Details of the Drug

General Information of Drug (ID: DMA8Z4N)

Drug Name
SB-318
Indication
Disease Entry ICD 11 Status REF
Mucopolysaccharidosis 5C56.3 Phase 1/2 [1]
Drug Type
Gene therapy
Cross-matching ID
TTD ID
DYWK46

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Alpha-L-iduronidase (IDUA) TT0IUKX IDUA_HUMAN Replacement [1]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 ClinicalTrials.gov (NCT02702115) Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I. U.S. National Institutes of Health.