Programs & Pipeline

Translating leading science into life-changing solutions.

We’re building a promising pipeline of potentially transformative programs focused on leveraging our platforms for maximum impact.

Huntington’s disease

Our AAV5 gene therapy platform shows promise for Huntington’s disease, a devastating and fatal neurodegenerative disorder, through uniQure’s proprietary miQURE™ gene-silencing technology.

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Hemophilia Type B

We are advancing our innovative gene therapy product candidate for hemophilia B, etranacogene dezaparvovec (AMT-061), to the next stage with a licensing agreement with global leader CSL Behring.

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Clinical trials

We continue to make progress in advancing AMT-130, our investigational gene therapy for Huntington’s disease currently in multiple-cohort Phase 1/2 clinical studies.

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Research programs

We are looking farther out on the horizon for new, life-changing applications of our expertise and technologies as we focus on advancing our first two potentially transformative gene therapies.

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Pipeline

We are executing an ambitious strategy to expand our pipeline and advance a new portfolio of next-generation gene therapies into clinical-stage development.

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Programs & Pipeline

Huntington’s disease

Hemophilia Type B

Clinical trials

Research programs

Pipeline

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