NGN 101
Alternative Names: NGN-101Latest Information Update: 09 Jan 2024
At a glance
- Originator Neurogene Inc
- Class Gene therapies
- Mechanism of Action Ceroid-lipofuscinosis neuronal protein 5 replacements; Gene transference
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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 Neuronal ceroid lipofuscinosis
Most Recent Events
- 05 Jan 2024 Neurogene plans a clinical/regulatory strategy meeting with the US FDA to discuss registrational pathway for NGN 101 for Neuronal ceroid lipofuscinosis in second half of 2024
- 31 Dec 2023 Neurogene in-license four pending patent applications from the University of North Carolina at Chapel Hill for NGN 101 Worldwide
- 08 Feb 2022 Phase-I/II clinical trials in Neuronal ceroid lipofuscinosis (In children) in USA (Intraventricular) (NCT05228145)