Editas: promises therapy with EDIT-301 in the first patient with Sickle Cell Disease

Editas Medicine, Inc. (Nasdaq: EDIT), announced that the gene editing therapy EDIT-301 is promising in the first patient with sickle cell disease (SCD). The experimental treatment is given to the patient according to the Phase 1/2 RUBY clinical trial. The EDIT-301 therapy uses a proprietary enzyme called AsCas12a and noted that is the first time has been used for genetic editing of human cells in a clinical trial. The first patient dosed in the RUBY clinical trial has shown signs of engraftment determined by analyses of the patient’s neutrophils and platelets. Editas Medicine, also announced that the U.S. Food and Drug Administration (FDA) has removed a partial hold on the RUBY trial, given the go-ahead for planned assessments of efficacy. “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,” Gilmore O’Neill, Editas president and CEO, said. For more information press release.