AVR-RD-03

**AVR-RD-03** is an investigational lentiviral-based hematopoietic stem cell (HSC) gene therapy developed by AVROBIO (now Tectonic Therapeutic) for **Pompe disease**, a rare lysosomal storage disorder caused by deficiency of acid alpha-glucosidase (GAA), leading to glycogen accumulation. It uses a proprietary **Glycosylation-Independent Lysosomal Targeting (GILT)-tag** system to enhance lysosomal delivery of the codon-optimized GAA transgene integrated into autologous HSCs via lentiviral vector, aiming for sustained systemic enzyme production and CNS penetration to reduce toxic glycogen buildup. Preclinical data demonstrate significant glycogen reduction in diseased tissues, including the central nervous system, with plans for clinical trials discussed with regulators targeting initiation around 2023.[1][4][7][13][15][17]