Delivery of Macromolecular Therapeutics: Tackling a Billion Year Old Barrier
Presenter: Steven Dowdy (UCSD School of Medicine)
Date: December 17, 2019
Time: 9:00 PST/12:00 EST/17.00 GMT/18:00 CET/22:30 IST/Dec 18th 1:00 CST/2:00 JST/4:00 AEDT
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All macromolecular therapeutics, including ASOs, siRNAs, peptides, proteins, CRISPR, mRNA and non-viral DNA vectors, are taken up into cells by endocytosis. However, <1% to none of the endocytosed therapeutic cargo escapes into the cytoplasm and nucleus of the cell. Thus, for all macromolecular therapeutics, endosomal escape remains the rate-limiting delivery step that prevents their effective use to treat cancer, pandemic influenza, and other diseases. Our research is focused on addressing this problem by developing new chemistry to synthesize novel universal endosomal escape domains (uEEDs) with the goal of enhancing endosomal escape of macromolecular therapeutics by 10- to 100-fold in the absence of toxicity.
Dr. Dowdy is a cancer biologist, specializing in RNAi therapeutics and G1 cell cycle control. He received his PhD in Molecular Genetics from UC Irvine with Eric Stanbridge (1990) working on tumor suppressor genes and performed his postdoctoral fellowship with Bob Weinberg at MIT working on the biochemistry of the Rb tumor suppressor gene (1990-1994). From 1994-2001, He was an Assistant Professor at Washington University School of Medicine. In 2001, He moved his lab to UCSD School of Medicine, where he is a Professor in the Dept. of Cellular & Molecular Medicine. He was a Howard Hughes Medical Institute Investigator for 18 years (1994-2012). Dr. Dowdy’s lab developed the synthesis of RNAi triggers containing neutral, bioreversible phosphotriester groups to increase stability and deliverability. He serves/has served on Science Advisory Boards for biotechs, pharmas and non-profit institutes. He is currently a co-founder and Board Director of Solstice Biologics, an RNAi biotech.