AMT 150
Alternative Names: AMT-150Latest Information Update: 28 Jul 2022
At a glance
- Originator uniQure
- Class Exosome therapies; Gene therapies
- Mechanism of Action Gene silencing
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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
Highest Development Phases
- No development reported Ataxia
Most Recent Events
- 28 Jul 2022 No recent reports of development identified for preclinical development in Ataxia in Netherlands (Intrathecal)
- 19 Nov 2018 Preclinical data for Ataxia released by uniQure
- 17 Nov 2018 uniQure announces intention to file for orphan drug designation for Ataxia (Spinocerebellar ataxia type 3)