We are reimagining a future that is filled with treatments for patients and families affected by rare neurological diseases.
Our most advanced programs utilize an adeno-associated virus (AAV) to deliver a healthy copy of a gene to people with a mutation in the disease-causing gene. Our product pipeline of gene therapy candidates addresses distinct monogenic neurological diseases.
*POC= proof of concept
**Neurogene has an exclusive option to in-license this program
Natural HISTORY STUDIES
Visit ClinicalTrials.gov for enrolling natural history studies sponsored by Neurogene:
A Natural History Study of Late Infantile Variant CLN5 And CLN7 Disease
A Natural History Study of Charcot-Marie Tooth 4J (CMT4J)