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Amicus Therapeutics signs $50 million pact to expand collaboration with Penn’s Gene Therapy Program

The five-year agreement will serve to develop new cures for lysosomal disorders and other rare diseases.

John F. Crowley, Chairman of the Board and Chief Executive Officer of Amicus Therapeutics. Amicus has extended a collaboration with Penn's Gene Therapy Program.
John F. Crowley, Chairman of the Board and Chief Executive Officer of Amicus Therapeutics. Amicus has extended a collaboration with Penn's Gene Therapy Program.Read moreCourtesy Amicus Therapeutics
    by Sam Wood
    Published 

Amicus Therapeutics, a Cranbury, N.J.-based biotechnology company, announced Wednesday it was expanding its collaboration with the University of Pennsylvania’s Perelman School of Medicine.

The new pact will span at least five years with Amicus investing $10 million annually into Penn’s Gene Therapy Program with an option to extend the agreement. The collaboration focuses on creating novel drugs to treat or cure rare and lethal metabolic diseases. The alliance already has has generated significant discoveries.

The deal deepens the commitment that Amicus has already made to Philadelphia. In February, the company launched a new Global Research and Gene Therapy Center at uCitySquare, at 3675 Market St.. The Amicus facility is adjacent to Penn’s Wilson Lab, which specializes in gene transfer technologies. The relationship between the two research nodes will continue to combine Amicus’ protein engineering abilities with the Wilson Lab’s expertise in vector technologies.

The $50 million in funding that Amicus provides to Penn will advance the Wilson Lab’s preclinical research programs and give Amicus the right to license certain technologies invented during the collaboration. Treatments for more than a dozen genetic disorders are under investigation. Those targeted ailments include Pompe disease, Fabry disease, Rett Syndrome, Sanfillip Syndrome and a number of other lysosomal disorders. Lysosomal diseases are inherited metabolic disorders characterized by an abnormal build-up of toxic materials in the body’s cells as a result of enzyme deficiencies.

“This agreement is a significant step forward in creating a world class industry-academia gene therapy partnership in rare diseases,” said James M. Wilson, director of Wilson Lab and a professor of medicine and pediatrics at the Perelman School of Medicine. “We have already seen highly encouraging preclinical results and proof-of-concept in Pompe disease through our existing collaboration and are excited by what we can further achieve together.”

By 2023, Amicus expects its therapies will treat 5,000 patients and generate $1 billion in global sales.