Skip to Main Content

Sarepta Therapeutics’ clinical trial for a gene therapy to treat Duchenne muscular dystrophy has been paused by the Food and Drug Administration because some of the materials used in the treatment weren’t as they should be, the company announced Wednesday.

A stray DNA fragment was found “in connection with routine quality assurance testing” of the plasmids used in the company’s gene therapy, said Douglas Ingram, Sarepta’s president and CEO. Plasmids carry the genetic material used in gene therapy into a cell. The FDA notified Nationwide Children’s Research Institute, where the trial is being conducted, of the clinical hold on Tuesday.


The lot that was tested has not been used in patients. “We can confirm that these plasmids were not used in any other trial,” said Gina Bericchia, a spokesperson for Nationwide.  Four patients received a dose from a prior lot from the same supplier. The fragment wasn’t found in biopsies from any of those patients, and no side effects from the fragment have been reported. “There’s nothing particular in the sequence that was troubling,” Ingram said — the issue was merely that the fragment existed.

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

  • I believe you have some information wrong. the company isn’t starting a new trial by the end of the year. The company is continuing the trial that you referenced that has reported some interim data. That June study is ongoing. That’s the actual study that’s currently on clinical hold.

    Also you’ve truncated the name of the treatment. You’re just referencing the AAV stereotype and the promoter. You aren’t even naming the transgene.

Comments are closed.