CRANBURY, N.J. & PHILADELPHIA—Capitalizing on momentum is the name of the game in a recent collaboration expansion, with Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania doubling the size of their existing partnership. The collaboration, aimed at researching and developing novel gene therapies, has been expanded from three programs for rare genetic diseases to six, and now includes Pompe disease, Fabry disease, CDKL5 deficiency disorder (CDD) and Niemann-Pick type C, as well as mucopolysaccharidosis type IIIB (MPS IIIB) and a next-generation program in mucopolysaccharidosis type IIIA (MPS IIIA)—both forms of MPS are known as part of Sanfillipo syndrome.
Additionally, a discovery research agreement has been established that grants Amicus exclusive disease-specific access to rights to collaborate with Penn's Gene Therapy Program (GTP) to develop new gene therapy platform technologies and programs for lysosomal disorders and 12 additional rare diseases.
“This agreement is a significant step forward in creating a world-class industry-academia gene therapy partnership in rare diseases,” said Dr. James M. Wilson, 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.”
In terms of specifics regarding the expanded agreement, the deal involves funding provided to Penn to support the preclinical research programs in the Wilson Lab and to license select technologies developed under the auspices of the collaboration. Pompe disease, Fabry disease, CDD and an undisclosed rare metabolic disorder comprised the original program lineup for the agreement.
The next-generation research program will consist of a new five-year agreement under which Penn will conduct discovery research to develop new gene therapy technologies, while Amicus will continue to advance its own research and technology platforms to combine with Penn's. Per the terms of the agreement, Amicus will make a $10-million annual investment to GTP's program each year for five years, with the possibility for an extension, and in return gains exclusive disease-specific rights to collaborate with GTP to research and develop products for lysosomal disorders. Amicus' rights also cover rare diseases such as Rett syndrome, Angelman syndrome, myotonic dystrophy and certain other muscular dystrophies.
“The major expansion of the collaboration … is a bold move for Amicus, and it reflects our unwavering commitment to develop potential cures that may alleviate an enormous amount of suffering for many more thousands of people living with rare genetic diseases, many of them children. This collaboration also reflects the extraordinary scientific capabilities at Amicus, as well as the success that we have seen with the work that we have done in collaboration with Dr. Wilson and his team,” John F. Crowley, chairman and CEO of Amicus, noted in a conference call. “Stated simply, we see great value for patients and shareholders alike in aligning as strongly and as deeply as possible with Penn in these diseases.”
A key focus of this collaboration—before the expansion and continuing forward—is combining Amicus' protein engineering and glycobiology work with Penn's gene transfer technologies to enable novel gene therapies with better uptake, targeting, dosing, safety and manufacturability.
“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,” remarked Dr. J. Larry Jameson, 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.”
“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,” said Crowley.