Crispr surges 21% on results of in-human study using gene-editing tool
Crispr is also studying medicines in both solid and blood cancers and is in the early stages of developing treatments for diabetes and cystic fibrosis
New York — A first in-human study using the gene-editing tool Crispr has a company named after the technology and its partner, Vertex Pharmaceuticals, claiming an early victory in treating two inherited blood disorders.
Crispr Therapeutics and Vertex said a pair of patients — one suffering from beta thalassemia and the other with severe sickle cell disease — saw benefits from one-time treatment with CTX001. While they’ve been followed for just nine months and four months, respectively, their painful flare-ups and once-routine need for blood transfusions stopped after getting the therapy. Both developed severe complications including infections that were deemed unrelated to the gene-editing treatment...