and sustained increases in fetal hemoglobin,” said Baisong Mei, MD, Ph.D., Senior Vice President and Chief Medical Officer, Editas Medicine. “I would like to thank the clinical trial participants, their families, clinicians, and colleagues at collaborating institutions that contribute to the RUBY and EdiTHAL trials. We remain on-track to dose 20 RUBY patients by year-end, and we look forward to sharing further RUBY and EdiTHAL clinical updates later this year.”
“I am encouraged by the results from the RUBY trial, which indicate this investigational gene editing treatment has been well-tolerated and efficacious in treated trial participants thus far. While we need to let the trial finish and enroll many other patients to understand the overall benefits and risks of this treatment, I am pleased to see transformative results at this stage in the study,” said Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children’s.
EDIT-301 uses AsCas12a, a novel, proprietary, highly efficient, and specific gene editing nuclease, to edit the promoter regions of gamma globin gene 1 and 2 to increase the expression of HbF to mimic the naturally occurring mechanism of hereditary persistence of fetal hemoglobin to treat SCD. The RUBY trial marks the first time AsCas12a was used to successfully edit human cells in a clinical trial.
EHA Oral Presentation Details:
Title: EDIT-301 Shows Promising Preliminary Safety and Efficacy Results in the Phase I/II Clinical Trial (RUBY) of Patients with Severe Sickle Cell Disease Using Highly Specific and Efficient AsCas12a Enzyme
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: Saturday, June 10, 2023, 4:30 – 5:45 p.m. CEST/ 10:30 – 11:45 a.m. EDT
Location: Harmonie 1, Messe Frankfurt
Session: s437 Gene therapy and cellular immunotherapy – Clinical
Virtual Event Information
Editas Medicine will host a virtual event on Monday, June 12, at 8:00 a.m. ET to present the data for the RUBY and EdiTHAL trials. The live and archived webcast of the presentation will be accessible through this webcast link, or through the Events & Presentations page of the “Investors” section of the Company’s website.
EDIT-301 is currently being investigated in clinical studies in patients with severe sickle cell disease (RUBY trial, NCT04853576) and transfusion-dependent beta thalassemia (EDITHAL trial, NCT05444894). In addition to the clinical data update from the RUBY trial at EHA and in a Company-sponsored webinar, the Company expects to present a further clinical update from the RUBY and EdiTHAL trials by year-end.