Drug Profile
Research programme: CNS gene therapies - uniQure
Alternative Names: AAV 5; AAV5 vector; AAV5-miC; AMT-090Latest Information Update: 28 Mar 2023
Price :
$50
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At a glance
- Originator Amsterdam Molecular Therapeutics
- Developer Lund University; uniQure; Universite de Toulouse; University of Cambridge
- Class Antidementias; Antiparkinsonians; Gene therapies; Neuroprotectants
- Mechanism of Action Gene silencing; Gene transference; Glial cell line-derived neurotrophic factor agonists
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
- Available For Licensing Yes
Highest Development Phases
- No development reported Amyotrophic lateral sclerosis; Dementia; Hearing loss; Multiple system atrophy; Parkinson's disease
Most Recent Events
- 28 Mar 2023 No recent reports of development identified for preclinical development in Amyotrophic-lateral-sclerosis in Netherlands (Parenteral)
- 28 Mar 2023 No recent reports of development identified for preclinical development in Dementia in Netherlands (Parenteral)
- 15 Feb 2019 Preclinical trials in Amyotrophic lateral sclerosis in Netherlands (Parenteral) before February 2019