Ocugen presents positive OCU400 clinical data: 84% less atrophy area in treated vs untreated

Ocugen, Inc.

OCU400 (AAV encoding NR2E3) shows restoration of phototransduction genes and improved retinal structure in multiple RP/LCA models.
Mean change in total atrophy area from baseline: +0.12 mm² treated vs +0.73 mm² untreated (84% improvement).
LL VA change from baseline: treated patients improved while untreated declined over 12 months.
Preclinical ERG and histology data show slowed degeneration across five animal models (Rho-/-, RhoP23H+/-, Rd7, Rd1, Rd16).

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