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 Abstract:

RNA therapeutics hold immense clinical potential for revolutionizing the treatment of a broad range of diseases. The ability to deliver therapeutic RNA to either express or inhibit specific proteins in situ opens up numerous clinical applications, including vaccine development, protein replacement therapy, cancer immunotherapy, and gene editing. In my talk, I will discuss modular platforms designed to expand the utility of mRNA drugs beyond their current application in vaccines. First, I will present the development of a library of extracellular vesicle-inspired lipid nanoparticles designed to enable the targeted delivery of RNA-based drugs to central nervous system (CNS) organs while minimizing off-target effects. I am currently leveraging this technology to address challenging CNS disorders, such as autism spectrum disorders and brain tumors. The success of this approach has led to the establishment of a spin-off company, Script Biosciences, which is focused on developing genetic medicines for the treatment of Huntington’s disease. Second, I will highlight the discovery of a macrophage-mediated mRNA delivery mechanism in the lungs, which can be harnessed to target lung diseases. Finally, I will discuss the development of an injectable hydrogel platform for localized delivery of RNA medicines and gene-editing tools. This platform represents a significant advancement in RNA medicine, with applications ranging from tissue engineering to protein replacement therapies and immunoengineering. My future research program will focus on combining my expertise in extracellular vesicles, biomaterials, bioprinting, and gene therapies to engineer bioinspired strategies for studying and treating neurological diseases.

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