Editas Medicine to Present Clinical Data from the RUBY and EdiTHAL Trials of EDIT-301 at the ASH 2023 Annual Meeting and in a Company-sponsored Webinar - Seite 2

Webinar Presentation Details:
The live and archived webcast of the Company’s webinar presentation will be accessible through this webcast link, or through the Events & Presentations page of the “Investors” section of the Company’s website.

A replay of the webinar will be available upon conclusion of the webinar in the Investors section of the Editas Medicine website at https://www.editasmedicine.com/.

ASH Presentation Details:
Title: AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia
Presenting Author: Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children’s, Cleveland, OH, United States
Date/Time: Monday, December 11, 2023, 6:00 – 8:00 p.m. PT/9:00 – 11:00 p.m. ET
Location: San Diego Convention Center, Halls G-H
Session: 801. Gene Therapies: Poster III
Publication Number: 4996

The abstract can be accessed on the ASH website.  

EDIT-301 is currently being investigated in a clinical study in patients with severe sickle cell disease (RUBY trial, NCT04853576) and transfusion-dependent beta thalassemia (EDITHAL trial, NCT05444894).

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 (HbS). In sickle cell disease, the red blood cells are misshapen in a sickle shape instead of a typical disc shape. The abnormal shape causes the red blood cells to have shortened lifespan and to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in the United States currently living with sickle cell disease. Higher levels of fetal hemoglobin (HbF) inhibit HbS polymerization, thus reducing the manifestation of sickling.

About EDIT-301
EDIT-301 is an experimental gene editing medicine under investigation 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 gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34⁺ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT.