Details of the Drug

General Information of Drug (ID: DMNJEGV)

Drug Name
PBGM01
Indication
Disease Entry ICD 11 Status REF
GM1 gangliosidosis 5C56.00 Phase 1/2 [1]
Drug Type
Gene therapy
Cross-matching ID
TTD ID
D93JPW

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Beta-galactosidase (GLB1) TTNGJPH BGAL_HUMAN Not Available [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

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