Data support novel approach to develop a best-in-class, durable medicine for people living with hemoglobinopathies

CAMBRIDGE, MA, USA I December 09, 2019 I Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced in vivo proof-of-concept data supporting the development of EDIT-301 as a potentially best-in-class, durable medicine to treat sickle cell disease and beta-thalassemia. EDIT-301 is the first experimental medicine in development using Cas12a (formerly known as Cpf1). The Company reported these data today at the 61st Annual Meeting and Exposition of the American Society of Hematology (ASH) in Orlando, Fla.

Sickle cell disease is caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein (HbS). Fetal hemoglobin (HbF) protects against sickle cell disease by inhibiting HbS polymerization. Individuals with high levels of HbF are protected from sickle cell disease. EDIT-301 is an experimental, autologous cell therapy comprising CD34+ cells genetically modified using a Cas12a ribonucleoprotein (RNP) that targets the HBG1/2 promoter in the beta-globin gene to stimulate HbF production. 

In this study, when EDIT-301 was infused into NBSGW mice, HbF levels in human red blood cells were increased by approximately 50 percentage 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.    

“We are very encouraged by these in vivo findings as the data further support our novel approach to developing a best-in-class and durable medicine for the potential treatment of sickle cell disease and beta-thalassemia. IND-enabling activities were initiated earlier this year for and are ongoing for EDIT-301,” said Charles Albright, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “If these preclinical results translate to humans, we believe our editing approach may yield a safer and more effective medicine, addressing a significant need for a transformative, durable treatment for people living with sickle cell disease and beta-thalassemia.”

About Editas Medicine
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (formerly known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit

SOURCE: Editas Medicine