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Antibody therapeutics for Duchenne Muscular Dystrophy

Genomic Applications Partnership Program
GAPP Round: 
Genome Centre(s):
Genome British Columbia
Project Leader(s):
Fabio Rossi & Michael Underhill (University of British Columbia)
Receptor Leader(s):
Carl Hansen (AbCellera Biologics)
Fiscal Year Project Launched: 
Project Description: 

AbCellera, a BC-based biotechnology company, will work with the laboratories of Dr. Fabio Rossi and Dr. Michael Underhill of the University of British Columbia to develop new antibody-based therapeutics for the treatment of fibrosis associated with Duchenne Muscular Dystrophy (DMD).  The collaboration will use AbCellera’s leading antibody discovery and development capabilities to discover and develop therapeutic antibodies against three novel targets for fibrosis.  This research builds on new insights in stem cell science from the Rossi and Underhill groups using a combination of innovative genomics tools and unique animal models.  An Investigational New Drug (IND) application for a first therapeutic will be made by the end of the project. Two other antibody therapeutics will be advanced to the point of IND-enabling studies, ensuring the research will translate to potential therapies for patients.

Duchenne muscular dystrophy (DMD) is one of the most common congenital diseases in the world, affecting 1 in 3500-6000 males (1 in 3500 in Canada). DMD is caused by mutations in the dystrophin gene that results in progressive muscle degeneration. There are currently no effective treatments for DMD.  A major contributor to DMD progression is fibrosis, the accumulation of scarring in muscle tissue, which contributes to progressive muscle stiffness and weakness.  Effective treatments that prevent fibrosis would be of great benefit to DMD patients, slowing disease progression and improving quality of life.  Beyond DMD, the same anti-fibrosis therapies have potential for uses in a variety of other high-burden diseases, including fibrosis of the liver, lungs, and heart.

In addition to creating new therapies that address an unmet medical need, this project will promote economic growth in the Canadian biotechnology sector by attracting major investment and new partnerships. The accessible market for new antibody therapies for DMD is estimated at over $900M, and broader application in fibrosis represents a combined market opportunity of over $40B. Over the five years following completion of these studies, this work will contribute to the creation of more than 100 new high-tech jobs in Canada, and will attract between $50-100 million of new foreign investment.