Rocket Pharma

Solid Biosciences

This life science company develops gene therapy candidates for patients living with rare neuromuscular and cardiac diseases.

Ultragenyx

This biopharmaceutical company develops and commercialises therapies for patients with serious rare and ultra-rare genetic diseases.

Phlox Therapeutics

This company develops RNA-based gene therapies to treat patients with rare genetic heart diseases, including cardiomyopathies and laminopathies.

Bluebird Bio

This company develops and manufactures gene therapies for patients with severe genetic diseases, including sickle cell disease and beta-thalassaemia.

Lexeo Therapeutics

This clinical-stage company develops gene therapies for patients with genetically defined cardiovascular diseases and Alzheimer's disease.

Stridebio

This company develops engineered viral vectors and gene therapies for patients with rare genetic diseases.

Affinia Therapeutics

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

4DMT

This company develops targeted gene therapy medicines for patients with severe genetic and retinal diseases.