Our story
The world’s first approved gene therapy. The first commercially licensed gene therapy manufacturing facility. The first AAV-delivered gene silencing therapy for Huntington’s disease in clinical development. This is the story of a gene therapy pioneer with a history of firsts, accelerating into the future.
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Our team
Our executive team and board of directors are a distinguished group of highly experienced and talented leaders, working together to realize our vision to deliver potentially curative therapies that transform the lives of patients.
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Our partners
We have a long track record of collaborating with other industry leaders—international pharma companies, early-stage innovators, academic institutions and other research organizations—to make gene therapy a reality.
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