Details of the Drug

General Information of Drug (ID: DM57HM7)

Drug Name
PBKR03
Indication
Disease Entry ICD 11 Status REF
Globoid cell leukodystrophy 8A44.4 Phase 1/2 [1]
Drug Type
Gene therapy
Cross-matching ID
TTD ID
D0LJ4H

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Galactocerebrosidase (GALC) TT5IZRB GALC_HUMAN Not Available [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

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.
2 Clinical pipeline report, company report or official report of Passage Bio