Ocugen is a clinical-stage biotechnology company developing gene therapies for inherited and degenerative retinal diseases. The company's core platform is a "modifier gene therapy" technology licensed from the Schepens Eye Research Institute, which delivers a nuclear hormone receptor gene to the retina to act as a master regulator across entire gene networks. Unlike conventional gene therapies that replace a single defective gene and thus treat only patients with that specific mutation, Ocugen's approach is designed to be mutation-agnostic, meaning a single product can potentially treat patients across a wide range of mutations. Ocugen has three lead programs: OCU400 for retinitis pigmentosa (Phase 3 enrollment complete, BLA submission targeted Q3 2026), OCU410ST for Stargardt disease (Phase 2/3 enrolling, BLA targeted H1 2027), and OCU410 for geographic atrophy, the advanced form of dry AMD (Phase 3 planned for 2026, BLA targeted 2028). All three are designed as one-time therapies delivered via a single subretinal injection. Ocugen plans to commercialize directly in the U.S. and Europe, and license rights in smaller geographies to regional partners. Manufacturing is handled by CanSinoBIO, which holds commercialization rights in China and surrounding territories in exchange for supplying clinical and commercial product. Ocugen has no approved products or commercial revenue today, and funds operations through equity raises and debt.
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