FDA clears Opus Genetics IND for RDH12 childhood blindness therapy

Opus Genetics, Inc. (Nasdaq: IRD) announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug application for OPGx-RDH12, a gene therapy candidate targeting RDH12-associated Leber congenital amaurosis, a rare inherited retinal disease that causes progressive vision loss beginning in early childhood.

The company said it plans to initiate a Phase 1/2 clinical trial during the current quarter. OPGx-RDH12 is an adeno-associated virus-based therapy designed to deliver a functional copy of the RDH12 gene to photoreceptor cells in the retina. The condition is estimated to affect approximately 30,900 people globally, with structural retinal changes often appearing by age two and rapid progression during adolescence.

The IND clearance follows a strategic collaboration established in July 2025 between Opus Genetics and the Global RDH12 Alliance, which comprises patient advocacy organizations RDH12 Fund for Sight in the United States and Eyes on the Future in the United Kingdom. Under that agreement, the Global RDH12 Alliance committed up to $1.6 million toward development of OPGx-RDH12, with eligible costs reimbursed as activities are completed subject to certain milestones and diligence obligations.

Preclinical studies in cell and mouse models showed restoration of RDH12 activity and functional improvements, according to the company's statement.

"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, M.D., Chief Executive Officer of Opus Genetics.

Opus Genetics is based in Research Triangle Park, N.C., and is developing seven AAV-based programs targeting inherited retinal diseases. OPGx-RDH12 joins OPGx-LCA5 and OPGx-BEST1 as programs that have entered clinical development, with additional candidates targeting MERTK, RHO, CNGB1, and NMNAT1 in the pipeline.

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