uniQure (QURE) AI Stock Analysis

• Market Cap: $1.38B
• Dividend Yield: N/A
• Average Volume (3M): 2.58M
• Price to Earnings (P/E): ―
• Beta (1Y): 1.13
• Revenue Growth: -44.90%
• EPS Growth: 11.49%
• Country: US
• Employees: 209
• Sector: Healthcare
• Sector Strength: 45
• Industry: Biotechnology
• EPS (TTM): -1.38
• Shares Outstanding: 61,603,770
• 10 Day Avg. Volume: 1,784,213
• 30 Day Avg. Volume: 2,579,206
• PEG Ratio: 0.15
• Price to Book (P/B): -127.24
• Price to Sales (P/S): 31.68
• P/FCF Ratio: -4.62
• Enterprise Value/Market Cap: 2.10
• Enterprise Value/Revenue: 183.14
• Enterprise Value/Gross Profit: 207.82
• Enterprise Value/Ebitda: -19.72
• 1Y Price Target: $50.44
• Price Target Upside: 125.68% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 11
• EPS Forecast (FY): -3.21
• Revenue Forecast (FY): $51.88M

• Rating: 46 Neutral
• Price Target: $22.50 ▲(0.67% Upside)

The score is held down mainly by weak financial performance (ongoing losses and negative cash flow) and bearish technical trends. This is partially offset by a stronger earnings-call backdrop driven by meaningful clinical progress and a sizable cash position, though regulatory uncertainty around the AMT-130 BLA pathway remains a significant risk.

Positive Factors

Proprietary AAV platform and diversified pipeline

uniQure's proprietary AAV platform and an approved hemophilia B therapy underpin durable competitive advantages. A diversified pipeline (AMT-130, AMT-260, AMT-191) across neurological and metabolic rare diseases increases the odds that one program attains long-term commercialization and partnership value.

Strong liquidity position

A $649.2M cash and investment base provides meaningful runway to fund ongoing pivotal work and multiple near-term readouts without immediate dilution. This liquidity supports continued R&D, regulatory engagement and strategic optionality while management addresses FDA feedback and advances programs.

High gross margins indicate scalable economics

An 88% gross margin suggests favorable product economics and scalable manufacturing once commercialized. Combined with initial revenue growth and an existing commercial product, this supports the potential for operating leverage over time if R&D spend stabilizes and commercialization expands.

Negative Factors

Regulatory uncertainty on AMT-130 BLA

The FDA's view that Phase 1/2 data may be inadequate for a BLA creates structural timeline and evidentiary risk for AMT-130. This can require additional trials or data, postpone potential approval and revenue, and increase development costs, materially affecting the program's long-term commercial trajectory.

Persistent negative cash flow and profitability strain

Ongoing negative operating and free cash flow and deeply negative ROE indicate the company is burning cash despite strong gross margins. Over the medium term, sustained losses risk future dilution if pipeline programs don't generate revenue or partnerships, constraining reinvestment and strategic flexibility.

Clinical safety setback: AMT-162 paused

The AMT-162 pause for a dose-limiting toxicity is a material safety signal that may require additional studies, altered dosing or program discontinuation. Safety issues can delay timelines, increase costs and raise platform-level risk that affects investor, regulator and partner confidence in gene therapy programs.

uniQure Business Overview & Revenue Model

Company Description

uniQure N.V., a gene therapy company, engages in the development of treatments for patients suffering from genetic and other devastating diseases. Its lead program is Etranacogene dezaparvovec (AMT-061), which is in Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also engages in developing AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease; AMT-060, which is in Phase I/II clinical trial for the treatment of hemophilia B; AMT-210, a product candidate for the treatment of Parkinson's disease; AMT-260 for temporal lobe epilepsy; AMT-240, a preclinical product candidate for the treatment of autosomal dominant Alzheimer's disease; and AMT-161 for the treatment of amyotrophic lateral sclerosis. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

How the Company Makes Money

uniQure generates revenue primarily through the sale of its gene therapy products, with significant income derived from its approved treatment for hemophilia B. The company also earns revenue through partnerships and collaborations with other biopharmaceutical companies, which may involve upfront payments, milestone payments based on clinical and regulatory achievements, and royalties on sales of partnered products. Additionally, uniQure may receive funding from government grants or research initiatives aimed at advancing gene therapy technologies, which contribute to its overall financial performance.

uniQure Financial Statement Overview

Summary

uniQure faces significant financial challenges, particularly in achieving profitability and positive cash flow. Despite a strong gross profit margin, high operating expenses and negative net profit margins indicate struggles with profitability. The balance sheet shows some improvement in equity, but negative cash flow and return on equity remain concerns.

Income Statement

uniQure's income statement shows significant challenges with profitability, as evidenced by negative net profit margins and EBIT margins over the TTM period. Despite a modest revenue growth rate of 9.86% in the TTM, the company has struggled to convert this into profit, with a net profit margin of -14.93%. The gross profit margin is relatively strong at 88.13%, indicating effective cost management at the gross level, but high operating expenses are impacting overall profitability.

Balance Sheet

The balance sheet reflects a moderate debt-to-equity ratio of 0.29, suggesting manageable leverage levels. However, the return on equity is significantly negative at -373.73%, indicating that the company is not generating returns on shareholder equity. The equity ratio is not directly provided, but the company's equity position has improved from negative to positive, indicating some recovery in financial stability.

Cash Flow

Cash flow analysis reveals challenges with negative operating cash flow and free cash flow in the TTM. The free cash flow to net income ratio is slightly above 1, indicating that cash flow is somewhat aligned with net income, albeit both are negative. The free cash flow growth rate is negative, suggesting declining cash generation capabilities.

Breakdown	TTM	Dec 2024	Dec 2023	Dec 2022	Dec 2021	Dec 2020
Income Statement
Total Revenue	15.75M	27.12M	15.84M	106.48M	524.00M	37.51M
Gross Profit	13.88M	8.79M	2.21M	103.14M	499.03M	-84.89M
EBITDA	-146.30M	-160.75M	-253.10M	-108.02M	319.00M	-126.97M
Net Income	-235.15M	-239.56M	-308.48M	-126.79M	329.59M	-125.02M
Balance Sheet
Total Assets	888.38M	556.54M	831.69M	704.96M	809.18M	340.39M
Cash, Cash Equivalents and Short-Term Investments	694.25M	367.52M	617.89M	352.84M	556.26M	244.93M
Total Debt	66.16M	66.06M	138.41M	142.89M	135.72M	71.54M
Total Liabilities	659.64M	563.29M	624.02M	228.96M	213.40M	96.49M
Stockholders Equity	228.75M	-6.75M	207.67M	476.01M	595.78M	243.91M
Cash Flow
Free Cash Flow	-153.62M	-186.10M	-153.08M	-162.75M	270.52M	-144.31M
Operating Cash Flow	-153.24M	-182.73M	-145.93M	-145.06M	287.96M	-134.83M
Investing Cash Flow	93.13M	162.97M	-205.69M	-182.73M	-67.39M	-9.48M
Financing Cash Flow	406.43M	-59.49M	362.72M	1.45M	94.86M	7.44M

uniQure Technical Analysis

• Technical Analysis Sentiment: Negative
• Last Price: 22.35
• 50DMA: Negative
• 100DMA: Negative
• 200DMA: Negative
• MACD: Negative
• RSI: Neutral
• STOCH: Positive

Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For QURE, the sentiment is Negative. The current price of 22.35 is below the 20-day moving average (MA) of 23.56, below the 50-day MA of 25.01, and below the 200-day MA of 23.47, indicating a bearish trend. The MACD of -1.15 indicates Negative momentum. The RSI at 41.22 is Neutral, neither overbought nor oversold. The STOCH value of 10.14 is Positive, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for QURE.

uniQure Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
IMNM	58 Neutral	$2.29B	-7.73	-93.04%	―	-4.44%	63.58%
PHVS	56 Neutral	$1.64B	-7.97	-51.81%	―	―	-20.08%
AMLX	53 Neutral	$1.55B	―	-56.52%	―	-100.34%	52.85%
BCRX	52 Neutral	$1.43B	-151.59	―	―	45.38%	92.85%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
AGIO	49 Neutral	$1.68B	-4.00	-27.57%	―	36.26%	-158.98%
QURE	46 Neutral	$1.38B	-5.01	-165.27%	―	-44.90%	11.49%

uniQure Corporate Events

uniQure updates gene therapy pipeline and regulatory outlook

In January 2026, uniQure N.V. updated its corporate presentation to highlight its positioning as a leader in gene therapy, underscoring a robust clinical pipeline and substantial liquidity to fund development across multiple rare and serious neurological and metabolic diseases. The refreshed materials emphasize AMT-130 as a first potential disease-modifying, one-time gene therapy for Huntington’s disease with so-called blockbuster potential, outline upcoming data readouts for AMT-260 in mesial temporal lobe epilepsy and AMT-191 in Fabry disease over the next three to six months, and signal focused engagement with the U.S. Food and Drug Administration on a potential biologics license application pathway, developments that collectively underscore the company’s bid to consolidate its competitive position in gene therapy and may be closely watched by investors, patients and partners for signs of clinical and regulatory progress.

The most recent analyst rating on (QURE) stock is a Hold with a $22.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.

uniQure prepares for FDA Type A meeting on AMT-130

On January 9, 2026, uniQure announced that the U.S. Food and Drug Administration has scheduled a Type A meeting with the company to discuss the Biologics License Application data package supporting a potential accelerated approval of AMT-130, its investigational gene therapy for Huntington’s disease. Management highlighted the significant unmet medical need in the Huntington’s disease community and its commitment to timely patient access, signaling that a favorable regulatory outcome could be a key milestone for uniQure’s Huntington’s program and broader gene therapy pipeline; the company plans to issue a regulatory update after it receives the FDA’s official meeting minutes.

The most recent analyst rating on (QURE) stock is a Buy with a $60.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.

uniQure Faces Setback in Gene Therapy Approval

On December 4, 2025, uniQure announced a regulatory update on its investigational gene therapy, AMT-130, for Huntington’s disease. Following a pre-Biologics License Application meeting with the FDA on October 29, 2025, the agency indicated that data from Phase I/II studies are unlikely to support a BLA submission. uniQure plans to urgently request a follow-up meeting with the FDA in early 2026 to address this feedback and advance the therapy for patients.

The most recent analyst rating on (QURE) stock is a Hold with a $27.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.

uniQure Faces FDA Setback on Huntington’s Therapy

On November 3, 2025, uniQure announced a regulatory update regarding its investigational gene therapy for Huntington’s disease, AMT-130, following a pre-Biologics License Application meeting with the FDA. The company revealed that the FDA no longer agrees that data from Phase I/II studies of AMT-130 in comparison to an external control may be adequate for a BLA submission, creating uncertainty around the timing of the submission. Despite this setback, uniQure is committed to working with the FDA and other regulatory bodies to find a path forward for AMT-130’s approval, emphasizing the therapy’s potential benefits for patients with Huntington’s disease.

The most recent analyst rating on (QURE) stock is a Hold with a $67.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.