RGX 501Alternative Names: Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor
Latest Information Update: 09 Jul 2016
At a glance
- Originator REGENXBIO
- Developer REGENXBIO; University of Pennsylvania
- Class Gene therapies; Proteins
- 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
- Phase I/II Hyperlipoproteinaemia type IIa
Most Recent Events
- 05 Jul 2016 Phase-I/II clinical trials in Hyperlipoproteinaemia type IIa in USA (IV)