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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: 08 Sep 2026

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At a glance

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

Most Recent Events

  • 02 Sep 2026 According to an uniQure media release, the Company intends to present a four-year data analysis from the ongoing Phase I/II clinical studies before the end of the current third quarter.
  • 02 Sep 2026 According to an uniQure media release, the company announced the submission of a Biologics License Application to the United States FDA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), for the treatment of Huntington's disease. The Company also announced that its Marketing Authorisation Application for ifezuntirgene inilparvovec has been submitted to the United Kingdom's Medicines and Healthcare products Regulatory Agency. The company has requested priority review for BLA.
  • 29 Jul 2026 According to an uniQure media release,Company plans to present data from its ongoing Phase I/II studies of AMT-130.

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