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Amicus Therapeutics is expanding the focus its drug development work with the purchase of a portfolio of early-stage gene therapies targeting rare neurologic diseases, the company announced Thursday.

Two of the 10 gene therapies programs being acquired by Amicus have already entered human clinical trials as potential, one-time cures for different forms of Batten disease, an inherited and fatal neurodegenerative disease in children.


Amicus is paying $100 million upfront to purchase the gene therapy programs, financed through newly issued debt. The seller is Celenex, a privately held biotech spun out of Nationwide Children’s Hospital and Ohio State University, both in Columbus, Ohio, where the gene therapies were invented.

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