Expert Interview
A look at the Phase I/II results for AMT-130, uniQure’s investigational gene therapy for Huntington’s disease.
Ticker(s): QUREInstitution: Banner Sun Health Research Institute
- Board-certified neurologist at Banner Sun Health Research Institute specializing in movement disorders
- Currently manages 10+ patients with Huntington's Disease and 30 patients for tardive dyskinesia
- Research interests center on advancing treatments for movement disorders, particularly tardive dyskinesia, through clinical trials and investigations into novel therapies like INGREZZA (valbenazine).
Which patient subpopulations (e.g. by CAG repeat length, disease stage at infusion, baseline cUHDRS) appear to derive the most benefit, and are there any biomarker correlates (e.g. MRI, CSF huntingtin levels) predictive of response or safety?
Added By: sara_adminHow do the observed effects of AMT-130 compare with existing or emerging therapies in Huntington’s disease, and where do you envision this gene therapy fitting into standard of care (e.g. combination with symptomatic treatments, timing of intervention)?
Added By: sara_adminGiven that AMT-130 is delivered via an intrastriatal AAV vector, what have we learned about optimal dosing, vector distribution, and potential immunogenicity risks that could influence broader clinical use or future gene therapy approaches in Huntington’s disease?
Added By: sara_adminAre You Interested In These Questions?
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