Effective Delivery of Nucleic Acid Therapeutics via Designed Nanocarriers

ongoing

Delivery of Nucleic Acid Therapeutics to desired organs has become the current challenge of biotechnological applications. Advancements on the chemical synthesis or targeting ligands, formulation of complex lipid systems, and their bioassays are essential to establish new gene therapies. Therefore, the main aim of NATPRIME DN is to establish a multidisciplinary training network on the emerging topic of nanoparticle-based adjuvants to deliver nucleic acid therapeutics (NAT). In the last decade, great efforts have been spent on the development of synthetic strategies for the creation of RNA based therapies/vaccines, and these efforts have been acknowledged by the Nobel committee in 2023. The next important step is targeted delivery, which is to carry NATs to the desired tissue. For this purpose, various nanoassemblies, i.e lipid nanoparticles, polyplexes, liposomes, that are modified with targeting ligands will be utilised to encapsulate NATS. Antibodies, peptides, glycans, and glycopolymers open up greater possibilities in the precise targeting of nanoparticles to desired organs/tissues/cells. The combined molecular toolbox of targeting ligands, charged lipids, helper lipids and PEG-replacement lipids need a high-throughput formulation-screening to ensure increased uptake of such particles in the desired organs. 15 interdisciplinary researchers will be trained on the design, synthesis, and characterisation of such complex targeting ligands, their formulation in nanoparticles, and their utilisation in the nucleic acid based treatments. Last but not least, biophysical understanding of molecular interactions of formulation components will bridge the gap between fundamental and applied research while broadening the horizon of ESRs from an academic lab to good manufacturing practice (GMP) synthesis facilities that can positively impact the well-being of future generations.