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November 4, 2026

FDA to review uniQure’s BLA for Huntington’s gene therapy

The US Food and Drug Administration (FDA) has accepted a biologics licence application (BLA) under its accelerated approval pathway from uniQure for ifezuntirgene inilparvovec, an investigational gene therapy to treat Huntington’s disease.

Huntington’s disease is an inherited disorder that causes motor symptoms, including chorea, behavioural changes, and progressive cognitive decline. Credit: H_Ko / Shutterstock.com.