Passage Bio reports PBGM01 interim data: MRI stabilization, biomarker decreases in GM1 gangliosidosis trial

Passage BIO, Inc.

All six treated patients showed stabilization of MRI severity scores over 6-12 months follow-up.
High-dose PBGM01 resulted in 3.6-5.2x increase in CSF β-gal activity, sustained up to 12 months.
No treatment-related serious adverse events, DRG toxicity, or complications from ICM delivery.
Urine Dp5 levels decreased in high-dose patients, indicating peripheral β-gal activity.
Milder developmental delay at baseline associated with improved treatment response.

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