Group Leader, Arrowhead Research Corporation
Arrowhead Madison, Suite C
465 Science Drive
Madison, WI 53711
After receiving a Ph.D. in Cell and Molecular Biology with Richard Burgess at McArdle in 1999, I worked at Mirus Bio Corporation on development of gene therapies. Initially I applied my training in transcription mechanisms to developing expression vectors that produce high levels of stable gene expression in the liver and in muscle, followed by vectors that expressed short interfering RNA (siRNA) or short hairpin RNA for RNA interference. From 2006-2010, I worked with Jon Wolff, M.D., University of Wisconsin-Madison and Mirus Bio, to develop a gene therapy for Duchenne muscular dystrophy, funded by the Association Française contre les Myopathies. Using the mdx mouse model of Duchenne muscular dystrophy, as well as rodent and nonhuman primate models for delivery, I led the pharmacology and delivery animal studies. Mirus Bio was acquired by Hoffmann-La Roche, becoming Roche Madison, and was then transferred to Arrowhead Research Corporation. At Arrowhead I have been Group Leader responsible for pharmacology in the development of RNA interference therapeutics to treat chronic hepatitis B virus infection and liver disease due to the Z mutant form of alpha-1 antitrypsin. I lead my research teams and collaborators to demonstrate efficacy of ARC-520 in animal models of HBV infection and efficacy of ARC-AAT for treatment of Z-AAT liver storage disease in the PiZ mouse model. Both of these therapeutics are now in clinical trials.