Amicus bolsters rare disease gene therapy pipeline with expanded UPenn deal

Amicus has added three more rare disease gene therapy programs to its pipeline through an expansion of its deal with the University of Pennsylvania, with the option to add at least a dozen more. The company also proposed Wednesday to raise $150 million in a follow-on underwritten by J.P. Morgan, Goldman Sachs and SVB Leerink.

Amicus Therapeutics Inc. (NASDAQ:FOLD) paid the university an option fee in the low single-digit millions to license gene therapy programs for three lysosomal disorders: Niemann-Pick disease type C and mucopolysaccharidosis types IIIB and IIIA. Amicus will fund preclinical research activities at Jim Wilson's lab at UPenn, and then be responsible for clinical development and commercialization of the programs. The university is eligible for milestones, plus royalties...