Sanofi is betting as much as $750 million on the power of tiny gene-regulating molecules to conquer the connective tissue disease fibrosis and other therapeutic areas as part of a new partnership with Regulus Therapeutics.

The molecules, called microRNAs, regulate the expression of more than a third of human genes, making them ripe targets for influencing the mechanisms of certain diseases, such as cardiovascular, metabolic, certain cancers. The company has already partnered it's hepatitis C and immuno-inflammatory disease programs with GSK.

 

"MicroRNAs are believed to be extremely important in human development and physiology," says Marc Cluzel, EVP of research and development for Sanofi. "Together with Regulus we will develop therapeutics which could potentially open a new paradigm in the treatment of major diseases and could offer an attractive new therapeutic approach for patients."

Sanofi's deal with Regulus, which will initially focus on fibrosis, is the largest microRNA partnership formed to date according to the companies. It includes a $25 million upfront fee for Regulus, a $10 million equity investment, and annual research support for up to five years.

 

Regulus was formed in 2007 as a joint venture between Alnylam and Isis Pharmaceuticals. The company says its pre-clinical fibrosis program has already shown promise in reversing fibrosis and significantly improving cardiac function in mice with failing hearts.

 

Cystic fibrosis, an inherited chronic disease affecting the lungs and digestive system, affects about 30,000 children and adults in the United States and about 70,000 people worldwide, according to the Cystic Fibrosis Foundation.

 

Idiopathic pulmonary fibrosis, another fatal form of the disease also affecting the lungs, affects about 128,000 people in the United States, with about 48,000 new cases diagnosed annually, according to the Coalition for Pulmonary Fibrosis.