Research programme: rare liver disease therapeutics - Dimension Therapeutics/University of PennsylvaniaAlternative Names: DTX 501; DTX 601; DTX 701; DTX501 AAV PAH; DTX601 AAV ASS1; DTX701 AAV ATP7B
Latest Information Update: 16 May 2016
At a glance
- Originator Dimension Therapeutics; University of Pennsylvania
- Developer University of Pennsylvania
- 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 Yes
Highest Development Phases
- Research Liver disorders