Neurogene Inc

Uncommon Therapeutics

This company develops gene-based medicines for patients with rare neurodevelopmental disorders, with an initial focus on treating Rett Syndrome.

Mahzi Therapeutics

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

Lysogene

Lysogene develops gene therapies to treat rare central nervous system diseases in paediatric patients.

Affinia Therapeutics

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

GenSight Biologics

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

Encoded Therapeutics

This biotechnology company develops precision genetic medicines for patients with severe neurological disorders, including Dravet syndrome.

AveXis

This biotechnology firm develops gene therapies for patients with rare and life-threatening neurological genetic diseases including spinal muscular atrophy.

CureAge

This company develops curative gene therapies for patients with neurofibromatosis type 1 and other peripheral nerve diseases using lipid nanoparticle technology.