Amicus, Penn expand gene therapy research agreement into five-year collaboration
Apart from getting the FDA to have a change of heart on its Fabry disease therapy, Galafold, last year, Amicus Therapeutics joined forces with the University of Pennsylvania to work on gene therapies for four different disorders — Pompe disease, Fabry disease, CDKL5 deficiency and an undisclosed rare metabolic disorder. On Wednesday, the two expanded their collaboration — to include additional lysosomal disorders and rare diseases.
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