Editas Medicine (NASDAQ:EDIT) announces in vivo proof-of-concept data supporting the development of EDIT-301 as a durable medicine to treat sickle cell disease and beta-thalassemia.
In this study, when EDIT-301 was infused into NBSGW mice, HbF levels in human red blood cells were increased by approx. 50% points above background at 16 weeks post-engraftment with pancellular distribution and no lineage skewing.
These elevated HbF levels were observed after editing with Cas12a which created an editing profile that enriched genomic changes that favored high and persistent HbF levels.
EDIT-301 is the first experimental medicine in development using Cas12a (formerly known as Cpf1).
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