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Source document· March 2, 2026

Ocugen Announces Phase 3 liMeliGhT Enrollment Completion for OCU400, a Novel Modifier Gene Therapy for Broad Retinitis Pigmentosa

View original at globenewswire.com
Ocugen Announces Phase 3 liMeliGhT Enrollment Completion for OCU400, a Novel Modifier Gene Therapy for Broad Retinitis Pigmentosa Enrollment for liMeliGhT, the first and largest gene therapy registrational trial for broad retinitis pigmentosa (RP) patients, was completed, reflecting strong interest from investigators a…
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  • Approximately 98% of people living with RP are not candidates for the approved gene therapy for RP, representing significant treatment gap

    80% confidence
  • Current gene replacement therapies are limited to treating just a single mutation and may not eliminate underlying genetic defect

    80% confidence
  • There are no approved treatment options that slow or stop progression of multiple forms of RP

    80% confidence
  • OCU400 is designed to reset dysfunctional gene network in RP patients to reestablish healthy cellular homeostasis, which has potential to improve vision

    80% confidence
  • Dedication of investigators and clinical teams has potential to shift treatment paradigm for RP by targeting multiple genetic mutations with single therapeutic approach

    80% confidence
  • Enrollment completion brings Ocugen closer to potentially delivering first novel modifier gene therapy candidate to market and providing one-time treatment for life to hundreds of thousands of RP patients globally with unmet medical need

    80% confidence
  • Enthusiastic about possibility of offering RP patients a safe, effective and durable treatment option that could potentially stabilize vision loss or improve vision

    80% confidence
  • Novel therapeutic approaches targeting broader RP disease in gene-agnostic manner offer additional hope for patients, especially when multiple and unknown genes are involved

    80% confidence
  • The Phase 3 liMeliGhT clinical trial patient response will support the gene-agnostic mechanism of action of Ocugen's novel modifier gene therapy platform

    80% confidence
Ocugen Announces Phase 3 liMeliGhT Enrollment Completion for OCU400, a Novel Modifier Gene Therapy for Broad Retinitis Pigmentosa — Source | Via News | Via News