Research programme: mucopolysaccharidoses gene therapies - ESTEVE/Universitat Autonoma de Barcelona

Drug Profile

Research programme: mucopolysaccharidoses gene therapies - ESTEVE/Universitat Autonoma de Barcelona

Alternative Names: EGT 201; EGT 301; NAV rAAv9 iduronate-2-sulfatase gene therapy - Esteve/UAB; NAV rAAv9 N-acetylglucosaminidase alph gene therapy - Esteve/UAB; The Sanfilippo programme - ESTEVE

Latest Information Update: 02 Mar 2016

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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
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis III; Mucopolysaccharidosis II
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Mucopolysaccharidosis II; Mucopolysaccharidosis III

Most Recent Events

  • 25 Feb 2016 Preclinical trials in Mucopolysaccharidosis II in Spain (Intracerebroventricular)
  • 25 Feb 2016 Preclinical trials of EGT 201 in Mucopolysaccharidosis III in Spain (Intracerebroventricular)
  • 25 Feb 2016 ESTEVE plans a phase I/II trial for Mucopolysaccharidosis IIIA in Spain
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