CRISPR gene editing has been a hot area of biotechnology for the past few years despite the absence of data in humans, but the field finally crossed a new threshold with initiation of the first industry-sponsored clinical trial, from CRISPR Therapeutics AG and partner Vertex Pharmaceuticals Inc. And, with aggressive investment in gene-editing technologies across the industry, this should be the start of a wave of clinical trials.
CRISPR Therapeutics and Vertex kicked off a 30-patient Phase I/II trial for CTX001 in transfusion-dependent beta-thalassemia, according to an Aug. 31 clinicaltrials.gov listing