Patients Treated With CRISPR Still "Cured" Three Years Later

A long-running human trial has shown that it is possible to modify genes to treat serious conditions.

The trial found that a new therapy called exagamglogene autotemcel, or ex-cel for short, was able to cure patients with transfusion- dependent alpha-thalassemia.

It is a new use of technology. The results suggest that tinkering with genetic code could become a new area of medicine.

New long-term data from the ongoing trial shows that 42 of 44 patients no longer need blood transfusions. There was a reduction of 75 and 89 percent in the amount of blood that was used.

During long-term follow-ups, all 31 patients with severe SCD no longer experienced symptoms such asVOCs, a painful inflammation of the body due to blood deprivation.

"These robust data from 75 patients, of which 33 have one year or more of follow-up after exa-cel injection, further demonstrate the potential of this therapy as a one-time functional cure for patients with severe SCD," said Carmen Boz.

Past results from the ongoing study were published in The New England Journal of Medicine last year.

The technique uses stem cells from the patient's blood to make a single genetic edit in the cells and then injects them into the patient's bloodstream.

Haydar Frangoul, medical director at the TriStar Centennial Medical Center in Tennessee, said that exa-cel restores fetal hemoglobin production and can be used to treat diseases.

The FDA gave the therapy a Fast Track designation, which means that it could be approved by the end of the year.

Exa-cel is a promising use case for the technology and a big win in the field of medical science if it were to be approved.

America's first CRISPR trial is still effective for 3 years.

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