The first clinical trial of CRISPR-Cas9 sponsored by U.S. companies has launched, testing the genome-editing technique in patients with the blood disorder beta thalassemia, according to an announcement posted Friday on the U.S. clinical trials website.
The Phase 1/2 clinical trial, co-sponsored by Vertex Pharmaceuticals and using an experimental treatment from CRISPR Therapeutics, will be conducted at a single hospital in Regensburg, Germany, and aims to recruit up to 12 adults with the inherited disease. Although it was only a matter of time before the start of the first company-sponsored CRISPR clinical trial, Editas Medicine’s experimental treatment for a rare form of blindness was widely expected to be the first in the clinic.
The gene-editing therapy that will be tested, called CTX001, is intended to treat both beta thalassemia and sickle cell disease.
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I would like updates regarding this trial!! Personal connection to SCD. Thank you!!
We would appreciate it if you would notify us about this clinical trial as we are following many patients suffering hemoglobinopathies at the Hereditary Blood Disorders Clinic and Research Team at the National Research Centre in Egypt
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please inform us when you start clinical trails.