~ Data follow the historic approval of the first gene therapy for hemophilia B, which has been shown in clinical trials to reduce the rate of annual bleeds, reduce or eliminate the need for prophylactic
~ Historic approval represents the first gene therapy in Europe to treat hemophilia B and provides a new treatment option for patients that reduces the rate of annual bleeds, reduces or eliminates the
~ License of APB-102 further strengthens uniQure's pipeline of innovative gene therapies to treat neurological disorders and miRNA-based gene silencing programs ~ ~ APB-102 and uniQure's c9orf72-ALS
~ IND Submission and Clinical Development Expected to Begin in 2023 ~ ~ Advancements in Manufacturing and Technology Platform Also Highlighted ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands