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Repairing and modifying disease using a cell’s blueprint for building proteins
Messenger RNA (mRNA) is the cell’s blueprint for building proteins. It is a single stranded RNA molecule that encodes genetic information from DNA to be translated into functional protein molecules. By chemically modifying or creating synthetic variants of mRNA, they have the potential to be less immunogenic than endogenous mRNA when delivered systemically.
mRNA is a compelling therapeutic modality because of its ability to drive high-efficiency, dose-dependent, protein expression which represents a unique approach for regulating aberrant or absent protein function. Combined with state-of-the-art drug delivery systems, such as lipid nanoparticles (LNPs), they offer opportunities for the delivery of a wide range of next generation medicines to patients.1
Together with Moderna, we are investigating the potential of mRNA in a number of therapy areas. One of our mRNA therapies is designed to stimulate the formation of new blood vessels to protect heart muscle cells (cardiomyocytes) in patients with heart failure or after a heart attack and other ischaemic vascular diseases.2 This asset has now entered the clinical phase of development.
The development of mRNA therapies is a hugely exciting and innovative area. As our early research has suggested, they may one day provide reparative and disease modifying treatment options for patients with heart failure, diabetic foot ulcers and other ischaemic vascular diseases.
As part of our collaboration with Moderna, we are also investigating another mRNA therapy in patients with advanced solid tumours. In this case, the therapy is injected directly into a tumour. Localising treatment in this manner may prevent systemic toxicity that may otherwise occur.
At AstraZeneca, we are also advancing the use of novel lipid chemistries and LNPs to promote delivery of mRNA therapeutics.1 Our in vitro and in vivo research is also examining the mechanisms that transport mRNA and LNPs into the cell, or deliver them to specific cell types, when conjugated to specific peptides or ligands.
References
1. Yanez Arteta, Marianna, Tomas Kjellman, Stefano Bartesaghi, Simonetta Wallin, Xiaoqiu Wu, Alexander J. Kvist, Aleksandra Dabkowska, et al. 2018. “Successful Reprogramming of Cellular Protein Production through mRNA Delivered by Functionalized Lipid Nanoparticles.” Proceedings of the National Academy of Sciences of the United States of America 115 (15): E3351–60.
2. Gan, Li-Ming, Maria Lagerström-Fermér, Leif G. Carlsson, Cecilia Arfvidsson, Ann-Charlotte Egnell, Anna Rudvik, Magnus Kjaer, et al. 2019. “Intradermal Delivery of Modified mRNA Encoding VEGF-A in Patients with Type 2 Diabetes.” Nature Communications 10 (1): 871.
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We know that however innovative our science, however effective our medicines and delivery, to achieve all we want to achieve, we cannot do it alone.