Bluebird Bio

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.

Forma Therapeutics

This company develops medicines for patients with sickle cell disease and rare blood disorders.

Beam Therapeutics

The company develops precision genetic medicines using base editing technology to treat patients with serious genetic diseases such as sickle cell disease.

Global Blood Therapeutics

This biopharmaceutical company develops and manufactures oral medicines to treat sickle cell disease for patients and healthcare providers.

Imara

This biopharmaceutical company develops oral small-molecule therapies to treat patients with rare genetic blood disorders, including sickle cell disease and beta thalassemia.

Kamau Therapeutics

This company develops clinical-stage gene correction therapies using targeted DNA integration to treat patients with sickle cell disease and other genetic conditions.

Rocket Pharma

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

Be Biopharma

This company develops engineered B cell medicines to treat patients with hemophilia B, genetic diseases, and cancer.