Drug intelligence / Profile preview

AT002

Development stage: Preclinical
Lead developer: Astellas Gene Therapies
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Oral

Overview

AT002 is an adeno-associated virus (AAV) gene therapy developed by Audentes Therapeutics for the treatment of Pompe disease. It is designed to deliver a functional copy of the gene encoding acid alpha-glucosidase (GAA), the enzyme deficient in patients with Pompe disease, to restore its activity and break down accumulated glycogen.

Other names: AAV-GAA

Targets

GAA (Lysosomal acid alpha-glucosidase)

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