PBGM01

PBGM01 is an investigational gene therapy designed for the treatment of infantile GM1 gangliosidosis, a rare neurodegenerative disorder caused by mutations in the GLB1 gene that result in little or no residual beta-galactosidase enzyme activity. The therapy uses a next-generation adeno-associated virus serotype hu68 (AAVhu68) vector to deliver a functional copy of the human GLB1 gene directly to the brain and peripheral tissues via intra-cisterna magna administration. By restoring beta-galactosidase activity, PBGM01 aims to reduce toxic accumulation of GM1 gangliosides, reverse neuronal toxicity, and restore developmental potential. Preclinical studies have shown broad distribution and uptake of the enzyme in both central nervous system and peripheral organs. Passage Bio is developing PBGM01, which has received Orphan Drug Designation from both FDA and EMA as well as Rare Pediatric Disease designation from FDA[1][3][5][7].