Novartis, to explore targeted therapy to restore liver function, collaborates with Alnylam

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Novartis, on Thursday, announced a collaboration with Alnylam to leverage Alnylam’s proven, proprietary siRNA technology to inhibit a target discovered at the Novartis Institutes for BioMedical Research, potentially leading to the 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.

As a part of the three-year exclusive research agreement, Alnylam will develop and test potential siRNAs leveraging Novartis’ target-specific assays. Novartis will conduct development and clinical testing after the selection of a lead candidate.

“There remains an enormous unmet need for new types of medicines to address end-stage liver disease,” 

Jay Bradner, President, Novartis Institutes for BioMedical Research

He added, “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.”

End-stage liver disease (ESLD) is a progressive illness defined by the destruction of healthy liver tissue and the loss of critical liver function. It is most commonly caused by cirrhosis. The disease has a massive impact on patients’ quality of life and is responsible for approximately one million deaths per year around the world.

“We are so pleased to collaborate with Novartis. 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.”

Kevin Fitzgerald, Ph.D., Chief Scientific Officer at Alnylam

Currently, the only remedy for ESLD is liver transplantation, however, transplants are invasive procedures with a limited supply of organs accessible for those in need. There is a great demand for medical alternatives to transplantation that regenerate liver tissue and restore the liver’s critical metabolic and synthetic functions.

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