Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D93JPW
|
|||
| Drug Name |
PBGM01
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | GM1 gangliosidosis [ICD-11: 5C56.00] | Phase 1/2 | [1] | |
| Company |
Passage Bio
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Beta-galactosidase (GLB1) | Target Info | . | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04713475) Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis. U.S.National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Passage Bio | |||
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