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