Amicus Therapeutics and the University of Pennsylvania Announce Major Expansion of Gene Therapy Collaboration

Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania (Penn) announced a major expansion to their collaboration with rights to pursue collaborative research and development of novel gene therapies for lysosomal disorders (LDs) and 12 additional rare diseases. The collaboration has been expanded from three to six programs for rare genetic diseases and now includes: 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 three new programs, a discovery research agreement provides Amicus with exclusive disease-specific access to rights to collaborate with Penn’s Gene Therapy Program (GTP) to develop potentially disruptive new gene therapy platform technologies and programs for the majority of lysosomal disorders and 12 additional rare diseases.

John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, “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. With a globally approved precision medicine product for Fabry, a late-stage biologic product with breakthrough therapy designation for Pompe, and now the industry’s largest rare disease gene therapy pipeline, Amicus is well-positioned to become a leading global biotechnology company at the forefront of human genomic medicine.”

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

“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 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. We are looking forward to expanding the relationship further for additional preclinical programs and committing to the research required to further advance the technology platforms at Penn. 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.”