Editas Medicine announces positive clinical data in the development of EDIT-301 for sickle cell disease

Editas Medicine, Inc. announced positive initial clinical data from the first two patients with sickle cell disease (SCD) treated with EDIT-301 in the Phase 1/2 RUBY trial. EDIT-301 is an investigational medicine supported by the Company's proprietary engineered AsCas12a enzyme to generate high efficiency edits in the HBG1/2 promoter site and disrupt the binding site of BCL11a, consistent with observed naturally occurring human mutations that lead to high levels of fetal hemoglobin. The clinical data includes safety data from the first two patients and efficacy data from the first patient treated. EDIT-301 was well-tolerated in the two patients and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. No serious adverse events occurred, and no adverse events reported were related to treatment with EDIT-301. For more information: press release