By using genetic therapy based on RNA interference and a new piece of medical hardware, a pair of biotechnology companies are working together to deliver gene therapy directly to the brain. The companies are looking to treat Huntington’s disease, a neurological problem caused by the loss of neurons due to a toxic protein made by a tainted gene. “The idea behind the new treatment is to stop at least some of that protein’s production so that it cannot damage the brain.” The procedure would take advantage of a medical device already implanted in over 250,000 patients.
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What’s the Big Idea?
One of the companies, Alnylam, has just successfully completed a clinical trial in which gene therapy produced lasting results by removing protein deposits which can build up dangerously in the liver. “The positive results add weight to the notion that RNAi therapeutics could eventually help patients with a range of genetic diseases.” Barry Greene, president and chief operating officer of Alnylam, said: “Today’s platforms target the protein that causes the disease and bind to that protein. We stop the protein from being made in the first place.”