General Information of Drug (ID: DMPD79C)

Drug Name
LYS-GM-101 Drug Info
Indication
Disease Entry ICD 11 Status REF
GM1 gangliosidosis 5C56.00 Phase 1/2 [1]
Cross-matching ID
TTD Drug ID
DMPD79C

Molecule-Related Drug Atlas

Molecule-Related Drug Atlas
Molecule Type:
DTT
Drug Status:
Clinical Trial Drug(s)
Preclinical Drug(s)
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 [2]
AXO-AAV-GM1 DMFBMI7 GM1 gangliosidosis 5C56.00 Phase 1/2 [3]
5N,6S-(N'-butyliminomethylidene)-6-thio-1-deoxygalactonojirimycin DM7RHOC GM1 gangliosidosis 5C56.00 Preclinical [4]
N-Octyl-4-epi-beta-valienamine DMTQ2UD GM1 gangliosidosis 5C56.00 Preclinical [5]

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Beta-galactosidase (GLB1) TTNGJPH BGAL_HUMAN Not Available [1]

References

1 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.
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 A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis. Mol Ther. 2013 Mar;21(3):526-32.
5 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.