Editas Medicine Announces Positive Initial EDIT-301 Safety and Efficacy Data from the First Four Patients Treated in the RUBY Trial and the First Patient Treated in the EdiTHAL Trial

EDIT-301 was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant

First two RUBY patients achieved normal levels of total hemoglobin and fetal hemoglobin of >40% at 5 months and maintained these levels after 10 and 6 months of follow up

All five patients treated with EDIT-301 successfully engrafted and all four RUBY patients treated are free of vaso-occlusive events since infusion

RUBY clinical data to be presented at the European Hematology Association (EHA) Hybrid Congress on Saturday, June 10, at 5:30 p.m. CET/11:30 a.m. ET

Company to host a virtual event on the RUBY and EdiTHAL data on Monday, June 12 at 8:00 a.m. ET

CAMBRIDGE, Mass., June 09, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced positive initial safety and efficacy data from the first four patients with sickle cell disease (SCD) treated with EDIT-301 in the RUBY trial and from the first transfusion-dependent beta thalassemia patient treated in the EdiTHAL trial.

The RUBY trial data will be presented in an oral presentation at European Hematology Association (EHA) Hybrid Congress in Frankfurt, Germany, and via live stream on Saturday, June 10, at 5:30 p.m. CEST/11:30 a.m. EDT. Editas Medicine will present the RUBY trial data and the EdiTHAL trial data on Monday, June 12, at 8 a.m. ET in a Company-sponsored webinar.

In the RUBY trial, Patients 1 and 2 reached normal hemoglobin levels five months post-treatment with EDIT-301, and both patients have maintained a normal hemoglobin level at ten- and six-month follow-up, respectively. Additionally, each of these patients has seen fetal hemoglobin levels of greater than 40% persist during the same time frame. Patients 3 and 4 in the RUBY trial saw increases in total hemoglobin and fetal hemoglobin at three and two months of follow up, respectively, that follow similar trajectories as those seen in the first two patients. All four treated RUBY patients are also free of vaso-occlusive events (VOEs) since infusion.

In the EdiTHAL trial, the first patient demonstrated successful neutrophil and platelet engraftment, and, at one and a half months post-infusion, the patient’s response resembles that of the first four RUBY patients.

EDIT-301 was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by the four patients in the RUBY trial and the first patient in the EdiTHAL trial. After EDIT-301 infusion, no serious adverse events occurred, and no adverse events reported were related to treatment with EDIT-301.