CAMBRIDGE, Mass., Dec. 01 10, 2022 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that it will host a live webinar on Tuesday, December 6, at 8:00 a.m. ET to present initial clinical data from the RUBY Phase 1/2 trial of EDIT-301, being developed for the treatment of severe sickle cell disease. Clinical data will include safety data from the first two patients and efficacy data from the first patient treated with EDIT-301.
The live and archived webcast of the presentation will be accessible via this webcast link or via the Events and Presentations page in the “Investors” section of the Company’s website. The presentation will also be available for download shortly after the webinar.
A replay of the webinar and webinar material will be available at the end of the webinar in the Investors section of the Editas Medicine website at https://www.editasmedicine.com/.
About sickle cell disease
Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that causes the polymerization of sickle hemoglobin (HbS) protein. In sickle cell disease, the red blood cells are deformed into a sickle shape instead of a typical disc shape. The abnormal shape shortens the lifespan of red blood cells and blocks 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 cell therapy drug being investigated for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). EDIT-301 consists of patient-derived CD34+ Hematopoietic stem and progenitor cells edited at the promoters of the gamma globin gene (HBG1 and HBG2), where mutations inducing fetal hemoglobin (HbF) naturally reside, by a highly specific and efficient patented AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34+ demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide both one-time and long-lasting therapeutic benefit to individuals with severe SCD and TDT.
The RUBY trial is a multicenter, open-label, single-arm, Phase 1/2 study designed to evaluate the safety and efficacy of EDIT-301 in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301. Additional details are available at www.clinicaltrials.gov (NCT#04853576).
About Edited by Medicine
As a clinical-stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome-editing systems into a robust portfolio of treatments for sufferers. serious illnesses around the world. Editas Medicine aims to discover, develop, manufacture and commercialize transformative, sustainable and precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of the Broad Institute and Harvard University Cas9 patent domains and the Broad Institute Cas12a patent domain for human medicines. For the latest scientific information and presentations, please visit www.editasmedicine.com.
CONTACT: Contacts: Media Cristi Barnett (617) 401-0113 firstname.lastname@example.org Investors Ron Moldaver (617) 401-9052 email@example.com
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