Amicus, UPenn expand collaboration to develop gene therapies for rare diseases

Amicus Therapeutics, a Cranbury, N.J.-based biotech company, has expanded its partnership with the University of Pennsylvania's Perelman School of Medicine in Philadelphia to research and develop gene therapies for a broad swath of rare diseases.

Within the partnership, researchers at UPenn use gene transfer technology to identify potential molecular targets, to which Amicus applies protein-engineering methods in order to develop gene therapy candidates. The collaboration initially focused on developing potential treatments for three lysosomal storage disorders — Pompe disease, Fabry disease and CDKL5 deficiency disorder — and one other undisclosed rare metabolic disorder.

Under the newly announced expansion, Amicus and UPenn will study three more lysosomal disorders — Niemann-Pick Type C and two types of Sanfilippo syndrome — as well as 12 other rare diseases including Rett syndrome, Angelman syndrome and several forms of muscular dystrophy.

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