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European Regulators Approve CRISPR Trial
Drugs Submissions and Approvals
CRISPR Therapeutics has slated a European clinical trial of its gene-editing technology for a common blood disorder this year.
Researchers aim to use the tool for patients with the inherited blood disorder beta thalassemia, which can cause bone deformities and anemia and affects thousands of children every year. Researchers will harvest stem cells from patients and engineer them with a goal of increasing levels of fetal hemoglobin, after which they will return the cells to the patients’ bodies.
If the procedure is successful, beta thalassemia will be the first human disease treated with the gene-editing technology in Europe. Researchers have not yet established a location for the trials.