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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: 20 May 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

  • 07 May 2026 Results published in the Media Release
  • 07 May 2026 According to an Opus Genetics media release, company presented data from this study at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2026 in Denver, Colorado.
  • 10 Mar 2026 According to an Opus Genetics media release, company will present Phase 1/2 six-month pediatric cohort data at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) conference in May 2026.

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