Three gene therapy BLAs targeted by 2028, but OCU400 Phase 3 readout is the first test of whether mutation-agnostic RP therapy works at registrational scale

Ocugen is a clinical-stage gene therapy company developing treatments for inherited retinal diseases, with its lead program for retinitis pigmentosa not yet approved.

Ocugen is running three simultaneous gene therapy pivotal programs for inherited retinal diseases, with OCU400's Phase 3 liMeliGhT (retinitis pigmentosa, 140 subjects) the nearest readout to determine whether mutation-agnostic efficacy seen in Phase 1/2 translates to a registrational trial. The thesis resolves on whether OCU400 meets its primary endpoint and OCU410 replicates Phase 2's 31% lesion reduction in 237 subjects. Cash of $100M extends into 2028, covering all three readouts, but the convertible debt load and absence of revenue mean any clinical miss forces dilutive financing.