Silencing protein production inside cells


Small interfering RNA (siRNA) are double-stranded RNA molecules that offer new opportunities for therapeutic intervention because they act inside the cell to reduce protein expression. They do this by targeting RNA to prevent the production of disease-associated proteins.1-3

Through our collaboration with Silence Therapeutics, we aim to develop novel, targeted treatments to address significant unmet need for the treatment of Cardiovascular, Renal, Metabolic and Respiratory diseases.

Harnessing Silence’s established siRNA platform, the collaboration will identify and progress liver-based targets, as well as develop new delivery approaches for targeting other tissues such as the heart, lungs and kidneys. Targeted delivery to these other tissues represents a new opportunity to treat Cardiovascular, Renal, Metabolic and Respiratory diseases.


The collaboration with Silence adds an exciting modality, siRNA, to our toolbox to help us develop novel therapeutics and address unmet medical needs.

Regina Fritsche Danielson Senior Vice President, Research and Early Development, Cardiovascular, Renal and Metabolism, BioPharmaceuticals R&D


References

1. Dana H, Chalbatani GM, Mahmoodzadeh H, et al. Molecular Mechanisms and Biological Functions of siRNA. Int J Biomed Sci. 2017;13(2):48-57.

2. Humphreys SC, Thayer MB, Campbell J, et al. Emerging siRNA Design Principles and Consequences for Biotransformation and Disposition in Drug Development. J Med Chem. 2020;63(12):6407-6422.

3. Setten RL, Rossi JJ, Han SP. The current state and future directions of RNAi-based therapeutics [published correction appears in Nat Rev Drug Discov. 2019 Mar 18;:] [published correction appears in Nat Rev Drug Discov. 2019 Apr 24;:]. Nat Rev Drug Discov. 2019;18(6):421-446.



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