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

General Information of Drug (ID: DMTQ2UD)

Drug Name

N-Octyl-4-epi-beta-valienamine Drug Info

Synonyms

CHEMBL1922581; (1s,2s,3s,6r)-4-(Hydroxymethyl)-6-(Octylamino)cyclohex-4-Ene-1,2,3-Triol; CHEMBL1922579; N-Octyl-4-epi-beta-Valienamine; BDBM50358321; Q27463709

Indication

Disease Entry	ICD 11	Status	REF
GM1 gangliosidosis	5C56.00	Preclinical	[1]

Cross-matching ID

PubChem CID: 10039700
TTD Drug ID: DMTQ2UD

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
PBGM01	DMNJEGV	GM1 gangliosidosis	5C56.00	Phase 1/2	[3]
AXO-AAV-GM1	DMFBMI7	GM1 gangliosidosis	5C56.00	Phase 1/2	[4]
LYS-GM-101	DMPD79C	GM1 gangliosidosis	5C56.00	Phase 1/2	[5]
5N,6S-(N'-butyliminomethylidene)-6-thio-1-deoxygalactonojirimycin	DM7RHOC	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)

DTT Name	DTT ID	UniProt ID	MOA	REF
Beta-galactosidase (GLB1)	TTNGJPH	BGAL_HUMAN	Inhibitor	[2]

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References

1	Lysosomes as a therapeutic target. Nat Rev Drug Discov. 2019 Dec;18(12):923-948.
2	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.
3	Clinical pipeline report, company report or official report of Passage Bio
4	Clinical pipeline report, company report or official report of Axovant Gene Therapies
5	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.
6	A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis. Mol Ther. 2013 Mar;21(3):526-32.