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Vertex, CRISPR to work on gene-editing treatments for muscular dystrophy

Vertex Pharmaceuticals, the Boston drug maker best known for its cystic fibrosis medicines, is entering the market to develop treatments for two forms of muscular dystrophy.

The company announced late Thursday that it was expanding its collaboration with Switzerland-based CRISPR Therapeutics to discover and develop gene-editing treatments for Duchenne muscular dystrophy and myotonic dystrophy, the most common form of muscular dystrophy that starts in adulthood.

Vertex will pay CRISPR $175 million up front with the potential for additional payments if the collaboration meets certain goals.

In addition, Vertex said that it was buying Watertown-based Exonics Therapeutics for $245 million. Exonics is a privately held firm working on gene-editing therapies to fix mutations that cause Duchenne and other severe neuromuscular diseases.


“Through the expanded collaboration with CRISPR and the acquisition of Exonics, we are bringing together the intellectual property, technologies, and scientific expertise needed to establish a leading gene editing platform” for the devastating muscle diseases, said Jeffrey Leiden, CEO of Vertex.

In 2016, Cambridge biotech Sarepta Therapeutics won approval for the first treatment of Duchenne after families of boys with the deadly muscle-wasting disease urged the government to overrule a panel’s finding that questioned whether the drug worked.

Last month, however, the Institute for Clinical and Economic Review, a nonprofit Boston-based drug-cost watchdog, issued its first-ever review of Duchenne drugs, including that medicine and a second experimental one for which Sarepta is seeking approval.

The report was preliminary, but its conclusion is withering: Evidence that the two medicines work was so scant that the watchdog couldn’t even say how much they should cost.

In a statement that same day, Sarepta called ICER’s approach “fatally flawed” and that “ICER’s model is unfit to evaluate rare disease populations in a manner that would encourage innovation.”

Jonathan Saltzman can be reached at jsaltzman@globe.com