Editas (EDIT) Reports Upbeat New Data From Reni-Cel Studies

Core Insights - Editas Medicine has reported positive data from its RUBY and EdiTHAL studies, demonstrating the efficacy and safety of its lead candidate, reni-cel, in treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) [6][12][20] Company Progress - Editas has dosed over 20 SCD patients and completed adult cohort enrollment while also enrolling patients in the adolescent cohort of the RUBY study, indicating significant progress [2] - All patients in both studies showed successful neutrophil and platelet engraftment after treatment with reni-cel [3] Efficacy Results - In the RUBY study, SCD patients experienced a sustained increase in the mean percentage of HbF-containing red cells, remaining above 90% from month 4 onward for 12 patients with at least four months of follow-up [7] - The mean corpuscular fetal hemoglobin of F-cells remained above the anti-sickling threshold of 10 pg/F-cell by month 3 post-reni-cel infusion for 14 patients with at least three months of follow-up [7] - In the EdiTHAL study, all TDT patients experienced early increases in total hemoglobin (Hb) levels, rising above the transfusion independence threshold of 9.0 g/dL, with all seven patients remaining transfusion-free at last follow-up for 4.1 to 12.8 months [14] Safety Profile - Safety data from both studies indicated that reni-cel was well-tolerated, with a safety profile consistent with myeloablative conditioning and autologous hematopoietic stem cell transplant, and no serious treatment-related adverse events reported [20] Competitive Landscape - Vertex and CRISPR Therapeutics are conducting new phase III studies for SCD and TDT, and their gene therapy, Casgevy, has been approved in multiple regions, including the US and EU [4][9][21]