The first CRISPR trial spearheaded by US companies has launched, Stat News reports.
Vertex Pharmaceuticals and CRISPR Therapeutics are collaborating on a phase 1/2 clinical trial of a CRISPR-based treatment for transfusion-dependent β-thalassemia, it says. The trial is taking place at a single site in Germany and is to enroll about a dozen patients. Stat News adds that the gene-editing approach the companies are testing, CTX001, involves removing cells from patients and altering them in the lab before returning the edited versions back to the participants. The aim, it notes, is to prod patients' cells into producing the fetal version of hemoglobin.
As Wired reported late last year, CRISPR Therapeutics is also pursuing this gene-editing approach for sickle-cell disease. CRISPR Therapeutics' Samarth Kulkarni told Technology Review last year that the company was able to successfully edit about 85 percent of stem cells they obtained from sickle-cell patients. Stat News notes that the sickle-cell trial Vertex Pharmaceuticals and CRISPR Therapeutics are planning in the US is on a "clinical hold," but that the β-thalassemia one in Europe is now recruiting.
Researchers in China have already launched CRISPR-based gene-editing trials.