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An Open-label, Phase 2 Trial to Evaluate the Efficacy and Safety of a Single Intravenous Administration of GNT0003 (an Adeno-associated Viral (AAV) Vector Expressing the UGT1A1 Transgene) Following Imlifidase Pre-treatment in Adult Participants With Severe Crigler-Najjar Syndrome (CNS) Requiring Daily Phototherapy and Presenting Pre-existing Anti-AAV8 Antibodies

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Trial Profile

An Open-label, Phase 2 Trial to Evaluate the Efficacy and Safety of a Single Intravenous Administration of GNT0003 (an Adeno-associated Viral (AAV) Vector Expressing the UGT1A1 Transgene) Following Imlifidase Pre-treatment in Adult Participants With Severe Crigler-Najjar Syndrome (CNS) Requiring Daily Phototherapy and Presenting Pre-existing Anti-AAV8 Antibodies

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 14 Jul 2026

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At a glance

  • Drugs GNT 0003 (Primary) ; Imlifidase (Primary)
  • Indications Crigler-Najjar syndrome
  • Focus Therapeutic Use
  • Sponsors Genethon

Most Recent Events

  • 10 Oct 2025 According to a Genethon media release, company announced that patient has been successfully treated with Genethon's AAV-based GNT0003 gene therapy for Crigler-Najjar syndrome, following prior administration of imlifidase, encouraging result, achieved in a clinical trial, is a significant advance in the treatment of patients with immunity to AAVs who were previously ineligible for clinical trials and existing gene therapy treatments.
  • 10 Oct 2025 According to a Genethon media release, data from this study presented at the 2025 congress of the European Society of Gene & Cell Therapy (ESGCT),
  • 01 Oct 2025 According to a Genethon media release, data from this study will be presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), From October 7 to 10, 2025.

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