Alnylam Pharmaceuticals, Inc. announced a collaboration with Novartis to leverage Alnylam's proven, proprietary siRNA technology to inhibit a target discovered at the Novartis Institutes for BioMedical Research, potentially leading to development of a treatment designed to promote the regrowth of functional liver cells and to provide an alternative to transplantation for patients with liver failure. End-stage liver disease (ESLD) is a progressive illness, most often resulting from cirrhosis, that is characterized by the destruction of healthy liver tissue and the loss of critical liver function. The disease has a profound impact on patients' quality of life, and accounts for over one million deaths globally each year.

Currently, liver transplantation is the only treatment for ESLD, but transplants are invasive procedures and there is a limited supply of organs available for patients in need. A significant need exists for medicinal alternatives to transplantation that regenerate liver tissue and restore the essential metabolic and synthetic processes that are managed by the liver. During the exclusive three-year research collaboration, Alnylam will develop and test potential siRNAs using target-specific assays developed by Novartis.

Once a lead candidate is identified, further development and clinical research will be conducted by Novartis. RNAi (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. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine.

By harnessing the natural biological process of RNAi occurring in cells, Alnylam developed a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.