx-RDH12, enabling clinical development of the investigational gene therapy for RDH12-LCA

  • A phase 1/2 clinical trial is planned for the fourth quarter of 2026, supported by preclinical findings demonstrating restored RDH12 activity

  • The Global RDH12 Alliance has committed up to $1.6 million to support development through a patient advocacy–industry collaboration

Opus Genetics announced that the FDA has cleared its investigational new drug (IND) application for OPGx-RDH12, an investigational gene therapy for RDH12-associated Leber congenital amaurosis (RDH12-LCA), a rare inherited retinal disease characterized by progressive vision loss beginning in early childhood.

The clearance allows the company to advance OPGx-RDH12 into clinical development, with plans to initiate a phase 1/2 trial in the fourth quarter of 2026. The program represents Opus Genetics' third gene therapy candidate to enter clinical development.

OPGx-RDH12 is an adeno-associated virus (AAV)-based gene therapy designed to deliver a functional copy of the RDH12 gene directly to retinal photoreceptor cells. Preclinical studies in cell and mouse models demonstrated restoration of RDH12 activity and functional improvements, supporting further clinical investigation.

RDH12-LCA affects an estimated 30,900 individuals worldwide. Mutations in the RDH12 gene impair retinal protein function, resulting in early visual deterioration, often accompanied by structural retinal changes by age 2 and rapid disease progression during adolescence.

“We are pleased to advance our third gene therapy pipeline program into clinical development and plan to initiate a phase 1/2 trial this quarter,” said George Magrath, MD, Chief Executive Officer, Opus Genetics. “RDH12-LCA causes progressive vision loss beginning at a very young age, underscoring the urgency of developing treatments that may preserve vision before further deterioration occurs. Receiving IND clearance for OPGx-RDH12 represents an important step toward bringing a potentially transformative gene therapy to children and families affected by this devastating disease. The program is being developed in collaboration with the Global RDH12 Alliance, comprising the US-based RDH12 Fund for Sight and UK-based Eyes on the Future."

Under a strategic partnership established in July 2025, the alliance committed up to $1.6 million toward OPGx-RDH12 development, with funding tied to eligible development activities and milestones.

The collaboration also incorporates patient and family perspectives into the clinical and regulatory development strategy, with the goal of accelerating access to a potential treatment for RDH12-LCA.