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.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!

GET STARTED

What is it?

STAT Plus is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What's included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • Online intelligence briefings
  • Frequent opportunities to engage with veteran beat reporters and industry experts
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.

Leave a Comment

Please enter your name.
Please enter a comment.

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy