Editas Medicine Announces Preclinical Data and Large-Scale Manufacturing Process for EDIT-301, in Development for the Treatment of Sickle Cell Disease and Beta-Thalassemia
Data support novel approach to develop and manufacture a best-in-class, durable medicine for people living with hemoglobinopathies
IND filing for EDIT-301 planned by end of 2020
CAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced preclinical data and successful development of a large-scale manufacturing process for EDIT-301, a potentially best-in-class, one-time, durable, autologous cell therapy medicine to treat sickle cell disease and beta-thalassemia. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing. The Company reported these data today at the 62nd Annual Meeting and Exposition of the American Society of Hematology (ASH) being held virtually.
The data showed that high levels of editing in CD34+ cells from normal donors and sickle cell patients were achieved with CRISPR/Cas12a at the HBG1 and HBG2 promoters, leading to robust fetal hemoglobin (HbF) induction in their erythroid progeny in a pan-cellular fashion. Red blood cells derived from edited sickle cell patient CD34+ cells showed remarkable correction of sickle cell disease phenotypes, including a reduction in sickling and improved rheological properties when deoxygenated.
In addition, the Company’s large-scale manufacturing process was shown to be consistent and robust. When infused into immunodeficient mice, edited CD34+ cells from normal donors manufactured at large-scale led to long term multi-lineage hematopoietic reconstitution that was comparable to unedited control cells. The engraftment was stable and highly polyclonal with high levels of editing detected throughout the course of the study.
“These findings are very encouraging and further support our novel approach to developing and manufacturing EDIT-301 as a best-in-class and durable medicine for the potential treatment of sickle cell disease and beta-thalassemia,” said Charles Albright, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “If these preclinical results translate to the clinic, we believe our editing approach may yield a safer and more effective medicine, addressing a significant unmet need for a transformative, durable treatment with the potential to transform the lives of people living with sickle cell disease and beta-thalassemia. ”