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Amicus, UPenn ally to develop rare disease gene therapies

Executive Summary

Amicus Therapeutics Inc. is teaming up with the University of Pennsylvania to research and develop adeno associated virus (AAV) gene therapies for Pompe disease, Fabry disease, CDKL5 deficiency (seizures), and one undisclosed rare metabolic disorder.
Deal Industry
  • Biotechnology
    • Gene Therapy, Cell Therapy
  • Pharmaceuticals
Deal Status
  • Final
Deal Type
  • Alliance
    • R+D and Marketing-Licensing

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