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GNT 0003

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Drug Profile

GNT 0003

Alternative Names: Adeno-associated Viral Vector expressing UGT1A1 transgene; GNT-0003; rAAV8-hUGT1A1

Latest Information Update: 09 Dec 2024

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At a glance

  • Originator Genethon
  • Developer Genethon; Hansa Biopharma AB
  • Class Gene therapies
  • Mechanism of Action Gene transference; UGT1A1 enzyme replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase II Crigler-Najjar syndrome

Most Recent Events

  • 08 Nov 2024 Phase-II clinical trials in Crigler-Najjar syndrome in France (IV) (NCT06518005)
  • 26 Jul 2024 Hansa Biopharma and Genethon plans a phase II trial for Crigler-Najjar syndrome (IV) in France (NCT06518005)
  • 27 Apr 2023 Hansa Biopharma and Genethon entered into research and development collaboration agreement for development of imlifidase as pre-treatment to gene therapy for the treatment of Crigler-Najjar syndrome

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