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EGT 101

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Drug Profile

EGT 101

Alternative Names: AAV9-CAG-coh-SGSH; AAV9-hsulfamidase; EGT-101; NAV rAAv9 sulfamidase gene therapy; Sulfamidase gene therapy - Esteve

Latest Information Update: 15 Feb 2022

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At a glance

  • Originator Universitat Autonoma de Barcelona
  • Developer ESTEVE; Universitat Autonoma de Barcelona
  • Class Gene therapies
  • Mechanism of Action Gene transference; N-sulfoglucosamine sulfohydrolase expression stimulants
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis III
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Mucopolysaccharidosis III

Most Recent Events

  • 15 Feb 2022 Phase I/II development in Mucopolysaccharidosis III (In adolescents, In children) is ongoing in Spain (EudraCT2015-000359-26)
  • 29 Apr 2016 Phase-I/II clinical trials in Mucopolysaccharidosis III (In children, In adolescents) in Spain (Intracerebroventricular) (EudraCT2015-000359-26)
  • 25 Feb 2016 Preclinical trials in Mucopolysaccharidosis III in Spain (Intracerebroventricular)

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