uniQure’s Huntington’s gene therapy holds blockbuster potential, says analyst

Dutch biotech uniQure (Nasdaq: QURE) recently announced that its novel gene therapy, AMT-130(ifezuntirgene inilparvovec), demonstrated statistically-significant disease slowing in a Huntington’s disease (HD) pivotal trial. The results indicate that AMT-130 holds blockbuster potential in the disease space, according pharma analytics company GlobalData.

Pippa Salter, managing neurology analyst at GlobalData, commented: “In a market where the only available therapies are for the symptomatic management of chorea, the efficacy results of AMT-130 are unprecedented.

Key opinion leaders (KOLs) previously interviewed by GlobalData consistently highlighted the significant unmet need in the HD market for a therapy that can modify the disease, and slow or prevent progression, with AMT-103 poised to become the first treatment option for HD that meets this need giving it blockbuster potential.”