A lackluster efficacy signal has prompted Editas Medicine Inc. to pause enrollment in a phase I/II trial of its CRISPR/Cas9-based gene editing therapy, EDIT-101, which is in development for patients with a particular form of Leber congenital amaurosis type 10.
An experimental gene editing therapy for an inherited form of blindness has become the first in vivo CRISPR medicine to be administered to patients, according to Editas Medicine Inc. and its partner, Allergan plc, which licensed the candidate in 2018.