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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Temporal Lobe Epilepsy (TLE)
We are excited to begin the clinical investigation of AMT-260, our one-time administered gene therapy for refractory mesial temporal lobe epilepsy.
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Fabry disease
AMT-191 represents a novel potential one-time administered approach to treating Fabry disease.
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ALS (SOD1)
The clinical development of AMT-162 is based on nearly 30 years of research demonstrating the link between the SOD1 gene mutation and ALS.
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Hemophilia Type B
In 2022 our gene therapy for hemophilia B became the world’s first approved gene therapy for hemophilia.
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Clinical trials
We continue to make progress in advancing AMT-130 for Huntington’s disease, currently in multiple-cohort Phase 1/2 clinical studies, and AMT-260 for refractory mesial temporal lobe epilepsy.
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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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