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.

Indication
Discovery
Preclinical
IND Enabling
Phase I/II
Batten Disease
Undisclosed
 
Other Lysosomal Storage Disorders
Undisclosed
Undisclosed
 
Platform Technology
Neurology

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
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A Natural History Study of Charcot-Marie Tooth 4J (CMT4J)

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For MEDICAL INQUIRIES

 1-877-237-5020

 medicalinfo@neurogene.com