First CRISPR Trial to Edit DNA in Human Body

Biotech companies Allergan and Editas Medicine have announced that the Brilliance Phase 1/2 clinical trial of AGN-151587 (EDIT-101) is open for patient enrollment. The clinical trial will be the world’s first in vivo study of a CRISPR-based genome editing medicine, where the editing takes place inside the human body.

“Beginning patient enrollment in the AGN-151587 clinical trial with our partners at Editas is an important step toward our goal of developing a game-changing, transformative, CRISPR-based medicine,” said Allergen researcher David Nicholson.

AGN-151587 is an experimental medicine under development for the treatment of Leber congenital amaurosis 10 (LCA10), an inherited form of blindness caused by mutations in the CEP290 gene.

SingularityHub emphasizes that, while CRISPR-based medicine has been tested in humans, this is the first study to use CRISPR to edit DNA inside the human body.

The clinical trial patients will have the treatment, which was approved by the FDA in December, injected behind their retinas. The hope is that the patients’ DNA will repair itself in a way that restores normal protein function, ultimately fixing their photoreceptor cells and letting them see.

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