CRISPR, Vertex dose first β-thalassemia patient, begin enrolling sickle cell patients in Phase I trials of CRISPR therapy

CRISPR Therapeutics AG (NASDAQ:CRSP) and Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) dosed the first patient in a Canadian and European Phase I/II trial of CTX001 to treat transfusion-dependent β-thalassemia, and enrolled the first patient in a U.S. Phase I/II trial of CTX001 to treat sickle cell disease. CTX001 is an ex vivo gene therapy comprising autologous CRISPR-modified human hematopoietic stem and progenitor cells.

In October, FDA lifted a clinical hold placed in May on an IND for the sickle cell trial (see "FDA Lifts Hold on IND for CRISPR's Sickle Cell Trial")...