uniQure’s AMT-130 Gene Therapy for Huntington’s Disease on Track for Q1 2026 BLA Submission After FDA Alignment

uniQure (NASDAQ:QURE) is one of the biotech stocks to buy according to Wall Street analysts. Earlier on June 2, uniQure provided a regulatory update on AMT-130, which is the company’s investigational gene therapy for Huntington’s disease, following recent Type B meetings and guidance from the US FDA.

The company has reached alignment with the FDA on key aspects of the statistical analysis plan/SAP and Chemistry, Manufacturing and Controls/CMC information, paving the way for a Biologics License Application/BLA submission, which is now expected in Q1 2026. This accelerated approval pathway is supported by multiple years of clinical data.

uniQure's AMT-130 Gene Therapy for Huntington's Disease on Track for Q1 2026 BLA Submission After FDA Alignment

A surgeon wearing scrubs with a stack of pharmaceuticals in the background.

Key upcoming milestones for the BLA submission include: submitting the updated SAP to the FDA in Q2 2025, initiating the PPQ run and presenting topline Phase I/II data per the SAP in Q3 2025, and holding a pre-BLA meeting with the FDA in Q4 2025.

uniQure (NASDAQ:QURE) develops treatments for patients suffering from rare and other devastating diseases in the US.

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Disclosure: None. This article is originally published at Insider Monkey.