Ocugen Announces Positive Opinion of EMA’s Committee for Advanced Therapies for ATMP Classification for Novel Modifier Gene Therapy Candidate OCU410 for Geographic Atrophy and OCU410ST for Stargardt Disease

Core Insights - Ocugen, Inc. has received a positive opinion from the European Medicines Agency's Committee for Advanced Therapies for its gene therapy candidates OCU410 and OCU410ST, aimed at treating vision loss due to geographic atrophy and Stargardt disease respectively [1][3] Group 1: Product Development and Clinical Trials - OCU410 is designed for patients with geographic atrophy, affecting 2-3 million people in the U.S. and Europe, while OCU410ST targets Stargardt disease, which affects approximately 100,000 people in the same regions [2] - The Phase 2 clinical trial for OCU410 has been completed, with plans to initiate a Phase 3 trial next year, targeting Marketing Authorization Application and Biologics License Application filings in 2028 [3] - Preliminary data from the Phase 1 trial of OCU410 showed a 44% reduction in lesion growth and a clinically meaningful improvement in visual function [4] - The Phase 1 trial data for OCU410ST indicated a 52% reduction in lesion growth and a statistically significant improvement in visual function [5] Group 2: Mechanism and Technology - Both OCU410 and OCU410ST utilize an adeno-associated virus platform to deliver the RORA gene, which plays a crucial role in lipid metabolism and has anti-inflammatory properties [6] - The gene therapy is administered through a single subretinal injection, offering a potential one-time treatment solution [6] Group 3: Company Overview - Ocugen, Inc. focuses on developing innovative gene and cell therapies, biologics, and vaccines to address unmet medical needs globally [7]