Details of the Drug

General Information of Drug (ID: DMUQWRD)

Drug Name

LYS-SAF302

Synonyms

SRP-9002

Indication

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

Gene therapy

Cross-matching ID

Molecular Interaction Atlas of This Drug

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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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

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.