Drug Profile
ADVM 043
Alternative Names: AAVrh 10halpha1AT; AAVrh-10hA1AT; AAVrh.10hA1AT; AAVrh.10halpha1AT; ADVM-043- Adverum; ANN-001Latest Information Update: 11 Apr 2022
Price :
$50
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At a glance
- Originator Weill Cornell Medical College
- Developer Adverum Biotechnologies
- Class Gene therapies
- Mechanism of Action Gene transference
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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
- Discontinued Alpha 1-antitrypsin deficiency
Most Recent Events
- 11 Jan 2019 Adverum Biotechnologies initiates enrolment in a long-term follow-up trial for Alpha 1-antitrypsin deficiency in USA (NCT03804021)
- 02 Nov 2018 Discontinued - Phase-I/II for Alpha 1-antitrypsin deficiency in USA (IV, Intrapleural)
- 02 Nov 2018 Adverse events and pharmacodynamics data from the phase I/II ADVANCE trial in Alpha 1-antitrypsin deficiency released by Adverum Biotechnologies