CRISPR, Vertex launch first company-backed gene editing trial in humans

CRISPR, Vertex launch first company-backed gene editing trial in humans

Cambridge biotech CRISPR Therapeutics and Boston-based Vertex Pharmaceuticals have launched the first company-sponsored study of a gene editing drug in humans, a landmark moment for the experimental technology.

CRISPR (Nasdaq: CRSP) and Vertex (Nasdaq: VRTX) began the early-stage study earlier this week in Regensburg, Germany, according to a posting Friday on the U.S. clinical trials website. The treatment targets beta thalassemia, a rare blood disorder that reduces the amount of oxygen-carrying red blood cells in a person’s body, causing fatigue, life-threatening anemia and other serious complications.

Spokespeople for Vertex and CRISPR confirmed that the companies have opened up enrollment in the study, but that no patients have been enrolled or received a dose of the drug as of Friday.

“This is one important step of many toward bringing the promise of this new technology to patients with serious diseases like (sickle cell disease) and beta thalassemia, and we are thrilled to be at the forefront of what we believe may be a fundamental change in the treatment of disease,” the companies said in a joint statement.

CRISPR is one of several Cambridge biotechs, along with Editas Medicine (Nasdaq: EDIT) and Intellia Therapeutics (Nasdaq: NTLA), that is developing drugs using a gene-altering technology called CRISPR/Cas9. Prior to this week, no company-sponsored clinical studies of gene editing treatments had officially begun, though researchers at the University of Pennsylvania and in China have already been testing the technology in humans.

Vertex and CRISPR are also hoping to launch a U.S. trial of their drug in patients with sickle cell disease, but the FDA halted the highly anticipated study in May. The companies are continuing to work with the regulator to remove the hold, according to a Vertex spokesperson.

The beta thalassemia trial is expected to run through May 2022.


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