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Editas Medicine Presents Preclinical Data Supporting the Initiation of the EDIT-301 Phase 1/2 RUBY Clinical Trial for the Treatment of Sickle Cell Disease at the European Hematology Association Congress
Preclinical data demonstrated robust fetal hemoglobin (HbF) induction in erythroid progeny cells with no detection of off-target editing; cells showed reduced
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[{"type":"text","content":"Preclinical data demonstrated robust fetal hemoglobin (HbF) induction in erythroid progeny cells with no detection of off-target editing; cells showed reduced sickling and improved rheological behavior RUBY trial of EDIT-301 for sickle cell disease active and recruiting CAMBRIDGE, Mass., June 11, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced preclinical data supporting the initiation of the EDIT-301 Phase 1/2 RUBY clinical trial to evaluate EDIT-301, a one-time, durable, autologous cell therapy medicine to treat sickle cell disease. EDIT-301 is the first experimental medicine generated using CRISPR/Cas12a gene editing. The Company reported the data in an oral presentation at the 26th Congress of the European Hematology Association (EHA) being held virtually. In these preclinical studies, CD34+ cells from normal donors and sickle cell patient donors were edited at the HBG1 and HBG2 promoters with Editas-engineered highly efficient and specific AsCas12a ribonucleoprotein (RNP) at research-scale and large-scale. The data demonstrated that high levels of editing were achieved, resulting in robust fetal hemoglobin (HbF) induction in erythroid cells with no detectable off-target editing. The red blood cells derived from edited sickle cell patient CD34+ cells showed significant reduction of sickling and improved rheological behavior. In addition, data from the Company’s current Good Manufacturing Practices (cGMP) clinical scale process to support EDIT-301 manufacturing demonstrated successful scale-up production, with consistent and high-level editing of the HBG1 and HBG2 promoters while maintaining high specificity and robust HbF induction. Furthermore, stable and polyclonal long-term engraftment was observed when cells manufactured from the representative scale-up process were infused into immunodeficient mice. “High levels of editing were achieved in CD34+ cells using the highly specific, Editas-engineered Cas12a RNP, leading to potentially therapeutically relevant levels of HbF expression. These findings further support our novel approach to developing EDIT-301 as a transformative, durable medicine for the potential treatment of sickle cell disease,” said Kate Zhang, Ph.D., Vice President, Biological Development, Editas Medicine. “With the current non-clinical study...