Editas Medicine to Present Data Demonstrating Progress Towards Transformative Gene Editing Medicines for the Treatment of Hemoglobinopathies and Cancer at the ASH Annual Meeting and Exposition
EDIT-301 preclinical data support differentiated approach to develop a transformative medicine for people living with transfusion-dependent beta thalassemia
Preclinical data demonstrating proprietary CRISPR/Cas12a multiplexed editing of iPSCs enhances iNK tumor killing ability, supporting promise as a potential therapeutic approach for solid tumors
- Edited peripheral blood CD34+ cells mobilized from transfusion-dependent beta thalassemia (TDT) patients demonstrated significantly improved red blood cell production and increased hemoglobin content, supporting the development of EDIT-301 for the treatment of TDT; and
- Induced pluripotent stem cells (iPSC)-derived natural killer cells (iNKs), edited with proprietary CRISPR/Cas12a to double knock-out (DKO) CISH and TGFβR2, demonstrated robust tumor reduction in vivo as compared to wild type iNKs, supporting the development of DKO iNKs as a potent allogeneic cell-based medicine for cancer.
“At ASH, we will present preclinical data from our EDIT-301 program that reinforces our belief that our differentiated therapeutic strategy leveraging our highly-specific engineered Cas12a enzyme with more physiologic targeting has great potential for transfusion-dependent beta thalassemia. We believe that EDIT-301 has the potential to be an efficacious autologous cell therapy for TDT, and we remain on track to file an IND by year-end,” said
The complete list of
Title: Preclinical Development of EDIT301, an Autologous Cell Therapy Comprising AsCas12a-RNP Modified Mobilized Peripheral Blood-CD34+ Cells for the Potential Treatment of Transfusion Dependent Beta Thalassemia
Date and Time:
Title: Deletion of CISH and TGFβR2 in iPSC-Derived NK Cells Promotes High Cytotoxicity and Enhances In
Date and Time:
EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 is comprised of sickle patient (for SCD) or normal donor (for TDT) 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 one-time, durable treatment benefit for people living with sickle cell disease and TDT.
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. 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 preclinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from preclinical 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 Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings with the
Cristi Barnett(617) 401-0113 email@example.com Investors Ron Moldaver(617) 401-9052 firstname.lastname@example.org
Source: Editas Medicine, Inc.