Expert Interview
A Second look at the Phase I/II results for AMT-130, uniQure’s investigational gene therapy for Huntington’s disease.
Ticker(s): QUREInstitution: Georgetown University
- Neuropsychiatrist and Director of the Huntington’s Disease Care, Education and Research Center at MedStar Georgetown University Hospital
- Manages over 250 patients with Huntington's Disease
- Currently the co-principal investigator on several clinical trial studying Huntington’s disease, tardive dyskinesia, Parkinson’s disease, brain injury and Alzheimer’s disease.
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?
Slingshot Insights Explained
Expert research benefits investors by giving them timely access to unbiased real world perspectives on highly specialized topics. Slingshot Insights' crowdfunded model makes this access available at a fraction of the cost of other expert networks.
Reason
*Slingshot Insights provides access to information, not investment advice. We work to support you and facilitate access to experts; however we are not responsible for monitoring calls for the disclosure of MNPI. You should obtain financial, legal and tax advice from your qualified and licensed advisers before deciding to invest in any security.
