Details of the Drug

General Information of Drug (ID: DM98YFK)

Drug Name
SB-913
Indication
Disease Entry ICD 11 Status REF
Mucopolysaccharidosis 5C56.3 Phase 1/2 [1]
Drug Type
Gene therapy
Cross-matching ID
TTD ID
DY9DX8

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Iduronate 2-sulfatase (IDS) TTNY2AP IDS_HUMAN Replacement [1]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 ClinicalTrials.gov (NCT03041324) Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II. U.S. National Institutes of Health.