uniQure N.V. (QURE) Stock Price & How to Invest

uniQure N.V. is a Netherlands-based, Nasdaq-listed clinical-stage biotech developing adeno-associated virus (AAV) gene therapies for serious central-nervous-system and other genetic diseases. Its lead program is AMT-130, a one-time gene therapy for Huntington's disease, delivered surgically into the brain, that met its primary endpoint at three years with roughly 75% slowing of disease progression versus external controls in a Phase I/II study (an updated analysis put the 36-month slowing at about 80% on cUHDRS). The rest of the pipeline is earlier stage: AMT-162 for SOD1 ALS, AMT-260 for refractory mesial temporal lobe epilepsy, and AMT-191 for Fabry disease. The company already validated its platform once by developing Hemgenix, the hemophilia B gene therapy now commercialized by CSL Behring, which produces royalty income.

The investment picture is that of a single-asset biotech. Revenue is minimal (about $18.7 million over the trailing twelve months, mostly license and royalty income), and the company runs a large net loss, about $252 million TTM, funding R&D and commercial preparation. What matters is AMT-130. After a June 2026 Type B meeting at which the FDA agreed that 36-month data from 12 high-dose patients could support a BLA under the accelerated-approval pathway, uniQure submitted the BLA. On September 29, 2026 it then released 48-month data: slowing on the cUHDRS measure fell to about 44% versus an updated external control and was not statistically significant, while TFC showed about 61% slowing. The company argued missing data and survivor bias in the control understated the effect, and a post-hoc analysis against the earlier control showed about 54% and 68% slowing. Five high-dose patients (17%) had treatment-related serious adverse events tied to CNS inflammation, all resolved. The stock fell about 37% that day, Barclays downgraded it to Equal Weight, and RBC cut its target to $48, though UBS raised its target to $48 in mid-October. As of October 16, 2026 QURE closed near $22.07, down about 50% in a month, 5.6% year to date and 63% over one year, for a market cap near $1.5 billion. Because Huntington's has no approved disease-modifying therapy and decades of prior failures, a first approval could still be transformative, while a rejection or a demand for more data would remove most of the thesis. Yahoo Finance lists about $810 million of cash against about $552 million of debt as of June 30, 2026.

uniQure is a single-asset biotech, so any negative surprise on AMT-130, whether a Complete Response Letter, a request for a controlled confirmatory trial before approval, manufacturing questions, or safety concerns from an invasive brain-delivery procedure, could sharply reduce the company's value. The September 2026 four-year data already showed this: a non-significant 44% cUHDRS slowing at 48 months and serious inflammation-related adverse events in 17% of high-dose patients took about 37% off the stock in a day. The accelerated-approval pathway still requires alignment with the FDA on a confirmatory study, and there is precedent for the agency demanding more evidence in neurology. The rest of the pipeline is early and unproven, so it offers limited support if the lead program falters. As a pre-profit company, uniQure lost about $71 million in Q2 2026 alone, carries about $552 million of debt, and will likely need to raise capital again, which can dilute shareholders, and the stock is highly volatile and prone to large swings around clinical and regulatory news. Competition in Huntington's is also intensifying as rival mechanisms advance.

12 analysts publish price targets on QURE, averaging $61.37 against a $21.78 price as of October 2026, or +181.8%. The published targets run from $28.08 to $91.81, a wide spread, and the ratings split 11 buy, 2 hold, 0 sell. Over the last six months there have been 7 raises and 4 cuts among the published actions. A price target is what an analyst published on a date, not a prediction, and sell-side ratings skew positive across the whole market.

Read the full QURE forecast and price target for the target table, the recent rating actions by firm, and how the consensus has shifted.

We give no verdict on uniQure N.V.. Both cases are real, which is why the question is contested at all, so here is the strongest version of each.

The case for buying. AMT-130 accelerated-approval review. The FDA agreed that AMT-130's three-year Phase I/II data could serve as the primary basis for a BLA under the accelerated-approval pathway, and uniQure has now submitted it, before the four-year data were available. The most optimistic published target, $91.81, assumes this works close to its best case.

The case against. uniQure is a single-asset biotech, so any negative surprise on AMT-130, whether a Complete Response Letter, a request for a controlled confirmatory trial before approval, manufacturing questions, or safety concerns from an invasive brain-delivery procedure, could sharply reduce the company's value. The most pessimistic target, $28.08, is roughly what QURE is worth if this bites instead.

Read the full bull and bear case on QURE, including what would have to change to break either one. Walnut is not an investment adviser.

No. uniQure N.V. (QURE) has not split its stock in the last 10 years. That is a statement about the window we check rather than about the company’s entire history, so an older split is possible. It also matters less than it once did: fractional shares mean a high price per share no longer keeps smaller investors out, which removed most of the practical reason to split.

A simple financial snapshot. These are approximations and refresh quarterly; for current figures see uniQure N.V.'s investor relations page or your broker.

• Revenue (TTM): ~$18.7 million (Q2 2026 ~$5.1 million, mostly license and royalty)
• Net loss (TTM): ~$252 million (Q2 2026 ~$71 million, EPS ~-$1.07 vs ~-$0.71 expected)
• Cash vs debt: ~$810 million cash, ~$552 million debt (June 30, 2026)
• Share price: ~$22.07 (October 16, 2026; YTD ~-5.6%, 1 year ~-63%)
• Market cap: ~$1.5 billion
• Price-to-book: ~4.7x

uniQure is pre-profit, so traditional valuation multiples like P/E do not apply and the stock is valued on the probability-weighted potential of AMT-130, which the market sharply marked down after the September 2026 four-year data. Revenue is minimal, mostly Hemgenix royalties and license deals, while losses run near $250 million a year. Analysts' mean target was about $61 across 12 analysts in October 2026, far above the share price, a gap that shows how much depends on the FDA outcome.

Other names that sit close to QURE: same theme, named as a direct competitor, or held beside it in the same funds. Each entry says which. Worth a look if you are thinking about diversification within a thesis rather than concentration on one ticker.

There are three common ways to get QURE exposure. Buy shares (or fractional shares) directly at any major broker. Hold an ETF that includes it, which spreads the position across many companies. Or build it into a focused thematic portfolio, so QURE sits alongside other stocks that express the same thesis.

Walnut takes the portfolio route. Describe a thesis where QURE fits (for example “AI infrastructure” or “dividend-growth large-caps”) and the AI proposes 5 to 6 constituents with target weights. You review the plan and fund it through your own broker when you're ready.

New to this? Start with how to invest in stocks, see how to analyze a stock with AI, or compare the best AI stock analyzers.

uniQure is a binary, catalyst-driven gene-therapy stock whose price hinges far more on the FDA's review of its Huntington's therapy AMT-130 than on current revenue. After the September 2026 four-year data reset expectations, the outcome distribution is still unusually wide in both directions.

Whether QURE is worth buying today depends more on your time horizon and what you already hold than on any single call. We walk through valuation, what would have to go right, and the risks in is QURE a buy or a sell?, and where the stock could go from here in the QURE stock forecast.

For income investors, whether QURE pays a dividend and how the payout looks is covered in does QURE pay a dividend? And to weigh QURE against a peer, read the full side-by-side comparisons: QURE vs IONS and QURE vs ASO.