AmicusFocussing on Pompe, Fabry, CDKL5 and one additional undisclosed rare metabolic disorder, Amicus Therapeutics begins a collaboration with the Gene Therapy Program at the Perelman School of Medicine at University of Pennsylvania. This will combine Amicus’s protein engineering and glycobiology expertise with Penn’s adeno associated virus (AAV) gene transfer technologies to develop gene therapies. John F. Crowley, Chairman and Chief Executive Officer of Amicus, hails this as a groundbreaking collaboration that has a real possibility to transform the lives of people affected by these four disorders.

There is synergy in their two programs, where Penn’s AAV vector technology is designed to improve targeting, tropism, safety, immunogenicity, and gene delivery, while Amicus’s protein engineering capabilities may optimize protein expression, secretion, targeting and uptake of the target protein. The agreement includes funding to Penn to further their preclinical program.

Amicus Therapeutics has now joined others in the business to develop gene therapies for Pompe disease, Fabry and CDKL5.


Tagged: gene therapy