Model Update

Research evidence excerpt

Model Update

July 1, 2026

Valuation

We use a probability-adjusted DCF analysis to value QURE shares. We model cash flows

out to 2035E. We assume a discount rate of 15% and a terminal value with a 2% long-

term growth rate. The total NPV is ~$4.4B, based on our analysis, and adding cash leads

us to our 12-month price target of $61/share. Our calculation includes estimated cash and

shares outstanding as of end-1Q27E.

Risks

uniQure is a clinical-stage company, and investment is subject to risk. These risks include,

but are not limited to:

Clinical trial risk: uniQure is a clinical-stage company and has one commercial product

marketed by CSL Behring (following Glybera being taken off the shelves in October

2017). uniQure is focusing on rare diseases, and any delays in patient recruitment or

enrollment can push back our sales estimates significantly. Early gene therapy trials

have reported serious adverse events and deaths. The key challenges for gene therapy

are the delivery and maintenance of the corrected genetic information without causing

unintended consequences (off-target activity).

Regulatory risk: Gene therapy is intended to provide a permanent correction for a

genetic error. Conceptually, and because of early safety observations, there is heightened

regulatory scrutiny over development and post-market surveillance. The regulatory

framework exists and literally hundreds of new gene-based trials are allowed to be

conducted annually. Although the unique nature of the therapy does not seem to slow

development, there could be unexpected observations that could lead to additional

regulatory hurdles. To-date, there have been few AAV-vector-based gene therapies

approved in the United States.