uniQure (QURE) Corporate presentation summary

Leadership and innovation in gene therapy

• Over 25 years of pioneering gene therapy, including firsts in AAV delivery, gene silencing for Huntington's, and FDA designations.

• Validated AAV platform and commercial-ready manufacturing capabilities.

• Strong management team with expertise across scientific, medical, and operational domains.

Financial position and capital allocation

• $694.2 million in cash, cash equivalents, and investment securities as of September 30, 2025.

• Completed a $300 million public offering with $345 million gross proceeds in September 2025.

• Refinanced $50 million debt and secured up to $125 million in non-dilutive funding.

Clinical pipeline and program highlights

• Lead programs: AMT-130 (Huntington's), AMT-260 (MTLE), AMT-191 (Fabry), AMT-162 (ALS), and Hemophilia B (approved).

• Robust clinical pipeline with upcoming data readouts in MTLE and Fabry within 3-6 months.