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LV-GAAco
- Development stage
- Preclinical
- Lead developer
- Erasmus MC
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
LV-GAAco is an experimental lentiviral gene therapy candidate designed for the treatment of Pompe disease (Glycogen Storage Disease Type II). It consists of a lentiviral vector encoding a codon-optimized version of the human acid alpha-glucosidase (GAA) enzyme. Pompe disease is a lysosomal storage disorder characterized by a deficiency in GAA, which leads to the toxic accumulation of glycogen in skeletal and cardiac muscles. LV-GAAco aims to provide a functional copy of the GAA gene to restore enzyme activity and reduce glycogen levels. In preclinical studies, LV-GAAco has been shown to partially correct the muscle proteome in GAA-deficient mouse models. It is frequently used as a foundational construct or baseline comparator for enhanced versions, such as LV-IGF2.GAAco, which incorporates an IGF2 peptide tag to improve cellular uptake via the cation-independent mannose-6-phosphate/IGF2 receptor.
- Other names
- lentiviral codon-optimized acid alpha-glucosidaseLV-GAA
02
Targets
GAA (Lysosomal acid alpha-glucosidase)
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