California Institute of Regenerative Medicine

Sangamo Therapeutics is a clinical-stage biotechnology company focused on developing genomic therapies aimed at improving the lives of patients with serious health conditions. The company utilizes advanced technologies in genome editing, gene therapy, gene regulation, and cell therapy to create innovative treatments. Sangamo's product pipeline addresses various medical areas, including hemophilia, central nervous system disorders, HIV, lysosomal storage disorders, and hemoglobinopathies. Additionally, the company has a priority neurology pipeline that includes therapies for chronic neuropathic pain, prion disease, and other neurological conditions, along with partnered programs targeting hemophilia A, oncology, amyotrophic lateral sclerosis, and Huntington's disease. Sangamo aims to leverage its scientific expertise to deliver transformative medicines for patients and their families.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.

Bluebird Bio is a clinical-stage biotechnology company focused on developing transformative gene therapies for severe genetic diseases and cancer. The company is engaged in researching and commercializing therapies that aim to genetically modify patients' cells to correct the underlying genetic causes of diseases. Key programs include LentiGlobin for β-thalassemia and sickle cell disease, and Lenti-D for cerebral adrenoleukodystrophy. In oncology, bluebird bio is developing CAR T cell therapies, such as bb2121 and bb21217, for multiple myeloma. The company has formed strategic collaborations with Bristol-Myers Squibb and Regeneron Pharmaceuticals to advance gene therapies in oncology, and works with various partners to develop innovative solutions in cancer treatment and in vivo genome editing for genetic disorders. Founded in 1992 and headquartered in Cambridge, Massachusetts, bluebird bio generates revenue through collaboration arrangements, research fees, license fees, and grants.