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)
Undisclosed Lysosomal Storage Disease (LSD)Undisclosed Lysosomal Storage Disease (LSD)
Undisclosed Lysosomal Storage Disease (LSD)Undisclosed Lysosomal Storage Disease (LSD)
Undisclosed Lysosomal Storage Disease (LSD)Undisclosed Lysosomal Storage Disease (LSD)
Undisclosed Lysosomal Storage Disease (LSD)Undisclosed Lysosomal Storage Disease (LSD)

For MEDICAL INQUIRIES

medicalinfo@neurogene.com