Skip to main content
Premium Trial:

Request an Annual Quote

Gene Snip Here and There

Editas Medicine says that it will be testing CRISPR-based gene editing in people by 2017, according to Technology Review.

At the EmTech conference, CEO Katrine Bosley said the startup — co-founded by the Broad Institute's Feng Zhang — plans to start a clinical trial within two years to test the use of gene editing to treat Leber congenital amaurosis, a rare form of blindness affecting the retina.

Though only some 600 people in the US have Leber congenital amaurosis, the University of Pennsylvania's Jean Bennett tells Tech Review that it presents a good test case. "The target that they have selected is fantastic; it has all the right characteristics in terms of making a correction easily," Bennett, who isn't associated with the Editas work, says.

Bosley notes that the company focused on the disease as the genetic mutation behind it is known and as the eye can be fairly easily targeted. She adds that they plan to use a gene therapy approach to deliver their CRISPR-based treatment.

That treatment would snip out about a thousand bases from the CEP290 gene in patients' photoreceptor cells. Preliminary lab tests indicate that the gene would then work correctly, Tech Review adds.