uniQure Schedules FDA Meeting for AMT-130 Accelerated Approval Discussion
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Jan 09 2026
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Source: NASDAQ.COM
- FDA Meeting Scheduled: uniQure N.V. announced a Type A meeting with the FDA to discuss the Biologics License Application (BLA) data package for AMT-130, highlighting the company's significant role in addressing the urgent need for Huntington's disease treatments.
- Therapeutic Potential: AMT-130, uniQure's lead candidate, utilizes a one-time adeno-associated virus gene therapy designed to deliver microRNA sequences directly into the brain to silence the huntingtin gene, with early clinical data suggesting its potential to lower mutant huntingtin protein levels.
- Breakthrough Therapy Designation: The FDA granted AMT-130 Breakthrough Therapy designation in April 2025 and Regenerative Medicine Advanced Therapy (RMAT) designation in May 2024, indicating the therapy's potential to meet serious unmet medical needs in the Huntington's disease space.
- Strong Financial Position: As per the latest quarterly update, uniQure reported approximately $694.2 million in cash, cash equivalents, and investments, ensuring financial stability as it advances the development of AMT-130.
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Analyst Views on QURE
Wall Street analysts forecast QURE stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for QURE is 49.88 USD with a low forecast of 33.00 USD and a high forecast of 70.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
10 Analyst Rating
8 Buy
2 Hold
0 Sell
Strong Buy
Current: 23.020
Low
33.00
Averages
49.88
High
70.00
Current: 23.020
Low
33.00
Averages
49.88
High
70.00
About QURE
Uniqure NV is a company based in the Netherlands specialized in gene therapy. It seeks to develop one-time administered treatments with potentially curative results for patients suffering from genetic and other devastating diseases. It develops, both internally and through partnerships, a pipeline of gene therapies. It produces adeno-associated virus based, or AAV-based, gene therapies in its own facilities with a proprietary, commercial-scale, current good manufacturing practices, compliant, manufacturing process. AMT-061, the Company’s lead product candidate for patients with hemophilia B, is going through a dosing phase of a pivotal study. AMT-130, the product candidate for patients with Huntington’s disease is in Phase I/II clinical study.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Analysis of QURE's 52-Week High and Low Points
- Price Range Analysis: QURE's 52-week low is $7.76 per share, while the high is $71.50, with the last trade at $23.88, indicating significant volatility in its stock price over the past year, reflecting varying market expectations for its future performance.
- Technical Indicator Focus: The current stock price is below the 200-day moving average, which may suggest increased selling pressure in the short term, prompting investors to monitor subsequent trends to assess potential investment risks.
- Market Sentiment Insight: Although the current stock price is significantly lower than the 52-week high, it remains above the low, indicating that the market still holds some confidence in QURE's fundamentals, potentially attracting investors looking for entry points at lower prices.
- Investor Strategy Adjustment: Investors should consider QURE's price volatility and technical indicators, along with market sentiment, to adjust their strategies in response to potential market changes.
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uniQure (QURE) Stock Plummets 50% After FDA Rejects BLA Submission for AMT-130
- FDA Notification Impact: On November 3, 2025, uniQure disclosed that the FDA indicated its clinical data for AMT-130, a gene therapy for Huntington's disease, was insufficient to support the Biologics License Application (BLA), leading to significant regulatory challenges for the company.
- Stock Price Volatility: Following this announcement, uniQure's stock plummeted over 50%, from $67.69 on October 31, 2025, to $34.29, reflecting extreme market pessimism regarding the company's future prospects.
- Investor Scrutiny: Kessler Topaz Meltzer & Check LLP is investigating potential violations of federal securities laws by uniQure, indicating heightened investor concerns about the company's compliance and transparency.
- Future Uncertainty: uniQure stated that the timing for the BLA submission for AMT-130 is now unclear, which could impact the company's fundraising and R&D efforts, posing a threat to its long-term strategic objectives.
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