Ocugen Clinical Trial Could Lead to Breakthrough One-Time Treatment for Advanced Dry Macular Degeneration

NORTHAMPTON, Ma., Dec. 19, 2023 — The American Macular Degeneration Foundation received news that the first patient has been dosed in a safety and efficacy clinical trial of a one-time gene therapy treatment for advanced dry macular degeneration (dry AMD), also known as Geographic Atrophy (GA).

What’s exciting about this trial is that, if successful, OCU410 is a “potential one-time treatment for life,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen, Inc. 

In other words, curative.

How Does OCU410 Work?

The development of OCU410 is based on the AMDF-supported research of Dr. Neena Haider. It is designed to prevent or halt AMD progression, and possibly to restore some vision if retinal cells are not damaged beyond recovery. OCU410 is a potential curative therapy, administered with a single sub-retinal injection, that modulates hundreds of genes and multiple pathways that are involved in dry AMD, including lipid metabolism, inflammation, oxidative stress, and complement activation. 

Currently available therapeutic options target only complement activation and require monthly or bi-monthly intravitreal injections.

What to Expect Next

While this news is very exciting, there are still several tests and steps before a treatment like this could be available to patients as a treatment option. Not all potential treatments that enter into clinical trials prove safe enough or effective enough to pass FDA approval. But we’ll be keeping an eye on this.

In the meantime, this news is all the more reason to make AMD-healthy lifestyle choices to help preserve your vision while curative therapies are on their way!  

“The fact that we are testing this game changing gene therapy in a human being is testament to the focused effort of my lab in identifying and developing a therapy that has a strong, broad impact. The National Eye Institute supported my early career as I sought to demonstrate this radically different approach to gene therapy. Our preclinical AMD work that informed these clinical trials was accomplished with combined support from Ocugen and the American Macular Degeneration Foundation (AMDF),” said Dr. Haider.  “The AMDF has provided the sustained support needed at many critical junctures in this journey, which was essential in creatively developing innovative solutions for this common and debilitating form of vision loss, and at times was crucial in keeping the lab open.  Ocugen is now spearheading the clinical trials necessary to bring this therapy closer to patients. Successful outcomes will provide tremendous hope and help to those whose vision is already deteriorating, and will prevent others from developing this debilitating condition. ”

About Dry Macular Degeneration and Geographic Atrophy

Dry AMD involves the slow deterioration of the retina with sub-macular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. Dry AMD accounts for 85-90% of the total AMD population.

Geographic Atrophy is an advanced form of dry age-related macular degeneration characterized by dead spots near and on the macula, affecting approximately 1 million people in the United States alone with vision loss.

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Read Ocugen’s press release here: https://ir.ocugen.com/news-releases/news-release-details/ocugen-inc-announces-first-patient-dosed-phase-12-clinical-1