RNA interference: Therapy by switching off genes
RNA interference (RNAi) was first observed in plants and some invertebrate animals (e.g. fruit flies). However, Thomas Tuschl and his colleagues from the Max Planck Institute for Biophysical Chemistry in Göttingen were the first to succeed in establishing the functioning of RNAi in mammalian cells as well. In the RNAi procedure developed by Thomas Tuschl, synthetically manufactured RNA strands are inserted into a cell. These bind themselves to complementary messenger RNAs (mRNAs), which are then removed or destroyed through a complex cellular mechanism. This reduces or prevents the implementation of genes in proteins. Due to RNAi, it is possible to specifically deactivate individual genes so that the information contained in the gene is not passed on and the protein concerned is not produced. The discovery of this “switch” represents an extremely promising approach to the treatment of a very wide range of diseases, some of which are currently still not accessible to treatment by drugs. Consequently, RNAi therapeutics constitutes a significant new class of drugs. In future, they could be used for the treatment of viral diseases, metabolic diseases and cancers, and could be of similar importance as monoclonal antibodies already today.
Alnylam, a leading company in the development of RNAi therapeutics, is out licensee. In 2018, Alnylam announced the very first FDA approval of an RNAi therapeutic - ONPATTRO ™ (Patisiran) - for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.