Drug Profile
AMT 021
Alternative Names: AAV5-AAT-PBGD; AAV5-PBGD gene therapy; AMT-020; AMT-021; rAAV2/5-PBGDLatest Information Update: 05 Sep 2017
Price :
$50
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At a glance
- Originator Digna Biotech; University of Navarra
- Developer uniQure
- Class Gene therapies
- Mechanism of Action Hydroxymethylbilane synthase stimulants
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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 Acute intermittent porphyria
Most Recent Events
- 28 Aug 2017 Protein Sciences Corporation has been acquired by Sanofi
- 16 Jul 2016 No recent reports of development identified for phase-I development in Acute-intermittent-porphyria in Spain (IV, Injection)
- 16 Jul 2016 No recent reports of development identified for preclinical development in Acute-intermittent-porphyria in Netherlands (IV, Injection)