PASG - Dosing underway in Passage Bio's PBGM01 study for infantile GM1 gangliosidosis

The first patient has been dosed in Passage Bio's (PASG) Phase 1/2 clinical trial program for PBGM01 for the treatment of infantile GM1 gangliosidosis (GM1). Imagine-1 is a dose-escalation study of PBGM01 administered by a single injection into the cisterna magna in pediatric subjects with early and late infantile GM1.The clinical program will enroll a total of four cohorts of two patients each, with separate dose-escalation cohorts for late infantile GM1 and early infantile GM1. The primary goal of the Phase 1/2 study is to first assess safety and tolerability and then efficacy of PBGM01 in patients.Passage Bio anticipates initial safety and 30-day biomarker data from the first cohort in mid-2021.GM1, a rare monogenic lysosomal storage disease, is caused by mutations in the GLB1 gene, which encodes the lysosomal enzyme beta-galactosidase (?-gal). Reduced ?-gal activity results in the accumulation of toxic levels of GM1 gangliosides in neurons throughout the brain, causing rapidly progressive

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Dosing underway in Passage Bio's PBGM01 study for infantile GM1 gangliosidosis