Amicus and Penn developing AVV gene therapies

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Amicus Therapeutics is working with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (Penn) to research and develop novel gene therapies for Pompe disease, Fabry disease, CDKL5 deficiency and one additional undisclosed rare metabolic disorder.

The collaboration will combine Amicus’ protein engineering and glycobiology expertise with Penn’s adeno associated virus (AAV) gene transfer technologies to develop AAV gene therapies designed for optimal cellular uptake, targeting, dosing, safety and manufacturability.

“This groundbreaking collaboration with Penn offers a new opportunity to potentially transform the lives of people living with these severe genetic disorders,” said John F. Crowley, Chairman and CEO of Amicus.

Penn’s AAV vector technology is designed to improve targeting, tropism, safety, immunogenicity, and gene delivery, while Amicus’ protein engineering capabilities may optimize protein expression, secretion, targeting and uptake of the target protein.