Ocugen, Inc. announced that the first patient has been dosed in the ArMaDa Phase 1/2 clinical trial of OCU410 (AAV-RORA), a modifier gene therapy product candidate being developed for dry AMD (dAMD). This Phase 1/2 trial will assess the safety and efficacy of OCU410 for geographic atrophy (GA) secondary to dAMD and will be conducted in two phases. Phase 2 is a randomized expansion phase in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 dose groups or to an untreated control group.

OCU410 is a potential curative therapy with a single sub-retinal injection that targets multiple pathways causing dAMD, including lipid metabolism, inflammation, oxidative stress, and complement activation. Currently, the other therapeutic options available target only complement activation and require approximately 6-12 intravitreal injections annually. The macula is the part of the retina responsible for clear vision in one's direct line of sight.

dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. dAMD accounts for 85-90% of the total AMD population. GA, an advanced form of dry age-related macular degeneration, affects approximately 1 million people in the United States alone.

Ocugen utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology.

Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA. Currently, the other therapeutic option available target only complement activation and requiring approximately 6-12 intrav it annually. The macula is The part of the retina responsible for Clear vision in one's direct line the retina involves the slow deterioration of the retinal retina with submacular drillusen (small white or yellow dots on the retina), atrophy, loss of Macular function and central vision impairment; dAMD accounts for 85- 90% of the totalAMD population.GA, an advanced form of dry Age-related macular degeneration.

affects approximately 1 million people in The United States alone.OCU410 utilizes an AAV delivery platform For the retinal delivery of theRORA (RAR RelatedOrphan Receptor A) gene; The RORA protein plays a important role in lipid metabolism, reduce lipofuscin deposits and oxidation stress, and demonstrates an anti -inflammatory role in-vivo (animal model) studies. These results demonstrated the ability for OCU410 To target multiple pathways linked with d AMD pathophysiology. Ocug is developing AAV-ROR A as a one-time gene Therapy for the treatment of GA.

currently, the other therapeutic options available targets only complement activation and require approximately6-12 intravitreal injection annually.