Product Description
AMT-150 is a AAV5-microRNA against ATXN3 to lower toxic ataxin-3 protein. (Sourced from: https://www.uniqure.com/R&D%20Day%202021%20Final%20PPT.pdf)
Mechanisms of Action: Gene Therapy,ATXN3
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: Injection
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: UniQure N.V.
Company Location: AMSTERDAM P7 1105 BP
Company CEO: Matthew Kapusta
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Highest Development Phases
Phase 0: Huntington Disease
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