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.
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It is a great news for the company and patients!! FDA has done its job!! However, there are more to do!! About 80 genes could cause multiple cancers in lots of families; gene therapy in germline or embryo is necessary!!
Gene editing of the germline is bad business for pharma companies. You can’t sell cures if there are no ill patients