General Information of Drug (ID: DMU87HW)

Drug Name
RGX-111 Drug Info
Indication
Disease Entry ICD 11 Status REF
Mucopolysaccharidosis 5C56.3 Phase 1 [1]
Cross-matching ID
TTD Drug ID
DMU87HW

Molecule-Related Drug Atlas

Molecule-Related Drug Atlas
Molecule Type:
DTT
Drug Status:
Clinical Trial Drug(s)
Drug(s) Targeting Alpha-L-iduronidase (IDUA)
Drug Name Drug ID Indication ICD 11 Highest Status REF
SB-318 DMA8Z4N Mucopolysaccharidosis 5C56.3 Phase 1/2 [3]

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Alpha-L-iduronidase (IDUA) TT0IUKX IDUA_HUMAN Replacement [2]

References

1 ClinicalTrials.gov (NCT03580083) RGX-111 Gene Therapy in Patients With MPS I. U.S. National Institutes of Health.
2 Clinical pipeline report, company report or official report of REGENXBIO.
3 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.