AveXis

Neurogene Inc

This company develops genetic medicines and gene therapies to treat patients and families affected by rare neurological diseases including Rett syndrome and Batten disease.

Audentes Therapeutics

The company develops gene therapy products to treat patients with serious and rare neuromuscular diseases.

GenSight Biologics

This company develops gene therapies for patients suffering from neurodegenerative retinal diseases and central nervous system disorders.

Mahzi Therapeutics

This biotechnology company develops gene and antisense oligonucleotide therapies for patients with rare genetic neurodevelopmental disorders.

ARTHEx Biotech

This clinical-stage biotechnology company develops RNA-based treatments for patients with rare neuromuscular genetic diseases, including myotonic dystrophy type 1.

Asklepios BioPharmaceutical

This clinical-stage company develops and manufactures adeno-associated virus gene therapies for patients with rare genetic, neuromuscular, central nervous system and cardiovascular disorders.

Minoryx

This biotechnology company develops orphan drugs and therapies for patients suffering from rare, life-threatening genetic diseases of the central nervous system.

Affinia Therapeutics

This company develops rationally designed gene therapies and viral vectors for patients with heart and central nervous system diseases.