Editas Medicine Announces the FDA has Cleared Initiation of the EDIT-301 Clinical Trial

EDIT-301 is in development as a best-in-class, durable medicine for people living with sickle cell disease

CAMBRIDGE, Mass., Jan. 11, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing.

“The FDA’s clearance for initiation for our EDIT-301 clinical trial is an exciting moment for us and the patients we hope to serve. We look forward to bringing this potentially best-in-class, one-time, durable medicine into the clinic and to patients,” said Cindy Collins, President and Chief Executive Officer, Editas Medicine. “We know patients are counting on us, and we believe EDIT-301 has the potential to transform the lives of people living with sickle cell disease, addressing a significant unmet need.”

Editas Medicine is preparing to initiate the RUBY clinical trial, a Phase 1/2 trial designed to assess the safety and efficacy of EDIT-301 for the treatment of sickle cell disease. The Company has identified a lead principal investigator and engaged a Clinical Research Organization (CRO). Clinical trial materials are being manufactured by Editas Medicine. The Company will need to resolve a partial clinical hold prior to commencement of the efficacy portion of the RUBY trial by developing an improved potency assay after the first patients are dosed in the safety portion of the trial before collection of data to support registration.

About Sickle Cell Disease
Sickle 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 red blood cells are misshapen, in a sickle shape instead of the disc shape. The abnormal shape causes the cells to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in the United States currently living with sickle cell disease. Fetal hemoglobin (HbF) protects against sickle cell disease by inhibiting HbS polymerization.