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Beam R&D pipeline
Beam's R&D pipeline can be divided into four categories, namely in vitro hematopoietic stem cell therapy, in vitro T cell therapy, in vivo LNP therapy, and AAV therapy
In vitro hematopoietic stem cell therapy: there are 2 models, namely BEAM-101 for sickle cell disease and β-thalassemia, and BEAM-102
In vitro T cell therapy: There are 2 models, namely BEAM-201 for acute T-lymphoblastic leukemia (T-ALL) and CD7-positive acute myeloid leukemia, which is a multi-gene editing CAR-T therapy
In vivo LNP therapy: The fastest progression is BEAM-301, which has been declared for IND, which uses LNP to deliver base-edited components to the liver in mRNA form to repair the R83C point mutation in the
AAV therapy: treatment of Stargardt disease, an autosomal negative genetic disorder caused by a single-base mutation, in which patients have decreased central vision due to the appearance of yellow spots on the fundus of
