Novartis Teams Up with Alnylam to Explore Targeted Liver Therapies

Novartis announced a collaboration with Alnylam Pharmaceuticals to develop a treatment that can restore liver function and offer an alternative to transplants in patients with end-stage liver disease. The collaboration will leverage Alnylam’s proven and proprietary siRNA technology to inhibit an unspecified drug target discovered at the Novartis Institutes for biomedical Research. 

Implications of End-stage Liver Disease (ESLD) 

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 most common causes of ESLD are alcoholic liver disease, hepatitis C and hepatic steatosis (fatty liver) resulting from obesity.  ESLD accounts for over one million deaths globally each year apart from having a profound impact on patients’ quality of life. 

Currently, liver transplantation is the only treatment for ESLD and a significant need exists for medicinal alternatives that regenerate liver tissue and restore the essential metabolic and synthetic processes that are managed by the liver. Transplants are not only expensive, invasive procedures, but also not feasible due to a limited supply of organs available for patients in need.

The Collaboration and Its Objectives

“There remains an enormous unmet need for new types of medicines to address end-stage liver disease,” said Jay Bradner, President of the Novartis Institutes for biomedical Research. 

“Building on a legacy of leadership in regenerative medicine, we have devised a restorative strategy that could potentially deliver a transformative benefit to patients with liver failure. We’re delighted now to work alongside Alnylam in this new collaboration, as the Alnylam siRNA platform is optimally suited to translate this concept to clinical investigation.”

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.

“We are so pleased to collaborate with Novartis,” said Kevin Fitzgerald, Ph.D., Chief Scientific Officer at Alnylam. “We believe collaborations like this serve as an excellent example of how Alnylam’s leadership in RNAi can fuel new frontiers of medicine with highly innovative targets coming from some of the most admired pharmaceutical companies.”

Author

Arvind C. Shekhar