Research programme: alpha-glucosidase gene therapy - Sanofi Genzyme

Drug Profile

Research programme: alpha-glucosidase gene therapy - Sanofi Genzyme

Alternative Names: Pompe disease gene therapy - Sanofi Genzyme

Latest Information Update: 20 Dec 2016

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At a glance

  • Originator Genzyme Corporation
  • Developer Duke University Medical Center
  • Class Gene therapies
  • Mechanism of Action Alpha glucosidase expression stimulants; Gene transference
  • 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

  • No development reported Glycogen storage disease type II

Most Recent Events

  • 02 Jul 2007 Preclinical development is ongoing
  • 27 Jun 2005 Preclinical trials in Glycogen storage disease type II in USA (IV)
  • 22 Jun 2005 Data presented at the 8th Annual Meeting of the American Society of Gene Therapy (ASGT-2005) have been added to the Metabolic Disorders pharmacodynamics section ,
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