All patients treated in the RUBY trial are free of vaso-occlusive events post-renizgamglogene autogedtemcel (reni-cel) infusion
Patients had early normalization of total hemoglobin with a mean within the normal range at >14 g/dL and rapid and sustained improvements in fetal hemoglobin well above levels of >40%.
Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant
EHA RUBY oral presentation on Saturday, June 15 at 11:30 a.m. CEST/5:30 a.m. EDT
CAMBRIDGE, Mass., June 14, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a clinical-stage genome editing company, today announced new safety and efficacy data in 18 patients living with sickle cell disease (SCD) treated with renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301) in the Phase 1/2/3 RUBY clinical trial. Reni-cel, the first investigational AsCas12a gene-edited cell therapy medicine, is being studied in the RUBY trial as a potential one-time, durable medicine for people living with severe SCD. The data will be presented in an oral presentation at the European Hematology Association (EHA) Hybrid Congress in Madrid, Spain and via livestream, on Saturday, June 15 at 11:30 a.m. CEST (5:30 a.m. EDT).
In the RUBY trial 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 (N=18). Since treatment with reni-cel, patients have been free of vaso-occlusive events (VOEs) (N=18) for up to 22.8 months of follow-up. Patients had early normalization of total hemoglobin (Hb) with a mean within the normal range at >14 g/dL and rapid and sustained improvements in fetal hemoglobin (HbF) well above levels of >40%. Patients in the RUBY trial underwent a median of 2.0 apheresis and mobilization cycles (min: 1.0, max: 4.0).
"These data confirm the observations from our prior clinical readouts and further support our belief that reni-cel has the potential to be a best-in-class and clinically differentiated, one-time, durable medicine that can provide life-changing clinical benefits to patients," said Baisong Mei, M.D., Ph.D., Chief Medical Officer, Editas Medicine. "Importantly, we continue to make significant progress in the development of reni-cel. In the RUBY trial, we have now dosed more than 20 patients, completed adult cohort enrollment, and opened and enrolled patients in the adolescent cohort. I would like to thank the participants, their families and caregivers, clinicians, and colleagues at collaborating institutions that contribute to the RUBY trial."
"I am encouraged by these results from the RUBY trial, demonstrating this investigational gene editing medicine has been well-tolerated and shows promising efficacy for people living with sickle cell disease. Treatment with reni-cel showed a favorable safety profile and promising preliminary efficacy, supporting further investigation as a differentiated gene-edited medicine for patients with SCD. We look forward to continuing to evaluate its effectiveness on this patient population in need of treatment options," said Rabi Hanna, M.D., Chairman of the Division of Pediatric Hematology Oncology and Blood and Marrow Transplantation at Cleveland Clinic Children's, and the RUBY presenting investigator.
Efficacy of reni-cel in Patients with Severe Sickle Cell Disease
All patients (N=18) are free of VOEs ...