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
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
About Sickle Cell Disease
Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein (HbS). In sickle cell disease, the red blood cells are misshapen, in a sickle shape instead of the disc shape. The abnormal shape causes the cells to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in
EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) that targets the HBG1 and HBG2 promoters in the beta-globin locus where naturally occurring fetal hemoglobin (HbF) inducing mutations reside. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in HbF production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.
About Editas Medicine
As a leading genome editing company,
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Company’s plans and expectations for EDIT-301, including filing an IND for EDIT-301 by the end of 2020. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Quarterly Report on Form 10-Q, which is on file with the
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Source: Editas Medicine, Inc.