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ADVM-043

Development stage: Discontinued
Lead developer: Adverum Biotechnologies
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intravenous, Parenteral

Overview

ADVM-043 is an adeno-associated virus (AAV)-based gene therapy candidate developed for the treatment of alpha-1 antitrypsin (A1AT) deficiency. Originally licensed from Cornell University by Annapurna Therapeutics (later acquired by Adverum Biotechnologies), the therapy utilizes an AAV vector to deliver a functional copy of the SERPINA1 gene, aiming to provide sustained expression of the A1AT protein to prevent lung damage. Adverum discontinued the program in late 2018 following a review of data from a Phase 1/2 clinical trial.

Other names: AAV-alpha1 gene therapyAAV-alpha-1 gene therapyAAV-alpha 1 gene therapyAAV.7m8-hAAT

Targets

AAVR (AAV receptor)

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