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OPGx 001

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Drug Profile

OPGx 001

Alternative Names: AAV8.hLCA5; Adeno-associated virus serotype 8 vector containing single-stranded DNA encoding the unmodified human lebercilin transgene - Opus Genetics; LCA5-LCA; OPGx-001; OPGX-LCA5

Latest Information Update: 16 Jan 2026

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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.

    Yes - Leber congenital amaurosis
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Leber congenital amaurosis

Most Recent Events

  • 08 Jan 2026 Opus Genetics plans a phase III trial in H2 2026
  • 06 Nov 2025 Opus Genetics plans accelerated approval pathway for OPGx 001
  • 06 Nov 2025 Opus Genetics plans to submit Rare Disease Evidence Principles (RDEP) review process application with the US FDA for OPGx 001

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