for medicines and diagnostics, announced Tuesday it has signed a deal with Vertex Pharmaceuticals, a biotech heavyweight best known for its cystic fibrosis therapies, to develop new treatments for genetic diseases delivered directly into the body, signaling more mainstream medical use of gene therapy after years of setbacks....
While gene therapy—a potentially curative treatment replacing faulty genes with healthy ones—has technically been possible for decades, it has typically not been precise enough to use clinically, something Crispr changes. David Altshuler, Vertex’s chief scientific officer, said Mammoth’s ultra-small Crispr systems will give the company “another set of tools to tackle many of the diseases we’re interested in” by directly editing genes inside patients. Crispr, standing for “clustered regularly interspaced short palindromic repeats,” is a powerful set of gene-editing tools derived from the immune systems of bacteria.
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