uniQure AMT-130 Gene Therapy Advances Toward UK Approval for Huntington's Disease Treatment

Key Takeaways

• uniQure successfully completed UK MHRA pre-submission meeting for AMT-130 gene therapy in Huntington’s disease
• Company plans to submit UK Marketing Authorization Application in Q3 2026, potentially bringing first gene therapy for Huntington’s disease to market
• FDA Type B meeting granted for Q2 to discuss Phase III trial design based on four-year AMT-130 data

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uniQure Progresses AMT-130 Gene Therapy Toward European Approval

uniQure N.V. (NASDAQ: QURE) announced April 30, 2026, that it successfully completed a pre-submission meeting with the UK Medicines and Healthcare products Regulatory Agency (MHRA) for AMT-130, its investigational gene therapy for Huntington’s disease. The biotechnology company expects to submit a UK Marketing Authorization Application in the third quarter of 2026.

Regulatory Momentum Building for Huntington’s Gene Therapy

The successful MHRA meeting represents a critical milestone for AMT-130, which could become the first approved gene therapy for Huntington’s disease. This rare, inherited neurodegenerative disorder affects approximately 30,000 people in the United States and has no current cure or disease-modifying treatments.

Concurrently, uniQure has secured a Type B meeting with the U.S. Food and Drug Administration (FDA) scheduled for the second quarter of 2026. This meeting will focus on discussing potential Phase III trial design and analysis plans based on AMT-130’s four-year clinical data.

Global Market Strategy Takes Shape

The company is actively pursuing additional regulatory pathways outside the United States to support international registration of AMT-130. This multi-regional approach could accelerate patient access to the therapy across key markets.

AMT-130 uses adeno-associated virus (AAV) gene therapy technology to deliver therapeutic genes directly to brain cells affected by Huntington’s disease. The treatment aims to reduce production of the mutant huntingtin protein that causes progressive neurodegeneration.

Market Impact and Investment Implications

The Huntington’s disease therapeutics market represents a significant unmet medical need, with current treatments only addressing symptoms rather than disease progression. Successful approval of AMT-130 could establish uniQure as a leader in neurological gene therapies and validate the company’s AAV platform technology for other central nervous system disorders.

The regulatory progress comes as gene therapy approvals have accelerated globally, with regulators increasingly receptive to innovative treatments for rare diseases with limited therapeutic options.

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Frequently Asked Questions

What does this regulatory progress mean for Huntington’s disease patients?

This brings AMT-130 closer to becoming the first gene therapy approved for Huntington’s disease, potentially offering patients a treatment that addresses the underlying cause rather than just symptoms.

When will AMT-130 be available to patients?

uniQure plans to submit for UK approval in Q3 2026. If approved, the therapy could be available in the UK by 2027, with U.S. approval potentially following after Phase III trials.

How does AMT-130 differ from current Huntington’s treatments?

Unlike existing treatments that only manage symptoms, AMT-130 is designed as a one-time gene therapy that could modify disease progression by reducing production of the toxic huntingtin protein.