CRISPR technology leaps from lab to industry

Helen Shen in Nature:

Instead of taking prescription pills to treat their ailments, patients may one day opt for genetic 'surgery' — using an innovative gene-editing technology to snip out harmful mutations and swap in healthy DNA. The system, called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), has exploded in popularity in the past year, with genetic engineers, neuroscientists and even plant biologists viewing it as a highly efficient and precise research tool. Now, the gene-editing system has spun out a biotechnology company that is attracting attention from investors as well.

Editas Medicine, based in Cambridge, Massachusetts, announced its launch on 25 November with an initial US$43 million venture capital investment. The company, founded by five leading CRISPR researchers, aims to develop therapies that directly modify disease-related genes. “This is a platform that could have a profound impact on a variety of genetic disorders,” says interim president Kevin Bitterman, a venture capitalist at Polaris Partners in Waltham, Massachusetts, which is one of Editas' backers. CRISPR piggybacks on an immune strategy that bacteria use to detect and chop up foreign DNA. The DNA-cutting enzyme Cas9 finds its target with the help of an RNA guide sequence that researchers can now engineer to home in on potentially any gene of interest. Editas is not disclosing its intended targets, but the technology might be tried first on diseases caused by a single faulty gene copy, says Feng Zhang, a neuroscientist at the Massachusetts Institute of Technology’s McGovern Institute for Brain Research in Cambridge, Massachusetts, and one of Editas’ founders. Simply disabling the disease-causing copy could clear the way for the good copy to take over.

More here. (Note: I sincerely believe that this is one of the most significant scientific discovery of our century; something as important as the discovery of shRNA or PCR!)