Gene therapy candidate restores vision in some patients

In a small trial of 14 patients, a CRISPR gene editing therapy improved vision among 11 participants, according to research led by a Mass General Brigham clinician. 

The phase 1/2 trial, sponsored by Editas Medicine, tested the drug's safety and efficacy in people with a rare eye disorder that causes blindness, the Somerville, Mass.-based system said in a May 6 news release. The inherited disorder is a form of Leber Congenital Amaurosis, which leads to severe vision loss at a young age. 

In the study, 12 adults and two children received an injection of the medicine, called EDIT-101, in one eye during a specialized procedure. The researchers looked for four measures: best-corrected visual acuity, dark-adapted full-field stimulus testing, visual function navigation and vision-related quality of life.

Eleven people experienced improvements in at least one outcome, and six patients noted two or more developments. The drug targets the CEP290 gene. 

The study "included the first patient to ever receive a CRISPR-based investigational medicine directly inside the body," the release said. 

The researchers are trying to find commercial partners to help conduct more trials. 

"While more research is needed to determine who may benefit most, we consider the early results promising," study lead author and Mass General Brigham clinician Eric Pierce, MD, PhD, said in the release. "To hear from several participants how thrilled they were that they could finally see the food on their plates — that is a big deal."

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