Editas presents new EDIT-301 safety and efficacy data in 17 patients

Editas Medicine announced new safety and efficacy data in 17 patients treated with EDIT-301, now known as renizgamglogene autogedtemcel, or reni-cel, in the RUBY trial for severe sickle cell disease, or SCD, and in the EdiTHAL trial for transfusion-dependent beta thalassemia, or TDT. The total dataset of 17 treated patients includes 12 additional patients since the data presentation at the European Hematology Association Annual Congress and in a company-sponsored webinar this past June. Reni-cel is being investigated in the RUBY and EdiTHAL clinical trials as a potential one-time, durable gene editing medicine for people living with severe SCD and TDT. Editas Medicine will present the RUBY and EdiTHAL trial data today at 1 p.m. ET in a company-sponsored webinar. The data will also be presented in a poster presentation at the American Society of Hematology Annual Meeting in San Diego, CA, at 9:00 p.m. ET. In both the RUBY and EdiTHAL trials to date, reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all patients in the two trials. Since treatment with reni-cel, all RUBY patients are free of vaso-occlusive events. All RUBY patients with greater than or equal to5 months follow-up have maintained a normal hemoglobin level and a fetal hemoglobin level of greater than40%. All EdiTHAL patients had early and robust increase of total hemoglobin, above the transfusion independence threshold of 9 g/dl. “These new and promising data with a larger patient cohort support our belief that reni-cel can be a clinically differentiated, one-time, durable medicine that can provide life-changing clinical benefits to patients with sickle cell disease and beta thalassemia, specifically driving early and robust correction of anemia and sustained increases in fetal hemoglobin. 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 look forward to dosing additional patients and sharing further RUBY and EdiTHAL clinical updates in mid-2024,” said Baisong Mei, MD, Ph.D., Senior Vice President and Chief Medical Officer, Editas Medicine.