Drug Profile
Research programme: Niemann-Pick type A disease gene therapy - Sanofi
Alternative Names: Sphingomyelinase gene therapyLatest Information Update: 31 Mar 2022
Price :
$50
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At a glance
- Originator Genzyme Corporation
- Developer Sanofi
- Class Gene therapies
- Mechanism of Action Gene transference; Sphingomyelin phosphodiesterase stimulants
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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 Niemann-Pick disease type A
Most Recent Events
- 26 Feb 2009 Discontinued - Preclinical for Niemann-Pick disease type A in USA (Injection)
- 01 May 2006 This programme is still in active development
- 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