The first clinical trial involving a CRISPR therapy created by U.S. companies is now recruiting patients. The CRISPR-Cas9 study, co-sponsored by CRISPR Therapeutics and Vertex Pharmaceuticals, will test the effectiveness of CTX001 in treating patients with beta thalassemia.
Beta thalassemia is caused by a mutation in the HBB gene, reducing a patient’s ability to produce hemoglobin. Using CRISPR, a technique that allows doctors to alter DNA to remove the underlying genetic cause of disease, CTX001 targets and removes a region of genetic code that stops production of fetal hemoglobin after the first months of life. If successful, the treatment should allow a patient to produce red blood cells that contain hemoglobin at normal levels, even if he or she has beta thalassemia or sickle cell disease.
The CRISPR-Cas9 trial will take place in Germany and will recruit up to twelve adult patients with beta thalassemia. Other CRISPR studies are already underway across the globe, including in the U.S. and China.
Source: STAT News, August 31, 2018.