First CRISPR therapy administered in landmark human trial

The first reported human trial outside of China using CRISPR gene editing is now officially underway...
 The first reported human trial outside of China using CRISPR gene editing is now officially underway testing the safety and efficacy of this groundbreaking technology(Credit:Bushko/Depositphotos)

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.

Following a setback in 2018 with the FDA temporarily hitting pause on the US arm of a Phase 1/2 clinical trial pending “the resolution of certain questions,” one of the first CRISPR human trials to take place outside of China has now commenced. 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.”


The CTX001 trials are not the only CRISPR therapies currently being tested. Another trial, being run out of the University of Pennsylvania, is set to commence any day now. This trial uses CRISPR-edited immune cells to target three types of cancer: multiple myeloma, sarcoma, and melanoma.


Inside China CRISPR human trials have been ongoing for at least two years now, although no data from these studies has been published and recent reports have begun to suggest significant procedural problems in the research. Last December it was revealed patients undergoing the experimental CRISPR treatments may not be consistently tracked following the trial.


It will still be several years before we gather clear data from these initial CRISPR human studies so it is unclear whether the technology will be the revolution in medicine it is purported to be. Still, these early human trials are undeniably milestone moments in the history of medical science.

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