Vertex, CRISPR show early benefits with therapy, but longer term data are needed to show how it stacks up

Vertex and partner CRISPR Therapeutics showed sustained benefits in the first two patients treated with their CTX001, but longer term data will be needed to understand how the ex vivo CRISPR therapy stacks up against more advanced programs in β thalassemia and sickle cell disease.

On Tuesday, Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) and CRISPR Therapeutics AG (NASDAQ:CRSP) announced data from a transfusion-dependent β thalassemia patient with a β0/IVS-I-110 genotype and a sickle cell disease patient treated with CTX001 in a Phase I/II study. CTX001 is a gene-edited autologous hematopoietic stem cell therapy. ...