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Main focus: Base editing for the treatment of genetic diseases and cancer
Company stage: Pre-clinical
Diseases: Sickle cell disease, beta-thalassemia, acute lymphoblastic leukaemia (ALL), alpha-1 antitrypsin deficiency, glycogen storage disorder-1, Stargardt disease
Genome-editing tool: Cytosine base editor, adesonine base editor, prime editing
Funding stage: Public (NASDAQ:BEAM)
Location: Cambridge, MA, USA
Beam Therapeutics is developing precision genetic medicines through base editing. Founded by leading scientists from the CRISPR field, Beam is pursuing therapies for serious diseases using its proprietary base-editing technology, which can make precise edits to single base pairs in DNA and RNA. The company's most advanced programme is in IND-enabling studies for sickle cell anaemia.