BEAM-102

BEAM-102 is an investigational gene editing therapy developed by Beam Therapeutics for sickle cell disease (SCD)[3][7][13]. It uses adenine base editing technology to directly convert the disease-causing hemoglobin S (HbS) mutation in the β-globin gene (HBB) into the naturally occurring Makassar variant (HbG-Makassar), which functions as normal hemoglobin[3][7][13]. By doing so, BEAM-102 aims to alleviate the clinical manifestations of SCD by restoring normal oxygen transport and red blood cell function. Preclinical data showed that BEAM-102 can efficiently convert the HbS mutation to HbG-Makassar in over 70% of gene copies, with the edited cells surviving post-implantation in animal models[3]. Beam Therapeutics intends for this therapy to be a one-time genetic cure for SCD[7][13].

Brand names

BEAM-102BEAM102BEAM 102

Other names

BEAM-102BEAM102BEAM 102