Ocugen Completes Dosing in Phase 1/2 GA Trial for OCU410 Gene Therapy

Ocugen, a biotechnology firm, has completed dosing in the initial group of its Phase 1/2 ArMaDa clinical trial for OCU410, a gene therapy being developed to treat geographic atrophy (GA), a condition that affects around 1 million people in the United States. GA is a severe form of dry age-related macular degeneration (dAMD), which impacts the macula, the part of the retina responsible for clear central vision.

Dr. Shankar Musunuri, the top executive at Ocugen, expressed optimism about OCU410's potential as a single-dose, long-term treatment delivered via a sub-retinal injection. He noted that while two approved treatments for GA exist, they require frequent intravitreal injections and only address the complement system. In contrast, OCU410 is designed to target a range of pathways associated with dAMD, including inflammation, oxidative stress, and lipid metabolism.

The clinical trial, which is being conducted at 13 top retinal surgery centers across the U.S., has enrolled its first group of three subjects who have received a low dose of OCU410. Lejla Vajzovic, a retinal surgeon and professor at Duke University Eye Center, highlighted the significance of OCU410 as a novel gene therapy that could revolutionize ophthalmology.

The ArMaDa trial is divided into two phases. Phase 1 is an open-label, dose-ranging study with three levels of dosage, and Phase 2 is a randomized study that includes an untreated control group. The trial aims to evaluate the safety of OCU410's unilateral subretinal administration.

The American Macular Degeneration Foundation (AMDF) has supported the research behind OCU410 and is encouraged by Ocugen's progress in advancing the therapy. The AMDF believes that OCU410 could preserve vision for those with deteriorating sight and potentially prevent vision loss in early-stage dAMD patients.

dAMD, which affects around 10 million Americans and over 266 million people globally, is characterized by the gradual degeneration of the retina. It accounts for 85-90% of all age-related macular degeneration cases.

OCU410 is based on an AAV delivery system for the RORA gene, which plays a crucial role in lipid metabolism and reducing oxidative stress. The gene therapy has shown anti-inflammatory properties in both in-vitro and in-vivo studies, indicating its potential to address the multiple pathways linked to dAMD.

Ocugen is a biotechnology company dedicated to discovering, developing, and commercializing innovative therapies and vaccines to improve global health. The company is committed to pioneering scientific advancements and addressing unmet medical needs in various disease areas, including retinal diseases, infectious diseases, and orthopedic conditions.