Earlier this month, the FDA approved an entirely new family of drugs, one so powerful that it could put CRISPR-based gene therapy to shame. Backed by two decades of research and a Nobel Prize, these drugs have the ability to cure inherited diseases—and do so without actually needing to edit the delicate genome.
The green-lit therapy is patisiran, a drug ten years in the making by the Cambridge-based Alnylam for genetic nerve damage. But in this rare case, the nature of the drug is perhaps more significant: patisiran is based on a technology called RNA interference, or RNAi, which allows doctors to silence genes that aren’t properly functioning.
Patisiran is just the tip of the iceberg. In theory, RNAi has the potential to cure any disease caused by “bad” proteins, such as stroke, Alzheimer’s, high cholesterol, or other neurodegenerative disorders. The potential is so grand that scientists have long dubbed the technology a way to “drug the undruggable.”
“This approval is part of a broader wave of advances that allow us to treat disease by actually targeting the root cause, enabling us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms,” said FDA commissioner Scott Gottlieb in a press release. “New technologies like RNA inhibitors, that alter the genetic drivers of a disease, have the potential to transform medicine, so we can better confront and even cure debilitating illnesses.”
With other RNA-based therapies hurtling down the drug development pipeline, it’s likely we’ll be seeing more of these drugs soon. Here’s what you need to know.
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