Lysogene

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.

GenSight Biologics

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

Minoryx

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

Genespire

This company develops off-the-shelf gene therapies for children and adults affected by inherited metabolic diseases and primary immunodeficiencies.

LogicBio

This company develops gene editing and delivery platforms to provide one-time treatments for patients with serious, early-onset rare genetic diseases.

Orchard Therapeutics

Orchard Therapeutics develops haematopoietic stem cell gene therapies for patients living with severe genetic diseases.

Coave Therapeutics

The company develops targeted gene therapies for patients with neurodegenerative and ocular diseases.

AveXis

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