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CTB-GAA

Development stage: Preclinical
Lead developer: University of Pennsylvania
Modality: Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
Administration: Oral

Overview

**CTB-GAA** is an orally administered, plant-made recombinant fusion protein investigated preclinically for antigen-specific immune tolerance in **Pompe disease**. It comprises the cholera toxin B subunit carrier fused to the N-terminal 410 amino acids of acid alpha-glucosidase, expressed in transplastomic tobacco chloroplasts. The construct is intended to deliver GAA epitopes across the intestinal mucosa and reduce anti-recombinant GAA antibody formation during enzyme replacement therapy; oral administration reduced GAA-specific immunoglobulin responses in a Pompe mouse model. ([pmc.ncbi.nlm.nih.gov](https://pmc.ncbi.nlm.nih.gov/articles/PMC4578979/))

Other names: CTB-GAA

Targets

GM1 (GM1 ganglioside)

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