Science, Space and Technology News 2024
Alnylam Pharmaceuticals is translating the promise of RNA interference research into a new class of powerful, gene-based therapies. In this rendering, the green strand is the targeted mRNA, and the white object is the RNA-induced silencing complex that can prevent the expression of the target mRNA’s proteins. The orange strand is RNAi. Credit: Courtesy of Alnylam Pharmaceuticalsprofessors and former postdocs, has developed five FDA-approved RNAi medicines, significantly impacting patient lives.
Today Alnylam has five medicines approved by the U.S. Food and Drug Administration and a rapidly expanding clinical pipeline. The company’s approved medicines are for debilitating, sometimes fatal conditions that many patients have grappled with for decades with few other options. “Alnylam has published more than 250 scientific papers over 20 years,” says Sharp, who currently serves as chair of Alnylam’s scientific advisory board. “Not only did we do the science, not only did we translate it to benefit patients, but we also described every step. We established this as a modality to treat patients, and I’m very proud of that record.”MIT’s involvement in RNAi dates back to its discovery.
Early on, Alnylam collaborated with MIT drug delivery expert and Institute Professor Bob Langer. Eventually, Alnylam developed the first lipid nanoparticles that could be used to encase RNA and deliver it into patient cells. LNPs were later used in the mRNA vaccines for Covid-19. Since that first treatment, Alnylam has updated its RNAi delivery system — including by conjugating small interfering RNAs to molecules that help them gain entry to cells — and earned approvals to treat other rare genetic diseases along with high cholesterol . All of those treatments primarily work by silencing genes that encode for the production of proteins in the liver, which has proven to be the easiest place to deliver RNAi molecules.
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