Details of the Drug Therapeutic Target (DTT)

General Information of Drug Therapeutic Target (DTT) (ID: TT5IZRB)

DTT Name Galactocerebrosidase (GALC)
Synonyms Galactosylceramide betagalactosidase; Galactosylceramide beta-galactosidase; Galactosylceramidase; Galactocerebroside betagalactosidase; Galactocerebroside beta-galactosidase; GALCERase
Gene Name GALC
DTT Type
Literature-reported target
 [1]
BioChemical Class
Glycosylase
UniProt ID
GALC_HUMAN
TTD ID
T72024
3D Structure
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2D Sequence (FASTA)
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3D Structure (PDB)
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EC Number
EC 3.2.1.46
Sequence
MAEWLLSASWQRRAKAMTAAAGSAGRAAVPLLLCALLAPGGAYVLDDSDGLGREFDGIGA
VSGGGATSRLLVNYPEPYRSQILDYLFKPNFGASLHILKVEIGGDGQTTDGTEPSHMHYA
LDENYFRGYEWWLMKEAKKRNPNITLIGLPWSFPGWLGKGFDWPYVNLQLTAYYVVTWIV
GAKRYHDLDIDYIGIWNERSYNANYIKILRKMLNYQGLQRVKIIASDNLWESISASMLLD
AELFKVVDVIGAHYPGTHSAKDAKLTGKKLWSSEDFSTLNSDMGAGCWGRILNQNYINGY
MTSTIAWNLVASYYEQLPYGRCGLMTAQEPWSGHYVVESPVWVSAHTTQFTQPGWYYLKT
VGHLEKGGSYVALTDGLGNLTIIIETMSHKHSKCIRPFLPYFNVSQQFATFVLKGSFSEI
PELQVWYTKLGKTSERFLFKQLDSLWLLDSDGSFTLSLHEDELFTLTTLTTGRKGSYPLP
PKSQPFPSTYKDDFNVDYPFFSEAPNFADQTGVFEYFTNIEDPGEHHFTLRQVLNQRPIT
WAADASNTISIIGDYNWTNLTIKCDVYIETPDTGGVFIAGRVNKGGILIRSARGIFFWIF
ANGSYRVTGDLAGWIIYALGRVEVTAKKWYTLTLTIKGHFTSGMLNDKSLWTDIPVNFPK
NGWAAIGTHSFEFAQFDNFLVEATR
Function
Enzyme with very low activity responsible for the lysosomal catabolism of galactosylceramide, a major lipid in myelin, kidney and epithelial cells of small intestine and colon. Hydrolyzes the galactose ester bonds of galactosylceramide, galactosylsphingosine, lactosylceramide, and monogalactosyldiglyceride.
KEGG Pathway
Sphingolipid metabolism (hsa00600 )
Metabolic pathways (hsa01100 )
Lysosome (hsa04142 )
Reactome Pathway
Glycosphingolipid metabolism (R-HSA-1660662 )

Molecular Interaction Atlas (MIA) of This DTT

Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This DTT
2 Clinical Trial Drug(s) Targeting This DTT
Drug Name Drug ID Indication ICD 11 Highest Status REF
FBX-101 DMZR3OJ Krabbe disease 8A44.4 Phase 1/2 [2]
PBKR03 DM57HM7 Globoid cell leukodystrophy 8A44.4 Phase 1/2 [3]
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References

1 Galactocerebrosidase deficiency in globoid cell leucodystrophy of late onset. Arch Dis Child. 1972 Jun;47(253):449-50.
2 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.
3 Clinical pipeline report, company report or official report of Passage Bio