The Medicine by Design Global Speaker Series invites established and emerging international leaders in regenerative medicine to engage with our extraordinary community of researchers and clinicians.
Medicine by Design, in partnership with the Ontario Institute for Regenerative Medicine, is pleased to welcome Daniel G. Anderson, PhD, the Samuel A. Goldblith Professor of Applied Biology, associate professor in the Department of Chemical Engineering and Institute for Medical Engineering and Science, and a member of the Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology. He will give a talk titled, “Delivery systems for in vivo genome editing and cell therapy.”
High throughput, combinatorial approaches have revolutionized small molecule drug discovery. Here we describe our work on the combinatorial development of biomaterials for medical devices ranging from nanoparticulate delivery systems to macroscopic devices. One focus of our work is on nanoparticulate, intracellular delivery systems for RNA therapy and gene editing. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated and screened for their ability to delivery macromolecular payloads inside of cells. A number of delivery formulations have been developed with in vivo efficacy, enabling gene suppression with siRNA, gene expression with mRNA, or permanent genetic editing using the CRISPR/Cas9 system. These formulations show potential therapeutic application for the treatment of disease in both rodent and primate models. A second focus of our work is on developing biomaterials that can avoid the fibrotic response common to implanted medical devices. Using combinatorial chemistry, we have developed new materials capable of avoiding fibrosis and scar tissue formation. These show particular promise as vehicles for the immune-isolation of transplanted cells, for the treatment of diabetes. When formulated into microcapsules these materials enable functional, long-term islet transplantation in immune competent, diabetic rodents, as well as normal non-human primates.
Learn more about Daniel G. Anderson