Opus Genetics has Type B RMAT meeting with FDA for its gene therapy candidate, OPGx-LCA5

Opus Genetics has completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the US Food and Drug Administration (FDA) regarding OPGx-LCA5, its gene therapy candidate for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene.

OPGx-LCA5 is designed to address a form of Leber congenital amaurosis that results from biallelic mutations in the LCA5 gene, which encodes the lebercilin protein, the investigators explained.

According to the company, the meeting provided feedback from the FDA on key elements of its registration strategy, including Chemistry, Manufacturing, and Controls (CMC), as well as pivotal trial design.

George Magrath, MD, CEO of Opus Genetics, commented on the meeting and the current phase 1/2 trial saying, “We expect to be able to advance our ongoing trial using an adaptive design that includes a phase 3 portion, which will avoid the requirement for a separate registrational trial. Given the severe nature of the disease, we are actively identifying patients who may qualify for phase 3 and enrolling them into the planned run-in period to monitor their disease. This productive RMAT interaction represents an important milestone as we continue working closely with the FDA to bring sight-restoring gene therapies to patients who currently have no approved treatment options.”

The trial is a phase 1/2 open-label, ascending-dose study of the safety and efficacy of OPGx-LCA5. Recently, the company released positive 3-month pediatric data, showing significant measurable gains in vision in teenagers and maintenance of visual gains to 18 months in adults who were treated earlier.

The company noted it will incorporate the FDA’s feedback into its updated clinical development and CMC plans for the phase 3 portion of the study to include “enrolling as few as 8 participants in a single-arm, 12-month study utilizing an adaptive design.” The company also stated that it expects the phase 3 portion of the trial to include a run-in period prior to dosing to evaluate the natural history of each participant to serve as their own control in the study. The company anticipates dosing to start in the second half of 2026, with topline results following a year later.

Additionally, the company announced its intent to apply for the Rare Disease Evidence Principles (RDEP) review process from the FDA. The process facilitates the approval of drugs to treat rare diseases with very small patient populations with significant unmet medical needs and with a known genetic defect that is the major driver of the pathophysiology.

References:

1. Opus Genetics announces successful FDA meeting supporting advancement of OPGx-LCA5 toward pivotal trial for LCA5-related inherited retinal disease. Published November 6, 2025. Accessed November 7, 2025. https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-presents-two-year-natural-history-data

2. Charters L. Positive pediatric data emerge from the OPGx-LCA5 phase 1/2 trial of Leber congenital amaurosis type 5. Published October 1, 2025. Accessed November 7, 2025. https://www.ophthalmologytimes.com/view/positive-pediatric-data-emerge-from-the-opgx-lca5-phase-1-2-trial-of-leber-congenital-amaurosis-type-5