Research programme: Galgt2 gene therapies - Nationwide Children's Hospital / SareptaAlternative Names: GalNAc tranferase gene therapies
Latest Information Update: 20 Jan 2017
At a glance
- Originator Nationwide Children's Hospital
- Developer Nationwide Children's Hospital; Sarepta Therapeutics
- Class Gene therapies
- Mechanism of Action 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 Duchenne muscular dystrophy