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Research programme: cognitive disorder gene therapy - PTC Therapeutics/University of South Florida

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Drug Profile

Research programme: cognitive disorder gene therapy - PTC Therapeutics/University of South Florida

Alternative Names: AGIL-AS - PTC Therapeutics/University of South Florida; Angelman syndrome gene therapy - PTC Therapeutics/University of South Florida; Angelman Syndrome Therapeutics - PTC Therapeutics/USF; AS gene therapy - PTC Therapeutics/University of South Florida; GT-AS; Reelin based therapy - PTC Therapeutics/University of South Florida; Reelin-based gene therapy - PTC Therapeutics/University of South Florida; UBE3a therapy - PTC Therapeutics/University of South Florida

Latest Information Update: 30 May 2023

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At a glance

  • Originator University of South Florida
  • Developer PTC Therapeutics; University of South Florida
  • Class Gene therapies; Neuroprotectants
  • Mechanism of Action Gene transference; Ubiquitin-protein ligase expression stimulants
  • Orphan Drug Status

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

    Yes - Angelman syndrome
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Angelman syndrome; Fragile X syndrome

Most Recent Events

  • 23 May 2023 Discontinued - Preclinical for Angelman syndrome in USA (unspecified route)
  • 23 May 2023 Discontinued - Preclinical for Fragile X syndrome in USA (unspecified route)
  • 16 Jan 2023 Research programme is still in preclinical phase for Angelman syndrome in USA (PTC Therapeutics website, January 2023)

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