globenewswire.com · Nov 6, 2025
Opus Genetics Announces Successful FDA Meeting Supporting Advancement of OPGx-LCA5 Toward Pivotal Trial for LCA5-Related Inherited Retinal Disease
Outcome of Regenerative Medicine Advanced Therapy (RMAT) meeting provides the potential for an accelerated regulatory pathway to approval of OPGx-LCA5 First participant enrolled in run-in period for planned adaptive Phase 3 trial Company intends to apply for the FDA's new Rare Disease Evidence Principles (RDEP) review process OPGx-LCA5 has the potential to be the first gene therapy and one-time treatment for Leber congenital amaurosis (LCA) type 5 Recent $23 million financing led by Perceptive Advisors and Balyasny Asset Management to advance LCA5 and BEST1 programs and fund current operating plans into second half of 2027 RESEARCH TRIANGLE PARK, N.C., Nov. 06, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs) and small-molecule therapies for other ophthalmic disorders, today announced the successful completion of a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the U.S. Food and Drug Administration (FDA) regarding OPGx-LCA5, its gene therapy candidate for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene.
