William Blair & Company initiated research coverage of Beam Therapeutics Inc. (BEAM $19.01), a gene-editing company focused on developing innovative, precision medicines for serious diseases using the company’s proprietary base editing technology.
Analyst Raju Prasad said, “Beam is focused on base editing, a next-generation gene-editing methodology that generates point mutations in genomic DNA or cellular RNA without giving rise to double-strand breaks. Two classes of DNA base editors have been developed: cytosine base editors, or CBEs, that convert a C-to-G base pair into a T-to-A base pair and adenine base editors, or ABEs, that convert an A-to-T base pair into a G-to-C base pair. To date, Beam has focused primarily on ABEs to advance into therapeutic development in its lead indications, with investigational new drug submissions expected starting in 2021. In our view, one of Beam’s biggest competitive advantages is the ability to evolve its platform—almost in real time—to improve editing efficiency and/or minimize any potential off-target editing. The company’s most advanced program involves the treatment of the blood disorders sickle cell disease and beta-thalassemia. Other programs in development cover diseases such as T-cell acute lymphoblastic leukemia, AML, A1ATD, glycogen storage disorder 1a, and Stargardt disease. And in June 2020, Verve Therapeutics presented in vivo proof-of-concept data in nonhuman primates that showed base editing (using Beam’s ABE) led to impressive reductions in two biomarkers associated with coronary heart disease.”
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