General Information of Drug (ID: DMU3PD7)

Drug Name
RGX-121 Drug Info
Indication
Disease Entry ICD 11 Status REF
Mucopolysaccharidosis 5C56.3 Phase 1/2 [1]
Cross-matching ID
TTD Drug ID
DMU3PD7

Molecule-Related Drug Atlas

Molecule-Related Drug Atlas
Molecule Type:
DTT
Drug Status:
Approved Drug(s)
Clinical Trial Drug(s)
Drug(s) Targeting Iduronate 2-sulfatase (IDS)
Drug Name Drug ID Indication ICD 11 Highest Status REF
Idursulfase DM30SXV Hunter syndrome 5C56.31 Approved [3]
Recombinant human iduronate-2-sulfatase DMOWQAF Hunter syndrome 5C56.31 Phase 3 [4]
DNL310 DMO3QAZ Mucopolysaccharidosis II 5C56.31 Phase 2/3 [5]
SB-913 DM98YFK Mucopolysaccharidosis 5C56.3 Phase 1/2 [6]

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Iduronate 2-sulfatase (IDS) TTNY2AP IDS_HUMAN Replacement [2]

References

1 ClinicalTrials.gov (NCT04571970) RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome). U.S. National Institutes of Health.
2 Clinical pipeline report, company report or official report of REGENXBIO.
3 2006 drug approvals: finding the niche. Nat Rev Drug Discov. 2007 Feb;6(2):99-101.
4 The effect of recombinant human iduronate-2-sulfatase (Idursulfase) on growth in young patients with mucopolysaccharidosis type II. PLoS One. 2014 Jan 13;9(1):e85074.
5 Clinical pipeline report, company report or official report of Denali
6 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.