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

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• 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

• 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

• Approximately 98% of people living with RP are not candidates for the approved gene therapy for RP, representing significant treatment gap

  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

• 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

• 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

• 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