Novartis, Alnylam Leverage siRNA Technology for Liver Disease
The companies will leverage siRNA technology to inhibit a target discovered at the Novartis Institutes for BioMedical Research.
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By - Novartis and Alnylam recently collaborated to leverage proprietary siRNA technology to develop liver-targeted therapy as an alternative to transplantation for patients experiencing liver failure.
During the three-year collaboration, the companies will leverage siRNA technology to inhibit a target discovered at the Novartis Institutes for BioMedical Research. Once the companies identify a lead candidate, Novartis will lead the development and clinical research.
“There remains an enormous unmet need for new types of medicines to address end-stage liver disease,” Jay Bradner, president of the Novartis Institutes for BioMedical Research, said in the announcement.
“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,” Bradner continued.
End-stage liver disease (ESLD) is a progressive illness that often results from cirrhosis. ESLD accounts for over one million deaths globally each year.
Currently, a liver transplant is the only treatment for ESLD. But a transplant is an invasive procedure and dependent on there is a limited supply of organs available for patients in need, a Novartis spokesperson explained.
Therefore, medical alternatives to regenerate liver tissues and restore the essential metabolic and synthetic processes are vital.
A 2017 study found that RNAi therapeutics may be effective in treating liver disease due to their ability to manipulate the expression levels of specific genes in liver pathology to treat liver disease effectively to effectively treat liver disease.
In April 2020, Alnylam and Dicerna announced the development and commercialization of investigational RNAi therapeutics to treat alpha-1 liver disease.
Under the collaboration, the companies will explore Alnylam’s ATL-AAT02 and Dicerna’s DCR-A1AT. Both of these investigational RNAi therapeutics are in Phase 1/2 development for the treatment of liver disease.
Dicerna has the experience and resources to lead the development and commercialization, while Alnylam retains an commercialization option outside the US.
