Research programme: lysosomal storage disorder therapeutics - Calporta
Latest Information Update: 14 Jul 2015
At a glance
- Originator University of Michigan
- Developer Calporta Therapeutics; University of Michigan
- Class Small molecules
- Mechanism of Action MCOLN1 protein modulators
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 Niemann-Pick disease type C
Most Recent Events
- 15 Jun 2015 Early research in Niemann-Pick disease type C in USA (unspecified route)