Anti-inflammatory mRNA-LNPs for the treatment of Multiple Sclerosis

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 aims to develop antigen-specific mRNA-LNP therapies for multiple sclerosis (MS) by leveraging non-inflammatory lipid nanoparticle formulations to selectively suppress myelin-specific immune responses without the systemic side effects of broad immunosuppression. Building on the collaborators' identification of a DC-targeted, non-adjuvant LNP formulation, this proposal will (1) optimize LNP delivery to dendritic cells to attenuate disease progression, (2) characterize CNS cellular responses to tolerogenic LNP treatment, and (3) evaluate the efficacy of diverse myelin antigens across preclinical MS models. Findings from these studies will lay the groundwork for next-generation, antigen-specific immunotherapies for MS and related autoimmune diseases of the CNS.