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An Open Label, Dose Exploration, Safety and Tolerability Study of a Subretinal Injection of an OPGx-001 Gene Vector to Participants With LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) With OCncurrent Non-Interventional Follow-Up of Untreated Patients

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Trial Profile

An Open Label, Dose Exploration, Safety and Tolerability Study of a Subretinal Injection of an OPGx-001 Gene Vector to Participants With LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) With OCncurrent Non-Interventional Follow-Up of Untreated Patients

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 21 Feb 2026

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At a glance

  • Drugs OPGx-001 (Primary)
  • Indications Leber congenital amaurosis
  • Focus Adverse reactions; First in man; Proof of concept; Therapeutic Use
  • Acronyms LCA5-IRD
  • Sponsors Opus Genetics

Most Recent Events

  • 19 Dec 2025 Planned number of patients changed from 15 to 22.
  • 02 Oct 2025 According to an Opus Genetics media release, company will present clinical results from this trial at the Cell and Gene Meeting on the Mesa, medical and industry conferences in October 2025.
  • 30 Sep 2025 According to an Opus Genetics media release, the company will host a webcast and conference call today at 8:30 a.m. Eastern Time to discuss the OPGx-LCA5 clinical trial data.

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