uniQure Stock Soars 196% on Huntington’s Disease Gene Therapy Potential Breakthrough – ( $QURE $IBB $XBI )

Published Sep 24, 2025
Biotech & Healthcare
Market News

By John F. Heerdink, Jr.

uniQure (NASDAQ: QURE) electrified Wall Street this week, with shares surging nearly 200% after the company reportedly announced transformative clinical data for its Huntington’s disease gene therapy, AMT-130. The biotech, which just secured up to $175 million in non-dilutive funding, is riding a wave of both clinical and financial milestones that have repositioned it as a leading contender in gene therapy innovation. Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Approximately 75,000 people have Huntington’s disease in the U.S.2, EU3, and the UK4, with hundreds of thousands of others at risk of inheriting the disease. Despite the clear etiology of Huntington’s disease, there are currently no approved therapies to delay the onset or to slow the disease’s progression.

Recent Press Releases and Developments

On September 24, 2025, uniQure announced the refinancing of $50 million in debt and access to an additional $125 million—funding that positions it to potentially launch AMT-130 without shareholder dilution. Results from the pivotal Phase I/II trial of AMT-130 showed a 75% disease progression slowing in high-dose Huntington’s patients over three years, and a significant 60% improvement in functional decline—data widely regarded as “groundbreaking” for the neurodegenerative disease secto.
Earlier in September, encouraging early clinical data for their Fabry disease gene therapy candidate, AMT-191, were highlighted, showing robust enzyme activity and the ability for patients to discontinue regular infusions, suggesting strong therapeutic potential.
In June 2025, the company made strategic leadership changes to support the commercialization of AMT-130 for Huntington’s disease.
Over the summer, regulatory progress was reported, including FDA alignment on statistical analysis plans and Chemistry, Manufacturing, and Controls (CMC) requirements—supporting a planned Biologics License Application submission in early 2026.
Additional clinical and regulatory updates were reported for the company’s pipeline therapies in epilepsy and ALS, with manufacturing improvements supporting broader clinical development.

Broader Market and Analyst Reactions

uniQure’s trial data has catapulted the company into the spotlight for 2025. Analysts have issued consensus “Buy” ratings with price targets in the mid-to-high $30s, and some calling for prices above $40 if commercialization hits the mark. uniQure remains pre-commercial, with revenue primarily driven by Hemgenix royalties, reporting $5.3 million in Q2 2025 sales and a net loss of $37.7 million. The company’s cash reserves appear sufficient for at least two years of operations.

Stock Performance: Today and Year-to-Date

On September 24, 2025, QURE shares soared 196%, trading at $39.59 and setting a new 52-week high after the Huntington’s data announcement. Year to date, QURE stock has more than doubled, outperforming most of its biotech peers and gaining well over 100% since January.

Outlook and Context

With AMT-130 likely heading for FDA submission in early 2026 and robust new funding supporting launch readiness, uniQure’s role in neurological gene therapy has evolved from speculative to potentially sector defining. Wall Street analysts and institutional investors are increasingly bullish, but emphasize the volatility inherent to biotech investing, where single catalysts can dramatically shift company trajectories.

The Sum…

This week’s events underscore not just the promise but also the risks of biotech investing, where groundbreaking clinical data can fuel a stock’s comeback and redefine market expectations almost overnight.