Ongoing Development of Lipid Nanoparticles as Drug Delivery Systems

Origin: The Tang Prize is awarded on a biennial basis, each with a cash reward of NT$40 million. Projects proposed by the laureates also receive a grant of up to NT$10 million. The 2022 grant for Biopharmaceutical Science is shared between Dr. Katalin Kariko, Dr. Drew Weissman and Dr. Pieter Cullis.

Project Summary: This research proposal has the overall goal of supporting the development of next-generation, rationally designed LNP systems with increased potency and reduced toxicity that enable transfection of both hepatic and extra-hepatic tissues following intravenous (i.v.) administration. The long-term goal is to achieve global gene therapy following an i.v. administration of LNP mRNA systems. Once achieved, such systems can be used to treat diseases, such as mitochondrial disorders, that affect every cell in the body. In pursuit of this endeavor, the specific goal of this proposal is to fund a series of pilot projects on LNP development for nucleic acid delivery. Data generated from successful pilot studies will be leveraged in larger grant applications in order to fund one or more comprehensive and large-scale research programs on rational design and therapeutic uses of LNP systems.