Expert Interview
A Second Look: Discussing the phase 2 SEQUOIA trial data on Arrowhead 's fazirsiran in treating A1AT liver disease
Ticker(s): ARWRInstitution: NYU Langone
- Assistant Professor of Gastroenterology and Hepatology at NYU Langone Health.
- Treats 750 patients with liver disease and 11 patients with A1ATD.
- Research interests include radiofrequency Ablation in a variety of gastrointestinal conditions.
Can you give an overview of your practice?
Added By: max_adminCan you give me an idea how many A1AD liver disease patients end up needing a liver transplant, and how long that usually takes?
Added By: max_adminOn a high level, can you discuss the approaches from Arrowhead and Vertex, siRNAs vs correctors?
Added By: max_adminHow do you think patients on existing augmentation therapy would affect efficacy, if at all?
Added By: max_adminDo you expect any variance in efficacy depending on fibrosis stage?
Added By: max_adminGiven that there are no approved therapies for the A1AD liver disease, what do you envision would be an appropriate phase 3 design, including primary endpoint?
Added By: max_adminLet’s assume fazirsiran is the 1st drug approved in AATD, either lung or liver, how do you imagine it being used vs the current SoC?
Added By: max_adminHow would you rate your enthusiasm about fazirsiran on a scale of 1-10, and you can you elaborate on why?
Added By: max_adminAre there any other approaches to A1AD, whether liver or lung disease, that you find interesting and are following?
Added By: max_adminAre there any opinions on gene therapy for A1ATD?
Added By: max_adminAre You Interested In These Questions?
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