Tough road ahead for uniQure's AMT-130 in Huntington's disease

30 June 2021

Dutch gene therapy company uniQure (Nasdaq: QURE) recently announced the enrolment of the first two patients in the second cohort of a Phase I/II clinical trial of AMT-130, a micro ribonucleic acid (miRNA) gene therapy, in development for Huntington’s disease (HD).

Considering the pressing need for a DMT and the limited pipeline candidates, AMT-130 will have a great opportunity to become a game changer for HD in the next ten years if it is deemed effective in trials, says data and analytics company GlobalData.

Though this is a promising step towards advancing the development of this disease-modifying therapy (DMT) in a disease with significant unmet clinical need, AMT-130 still has a long and challenging road to prove its safety and impact on modifying HD progression, while also tackling the anticipated high cost of the treatment.

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