Iduronate-2-sulfatase gene therapy

Drug Profile

Iduronate-2-sulfatase gene therapy

Alternative Names: Hunter syndrome gene therapy

Latest Information Update: 23 Sep 2013

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At a glance

  • Originator University of Minnesota
  • Class Iduronate sulfatases
  • Mechanism of Action Iduronate sulfatase replacements
  • Orphan Drug Status

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


Highest Development Phases

  • Discontinued Mucopolysaccharidosis II

Most Recent Events

  • 31 Dec 2007 Discontinued - Phase-II for Mucopolysaccharidosis II in USA (Infusion)
  • 31 Dec 2007 Discontinued - Preclinical for Mucopolysaccharidosis II in Italy (unspecified route)
  • 11 Dec 2003 No development reported - Phase-II for Mucopolysaccharidosis type II in USA (Infusion)
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