Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DMUQWRD)

Drug Name

LYS-SAF302 Drug Info

Synonyms

SRP-9002

Indication

Disease Entry	ICD 11	Status	REF
Mucopolysaccharidosis	5C56.3	Phase 2/3	[1]

Cross-matching ID

Molecule-Related Drug Atlas

Molecule Type:

DTT

Drug Status:

Clinical Trial Drug(s)

Download the Complete Related Drug List

Drug(s) Targeting N-sulphoglucosamine sulphohydrolase (SGSH)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
HGT-1410	DMHZ4JI	Sanfilippo syndrome	5C56.3Y	Phase 2	[3]
ABO-102	DMFJB35	Mucopolysaccharidosis	5C56.3	Phase 1/2	[4]
SOBI003	DMXNTR7	Sanfilippo syndrome	5C56.3Y	Phase 1/2	[5]
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Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Download the Complete Interaction Atlas for Visualization in Cytoscape

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
N-sulphoglucosamine sulphohydrolase (SGSH)	TTPJ2SH	SPHM_HUMAN	Replacement	[2]

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References

1	ClinicalTrials.gov (NCT03612869) Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance). U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of Lysogene.
3	ClinicalTrials.gov (NCT01155778) Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients. U.S. National Institutes of Health.
4	Clinical pipeline report, company report or official report of Abeona Therapeutics.
5	Clinical pipeline report, company report or official report of Swedish Orphan Biovitrum.