First CRISPR therapy administered in landmark human trial

The first confirmed CRISPR gene editing clinical trial to take place outside of China is officially underway, with pharmaceutical companies CRISPR Therapeutics and Vertex revealing a human patient has been administered the experimental treatment targeting a rare blood disease.

The gene-editing therapy, called CTX001, is designed to treat beta-thalassemia and sickle cell disease. Both conditions are caused by a mutation in a single gene. CTX001 involves engineering a patient's stem cells with a single genetic change designed to raise levels of fetal hemoglobin in red blood cells.

The beta-thalassemia trial is the first of two CTX001 trials to commence, currently taking place in Canada and Europe, with US patient enrolments beginning later this year. Initially the trial will sequentially administer the treatment to just two patients to evaluate an overall safety profile before expanding to a larger cohort.

A second CTX001 sickle cell trial will follow and utilize a similar process after being granted a Fast Track Designation by the FDA earlier this year. The first patient in the sickle cell trial has been enrolled and will be administered the treatment in the United States over the next few months.

"We have made tremendous progress with CTX001 and are pleased to announce that we've treated the first patient with beta thalassemia in this clinical study," says Samarth Kulkarni, CEO of CRISPR Therapeutics. "Treating the first patient in this study marks an important scientific and medical milestone and the beginning of our efforts to fully realize the promise of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases."