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Editas Medicine Announces Clinical Achievements in the Development of EDIT-301 for Sickle Cell Disease
Successful engraftment of first patient dosed with EDIT-301 for sickle cell disease FDA removed partial clinical hold for the RUBY trial in EDIT-301 First
About this update from Editas Medicine, Inc.
[{"type":"text","content":"Successful engraftment of first patient dosed with EDIT-301 for sickle cell disease FDA removed partial clinical hold for the RUBY trial in EDIT-301 First clinical use of Editas-engineered AsCas12a enzyme Initial clinical data from the RUBY trial expected by year-end CAMBRIDGE, Mass., July 27, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced the dosing and confirmed successful neutrophil and platelet engraftment of the first patient in the Phase 1/2 RUBY trial of EDIT-301 for the treatment of severe sickle cell disease (SCD). This dosing is the first time that the Company’s engineered AsCas12a enzyme, a proprietary, highly efficient, and specific gene editing nuclease, has been used to edit human cells in a clinical trial. The trial is enrolling additional study participants at multiple centers in the U.S. and Canada. The Company has successfully edited CD34+ cells from patients in preparation for reinfusion and remains on track to announce top-line clinical data by year-end. Additionally, the Company announced that the U.S. Food and Drug Administration (FDA) removed the previously disclosed partial clinical hold on the RUBY trial, which enables the Company to include efficacy data from patients in a marketing application for EDIT-301 in the future. “It is an exciting time at Editas as we continue to build momentum for our EDIT-301 program,” said Gilmore O’Neill, M.B., M.M.Sc., President and CEO, Editas Medicine. “Dosing and successful engraftment of the first patient coupled with the FDA’s removal of the partial clinical hold on the RUBY trial are important steps toward our goal of bringing this new and promising treatment to people living with sickle cell disease and thalassemia.” EDIT-301 is also being investigated in a clinical study in patients with transfusion-dependent beta thalassemia (TDT). Preparations to initiate the Phase 1/2 EDITHAL clinical trial designed to assess the safety, tolerability, and preliminary efficacy of EDIT-301 for the treatment of TDT are underway, and the Company remains on track to dose the first TDT patient in 2022. About Sickle Cell DiseaseSickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein (HbS). In sickle cell disease, the re...