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AT GTX 701

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Drug Profile

AT GTX 701

Alternative Names: AAV based Fabry gene therapy - Amicus Therapeutics/University of Pennsylvania; AT-GTX-701

Latest Information Update: 05 May 2026

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At a glance

  • Originator Amicus Therapeutics
  • Developer Amicus Therapeutics; University of Pennsylvania
  • Class Gene therapies
  • Mechanism of Action 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

  • No development reported Fabry's disease

Most Recent Events

  • 27 Apr 2026 Amicus Therapeutics has been acquired by BioMarin Pharmaceutical
  • 28 Mar 2023 No recent reports of development identified for preclinical development in Fabry's-disease in USA (Parenteral)
  • 31 Dec 2022 Amicus Therapeutics and University of Pennsylvania terminates its research, collaboration and license agreement for the development of AAV based gene therapies for Fabry's disease and Pompe disease

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