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Medical and health sciences
- Respiratory medicine
- In vitro testing
- Biopharmaceuticals
- Pharmaceutical technology
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Engineering and technology
- Gene and molecular therapy
Lipid nanoparticles (LNPs) have emerged as the dominant platform for RNA delivery, as exemplified by the COVID-19 mRNA vaccines. However, limited cytosolic RNA delivery efficiency and severe inflammation induced by ionizable lipids strongly hamper the extrapolation of RNA LNPs to other (chronic) therapeutic applications, including the lung.
This project envisions the development of innovative LNP designs for safe and effective RNA delivery to the lung via the introduction of peptides. First, we aim to further validate our technology for enhanced RNA delivery on a selection of LNPs and optimize peptide-LNPs to identify the best performing compositions and to strengthen an anticipated patent application. Second, we will work on the manufacturability and stability of lead formulations. Third, in vivo proof-of-concept studies will be conducted to demonstrate the superior RNA delivery over conventional ionizable LNP benchmarks in a pre-clinical animal model for inhalation therapy.