December 21, 2023

OCUGEN GAINS FDA ALIGNMENT ON KEY ASPECTS OF OCU400—MODIFIER GENE THERAPY—PIVOTAL PHASE 3 STUDY DESIGN

On December 21, 2023, Ocugen released a press statement titled, "Ocugen Gains FDA Alignment on Key Aspects of OCU400-Modifier Gene Therapy-Pivotal Phase 3 Study Design." One of Ocugen’s missions is to bring gene-agnostic therapies for retinitis pigmentosa (RP) to market, providing global access to patients. They reported that, after meeting with the FDA during an ongoing Phase 1/2 study, they received insights for their Phase 3 study design. These insights included the study endpoint, a broader patient enrollment strategy for RP patients, and a study duration of one year.

What this means for Usher syndrome: Since OCU400 is considered a gene-agnostic treatment, it can potentially be used by anyone with RP regardless of the genetic cause, including individuals with Usher syndrome.

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