Research programme: CNS gene therapies - uniQureAlternative Names: AAV5; AMT-090; AMT-130
Latest Information Update: 17 Jan 2017
At a glance
- Originator Amsterdam Molecular Therapeutics
- Developer Lund University; uniQure; Universite de Toulouse; University of Cambridge
- Class Gene therapies; Nerve growth factors; Small interfering RNA
- Mechanism of Action DNA-directed RNA polymerase inhibitors; Gene silencing; Gene transference; Glial cell line-derived neurotrophic factor agonists; RNA interference
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
- Available For Licensing Yes
Highest Development Phases
- Preclinical Hearing loss; Huntington's disease; Multiple system atrophy; Parkinson's disease