Ocugen, Inc. products

OCU400 (AAV-NR2E3) is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases (“IRDs”). It consists of a functional copy of a nuclear hormone receptor (“NHR”) gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral (“AAV”) vector.  As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration.

Ocugen and Bharat Biotech to Co-Develop COVAXIN™, a Whole-Virion Inactivated COVID-19 Vaccine, for the US Market.

OCU410 (AAV-RORA) is a modifier gene therapy product candidate being developed for the treatment of dry age-related macular degeneration (Dry AMD). OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. Various genes associated with AMD are regulated by RORA. The RORA protein plays an important role in lipid metabolism and demonstrates an anti-inflammatory role, which we believe could be a potential therapeutic candidate for dry AMD.

OCU200 is a biologic product candidate in preclinical development for treating severely sight-threatening diseases like Diabetic Macular Edema (DME), Diabetic Retinopathy (DR), and Wet Age-Related Macular Degeneration (Wet-AMD). Patients affected by these diseases share common symptoms, such as blurriness in vision and progressive vision loss as the disease progresses. The formation of fragile and leaky new blood vessels leads to fluid accumulation in and around the retina, causing damage to vision.

Ocugen’s modifier gene therapy platform is a cutting-edge technology licensed from the Schepens Eye Research Institute of Massachusetts Eye and Ear (Harvard Medical School) and involves targeted delivery and expression of one or more nuclear hormone receptor genes (NHRs) in the disease tissues.  NHRs play a vital role in regulating retinal cell development, maturation, metabolism, visual cycle function, survival, and maintaining the cellular and molecular homeostatic various tissues, including the retina.  Multiple animal models have shown that expression of NHRs within the retina can stabilize retinal cells and suppress/prevent the development of inherited diseases, demonstrating their modifier function.