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HDAd.CD63-GAA
- Development stage
- Preclinical
- Lead developer
- University of Washington
- Modality
- Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
HDAd.CD63-GAA is an experimental helper-dependent adenoviral (HDAd) gene therapy vector developed for the treatment of Pompe disease. Utilizing an HDAd5/35++L capsid, the vector is designed to transduce hematopoietic stem and progenitor cells (HSPCs) in vivo, effectively turning them into producer cells for engineered extracellular vesicles (EVs). These EVs carry a fusion protein consisting of the EV-sorting protein CD63 and the therapeutic enzyme acid α-glucosidase (GAA), separated by a pH-dependent self-cleaving mini-intein. This design facilitates the efficient packaging of GAA into the EV lumen and its subsequent release. The strategy aims to provide sustained, systemic delivery of GAA, including the ability to cross the blood-brain barrier, to achieve efficient glycogen clearance in multiple tissues such as the central nervous system, heart, and skeletal muscle.
- Other names
- HDAd-CD63-GAAHDAd-CD-63-GAAHDAd-CD 63-GAA
02
Targets
GAA (Lysosomal acid alpha-glucosidase)
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