Vertex becomes first U.S. company to enter clinic with CRISPR therapy

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) and partner CRISPR Therapeutics AG (NASDAQ:CRSP) have begun enrolling patients in the first U.S. company-sponsored trial using CRISPR/Cas9 editing. The European Phase I/II trial is evaluating CTX001, which comprises autologous CRISPR-modified human hematopoietic stem and progenitor cells, in transfusion-dependent β thalassemia patients.

Vertex spokesperson Heather Nichols said, "It's the first clinical trial of a CRISPR therapy in the Western world."...