The FDA approved antisense drug Kynamro to treat homozygous familial hypercholesterolemia, a rare, inherited disorder. The technology on which the drug is based was initially developed by Gilead Sciences, which sold its patents to Isis Pharmaceuticals. Sarepta Therapeutics and Prosensa are developing gene-silencing drugs to treat muscular dystrophy.
20 years and $2B later, FDA approves gene-silencing drug
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