Alnylam Announces Collaboration with Novartis to Explore Targeted Therapy to Restore Liver Function

– Agreement will Bring Alnylam’s Proprietary siRNA Technology to Bear on Liver Target Identified by Novartis Researchers –

– Alnylam and Novartis have Agreed to Collaborate on Discovery and Development of siRNA-based Targeted Therapy to Restore Functional Liver Cells in Patients with End-Stage Liver Diseases --

– Collaboration Aims to Develop Liver-Targeted Therapy as a Potential Alternative to Transplantation for Patients Experiencing Liver Failure –

CAMBRIDGE, MA, USA I January 6, 2022 I Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today 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.

“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.”

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.

“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.

About Alnylam RNAi Therapeutics
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 our 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.

About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO® (lumasiran), as well as Leqvio® (inclisiran), with Alnylam’s partner Novartis, which has obtained global rights to develop, manufacture and commercialize inclisiran under a license and collaboration agreement. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam, on LinkedIn, or on Instagram.

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.

SOURCE: Alnylam Pharmaceuticals

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