November 26, 2019
Novartis’ $9.7 billion move for inclisiran developer, the Medicines Co, and US approval for Alnylam’s Givlaarim have put RNA interference back in the headlines.
RNAi therapies treat disease by interfering with – or “silencing” – genes.
Drugs like inclisiran and Givlaarim bind mRNA transcribed from target genes, forming double stranded molecules that cannot be translated into protein.
image: iStock/kentoh
Inclisiran’s target mRNA encodes PCSK9, a protein that hampers the body’s ability to absorb low-density lipoprotein (LDL) [1]. By minimizing PCSK9 levels inclisiran allows the body to process more cholesterol.
The RNAi approach is analogous to “turning off the faucet” rather than clearing up the flood according to the Medicines Company’s Michael Blash, who says targeting the root cause of a disease has advantages.