Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0YO5Z
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| Former ID |
DIB007728
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| Drug Name |
UshStat
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| Synonyms |
Myosin 7A gene therapy (Usher syndrome 1B, LentiVector), Oxford BioMedica; Myosin VIIA gene therapy (Usher syndrome 1B, LentiVector), Oxford BioMedica
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| Indication | Deafness [ICD-11: AB52] | Phase 1/2 | [1] | |
| Retinitis pigmentosa [ICD-11: 9B70; ICD-10: H35.5] | Phase 1 | [2] | ||
| Company |
Sanofi us
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Myosin-7 (MYH7) | Target Info | Modulator | [3] |
| KEGG Pathway | cGMP-PKG signaling pathway | |||
| Cardiac muscle contraction | ||||
| Adrenergic signaling in cardiomyocytes | ||||
| Tight junction | ||||
| Hypertrophic cardiomyopathy (HCM) | ||||
| Dilated cardiomyopathy | ||||
| Viral myocarditis | ||||
| Panther Pathway | Cytoskeletal regulation by Rho GTPase | |||
| Inflammation mediated by chemokine and cytokine signaling pathway | ||||
| Nicotinic acetylcholine receptor signaling pathway | ||||
| Wnt signaling pathway | ||||
| Reactome | Translocation of GLUT4 to the plasma membrane | |||
| References | Top | |||
|---|---|---|---|---|
| REF 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. | |||
| REF 2 | Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA) | |||
| REF 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. | |||
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