Amicus Forms R&D Collaboration with Penn Focused on AAV Gene Therapies

October 8, 2018

Rare Daily Staff

Amicus Therapeutics said it entered into a collaboration with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania to pursue research and development of novel gene therapies for a set of rare, metabolic disorders.

The agreement between Amicus and Penn is a research, collaboration and license agreement, with funding provided to Penn to advance the preclinical research programs in the Wilson Lab and to license certain technologies invented under the funded research collaboration. The collaboration program will focus on developing innovative new AAV gene therapies for Pompe disease, Fabry disease, CDKL5 deficiency, and one additional undisclosed rare metabolic disorder.

Wilson said there are multiple and unique challenges in developing optimal gene therapy products for patients living with Pompe and Fabry diseases. The partners hope to leverage their complimentary expertise to address the unique challenges of developing a gene therapy to address these diseases.

“Amicus has developed unique abilities in drug development in the lysosomal storage disorders, particularly in Pompe and Fabry diseases,” said James Wilson, professor of medicine and pediatrics at the Perelman School of Medicine. “I believe that we can combine the technologies and capabilities from my research laboratory at Penn with the Amicus expertise in protein engineering, glycobiology and disease biology understanding to rapidly advance novel gene therapies to the clinic.”

The company said it will provide additional details and an overview of the collaboration during the Amicus Analyst Day on October 11.

“This groundbreaking collaboration with Penn offers a new opportunity to potentially transform the lives of people living with these severe genetic disorders,” said John Crowley, chairman and CEO of Amicus. “This is another important step in Amicus becoming the leader in gene therapy for rare metabolic disorders.”

October 8, 2018
Photo: John Crowley, CEO of Amicus Therapeutics


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