Sarepta Touts More Gene Therapy Data, Snaps Up Myonexus For $165M
Summary
Sarepta Therapeutics (NASDAQ: SRPT ) has been steadily building up its gene therapy capabilities for a few years now, and it took two more steps forward on that front this morning. In the deal, Sarepta paid Myonexus $60 million for the exclusive option to buy the company and its five experimental gene therapy programs—each for different forms of LGMD—upon getting a look at early human data. The hope is boosting beta-SG levels should change the trajectory of the disease, which causes a progressive loss of muscle function and often an early death from heart or lung problems. To be clear, Sarepta is only reporting results today that roughly two months after treatment, the three patients, so far, are producing more beta-SG and other markers indicative of a potential impact on the disease. Sarepta did see liver enzymes spike in two patients—a common side effect of gene therapies, though one that is typically quickly reversed with a course of steroids.
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