Vertex Pharmaceuticals expanded the company’s collaborative partnership with CRISPR Therapeutics to develop and commercialize a possible cure for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The focus is on the development of CTX001, an autologous, ex vivo CRISPR-CAS9 gene-edited therapy.
CRISPR Therapeutics and Vertex Pharmaceuticals announced positive interim data from the first patients in the companies’ Phase I/II clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001.
A European Medicines Agency panel recommended a conditional marketing approval for a gene therapy from Bluebird Bio Inc. as a genetic blood disorder treatment, setting the stage for the U.S. biotech to win the company’s first regulatory nod.
