Drug Profile

ADVM 043

Alternative Names: AAVrh 10halpha1AT; AAVrh-10hA1AT; AAVrh.10hA1AT; AAVrh.10halpha1AT; ADVM-043- Adverum; ANN-001

Latest Information Update: 15 Dec 2025

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At a glance

Originator Weill Cornell Medical College
Developer Adverum Biotechnologies
Class Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

Highest Development Phases

Discontinued Alpha 1-antitrypsin deficiency

Most Recent Events

09 Dec 2025 Adverum Biotechnologies has been acquired by Eli Lilly and Company
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)

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