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CRISPR treatment for rare genetic eye disorder gains FDA study approval

| | December 10, 2018

Days after a Chinese researcher incensed the world of science with claims of editing the genomes of twin girls, an American company is plotting a CRISPR trial of its own. But in place of the secrecy and stagecraft that marked the Chinese experiment, Editas Medicine went the old-fashioned way: waiting for approval from the Food and Drug Administration.

The company, headquartered in Cambridge, Mass., got the FDA’s blessing to test a CRISPR-based therapy on patients with a rare genetic disorder that leads to blindness. Editas, which is partnered with Botox maker Allergan, said it plans to enroll between 10 and 20 patients in a study to test the treatment’s safety and efficacy.

The timing is entirely coincidental: Editas asked the FDA’s permission back in October, long before He Jiankui went public with his controversial experiment. And the two ideas, while both employing CRISPR, are vastly different: While He edited embryos, making genomic changes that would be passed down to future children, Editas is targeting cells in the eye that play no role in reproduction.

Related article:  Viewpoint: Biologist Craig Mello knew about the CRISPR babies. Why his silence was 'not acceptable'

BIO, the industry’s lobbying group, issued a statement [November 28] calling the Chinese experiment “deeply troubling” and underlining the fact that none of its member companies are working on CRISPR projects that would result in heritable changes to anyone’s DNA.

Read full, original post: FDA signs off on Editas CRISPR study on patients with a rare genetic disorder

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