Benzinga | Jan 11, 2021 06:09AM EST

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

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.