Skip to main content
Early data reported for 2 trials testing CRISPR/Cas9 gene therapy

Preliminary safety and efficacy data from the first two patients participating in two Phase I/II clinical studies to assess CTX001, a gene therapy that uses CRISPR/Cas9 gene editing technology, has been made public by the treatment's manufacturers, CRISPR Therapeutics and Vertex Pharmaceuticals. The company unveiled nine months' worth of data testing the therapy as a treatment for beta thalassemia as well as data for the past four months testing the therapy in sickle cell disease.

Full Story: