US-based biotechnology firm Amicus Therapeutics has entered a research, collaboration and licence agreement with the Perelman School of Medicine at the University of Pennsylvania (Penn) for the development of new adeno associated virus (AAV) gene therapies.
Under the university’s Gene Therapy Program, Amicus’ protein engineering and glycobiology expertise will be combined with Penn’s AAV gene transfer technologies.
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The partners will focus on treatments for Pompe disease, Fabry disease, CDKL5 deficiency and one other undisclosed rare metabolic disorder.
Amicus and Penn aim to formulate the new therapies for optimal cellular uptake, targeting, dosing, safety and manufacturability.
Penn’s AAV vector technology is designed to enhance targeting, tropism, safety, immunogenicity and gene delivery.
In addition, Amicus’ protein engineering capabilities are expected to optimise protein expression, secretion, targeting and uptake of the target protein.
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By GlobalDataAmicus Therapeutics chairman and CEO John Crowley said: “This groundbreaking collaboration with Penn offers a new opportunity to potentially transform the lives of people living with these severe genetic disorders.
“Dr Wilson’s laboratory and The Gene Therapy Program at Penn has strong capabilities in its AAV vector technology, as well as manufacturing and immunology strategies to develop novel gene therapy candidates, and to successfully advance them through preclinical development.”
As part of the agreement, Penn will receive funding to advance the pre-clinical research programmes in the Wilson Lab as well as to licence certain technologies under the research collaboration.
Perelman School of Medicine professor James Wilson said: “I believe that we can combine the technologies and capabilities from my research laboratory at Penn with the Amicus expertise in protein engineering, glycobiology and disease biology understanding to rapidly advance novel gene therapies to the clinic.”
