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rAAV-GDE

Development stage: Preclinical
Lead developer: Généthon
Modality: Gene Therapies
Administration: Intravenous

Overview

rAAV-GDE is a liver-directed gene therapy candidate developed by Généthon for the treatment of Glycogen Storage Disease type III (GSDIII). GSDIII is caused by a deficiency in the glycogen debranching enzyme (GDE), leading to excessive glycogen accumulation in the liver, heart, and skeletal muscles. The therapy utilizes a recombinant adeno-associated virus (rAAV) vector to deliver a functional GDE transgene to hepatocytes. Research presented at ASGCT 2026 highlights that the persistence of transgene expression is influenced by hepatocyte proliferation and immune activation, and that CpG depletion of the expression cassette can enhance long-term stability in young animal models.

Other names: rAAV-GDE vectorAAV-GDE

Targets

AAVR (AAV receptor)AGL (Glycogen debranching enzyme)

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