Glucocerebrosidase gene therapy - Targeted Genetics

Drug Profile

Glucocerebrosidase gene therapy - Targeted Genetics

Latest Information Update: 25 Oct 2000

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At a glance

  • Originator Targeted Genetics
  • Class Gene therapies
  • Mechanism of Action Gene transference; Glucosylceramidase replacements
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Discontinued Gaucher's disease

Most Recent Events

  • 25 Oct 2000 Discontinued-I for Gaucher's disease in USA (IV-infusion)
  • 03 Mar 1998 Phase-I clinical trials for Gaucher's disease in USA (IV-infusion)
  • 20 Feb 1997 New profile
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