Beam Therapeutics Inc. is a biotechnology company developing precision genetic medicines through base editing. The Company's suite of gene editing technologies is anchored by base editing, a technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the deoxyribonucleic acid. Its lead programs are focused on sickle cell disease and alpha-1 antitrypsin deficiency, and it is also advancing programs in other genetic diseases. Its primary programs include BEAM-101, Engineered Stem Cell Antibody Paired Evasion (ESCAPE), BEAM-302 and BEAM-301. BEAM-101 is a patient-specific, autologous hematopoietic stem cell (HSC), investigational therapy. ESCAPE is a potentially non-genotoxic approach to HSC transplantation. BEAM-302 is a liver-targeting lipid nanoparticle formulation of base editing reagents designed to offer a one-time treatment to correct the E342K point mutation (PiZZ genotype).
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