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OCU 400

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Drug Profile

OCU 400

Alternative Names: AAV-NR2E3; AAV5-hNR2E3; NR2E3-AAV; OCU-400

Latest Information Update: 12 Mar 2026

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At a glance

  • Originator Schepens Eye Research Institute
  • Developer Ocugen; Schepens Eye Research Institute
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference; NR2E3 protein replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Retinitis pigmentosa; Leber congenital amaurosis; Retinal disorders
  • New Molecular Entity No

Highest Development Phases

  • Phase III Retinitis pigmentosa
  • Phase I/II Leber congenital amaurosis
  • No development reported Retinal disorders

Most Recent Events

  • 04 Mar 2026 Ocugen expects potential approval of BLA in 2027
  • 04 Mar 2026 Ocugen plans to submit BLA for Retinitis pigmentosa in third quarter of 2026
  • 02 Mar 2026 Efficacy and adverse events data from a phase I/II trial in Leber congenital amaurosis released by Ocugen

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