Details of the Drug Therapeutic Target (DTT)

General Information of Drug Therapeutic Target (DTT) (ID: TTNY2AP)

• DTT Name: Iduronate 2-sulfatase (IDS)
• Synonyms: Idursulfase; Iduronate 2sulfatase 14 kDa chain; IDS; AlphaLiduronate sulfate sulfatase
• Gene Name: IDS
• DTT Type: Successful target; [1]
• BioChemical Class: Sulfuric ester hydrolase
• UniProt ID: IDS_HUMAN
• TTD ID: T28993
• EC Number: EC 3.1.6.13
• Sequence:
  MPPPRTGRGLLWLGLVLSSVCVALGSETQANSTTDALNVLLIIVDDLRPSLGCYGDKLVR
  SPNIDQLASHSLLFQNAFAQQAVCAPSRVSFLTGRRPDTTRLYDFNSYWRVHAGNFSTIP
  QYFKENGYVTMSVGKVFHPGISSNHTDDSPYSWSFPPYHPSSEKYENTKTCRGPDGELHA
  NLLCPVDVLDVPEGTLPDKQSTEQAIQLLEKMKTSASPFFLAVGYHKPHIPFRYPKEFQK
  LYPLENITLAPDPEVPDGLPPVAYNPWMDIRQREDVQALNISVPYGPIPVDFQRKIRQSY
  FASVSYLDTQVGRLLSALDDLQLANSTIIAFTSDHGWALGEHGEWAKYSNFDVATHVPLI
  FYVPGRTASLPEAGEKLFPYLDPFDSASQLMEPGRQSMDLVELVSLFPTLAGLAGLQVPP
  RCPVPSFHVELCREGKNLLKHFRFRDLEEDPYLPGNPRELIAYSQYPRPSDIPQWNSDKP
  SLKDIKIMGYSIRTIDYRYTVWVGFNPDEFLANFSDIHAGELYFVDSDPLQDHNMYNDSQ
  GGDLFQLLMP
• Function: Required for the lysosomal degradation ofheparan sulfate and dermatan sulfate.
• KEGG Pathway:
  Glycosaminoglycan degradation (hsa00531)
  Metabolic pathways (hsa01100)
  Lysosome (hsa04142)
• Reactome Pathway:
  CS/DS degradation (R-HSA-2024101)
  HS-GAG degradation (R-HSA-2024096)
• BioCyc Pathway: MetaCyc:HS00286-MON

Molecular Interaction Atlas (MIA) of This DTT

1 Approved Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Idursulfase	DM30SXV	Hunter syndrome	5C56.31	Approved	[1]

4 Clinical Trial Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Recombinant human iduronate-2-sulfatase	DMOWQAF	Hunter syndrome	5C56.31	Phase 3	[2]
DNL310	DMO3QAZ	Mucopolysaccharidosis II	5C56.31	Phase 2/3	[3]
RGX-121	DMU3PD7	Mucopolysaccharidosis	5C56.3	Phase 1/2	[4]
SB-913	DM98YFK	Mucopolysaccharidosis	5C56.3	Phase 1/2	[5]

The Drug-Metabolizing Enzyme (DME) Role of This DTT

• DTT DME Name: Iduronate 2-sulfatase (IDS) DME Info
• Gene Name: IDS

1 Approved Drug(s) Metabolized by This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Chondroitin sulfate	DM0N19Y	N. A.	N. A.	Phase 4	[6]

References

• [1] 2006 drug approvals: finding the niche. Nat Rev Drug Discov. 2007 Feb;6(2):99-101.
• [2] 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.
• [3] Clinical pipeline report, company report or official report of Denali
• [4] Clinical pipeline report, company report or official report of REGENXBIO.
• [5] 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.
• [6] MFN human ref: Chondroitin sulfate degradation.