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 defect in the disease-causing gene. Our product pipeline of gene therapy candidates addresses distinct monogenic neurological diseases.

Aspartylglucosaminuria (AGU)Aspartylglucosaminuria (AGU)
Aspartylglucosaminuria (AGU)Aspartylglucosaminuria (AGU)
Charcot-Marie Tooth disease (CMT4J)Charcot-Marie Tooth disease (CMT4J)
Charcot-Marie Tooth disease (CMT4J)Charcot-Marie Tooth disease (CMT4J)
Batten Disease (CLN7)Batten Disease (CLN7)
Batten Disease (CLN7)Batten Disease (CLN7)
Batten Disease (CLN5)Batten Disease (CLN5)
Batten Disease (CLN5)Batten Disease (CLN5)

Natural HISTORY STUDIES

Visit ClinicalTrials.gov for enrolling natural history studies sponsored by Neurogene:

 

A Natural History Study of Aspartylglucosaminuria (AGU)

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

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A Natural History Study of Late Infantile Variant CLN7 and CLN5 Disease

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For MEDICAL INQUIRIES

medicalinfo@neurogene.com