AAVB1-GAA

AAVB1-GAA is a gene therapy vector composed of an engineered adeno-associated virus serotype B1 (AAVB1) delivering the human acid alpha-glucosidase (GAA) gene. It is designed to treat Pompe disease, a lysosomal storage disorder caused by GAA deficiency leading to glycogen accumulation in muscle and the central nervous system. The AAVB1 capsid targets muscle and respiratory tissues efficiently. Systemic (intravenous) delivery of AAVB1-GAA to GAA-deficient mice results in transduction of cardiac, skeletal, and some respiratory muscles, with restoration of GAA enzymatic activity, clearance of glycogen, improved muscle/respiratory function, weight gain, and increased survival. AAVB1-GAA may have superior transduction efficiency for certain tissues compared to other AAV serotypes, particularly in the upper airway and respiratory system. This vector is primarily being investigated in preclinical animal studies for Pompe disease[1][3][10].

Other names

AAVB1-GAAAAVB-1-GAAAAVB 1-GAA