General Information of Drug (ID: DM57HM7)

Drug Name
PBKR03 Drug Info
Indication
Disease Entry ICD 11 Status REF
Globoid cell leukodystrophy 8A44.4 Phase 1/2 [1]
Cross-matching ID
TTD Drug ID
DM57HM7

Molecule-Related Drug Atlas

Molecule-Related Drug Atlas
Molecule Type:
DTT
Drug Status:
Clinical Trial Drug(s)
Drug(s) Targeting Galactocerebrosidase (GALC)
Drug Name Drug ID Indication ICD 11 Highest Status REF
FBX-101 DMZR3OJ Krabbe disease 8A44.4 Phase 1/2 [3]

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Galactocerebrosidase (GALC) TT5IZRB GALC_HUMAN Not Available [2]

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
3 ClinicalTrials.gov (NCT04693598) A Phase 1/2 Clinical Study of Intravenous Gene Transfer With an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation (RESKUE). U.S.National Institutes of Health.