Beam Therapeutics Inc.

Beam Therapeutics is a biotechnology company focused on developing precision genetic medicines for serious genetic and hematologic diseases. The company's pipeline centers on two main therapeutic modalities: in vivo base editing and ex vivo gene editing of hematopoietic stem cells. Its lead program, Ristoglogene autogetemcel, is a patient-specific autologous HSC therapy in clinical development for sickle cell disease. The company also advances a series of liver-targeting lipid nanoparticle (LNP) programs, including BEAM-302 for severe alpha-1 antitrypsin deficiency, BEAM-304 for phenylketonuria, and BEAM-301 for glycogen storage disease type 1a.

The ESCAPE platform represents a key technology asset, combining antibody-based conditioning with multiplex gene-edited HSCs to enable broader applications across hematologic and genetic disorders. Supporting this platform is BEAM-103, an anti-CD117 monoclonal antibody in development.

Beam maintains research collaborations with several pharmaceutical and biotechnology partners, including Pfizer (focused on in vivo base editing for rare liver, muscle, and central nervous system diseases), Verve Therapeutics (cardiovascular disease), and Orbital Therapeutics (RNA design). Incorporated in Delaware in 2017 and headquartered in Cambridge, Massachusetts, the company operates primarily in the United States, though its therapeutic focus addresses rare genetic diseases with global patient populations.