Research programme: cognitive disorder gene therapy - Agilis Biotherapeutics/University of South Florida

Drug Profile

Research programme: cognitive disorder gene therapy - Agilis Biotherapeutics/University of South Florida

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

Latest Information Update: 27 Oct 2017

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At a glance

  • Originator University of South Florida
  • Developer Agilis Biotherapeutics; University of South Florida
  • 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.

    Yes - Angelman syndrome
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Angelman syndrome
  • Research Fragile X syndrome

Most Recent Events

  • 20 Oct 2017 Pharmacodynamics data from preclinical studies in Angelman syndrome released by Agilis Biotherapeutics
  • 03 Oct 2017 Agilis Biotherapeutics plans a clinical trial for Angelman syndrome in 2019
  • 04 May 2016 Research programme: cognitive disorder gene therapy - Agilis Biotherapeutics/University of South Florida receives Orphan Drug status for Angelman syndrome in European Union
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