AstraZeneca Partners with Silence Therapeutics to Develop siRNA Therapies for Cardiovascular, Renal, Metabolic, and Respiratory Diseases

March 25, 2020/BioValleyBIOON/--AstraZeneca(AstraZeneca) recently announced that it will collaborate with Silence Therapeutics to discover, develop, and commercialize small interfering RNA (siRNA) therapy for the treatment of cardiovascular, renal, metabolic, and respiratory diseases.

This multi-target collaboration will leverage Silence Therapeutics’ siRNA platform to identify and develop liver-based targets, as well as to develop novel delivery methods for targeting other tissues such as the heart, lungs, and kidneys. Targeted drug delivery to these tissues represents a new opportunity for the treatment of cardiovascular, renal, metabolic, and respiratory diseases.

Mene Pangalos, Executive Vice President of Biopharmaceuticals R&D at AstraZeneca, stated: “Our collaboration with Silence Therapeutics adds an exciting new modality—siRNA—to our drug discovery toolkit. Importantly, we can apply this therapeutic modality across key therapeutic areas in cardiovascular, renal, metabolic, and respiratory diseases to target novel pathways that are not amenable to more traditional drug discovery approaches.”

Iain Ross, Executive Chairman of Silence Therapeutics, stated: “We are pleased to announce our collaboration with AstraZeneca, as this further validates our proprietary siRNA platform. This partnership not only provides us with the opportunity to collaborate on specific liver-expressed gene targets, but also offers us the chance to work with a leading company to achieve targeted delivery of siRNA molecules to other tissues, including the heart, kidneys, and lungs.”

Small interfering RNA (siRNA) is a double-stranded RNA molecule that functions within cells via the RNA interference (RNAi) mechanism to modulate protein production, thereby offering new opportunities for therapeutic intervention. These agents exert their RNAi effects by targeting specific messenger RNAs (mRNAs), thereby preventing the synthesis of disease-causing proteins.

Currently, RNA interference (RNAi) has become a hotspot in new drug development, with Alnylam Pharmaceuticals as the global leader in this field. In August 2018, the company’s drug Onpattro (patisiran) received approval from the United States and the European Union, becoming the first RNAi therapeutic approved for market launch in the 20 years since the discovery of the RNAi phenomenon. This medication is indicated for the treatment of adult patients.GeneticsHereditary transthyretin-mediated (hATTR) amyloidosis. In November 2019 and March 2020, the company’s another drug, Givlaari (givosiran), was approved in the United States and the European Union, respectively, becoming the second RNAi therapeutic approved globally. This medication is indicated for the treatment of adult patients with acute hepatic porphyria (AHP). Furthermore, the company’s third RNAi therapeutic, lumasiran, has initiated a rolling submission of its New Drug Application (NDA) in the United States for the treatment of primary hyperoxaluria type 1 (PH1).

November 2019,NovartisSpending $9.7 billion to acquire The Medicines Company (TMC) and obtaining the RNAi drug inclisiran. This drug was developed by Alnylam Pharmaceuticals using its proprietary Enhanced Stabilization Chemistry-GalNAc conjugation technology (ESC-GalNAc), which enables GalNAc chemical modification of RNA fragments, enhancing stability and promoting liver-targeted delivery of the drug. TMC signed a licensing and collaboration agreement with Alnylam, securing global rights for the development, manufacturing, and commercialization of inclisiran.

Inclisiran is the first cholesterol-lowering therapy in the small interfering RNA (siRNA or sir-nah) class, targeting proprotein convertase subtilisin/kexin type 9 (PCSK9). In three pivotal Phase III clinical studies, inclisiran administered subcutaneously twice yearly after two initial doses demonstrated sustained and effective reduction of LDL-C.

In December 2019, TMC Inc. has submitted to the United StatesFDAA New Drug Application (NDA) for inclisiran has been submitted for the secondary prevention in patients with atherosclerotic cardiovascular disease (ASCVD) or familial hypercholesterolemia (FH). Under the signed agreement, Alnylam Pharmaceuticals will receive development milestone payments for inclisiran and royalties of up to 20%. (Bioon.com)

Original Source: AstraZeneca to discover and develop siRNA therapeutics for Cardiovascular, Renal, Metabolic and Respiratory diseases in new collaboration