Approved Commercial Product: Hemgenix: uniQure has an FDA and EMA approved gene therapy, Hemgenix (etranacogene dezaparvovec), for hemophilia B, which is the world's first and only gene therapy for this condition, providing significant revenue potential and market validation for their platform.

Validated AAV Gene Therapy Platform: The company's proprietary AAV5 vector platform has been clinically validated through the approval of Hemgenix, demonstrating its efficacy and safety profile and providing a strong foundation for future pipeline candidates.

Integrated In-House Manufacturing Capabilities: uniQure possesses state-of-the-art in-house manufacturing facilities, which are critical for controlling the quality, cost, and supply chain of complex gene therapies from clinical development through commercialization.