Alnylam scientists demonstrate new RNAi approach

In a paper published in Nature the scientists describe the rational design of a potential new class of chemically modified RNA-based drugs, called ‘antagomirs’, that specifically silence miRNAs across multiple tissue types following therapeutically relevant administration in animals.

The company believes this research creates the opportunity to design antagomirs that target miRNAs in the context of human diseases, such as cancer and viral infection.

“Our discovery of antagomirs represents the first-ever demonstration of a pharmacologic strategy to silence miRNAs. These antagomirs have remarkable properties including high potency, high specificity, broad bioavailability, and long-lasting effects, and we believe this may have profound implications for the treatment of many human diseases,” said Dr Markus Stoffel, professor of The Rockefeller University’s laboratory of metabolic diseases.

In the Nature paper, antagomirs that target miRNA-16, miRNA-122, miRNA-192, and miRNA-194 demonstrated specific in vivo silencing of the target miRNA in tissues including liver, lung, kidney, heart, and bone marrow. This silencing was shown to be sustained for over 20 days following a single treatment course.

Alnylam and The Rockefeller University have a collaboration agreement for research in the field of RNAi. Alnylam has taken an exclusive license to all of The Rockefeller University’s interest in antagomir technology.