First-in-class gene therapy for orphan indications
UniQure’s lead programme, Glybera® for the treatment of lipoprotein lipase deficiency obtained approval in 2012. Glybera® was the first gene therapy product approved by the EMA for any indication. UniQure has now designed and validated a scalable AAV manufacturing platform.
UniQure has partnered Glybera® with Chiesi, and is developing a proprietary clinical pipeline of gene therapy products for a range of orphan indications.
CEO Matt Kapusta