Marie Daghlian

Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania announced a major expansion to their rare disease gene therapy collaboration from three to six programs and includes rights to pursue collaborative research and development of novel gene therapies for lysosomal disorders and 12 additional rare diseases.

The three programs of the initial collaboration—Pompe disease, Fabry disease, and CDKL5 deficiency disorder—will be expanded to include Niemann-Pick Type C (NPC), Mucopolysaccharidosis Type IIIB (MPS IIIB), as well as a next generation program in Mucopolysaccharidosis Type IIIA (MPS IIIA), also known as part of Sanfillipo Syndrome.

Additionally, a discovery research agreement provides Amicus with exclusive disease-specific access to rights to collaborate with Penn’s Gene Therapy Program to develop potentially disruptive new gene therapy platform technologies and programs for the majority of lysosomal disorders and 12 additional rare diseases, including Rett Syndrome, Angelman syndrome, myotonic dystrophy, and select other muscular dystrophies. Amicus will invest $10 million annually into Penn’s gene therapy program’s discovery research with the ability to extend the partnership.

“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,” said John Crowley, chairman and CEO of Amicus.

The expanded collaboration builds on the initial success of Amicus’ protein engineering and glycobiology expertise with Penn’s gene transfer technologies to develop novel gene therapies that have already delivered initial preclinical proof-of concept data in Pompe disease. Penn’s vector technology is designed to improve targeting, tropism, safety, immunogenicity, and gene delivery, while Amicus’ protein engineering capabilities may optimize protein expression, secretion, targeting and uptake of the target protein.

“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,” said James Wilson, 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.”

As part of the research, collaboration, and license agreement, Amicus will provide funding to Penn to advance the preclinical research programs in the Wilson Lab and to license certain technologies invented under the funded deal. The agreement also includes a five-year research collaboration under which Penn will conduct discovery research to develop potentially disruptive new gene therapy technologies.

Amicus will continue to advance its own research and technology platforms to combine with Penn’s technologies, which can be used in the collaborative research programs. For many of these indications, there is a potential to apply protein-engineering and targeting motifs to enable cross-correction with next-generation gene therapy technology.

“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,” said Crowley.

Photo: John Crowley, chairman and CEO of Amicus

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