Sanofi and Alnylam have agreed to conclude the research and option phase of the companies’ RNAi therapeutics alliance in rare genetic diseases, which was established in 2014.

As part of the agreement, Alnylam will advance an additional investigational asset in an undisclosed rare genetic disease through the end of IND-enabling studies.

“Our alliance with Alnylam has successfully brought one important medicine to patients with ATTR amyloidosis and it also has spawned a molecule that is in pivotal clinical trials for people with haemophilia,” said John Reed, global head of Research and Development at Sanofi.

He continued, “As we wrap up this phase of our programme with Alnylam, we remain committed to advancing therapies for patients with rare diseases and rare blood disorders.”

RNAi, or RNA interference, is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines has been realised, known as RNAi therapeutics.

It is a biological process in which RNA molecules inhibit gene expression or translation, by neutralising targeted mRNA molecules. Since the discovery of RNAi and its regulatory potentials, it has become evident that RNAi has immense potential in suppression of desired genes.

Sanofi will be responsible for any potential further development or commercialisation of such asset, and if this product is approved, Alnylam will be eligible to receive tiered double-digit royalties on its global net sales.