Research programme: AAV-alpha-sarcoglycan gene therapy - GenethonAlternative Names: AAV-delivered human alpha sarcoglycan gene therapy - Genethon; Alpha sarcoglycanopathy gene therapy - Genethon; Alpha-sarcoglycan gene therapy - Genethon; LGMD2D gene therapy - Genethon
Latest Information Update: 02 Aug 2013
At a glance
- Originator Genethon
- Class Gene therapies
- Mechanism of Action Alpha sarcoglycan replacements; Gene transference
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- New Molecular Entity No
Highest Development Phases
- Preclinical Sarcoglycanopathies
Most Recent Events
- 01 Aug 2013 Preclinical development is ongoing in France
- 26 Jul 2011 Preclinical development is ongoing in France