Orchard Therapeutics

Bluebird Bio

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

Genetix Biotherapeutics

The company develops and commercialises gene therapies for patients suffering from severe rare genetic disorders such as sickle cell disease and beta thalassemia.

Rocket Pharma

This company develops gene therapies for patients with rare and devastating genetic cardiovascular and haematological diseases.

Genespire

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

Garuda Therapeutics

The company develops off-the-shelf blood stem cell therapies to treat patients with hematologic malignancies, sickle cell disease, and bone marrow failure disorders.

Lysogene

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

Castle Creek Biosciences

This company develops cell and gene therapies for patients suffering from rare genetic skin and connective tissue diseases.

Seamless Therapeutics

The company develops programmable recombinase-based gene editing therapies for patients with severe genetic diseases through its proprietary technology platform.