MALVERN, PA — Ophthalmology is on the crest of a wave; a paradigm shift heralded by Ocugen, Inc (NASDAQ: OCGN) may be imminent. The Pennsylvania-based biopharmaceutical firm recently crossed a crucial milestone in its Phase 1/2 ArMaDa clinical trial for its candidate modifier gene therapy, OCU410 (AAV-hRORA), targeting geographic atrophy (GA)—the bane of approximately one million American adults.
GA, an advanced stage of dry age-related macular degeneration (dAMD), has, until now, had limited treatment options. But scientists and industry watchers alike are keeping a keen eye on Ocugen’s promising developments, which CEO Dr. Shankar Musunuri believes could change the therapeutic game with a ‘one-time, single sub-retinal injection’ treatment approach.
Dr. Musunuri stated, “While there are currently two recently approved products for the treatment of GA, both require around six to twelve intravitreal injections annually and only target the complement system.” What sets OCU410 apart is its focus on a broad array of dAMD triggers, including lipid metabolism, inflammation, oxidative stress, and the complement system—the biological pathway that augments the body’s ability to clear microbes and damaged cells from an organism, promoting inflammation and attacking the pathogen’s cell membrane.
This ambitious trial, literally banking on a single shot, is now well underway in the United States, across 13 leading retinal surgery centers. Having completed dosing the first participant cohort, a triumvirate of trial subjects received a 200µL single subretinal administration of the low-dose OCU410.
The ArMaDa clinical trial comprises two phases. The ongoing Phase 1 is a multicenter, open-label, dose-ranging study incorporating three dose concentrations: low, medium, and high. The subsequent Phase 2 will proceed as a randomized, outcome accessor-blinded, dose-expansion study, where the participants will be randomly assigned to either one of two OCU410 treatment groups or to an untreated control group.
Lejla Vajzovic, MD, FASRS, Director of Duke Surgical Vitreoretinal Fellowship Program, said, “OCU410 is a novel modifier gene therapy approach that could initiate a paradigm shift in the field of ophthalmology.”
OCU410’s notable progress has been lauded not just by physicians but also by prominent industry bodies such as the American Macular Degeneration Foundation (AMDF). Matthew Levine, Director of Grants, Advocacy and Partnerships at AMDF, stated, “The continued advancement of OCU410 offers hope to those whose vision is already deteriorating that their remaining vision could be preserved and could potentially prevent others with an early dAMD diagnosis from developing any significant vision loss.”
Indeed, Ocugen’s therapeutic innovation could be a game-changer for those afflicted by GA. This progress is not just about a new product on the market but a testament to Ocugen’s commitment to exploring the hitherto largely uncharted territory of gene therapy for GA, giving hope to millions globally.
While industry watchers, physicians, and patients alike eagerly await further clinical updates, one thing remains apparent: as Ocugen continues its transformative work, the optics are bright for those hoping for a future where GA is less a debilitating certainty and more a treatable condition.
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