Details of the Drug

General Information of Drug (ID: DMI0H3B)

Drug Name

UshStat

Synonyms

Myosin 7A gene therapy (Usher syndrome 1B, LentiVector), Oxford BioMedica; Myosin VIIA gene therapy (Usher syndrome 1B, LentiVector), Oxford BioMedica

Indication

Disease Entry	ICD 11	Status	REF
Deafness	AB52	Phase 1/2	[1]
Retinitis pigmentosa	9B70	Phase 1	[2]
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Cross-matching ID

TTD ID: D0YO5Z

Repurposed Drugs (RPD)

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Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Myosin-7 (MYH7)	TTNIMDP	MYH7_HUMAN	Modulator	[3]

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Molecular Interaction Atlas (MIA)

MIA Detail

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References

1	ClinicalTrials.gov (NCT02065011) A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat in Patients With Usher Syndrome Type 1B. U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
3	EIAV-based retinal gene therapy in the shaker1 mouse model for usher syndrome type 1B: development of UshStat. PLoS One. 2014 Apr 4;9(4):e94272.