Jeffrey Chamberlain, PhD

Chief Scientific Advisor

Dr. Chamberlain is a co-founder of Kinea Bio and a professor in the Departments of Neurology, Medicine, and Biochemistry, the McCaw Endowed Chair in Muscular Dystrophy at the University of Washington School of Medicine. Dr. Chamberlain also serves as the Director of the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center of Seattle. Jeff’s research group has a long-standing interest in the muscular dystrophies, focusing on gene therapy. Research in his lab focuses on dystrophin and the dystrophin-glycoprotein complex and developing treatments for associated disorders. His group invented ‘micro-dystrophin’ (beginning in 1996) and discovered that AAV can be used for body wide, systemic gene delivery to muscle (2004). Together with the Hauschka lab, they isolated the first muscle specific enhancer element (from the MCK gene), which has been engineered to make hundreds of muscle specific expression cassettes (MSECs) for use in gene therapy. Several of their vector designs are being tested in human clinical trials for Duchenne muscular dystrophy, including the initial MCK-microdystrophin vector that was adapted by Sarepta and which became the first FDA approved gene therapy for a muscular dystrophy (2023). In addition to DMD, his group is studying gene therapy for several forms of Limb-Girdle Muscular Dystrophy.  In May 2023, Dr. Chamberlain was elected as President of American Society of Cell and Gene Therapy. In January 2024, Dr. Chamberlain received the prestigious Muscular Dystrophy Association (MDA) Legacy Award for his achievement in translational research on neuromuscular diseases.