Core Insights - Opus Genetics has successfully completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the FDA regarding its gene therapy candidate OPGx-LCA5 for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene [1][2][8] - The FDA provided constructive feedback on Opus's registration strategy, including Chemistry, Manufacturing and Controls (CMC), and pivotal trial design, acknowledging the significant unmet medical need for individuals with LCA5-related blindness [2][3] - The company plans to advance its ongoing trial using an adaptive design that includes a Phase 3 portion, which will avoid the need for a separate registrational trial [3][4] Clinical Development - Six late-stage participants have been treated in the Phase 1/2 trial of OPGx-LCA5, with all experiencing clinically meaningful improvements in vision [3][9] - The Phase 3 trial will include a run-in period to evaluate the natural history of each participant, serving as their own control [5][4] - Efficacy and safety will be assessed using various measures, including visual acuity and microperimetry, with dosing anticipated in the second half of 2026 [5][8] Financial Position - Opus Genetics has secured recent financing of $23 million, which will support the advancement of its LCA5 program and fund current operating plans into the second half of 2027 [6][8] Regulatory Environment - The FDA's introduction of the Rare Disease Evidence Principles (RDEP) review process aims to facilitate the approval of drugs for rare diseases, and Opus believes its LCA5 program meets the eligibility criteria for this process [7][8] - OPGx-LCA5 has the potential to be the first gene therapy and one-time treatment for LCA type 5, addressing a significant unmet medical need [8][11] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small-molecule therapies for other ophthalmic disorders [12] - The company's pipeline includes AAV-based gene therapies targeting conditions such as LCA and bestrophinopathy, with OPGx-LCA5 currently in a Phase 1/2 clinical trial [12][9]