Expert Interview
Examining uniQure’s investigational gene therapy for Huntington’s disease, AMT-130, amid Regulatory Questions from the FDA
Ticker(s): QURE- Chief of Behavioral and Cognitive Neurology and Professor of Neurology at major US academic center.
- Currently manages 10+ patients with Huntington's Disease 500 patients with Parkinson's Disease
- Head of his research lab which investigates the neurobiological basis neurodegeneration through innovative cognitive neuroscience, neuroimaging tools, and biomarker discovery using patient biofluids.
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 there rumblings amongst the community/experts on what sort of internal decision-making occurred to prompt the FDA's flip flop?
Added By: user09b50267What were initial impressions when evaluating early results against the sham-surgery arm? If the relative parity in results was attributed to variance (i.e. small sample size, not enough time for symptom progression, etc) - does the three year timeframe from September data still pose similar risks?
Added By: user09b50267The skepticism on some of the questionnaire based results is fair. What does he think of the NfL measures which the team referenced as a secondary endpoint?
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