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Research programme: AAV based gene therapies - Amicus Therapeutics/University of Pennsylvania

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Drug Profile

Research programme: AAV based gene therapies - Amicus Therapeutics/University of Pennsylvania

Alternative Names: Amicus/Penn engineered hGAA AAV gene therapy; Pompe gene therapy - Amicus Therapeutics/University of Pennsylvania

Latest Information Update: 28 Feb 2025

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At a glance

  • Originator Amicus Therapeutics
  • Developer Amicus Therapeutics; University of Pennsylvania
  • Class Gene therapies
  • Mechanism of Action Cyclin-dependent kinase 5 modulators; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Early infantile epileptic encephalopathy 2
  • No development reported Glycogen storage disease type II; Mucopolysaccharidosis III; Niemann-Pick diseases

Most Recent Events

  • 28 Feb 2025 No recent reports of development identified for preclinical development in Glycogen-storage-disease-type-II in USA (Parenteral)
  • 28 Feb 2025 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-III in USA (Parenteral)
  • 28 Feb 2025 No recent reports of development identified for research development in Niemann-Pick-diseases in USA (Parenteral)

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