The experimental drug has startling powers: It can turn down a mutant gene in a patient’s body, stopping the production of proteins that cause a terribly painful rare disease.
A crucial, late-stage clinical trial showed that the drug works — and that it’s safe. And now the biotech company behind it, Alnylam, is poised to bring this first-of-its-kind therapy to market.
The news has thrilled both patients and scientists, who have been working for decades on the technology to mute misbehaving genes, known as RNA interference, or RNAi. They’ve understood for two decades how the biology works. But it’s been a long, slow slog to figure out how to deliver RNAi therapies to the right cells safely and effectively. Alnylam alone has spent 15 years, and more than $1 billion, on the effort.
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