Details of the Drug

General Information of Drug (ID: DMU87HW)

• Drug Name: RGX-111

Indication

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

• Drug Type: Gene therapy
• Cross-matching ID: TTD ID: D8XHT1

Molecular Interaction Atlas of This Drug

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.