Drug Profile

NLX P101

Alternative Names: AAV-GAD; GAD gene therapy; NLX-P-101; STN AAV-GAD gene therapy

Latest Information Update: 19 May 2025

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At a glance

Originator Cornell University; Neurologix
Developer MeiraGTx
Class Antiparkinsonians; Gene therapies
Mechanism of Action GABA modulators; Glutamate modulators

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 Parkinson's disease

Most Recent Events

09 May 2025 NLX P101 receives Regenerative Medicine Advanced Therapy (RMAT) status for Parkinson's disease in USA
13 Mar 2025 MeiraGTx and Hologen enter into joint venture to develop AAV-GAD for Parkinson’s disease
15 Oct 2024 Efficacy and adverse events data from a phase I/II trial in Parkinson's Disease released by MeiraGTx

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