Ocugen Receives DSMB Approval for Medium Dose Enrollment in Phase 1/2 Trial of OCU410 Gene Therapy for Geographic Atrophy

Ocugen, Inc., a biotech firm specializing in innovative gene and cell therapies and vaccines, has received approval from the Data and Safety Monitoring Board (DSMB) to advance to the medium dose cohort in the Phase 1/2 clinical trial for OCU410, a potential treatment for geographic atrophy (GA). The DSMB's decision follows the safe and tolerable low dose administration in the trial, with no serious adverse events reported. 

Dr. Peter Chang, DSMB Chair for the OCU410 trial, highlighted the significance of this step towards establishing the optimal dosing strategy for OCU410. The company's Chief Medical Officer, Dr. Huma Qamar, expressed optimism about OCU410's potential as a one-time treatment administered via a single sub-retinal injection. 

Currently, treatments for GA are limited, necessitating frequent injections and targeting only one disease pathway. In contrast, OCU410 addresses multiple disease-related pathways, including lipid metabolism, inflammation, oxidative stress, and the complement system. The ArMaDa trial is divided into two phases: Phase 1, an open-label dose-ranging study with three levels, and Phase 2, a randomized, dose-expansion study with subjects allocated to treatment or control groups. GA, an advanced form of dry age-related macular degeneration (dAMD), impacts around 1 million people in the U.S., and dAMD affects approximately 10 million Americans and over 266 million individuals globally. 

OCU410 employs an AAV delivery system for the RORA gene, which has shown promise in targeting various pathways associated with dAMD. Ocugen is dedicated to developing solutions for inherited retinal diseases and conditions causing blindness, with a focus on gene therapy for GA treatment. The company's modifier gene therapy platform aims to treat multiple retinal diseases with a single product and is also exploring treatments for infectious and orthopedic diseases. 

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