Audentes Therapeutics (BOLD), a developer of experimental gene therapies, will announce Monday that it aims to create new treatments for two forms of muscular dystrophy: Duchenne muscular dystrophy and myotonic muscular dystrophy.
Duchenne, in particular, is one of the most competitive areas in the hot new field of gene therapy, which uses viruses to embed new genes in the cells of sick patients. Several other companies, including Sarepta Therapeutics (SRPT) and Pfizer (PFE), have active gene therapy efforts targeting the disorder, a genetic disease that afflicts boys and dramatically shortens their lives. The two new Audentes approaches have not yet been tested in humans.
“We’re going into it with what we believe is a differentiated program, versus the other programs, and one that we feel has great potential to be the best in class,” said Matthew Patterson, Audentes’ co-founder, chairman, and chief executive.