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Daniel J. Siegwart, PhD
University of Texas Southwestern Medical Center
Talk Title: Development of SORT lipid nanoparticles (LNPs) for genome correction of disease-causing mutations
Daniel J. Siegwart is a Professor in the Department of Biomedical Engineering at the University of Texas Southwestern Medical Center. He holds the W. Ray Wallace Distinguished Chair in Molecular Oncology Research and serves as the Director of the Program in Genetic Drug Engineering and Co-leader of the Chemistry and Cancer Program in the NCI-designated Simmons Comprehensive Cancer Center (SCCC). He received a B.S. in Biochemistry from Lehigh University (2003), and a Ph.D. in Chemistry from Carnegie Mellon University (2008), studying with Professor Krzysztof Matyjaszewski. He also studied as an NSF EAPSI Research Fellow at the University of Tokyo with Professor Kazunori Kataoka (2006). He then completed an NIH NSRA Postdoctoral Fellowship at MIT with Professor Robert Langer (2008-2012). He has been elected as a Fellow to the National Academy of Inventors (NAI), the American Institute for Medical and Biological Engineering (AIMBE) College of Fellows, and the Controlled Release Society (CRS) College of Fellows. He was also recognized as an Emerging Leader in Health and Medicine (ELHM) Scholar by the National Academy of Medicine (NAM). His research laboratory utilizes materials chemistry to enable targeted nanoparticle delivery of genomic medicines. Their efforts led to an understanding of the essential physical and chemical properties of synthetic carriers required for therapeutic delivery of siRNA, miRNA, tRNA, pDNA, mRNA, and gene editors. His lab has been at the forefront in the design of synthetic carriers for gene editing and has applied these technologies for correction of genetic diseases and treatment of cancer. For example, they reported the first non-viral system for in vivo CRISPR/Cas gene editing. They also developed the first strategy for predictable tissue specific mRNA delivery and gene editing, called Selective ORgan Targeting (SORT) lipid nanoparticles (LNPs), which are being utilized in two separate Phase 1b human clinical trials to treat genetic respiratory diseases. They ultimately aspire to utilize chemistry and engineering to make a beneficial impact on human health.
