On November 2, 2026, uniQure announced that the FDA has accepted for review the BLA for ifezuntirgene inilparvovec, an investigational gene therapy for the treatment of Huntington’s disease submitted under the Accelerated Approval pathway. The application has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of May 1, 2027.

Ifezuntirgene inilparvovec (AMT-130) is an AAV-based gene therapy encoding a miRNA designed to lower huntingtin protein expression by targeting human HTT mRNA. In September 2025, uniQure revealed positive topline data from its pivotal Phase I/II study of AMT-130. The study met its prespecified primary and key secondary endpoints: a single administration of high-dose AMT-130 into the brain slowed disease progression by 75% at 36 months, while maintaining a consistently manageable safety profile.

The FDA’s acceptance of ifezuntirgene inilparvovec for Priority Review marks a significant milestone in the development of gene therapies for neurodegenerative diseases. As innovative genetic medicines continue to advance toward clinical and regulatory milestones, robust development and manufacturing capabilities will remain essential to translating scientific breakthroughs into accessible treatments.

As part of the broader gene therapy ecosystem, Genevoyager provides end-to-end CRO/CDMO services, from drug discovery to large-scale manufacturing, leveraging both HEK293 and our proprietary Bac/Sf9 AAV production platforms to produce high-quality AAV vectors with minimal impurities and no detectable rcAAV. With 30+ drug development projects and multiple IIT/IND submissions supported, Genevoyager has helped advance AAV-based gene therapies and recombinant protein vaccines into clinical development, including programs targeting Parkinson’s disease, refractory epilepsy, hepatitis B, and cancer, with selected programs receiving FDA IND clearance and entering Phase I trials. Backed by extensive experience, we are committed to delivering safe, high-quality solutions to academic, pharmaceutical, and biotech partners worldwide.

Source:

https://www.uniqure.com/investors-media/press-releases

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https://poshuk.info/en/gene-therapy-huntingtons-disease-amt-130-promising-modern-neuromedicine/