Research programme: Niemann-Pick disease type B gene therapy - Sanofi Genzyme

Drug Profile

Research programme: Niemann-Pick disease type B gene therapy - Sanofi Genzyme

Latest Information Update: 16 Dec 2016

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At a glance

  • Originator Genzyme Corporation
  • Developer Genzyme Corporation; Mount Sinai School of Medicine
  • Class Gene therapies
  • Mechanism of Action Sphingomyelin phosphodiesterase stimulants
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued Niemann-Pick disease type B

Most Recent Events

  • 26 Feb 2009 Discontinued - Preclinical for Niemann-Pick disease type B in USA (unspecified route)
  • 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
  • 29 Jul 2003 Genzyme General, Genzyme Biosurgery and Genzyme Molecular Oncology are now called Genzyme Corporation
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