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A Phase Ib/II Randomized, Double-Blind Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered RAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington's Disease

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Trial Profile

A Phase Ib/II Randomized, Double-Blind Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered RAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington's Disease

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 21 Aug 2025

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At a glance

  • Drugs AMT-130 (Primary)
  • Indications Huntington's disease
  • Focus Adverse reactions; Proof of concept; Therapeutic Use
  • Sponsors uniQure

Most Recent Events

  • 29 Jul 2025 According to an uniQure media release, the company plans to Hold pre-BLA meeting with FDA in Q42025, and to submit BLA with a request for priority review designation in Q1 2026.
  • 29 Jul 2025 According to an uniQure media release, topline, three-year data from this trial is expected in September 2025.Also company plans to Initiate a fourth cohort evaluating high-dose AMT-130 in six patients with lower striatal volumes compared to those of patients enrolled in previous cohorts.
  • 02 Jun 2025 According to an uniQure media release, Type B meetings and further guidance from the U.S. Food and Drug Administration (FDA), the Company has reached alignment with the FDA on several key components of the statistical analysis plan and Chemistry, Manufacturing and Controls (CMC) information that will support a Biologics License Application

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