Sarepta all-in on gene therapy pipeline, adding 3 CNS treatments in $30M Lacerta deal

Sarepta’s lead gene therapy $SRPT may be on clinical hold, but the brash Duchenne muscular dystrophy company is continuing its deep dive into the field, handing over $30 million in an equity investment in Lacerta in exchange for a group of CNS therapies, including one program focused on Pompe disease.

The biotech reported the deal just after market close on Wednesday, along with a Q2 statement showing growing sales volume for Exondys 51.

In the deal with Lacerta — a spinout from the University of Florida with expertise in AAV vector tech — Sarepta will take over clinical development after the private company has wrapped preclinical work. The pact gives Sarepta 11 gene therapy programs, a big part of the 20 it has hustled into the pipeline.

The clinical hold for Sarepta’s gene therapy for DMD came just days ago, triggered by what the biotech said was a problem on the manufacturing side of things. The hold came shortly after the biotech whipped up some intense enthusiasm for their work based on strong, but very early, responses in the clinic.

Sarepta has been wheeling and dealing its way to an expanded pipeline in the wake of the controversial OK for Exondys 51, a landmark advance only made possible by Janet Woodcock’s insistence that the drug should be OK’d despite the lack of efficacy data. That decision proved unpopular inside the FDA, but wildly successful in the disease community that supported it through approval.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
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Flatiron Health New York City or San Francisco

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