Self-Delivering RNAi Compounds—a Novel Platform for Therapeutic Development
Joanne Kamens,
Senior Director of Discovery Research
RXi Pharmaceuticals
Additional Authors: Joanne Kamens, Wes Solomon, James Cardia, William Stanney, Pamela Pavco, Dmitry Samarsky, Anastasia Khvorova
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Abstract: Various patterns and compositions of chemical modifications on RNAi compounds have been used to improve diverse therapeutic properties of oligonucleotides including potency, stability, specificity, tissue distribution and cellular uptake. We have recently developed a new type of RNAi compound termed self-delivering-rxRNAs (sd-rxRNAs). These compounds are characterized by improved tissue distribution properties and spontaneous cellular uptake—sd-rxRNAs can enter cells and silence target gene expression with no additional formulation or cellular treatment. This novel functionality results in compounds with improved tissue distribution properties. Data on functionality in diverse cell types, kinetics of cell entry, and demonstration of in vivo uptake will be presented.
Presenter Biography: Dr. Kamens did her undergraduate work in Biology at the University of Pennsylvania. She received her Ph.D. in Genetics from Harvard Medical School's Division of Medical Sciences. Dr. Kamens spent 15 years in Worcester, MA at BASF Bioresearch Center which became Abbott Bioresearch Center in 2001. While at Abbott, she led discovery research projects on small molecule and antibody approaches to immunological and inflammatory diseases, ultimately serving as a Group Leader in Molecular Biology. Dr. Kamens directed the development of RNA interference (RNAi) methods in primary immune cells. In 2007 she joined RXi Pharmaceuticals and is now serving as Senior Director of Discovery Research. RXi is a discovery-stage biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNAi for the treatment of human diseases. Dr. Kamens founded the Boston chapter of AWIS (Association for Women in Science) and is now serving on the boards of WEST (Women Entrepreneurs in Science and Technology) and the Boston HBA Mentoring Committee (Healthcare Businesswomen’s Association).
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