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OPGx 001

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Drug Profile

OPGx 001

Alternative Names: AAV8.hLCA5; OPGx-001; OPGX-LCA5

Latest Information Update: 22 Aug 2024

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At a glance

  • Originator University of Pennsylvania
  • Developer Opus Genetics
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference; LCA5 protein replacements
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Leber congenital amaurosis

Most Recent Events

  • 21 Aug 2024 OPGx 001 receives rare paediatric disease designation for Leber congenital amaurosis in USA
  • 15 Jun 2023 Phase-I/II clinical trials in Leber congenital amaurosis in USA (Subretinal) (NCT05616793)
  • 01 Dec 2022 Opus submits IND application with the US FDA for a phase I/II trial in Leber congenital amaurosis in USA, prior to December 2022

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