Agreement will bring Alnylam’s proprietary siRNA technology to bear on liver target identified by Novartis researchers

Novartis and Alnylam have agreed to collaborate on discovery and development of siRNA-based targeted therapy to restore functional liver cells in patients with end-stage liver disease

Collaboration aims to develop liver-targeted therapy as a potential alternative to transplantation for patients experiencing liver failure
Basel, January 6, 2021 — Novartis today 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 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.

“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.”

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.1 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.

“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.”

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.

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” etc. etc..

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References

Potosek, Jamie et al. Integration of palliative care in end-stage liver disease and liver transplantation. Journal of Palliative Medicine. 2014;17(11):1271-1277. doi:10.1089/jpm.2013.0167
Asrani SK, Devarbhavi H, Eaton J, Kamath PS. Burden of liver diseases in the world. J Hepatol. 2019 Jan;70(1):151-171. doi: 10.1016/j.jhep.2018.09.014. Epub 2018 Sep 26. PMID: 30266282.
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