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Antibody Therapeutics for Duchenne Muscular Dystrophy

  • Project Leaders: Fabio Rossi, Carl Hansen, Kevin Heyries, Michael Underhill
  • Institutions: University of British Columbia (UBC)
  • Budget: $6506824
  • Program/Competition: Genomic Applications Partnership Program (GAPP)
  • Genome Centre(s): Genome Canada
  • Fiscal Year: 2017
  • Status: Closed

Duchenne Muscular Dystrophy (DMD) is the most prevalent genetic disorder in the world, affecting 1 in 3500 males in Canada. People with DMD have a mutation in a gene that makes dystrophin, a key component that links the muscle fiber cytoskeleton to the plasma membrane. Without functional dystrophin, normal contractions of the muscle lead to chronic damage, inflammation, and rigid deposits, and eventually muscle stiffness and loss of strength that can even affect cardiac tissue. DMD can impact children as young as three years old and is fatal in every case, with an average life expectancy of 25. Fibrosis is an associated condition that promotes the muscle degeneration in DMD and blocks tissue regeneration, thereby reducing the effectiveness of treatments.  

This collaborative project brought together critical R&D from UBC's Fabio Rossi and Michael Underhill with the therapeutics development tools of BC biotech company, AbCellera. By the end of the project, they successfully identified and generated a portfolio of antibodies that neutralized targets involved in fibrosis. These antibodies possess clinical potential in combination therapy for DMD or have the potential to become bi-specific reagents that target multiple disease pathways. This project contributed to fueling the rapid expansion of AbCellera and led to the creation of multiple positions in the company.