NGN 401
Alternative Names: NGN-401Latest Information Update: 12 Aug 2024
At a glance
- Originator Neurogene Inc
- Class Gene therapies
- Mechanism of Action Gene transference; Methyl-CpG-binding protein 2 gene 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 Rett syndrome
Most Recent Events
- 12 Aug 2024 The European Medicines Agency grants Advanced therapy medicinal product designation for Rett syndrome in European Union
- 07 Aug 2024 The United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) grants Innovative Licensing and Application Pathway (ILAP) designation for Rett syndrome in United Kingdom
- 18 Jun 2024 Adverse events data from a phase I/II trial in Rett syndrome released by Neurogene