- Passage Bio Inc PASG announced new interim safety, biomarker, and clinical development results from cohorts 1-3 in the Imagine-1 Phase 1/2 study of AAVhu68 gene therapy PBGM01 for GM1 Gangliosidosis (GM1).
- GM1 is a rare, fatal lysosomal storage disease in which mutations in the GLB1 gene result in the deficient activity of the enzyme beta-galactosidase (β-Gal), an enzyme that helps break down fats and sugars.
- PBGM01 administration resulted in dose-dependent increases in CSF β-Gal activity, with both patients who received the high dose (Cohort 2, late infantile) exhibiting increases in enzyme activity well above baseline.
- PBGM01 administration also resulted in dose-dependent decreases in CSF GM1 ganglioside levels.
- Patients with milder development delay at dosing showed a higher response to PBGM01 treatment.
- Patients 1 (late infantile, low dose) and 5 (early infantile, low dose), who both demonstrated modest developmental delay at baseline, showed increases in overall developmental age.
- On the safety front, no treatment-related serious adverse events (SAEs) were observed. All treatment-related adverse events (AEs) were mild to moderate in severity.
- Passage has completed dosing of PBGM01 in the dose-escalation portion of the study and will evaluate dosing further patients at the current high dose and/or a higher dose as data from cohorts 1-4 mature.
- Data from Cohort 4 is anticipated by mid-2023.
- Price Action: PASG shares are up 6.14% at $1.21 on the last check Wednesday.
Loading...
Loading...
PASGPassage Bio Inc
$7.6911.1%
Edge Rankings
Momentum
8.31
Price Trend
Short
Medium
Long
© 2025 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.