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Ocugen Inc company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. The company's pipeline includes Modifier Gene Therapy Platform, Novel Biologic Therapy for Retinal Diseases, Regenerative Medicine Cell Therapy Platform, Inhaled Mucosal Vaccine Platform. The company is developing a modifier gene therapy platform designed to fulfill unmet medical needs related to retinal diseases, including inherited retinal diseases ("IRDs"), such as RP, LCA, Stargardt disease, and multifactorial diseases such as dAMD and Geographic Atrophy ("GA").
Neurogene Inc is a clinical-stage biotechnology company committed to overcoming the limitations of neurological diseases into treatable conditions, by harnessing its proprietary transgene regulation technology, EXACTTM (Expression Attenuation via Construct Tuning). It is building a differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet needs. The drug candidates in the company's product pipeline include NGN-401, which is in development for the treatment of Rett syndrome, using its EXACT platform, and NGN-101, a non-EXACT, conventional gene therapy development candidate for the treatment of CLN5 Batten disease, designed to deliver the CLN5 gene and packaged in an AAV9 capsid.