The fascinating case of gene therapy for DMD

Yesterday, a company called Audentes Therapeutics joined the race to develop a gene therapy for Duchenne muscular dystrophy, meaning it will vie with Pfizer, Sarepta Therapeutics, and others to apply cutting-edge technology to a devastating disease.

But as STAT’s Matthew Herper reports, the nuances of DMD make developing gene therapies particularly challenging, forcing the various contenders to get creative. The classic approach to gene therapy is inserting a corrective copy of the faulty gene into a delivery capsule, usually a virus, and infusing it into patients. But in DMD, patients are missing a key protein whose attendant gene is simply too large to fit inside such viruses.

And so there are workarounds. Audentes’ forebears have gone with a shortened version of that gene, betting even a truncated protein can stop the muscle-wasting advance of DMD. But Audentes is instead trying to convince the body to skip over those genetic errors and produce the same protein healthy people do, an approach the company said could be better than its competition.

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