Gene Therapy Co. Announces Initial Data for Refractory Epilepsy Treatment, Gets Buy Rating

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1. Ownership and Share Structure Information

The information listed above was updated on the date this article was published and was compiled from information from the company and various other data providers.

On June 19, 2026, uniQure N.V. (QURE:NASDAQ) announced initial six-month follow-up data for its first, low-dose cohort of six patients in its Phase 1 and Phase 2 trial of AMT-260, an experimental treatment for refractory mesial temporal love epilepsy (MTLE). Results showed three of the six patients achieved meaningful reductions in seizures during months four through six of the trial, ranging from a 79% to 100% reduction from baseline. The other half of the patients in the cohort experienced variable changes, ranging from a 33% to 36% decline in debilitating seizures. So far, the company has observed no serious adverse effects from the medication trial.

Walid Abi-Saab, MD and Chief Medical Officer or Uniqure, said in the release: "While patient responses have varied, we believe the data generated to date provide preliminary evidence of biological activity of AMT-260. Although these findings are based on a limited number of patients and require longer follow-up, the favorable tolerability profile and observed responses support continued evaluation of AMT-260."

The trial drug is intended to be a one-dose, in vivo gene therapy that will reduce or entirely eliminate seizures in those with MTLE.

Temporal lobe epilepsy is a chronic neurological disorder affecting approximately 500,000 people in the U.S., and around 80% of cases involve the medial structures of the brain. According to the company, roughly 300,000 of the 500,000 patients are inadequately treated with simple anti-seizure medications.

Uniquire is a gene therapy trial company dedicated to developing single treatments with potentially curative results. So far, the company has had success with its gene therapy for treating hemophilia B and is currently working on treatments for Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, and others.

R&D Spending Down Despite Need

Epilepsy treatment has shifted more toward personalized treatment in the past few years, leveraging molecular diagnostics and data analytics to tailor treatments to specific patients' needs. It's a huge market, with Europe alone expected to grow from US$1.34 billion in 2024 to US$2.82 billion by 2035.

In February 2026, Iqvia discussed the global pharma market projection for 2026, noting that total drug usage is expected to surpass four trillion doses daily by 2030. They wrote, "The largest drivers of medicine spending growth through the next five years will continue to be the use in developed markets of innovative therapeutics, especially in oncology, immunology, diabetes, and obesity."

Pharma sector funding fell between 2024 and 2025, according to a March 26, 2026, article for Fierce Biotech by Nick Paul Taylor. He wrote that pharma funding had fallen from 2024 but noted that, "2025 was still the third-best year of the past decade. Similarly, overall funding was well above the pre-pandemic norm and only topped by 2020, 2021, and 2024."

BCG talked about trends biopharma companies need to be aware of in 2026 in order to stay competitive, saying, "Near term, companies need to continue to innovate to decrease the complexity and cost of these therapies, and governments can find ways to incentivize and pay for them. The longer-term challenge for companies is to factor operational and economic considerations into R&D decision making earlier, ensuring that trial designs match real-world usage, indication sequences match opportunity, and endpoints enable market access."

"Buy Rating" Issued, Experts Anticipate New Data

Patrick Trucchio of H.C. Wainwright & Co. released an updated research report on Uniqure on June 17, 2026, reiterating a "Buy" rating, with a price target of US$50.00. Trucchio noted, looking forward to further data on multiple drugs in the company's pipeline.

Chen Lin of What is Chen Buying? What is Chen Selling? commented on the company's news on June 23, 2026, saying: "Biotech is rallying thanks to more positive news from the FDA. For the first time, HHS(FDA) is rolling out sweeping reforms to help biotech to compete with China. As we know, the ADC and many areas in biotech in the U.S. are wiped out by China. For example, KYNB is trading way below cash, yet nobody cares. Investors believe it will be another victim crashed by the Chinese competitors. I do think it is very good news for small biotechs, many of which I own. This plus the recent news that FDA is getting easier for new drug approval, examples like OTLK and QURE . . . All these are setting up a powerful rally of biotechs this summer . . . We are not the COVID high yet, but it does look like we are going to retest it."

A Multi-Drug Pipeline in Development

Trucchio listed near-term catalysts as receiving final Type B meeting minutes from the company's new AMT-130 Huntington's drug, confirmatory study alignment, and a Q3 2026 BLA submission, saying, "We expect investors to focus on whether final minutes confirm the acceptability of the 3-year analysis as primary BLA evidence, whether a concurrent SoC control can replace sham in the confirmatory study, which endpoint will anchor the confirmatory trial, and whether FDA indicates any additional requirements around safety, chemistry, manufacturing, and controls (CMC), or long-term follow-up." 

Uniqure's website lists current drugs in its pipeline as:

• AMT-061 (approved) for the treatment of Hemophilia B
• AMT-130 for the treatment of Huntington's Disease
• AMT-191 for the treatment of Fabry Disease
• AMT-260 for the treatment of Temporal Lobe Epilepsy
• Other undisclosed programs in development

Ownership & Share Information¹

Uniqure NV has a market cap of US$3.08 billion, with 63.07 million shares outstanding. The company's 52-week range is US$8.73-US$71.50. Institutions own 89.45% of shares, with 3.51% of shares of shares belonging to Strategic Investors.

Management & Insiders own 1.32% of shares, and the remaining 5.72% of shares are held by Retail.