Passage Bio (NASDAQ:PASG) announces publication of data in a murine model of GM1 gangliosidosis (GM1) demonstrating that a single intracerebroventricular injection of an optimized adeno-associated virus (PBGM01) into the cerebral spinal fluid (CSF) resulted in significant expression of Beta-galactosidase (β-gal) in the brain and peripheral tissues, and demonstrated dose-related reductions in neuronal lysosomal storage lesions, neurological impairment and improvement in survival.
GM1 is a rare and often life-threatening lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid β-gal.
The mice received the single administration at age one month and were evaluated over 300 days. β-gal activity was increased significantly in the CSF and serum of the vector-treated mice compared to vehicle control-treated mice.
Considerable improvements in gait assessments and preservation of neurological function were observed throughout the study period.
There were significant decreases in lysosomal storage lesions of vector-treated animals, and by day 300, all