Drug Profile
Research programme: alpha-glucosidase gene therapy - Sanofi
Alternative Names: Pompe disease gene therapy - SanofiLatest Information Update: 31 Mar 2022
Price :
$50
*
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 ,