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] Company Developments - 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 individuals in the same regions [2][3] - The company has completed dosing for Phase 2 of the OCU410 ArMaDa clinical trial and plans to initiate Phase 3 trials next year, with potential regulatory submissions expected in 2028 [3] - The FDA has endorsed Ocugen's plan for a Phase 2/3 pivotal trial for OCU410ST, with potential submissions for marketing authorization anticipated in 2027 [3] Clinical Trial Results - Preliminary data from the Phase 1 trial of OCU410 showed a 44% reduction in lesion growth and a clinically meaningful improvement in visual function in treated eyes compared to untreated eyes [4] - Phase 1 data for OCU410ST indicated a 52% reduction in lesion growth and a statistically significant improvement in visual function [5] Technology and Innovation - Both OCU410 and OCU410ST utilize an adeno-associated virus platform for gene delivery, targeting multiple pathways associated with dry age-related macular degeneration and Stargardt disease [6] - The RORA gene, delivered via a single subretinal injection, plays a crucial role in lipid metabolism and has demonstrated anti-inflammatory properties [6] Company Overview - Ocugen, Inc. focuses on developing innovative gene and cell therapies, biologics, and vaccines to address unmet medical needs and improve patient health globally [7]