Amicus, Perelman School of Medicine Expand Collaboration to Develop Gene Therapies

Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania have expanded their research collaboration to develop new gene therapies for Pompe disease as well as other rare genetic diseases, the company announced.

Encouraged by the success of an ongoing collaboration, including promising preclinical data for a candidate gene therapy for Pompe disease, the research engine combines Amicus’ expertise in protein engineering and glycobiology (biology of sugars) with the gene transfer vector technology, manufacturing, and immunology capabilities of the University of Pennsylvania’s Gene Therapy Program, headed by James M. Wilson, MD, PhD, professor at the Perelman School of Medicine and director of Penn’s Gene Therapy Program.

Penn’s gene transfer vectors (carriers), based on adeno-associated virus (AAV), are designed to improve gene delivery, maintain safety and avoid immune responses, while Amicus’ protein engineering expertise can optimize protein production, release and uptake of the missing protein by the producing and neighboring cells, a mechanism known as cross-correction.

The Amicus-Penn collaboration has expanded from three to six gene therapy programs, now including the rare diseases Pompe disease, Fabry disease, CDKL5 deficiency disorder (CDD), Niemann-Pick Type C (NPC), and Sanfilippo syndrome, specifically MPS IIIA and MPS IIIB.

The agreement includes funding by Amicus provided to Penn to advance the preclinical research programs in the Wilson Lab and to license certain technologies invented under the collaboration.

“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,” John F. Crowley, chairman and CEO of Amicus, said in a press release.

“With a globally approved precision medicine product for Fabry, a late-stage biologic product with breakthrough therapy designation for Pompe (AT-GAA investigational therapy), 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,” Crowley said.

Amicus and Penn also signed a five-year license agreement to collaborate on discovery programs focusing on next-generation gene therapy technologies, targeting many lysosomal storage disorders and 12 other rare diseases, including Rett syndrome, Angelman syndrome, myotonic dystrophy, and other muscular dystrophies. Amicus will put a $10 million annual investment into the Gene Therapy Program, while receiving exclusive disease-specific rights.

“This agreement is a significant step forward in creating a world-class industry-academia gene therapy partnership in rare diseases,” Wilson said. “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.”