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An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia with Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

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Trial Profile

An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia with Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 18 Sep 2024

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At a glance

  • Drugs Timrepigine emparvovec (Primary)
  • Indications Choroideraemia
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms GEMINI
  • Sponsors Biogen; Nightstar Therapeutics; NightstaRx

Most Recent Events

  • 01 Aug 2024 Results assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia published in the Human Gene Therapy
  • 25 Jul 2022 Status changed from active, no longer recruiting to completed.
  • 24 Jun 2022 Planned End Date changed from 2 Jun 2022 to 29 Jun 2022.

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