The race to cure DMD hits a snag

Solid Biosciences, a company founded to halt the muscle-wasting Duchenne muscular dystrophy, has disappointing news from its first clinical trial: The firm’s gene therapy, designed to boost production of a key protein, performed well below expectations in a small study.

As STAT’s Matthew Herper reports, the three patients treated in Solid’s trial produced only small amounts of that protein, called dystrophin. Investors — and Solid’s management — had hoped to see much better results from the study, making this morning’s disclosure a stumble for the early-stage therapy.

But the process is hardly over. The patients in question received the lowest planned dose of Solid’s treatment, and three more are yet to be treated. And Solid, which is racing with Sarepta Therapeutics to develop a gene therapy for DMD, believes it’s still in the game.

“We believe we will remain competitive,” Ganot said. “I don’t think we ever stopped being competitive. I think the competitiveness of the situation will be determined by, of course expression. But also the quality of the protein. But also the ability to manufacture it at scale.”

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