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Sarepta CEO says he welcomes Vertex’s effort to develop muscular dystrophy drugs

Vertex Pharmaceuticals’ headquarters on Northern Avenue in Boston. Craig F. Walker/Globe Staff/Globe Staff

The head of Cambridge-based Sarepta Therapeutics said Friday he welcomed a foray by Vertex Pharmaceuticals into the effort to develop treatments for Duchenne muscular dystrophy and saw no threat to his biotech, which markets the first approved medicine for the deadly illness.

“I applaud anyone that’s focused on research into Duchenne muscular dystrophy, which is a really serious disease,” said Douglas Ingram, CEO of Sarepta. “Good for Vertex.”

Sarepta won a controversial approval in 2016 for the first treatment for the muscle-wasting disease and hopes to win approval of a second experimental drug, now known as golodirsen, some time in August.


Vertex, based in Boston, announced Thursday that it was expanding its collaboration with Switzerland-based CRISPR Therapeutics to discover and develop gene-editing treatments for Duchenne and myotonic dystrophy, the most common form of muscular dystrophy that starts in adulthood. Vertex will pay CRISPR $175 million up front.

Vertex, best known for its cystic fibrosis medicines, also said it was buying Exonics Therapeutics of Watertown for $245 million. Exonics is working on gene-editing therapies to fix mutations that cause Duchenne and other serious neuromuscular diseases.

In addition to its approved drug Exondys 51 and its yet-to-be approved golodirsen, Sarepta has 14 drug research and development programs for Duchenne, Ingram said. The potential treatments rely on gene editing, gene therapy, and RNA interference, or RNAi, medicine.

“We’re very confident in our approach,” he said, although he added that gene editing was “quite a ways out into the future.”

Jonathan Saltzman can be reached at