Passage Bio reports positive interim Phase 1/2 data for PBGM01 in GM1 gangliosidosis

Passage BIO, Inc.

No treatment-related SAEs; all AEs mild-moderate; no DRG toxicity or ICM administration complications.
Dose-dependent CSF β-Gal activity increases (up to 5.2-fold) and GM1 ganglioside decreases (up to 75%) in high-dose cohort.
Patients with milder baseline developmental delay showed meaningful improvement on Vineland and Bayley scales.
Cohort 4 fully dosed; data expected by mid-2023; conference call held December 14, 2022.

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