CANbridge Enters DMD Gene Therapy Research Agreement with UW School of Medicine

China based rare disease biotech CANbridge Pharmaceuticals entered into a two-year sponsored research agreement with the University of Washington School of Medicine for gene therapy research in Duchenne muscular dystrophy, a rare neuromuscular disease.

Photo: James Xue, founder, chairman and CEO of CANbridge Pharmaceuticals

The program will be under the direction of Jeffrey Chamberlain, professor in the Departments of Neurology, Medicine and Biochemistry, the McCaw Endowed Chair in Muscular Dystrophy at the University of Washington School of Medicine, and director of the Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center of Seattle. Guy Odom, research assistant professor in the Department of Neurology at the University of Washington, will serve as the co-principal investigator.

Chamberlain is a pioneer and one of the top researchers in the field of gene therapies for muscle diseases. His lab has been studying muscular dystrophy mechanisms, particularly dystrophin structure, and gene therapy approaches. They were the first to show that adeno-associated virus (AAV) vectors could be used for systemic gene delivery to muscle.

Duchenne muscular dystrophy (DMD) is a rare muscle disorder, but it is one of the most frequent genetic conditions that primarily affects males. DMD usually presents in early childhood and is characterized by rapidly progressive muscle degeneration and weakness, leading to loss of ambulation by about 12 years of age. Cardiomyopathy is a common cause of morbidity and death in DMD patients. The incidence of DMD is estimated to be 1/3,500 – 1/5,000 male births worldwide and 1/4,560 in China, according to the National Organization for Rare Disease and published peer review.

“Duchenne muscular dystrophy is the most common of the hereditary neuromuscular diseases and, despite recent approvals for exon-skipping therapies, remains severely underserved medically.,” said James Xue, founder, chairman and CEO of CANbridge Pharmaceuticals. “We believe that the best gene therapy for this devastating disease has not yet been discovered, and we look forward to working with Dr. Chamberlain and his team on their innovative research, as well as the new treatments that may arise from it.”

Author: Rare Daily Staff