Editas Medicine presents preclinical data supporting the
Preclinical data demonstrated robust induction of fetal hemoglobin (HbF) in erythroid progeny cells without detection of off-target editing; cells showed reduced sickling and improved rheological behavior
RUBY trial of EDIT-301 for active and recruiting sickle cell disease
CAMBRIDGE, Mass., June 11, 2021 (GLOBE NEWSWIRE) – Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced preclinical data supporting the launch of the clinical trial EDIT-301 Phase 1/2 RUBY trial to evaluate EDIT-301, an autologous, durable and unique cell therapy drug for treating sickle cell anemia. EDIT-301 is the first investigational drug generated using CRISPR / Cas12a gene editing. The Company reported the data in an oral presentation at the 26th Congress of the European Hematology Association (EHA) which was held virtually.
In these preclinical studies, CD34 + cells from normal donors and sickle cell patients were edited to HBG1 and HBG2 promoters with the highly efficient and specific ribonucleoprotein (RNP) AsCas12a designed by Editas at research and large scale. The data demonstrated that high levels of editing were achieved, resulting in a robust induction of fetal hemoglobin (HbF) in erythroid cells without detectable off-target editing. Red blood cells derived from modified CD34 + cells from sickle cell patients showed significant reduction in sickling and improved rheological behavior. Additionally, data from the company’s current clinical-scale Good Manufacturing Practices (cGMP) process to support the manufacture of EDIT-301 has demonstrated successful large-scale production, with consistent and high-level editing of the HBG1 and HBG2 promoters while maintaining high specificity and robust induction of HbF. In addition, long-term stable and polyclonal engraftment was observed when cells made from the representative scaling process were infused into immunodeficient mice.
“High levels of editing were achieved in CD34 + cells using the highly specific RNP Cas12a, designed by Editas, leading to potentially therapeutically relevant levels of HbF expression. These results reinforce our new approach to develop EDIT-301 as a transformative and sustainable drug for the potential treatment of sickle cell disease, ”said Kate Zhang, Ph.D., Vice President, Biological Development, Editas Medicine. “With the results of current non-clinical studies and the first successful manufacturing tests, we believe our approach can produce a safer and more effective drug with the potential to change the lives of people living with sickle cell disease. “
The RUBY clinical trial, a phase 1/2 trial designed to assess the safety and efficacy of EDIT-301 for the treatment of sickle cell anemia, is active and recruiting. The Company remains on track to begin dosing patients in the RUBY trial by the end of 2021.
About sickle cell anemia
Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that causes the sickle cell protein (HbS) to polymerize. In sickle cell anemia, the red blood cells are deformed, sickle shaped instead of disc shaped. The abnormal shape causes cells to block blood flow, causing anemia, pain attacks, organ failure, and premature death. It is estimated that 100,000 people in the United States are currently living with sickle cell disease. Higher levels of fetal hemoglobin (HbF) inhibit the polymerization of HbS, thereby reducing the manifestation of sickling.
EDIT-301 is an investigational autologous cell therapy drug under investigation for the treatment of sickle cell anemia. EDIT-301 is composed of CD34 + cells from sickle cell patients genetically modified using a highly specific and efficient ribonucleoprotein (RNP) CRISPR / Cas12a (also known as Cpf1) that selectively 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 the production of HbF, which has the potential to provide unique and lasting therapeutic benefit to people living with sickle cell disease.
The RUBY trial is a phase 1/2, single-arm, open-label, multicenter study designed to evaluate the safety and efficacy of EDIT-301 in people with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301.
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR / Cas9 and CRISPR / Cas12a (also known as Cpf1) genome editing systems into one strong pipeline of treatments for people living with serious illnesses around the world. . Editas Medicine aims to discover, develop, manufacture and commercialize transformative, sustainable and precision genomic drugs for a broad class of diseases. For the latest scientific information and presentations, please visit www.editasmedicine.com.
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”, “” ” can ” ” ‘estimate,’ ” ‘predict,’ ” ‘intend,’ ” ‘can,’ ” ‘plan,’ ” ‘potential,’ ” ‘predict,’ ” ‘project,’ ” ‘target,’ ” ‘should,’ ” ‘would,’ ‘and similar phrases are intended to identify forward-looking statements, although not all forward-looking statements contain these words. identification. Forward-looking statements contained in this press release include statements regarding the Company’s plans and expectations for EDIT-301, including the initiation of patient dosing in the RUBY trial by the end of 2021. The Company may do not actually carry out 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 due to various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development the company’s product candidates; the availability and timing of the results of preclinical studies and clinical trials; whether the interim results of a clinical trial will predict the final trial results or the results of future trials; expectations of regulatory approvals to conduct testing or to market products and the availability of sufficient funding for foreseeable and unpredictable operating expenses and capital expenditure needs of the Company. These and other risks are described in more detail under the heading “Risk Factors” included in the Company’s most recent annual report on Form 10-K, which is filed with the Securities and Exchange Commission, and in other documents that the Company may make with the Securities and Exchange Commission in the future. All forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or other.