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UPenn signs $50M expanded gene therapy collaboration with Amicus Therapeutics


By David Schwartz
Published: June 4th, 2019

The Perelman School of Medicine at the University of Pennsylvania has signed a major expansion of its collaboration with Amicus Therapeutics. The partners will now collaborate on developing novel gene therapies for lysosomal disorders (LDs) and 12 additional rare diseases.

The partnership now covers Pompe disease, Fabry disease, CDKL5 deficiency disorder (CDD), Niemann-Pick Type C (NPC), Mucopolysaccharidosis Type IIIB (MPS IIIB), as well as a next generation program in Mucopolysaccharidosis Type IIIA (MPS IIIA), both also known as part of Sanfillipo Syndrome.

In addition to these new programs, a discovery research agreement provides Amicus with exclusive disease-specific rights to collaborate with Penn’s Gene Therapy Program (GTP) on potentially disruptive new gene therapy platform technologies and programs for the majority of lysosomal disorders and 12 additional rare diseases.

John F. Crowley, Amicus chairman and CEO, said “the extension of our collaboration with Penn is a bold step forward in our commitment to create potential cures that may alleviate an enormous amount of suffering for countless numbers of people in the world living with rare diseases, many of them children. Together with Penn we are now able to focus on additional lysosomal disorders, as well as several more prevalent rare diseases for which we can apply our understanding of underlying disease biology in rare metabolic disease, Amicus’ protein-engineering and development expertise, and the world renowned capabilities of Dr. Jim Wilson’s laboratory to develop novel gene therapy candidates.”

Building off the initial success of the ongoing Amicus-Penn collaboration, including compelling initial preclinical proof-of-concept data in Pompe disease, the expanded relationship will continue to combine Amicus’ protein engineering and glycobiology expertise with Penn’s gene transfer technologies to develop novel gene therapies.

The five-year agreement will fund the preclinical research programs in the Wilson Lab, with an option to license certain technologies invented under the funded program. Terms include $10 million annual investments, each year for five years, into the Gene Therapy Program’s discovery research, with the ability to extend. Amicus gets exclusive disease-specific rights to collaborate with GTP to research and develop products for many lysosomal disorders as well as additional rare diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy and select other muscular dystrophies.

“This agreement is a significant step forward in creating a world class industry-academia gene therapy partnership in rare diseases,” said James M. Wilson, MD, PhD, professor of medicine and pediatrics at the Perelman School of Medicine. “We have seen the first results of our combined capabilities and platforms and I believe that we can further expand and accelerate our efforts to rapidly develop gene therapies for many more patients with unmet needs.”

“Penn Medicine has put Philadelphia on the map as the global epicenter of gene therapy research and development, and under the leadership and vision of Jim Wilson, our expanded agreement with Amicus is an exciting milestone for a field which is in the midst of transformative breakthroughs,” said J. Larry Jameson, MD, PhD, executive vice president of the University of Pennsylvania for the Health System and Dean of the Perelman School of Medicine. “We are thrilled to be part of this collaboration, which will help to bolster our city’s growing reputation as a magnet for talent and an engine for gene therapy innovation.”

Source: AP News

Posted under: University-Industry Engagement Week

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