Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DMNJEGV)

Drug Name

PBGM01 Drug Info

Indication

Disease Entry	ICD 11	Status	REF
GM1 gangliosidosis	5C56.00	Phase 1/2	[1]

Cross-matching ID

Molecule-Related Drug Atlas

Molecule Type:

DTT

Drug Status:

Clinical Trial Drug(s)

Preclinical Drug(s)

Download the Complete Related Drug List

Drug(s) Targeting Beta-galactosidase (GLB1)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
AXO-AAV-GM1	DMFBMI7	GM1 gangliosidosis	5C56.00	Phase 1/2	[3]
LYS-GM-101	DMPD79C	GM1 gangliosidosis	5C56.00	Phase 1/2	[4]
5N,6S-(N'-butyliminomethylidene)-6-thio-1-deoxygalactonojirimycin	DM7RHOC	GM1 gangliosidosis	5C56.00	Preclinical	[5]
N-Octyl-4-epi-beta-valienamine	DMTQ2UD	GM1 gangliosidosis	5C56.00	Preclinical	[6]
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Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Download the Complete Interaction Atlas for Visualization in Cytoscape

Drug Therapeutic Target (DTT)

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
3	Clinical pipeline report, company report or official report of Axovant Gene Therapies
4	ClinicalTrials.gov (NCT04273269) An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human beta-Galactosidase cDNA for Treatment of GM1 Gangliosidosis. U.S.National Institutes of Health.
5	A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis. Mol Ther. 2013 Mar;21(3):526-32.
6	Chemical modification of the beta-glucocerebrosidase inhibitor N-octyl-beta-valienamine: synthesis and biological evaluation of 4-epimeric and 4-O-(beta-D-galactopyranosyl) derivatives. Bioorg Med Chem. 2002 Jun;10(6):1967-72.