Drug Profile

AMT 130

Alternative Names: AAV5-miHTT; AMT-130; rAAV5-miHTT

Latest Information Update: 17 Jun 2025

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At a glance

Originator Amsterdam Molecular Therapeutics
Developer uniQure
Class Antidementias; Antiparkinsonians; Gene therapies; MicroRNAs; Neuroprotectants
Mechanism of Action Huntingtin protein expression inhibitors; RNA interference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Huntington's disease
New Molecular Entity No

Highest Development Phases

Phase I/II Huntington's disease

Most Recent Events

11 Jun 2025 uniQure plans for the launch of AMT 130 for Huntington’s disease in USA in 2026
02 Jun 2025 uniQure plans to submit the BLA for AMT 130 for Huntington’s disease in the first quarter of 2026.
17 Apr 2025 AMT 130 receives Breakthrough Therapy status for Huntington's disease in USA

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