Ocugen Completes Dosing in Phase 1/2 GA Trial's Cohort 1 with OCU410

Biotechnology firm Ocugen, Inc. has announced the completion of dosing for the initial group in its Phase 1/2 clinical trial for OCU410, a gene therapy candidate for geographic atrophy (GA), a severe form of dry age-related macular degeneration (dAMD). The trial, known as ArMaDa, is set to evaluate the safety and efficacy of a single sub-retinal injection of OCU410, which is designed to address various pathways associated with dAMD, such as complement, lipid metabolism, inflammation, and oxidative stress. In contrast to current treatments for GA, which require frequent intravitreal injections, OCU410 is intended to provide a one-time treatment solution.
Dr. Shankar Musunuri, CEO of Ocugen, expressed optimism about OCU410's potential to revolutionize treatment for GA with its multi-pathway approach. The clinical trial involves 13 prominent retinal surgery centers across the U.S., with the first cohort having completed enrollment and received a low dose of the therapy.
Lejla Vajzovic, MD, a retinal surgeon and director at Duke University Eye Center, highlighted the significance of gene therapy like OCU410, which could offer long-term benefits and potentially alter the current treatment paradigm in ophthalmology.
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 a control group. The American Macular Degeneration Foundation (AMDF) has supported the research behind OCU410 and is encouraged by the progress of the clinical trials, which could offer hope to millions of patients with deteriorating vision.
dAMD affects around 10 million Americans and over 266 million people globally, characterized by the gradual loss of central vision due to macular thinning. GA, which impacts about 1 million people in the U.S., is a significant cause of vision loss in those over 60.
OCU410 is based on an AAV delivery system to introduce the RORA gene into the retina, which is crucial for lipid metabolism and reducing oxidative stress. Ocugen is developing this gene therapy with the aim of providing a one-time treatment for GA.
Ocugen, Inc. is dedicated to developing innovative therapies and vaccines to improve global health. The company's modifier gene therapy platform aims to treat various retinal diseases and is also advancing research in infectious and orthopedic diseases.

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