Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0LJ4H
|
|||
| Drug Name |
PBKR03
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Globoid cell leukodystrophy [ICD-11: 8A44.4] | Phase 1/2 | [1] | |
| Company |
Passage Bio
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Galactocerebrosidase (GALC) | Target Info | . | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04771416) A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy). U.S.National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Passage Bio | |||
If You Find Any Error in Data or Bug in Web Service, Please Kindly Report It to Dr. Zhou and Dr. Zhang.