Beam Therapeutics

Base editing di precisione, la generazione successiva del CRISPR.

Beam Therapeutics Inc., a biotechnology company, engages in the development of precision genetic medicines for patients suffering from serious diseases in the United States. Its programs in hematology and genetic disease portfolio include Ristoglogene autogetemcel, a patient-specific, autologous hematopoietic stem cell (HSC) therapy for the treatment of sickle cell disease; BEAM-302, a liver-targeting lipid nanoparticle (LNP) for the treatment of severe alpha-1 antitrypsin deficiency; BEAM-304, a liver-targeting LNP for the treatment of phenylketonuria; and BEAM-301, a liver-targeting LNP formulation for the treatment of glycogen storage disease type 1a. The company also develops the ESCAPE platform, which combines antibody-based conditioning with multiplex gene edited HSCs. In addition, it develops BEAM-103, an anti-CD117 monoclonal antibody that enables ESCAPE. The company has research collaboration agreement with Pfizer Inc., focusing on in vivo base editing programs for targets rare genetic diseases of the liver, muscle, and central nervous system; Verve Therapeutics, Inc., for cardiovascular disease treatments; and Orbital Therapeutics to design RNA for the prevention, treatment, or diagnosis of human disease. Beam Therapeutics Inc. was incorporated in 2017 and is based in Cambridge, Massachusetts.