Amicus Therapeutics and the University of Pennsylvania’s Perelman School of Medicine have agreed to a major expansion of their gene therapy research collaboration.

Financial terms of the expanded research and licensing agreement include Amicus making a $10 million annual investment, each year for five years, into Penn’s gene therapy program in return for exclusive, disease-specific product development rights.

Other financial terms of the expanded relationship were not disclosed.

Amicus, of Cranbury, N.J., in February unveiled plans to establish a new global research and gene therapy center at uCity Square, where it now occupies temporary space at BioLabs@CIC. The company will occupy the top three floors under construction at the 3675 Market St. building, where it expects to have 200 employees there over the next few years.

Under its expanded gene therapy partnership:

  • Amicus (NASDAQ: FOLD) has acquired the rights for all next-generation Penn gene therapy technologies covering a majority of all lysosomal disorders. Lysosomal disorders are a group of about 50 rare inherited metabolic conditions that result from an abnormal build-up of various toxic materials in the body’s cells due to enzyme deficiencies.
  • The existing collaboration between Penn and Amicus first established in October 2018 has grown from three to six immediate programs for rare genetic diseases, including Pompe and Fabry diseases.
  • Amicus gains exclusive disease-specific worldwide rights to collaborate with the Penn’s gene therapy program to develop “potentially disruptive new gene therapy platform technologies and programs” for 12 additional rare diseases, including Rett Syndrome, Angelman syndrome, Myotonic Dystrophy and select Muscular Dystrophies.

John F. Crowley, chairman and CEO of Amicus, described the extension of his company’s collaboration with Penn as 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.”

The partnership — designed to combine Amicus’ protein engineering and glycobiology (the study of carbohydrates and their role in biology) expertise with Penn’s gene transfer technologies — has already produced what the organization is calling “compelling” initial preclinical proof-of-concept data of a potential therapy for Pompe disease. Pompe disease is an inherited disorder caused by the buildup of glycogen, a complex sugar, in the body’s cells resulting in skeletal muscle weakness, mobility problems and respiratory function impairment.

“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, a professor of medicine and pediatrics at the Perelman School of Medicine and the leader of Penn’s gene therapy program. “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.”


Source: Philadelphia Business Journal by John George | May 29, 2019