GNT 0003

Drug Profile

GNT 0003

Alternative Names: Adeno-associated Viral Vector expressing UGT1A1 transgene; GNT0003; rAAV8-hUGT1A1

Latest Information Update: 27 Oct 2017

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At a glance

  • Originator Genethon
  • Developer Genethon; Spark Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Liver disorders

Most Recent Events

  • 09 Oct 2017 Phase-I/II clinical trials in Liver disorders (In children, In adolescents, In adults, In the elderly) in Netherlands (IV) (EudraCT2017-000506-37)
  • 20 Sep 2017 Spark Therapeutics and Genethon agree to co-promote and co-develop adeno-associated viral gene therapy for Liver disorders
  • 20 Sep 2017 Early research in Liver disorders in USA, France (Parenteral)
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