Research programme: TDP-43 targeted neurodegenerative disorders therapy - Aquinnah PharmaceuticalsAlternative Names: TDP-43 stress granules
Latest Information Update: 27 Jul 2015
At a glance
- Originator Aquinnah Pharmaceuticals
- Class Neuroprotectants
- Mechanism of Action TDP-43 protein inhibitors
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
- Preclinical Alzheimer's disease; Amyotrophic lateral sclerosis