Drug Profile
GNT 0003
Alternative Names: Adeno-associated Viral Vector expressing UGT1A1 transgene; GNT-0003; rAAV8-hUGT1A1Latest Information Update: 07 Feb 2024
Price :
$50
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At a glance
- Originator Genethon
- Class Gene therapies
- Mechanism of Action Gene transference; UGT1A1 enzyme replacements
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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 Crigler-Najjar syndrome
Most Recent Events
- 02 Feb 2024 Hansa Biopharma and Genethon plans a phase I/II trial for Crigler-Najjar syndrome (IV) in 2024
- 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
- 07 Mar 2023 GNT 0003 receives Priority Medicine (PRIME) status for Crigler-Najjar syndrome in European Union