Duchenne muscular dystrophy gene therapy trial put on hold


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 [July 27].

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[July 26].

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“We know the source of the problem, we have been given clear guidance on how to address the issue, and we have a plan for our clinical development program to remain on track,” [said Douglas Ingram, Sarepta’s president and CEO.]


Results announced in June from an earlier trial in just three boys were promising. After receiving the treatment, the boys were producing enough dystrophin — the protein that is missing in Duchenne — to potentially stop or reverse the condition.

Read full, original post: Sarepta halts early-stage gene therapy trial for DMD

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