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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