QURE Share Price Performance

US$44.51

29.67 (200.03%)

US$69.38

Fair Value

US$44.51

29.67 (200.03%)

35.8% undervalued

US$69.38

Fair Value

US$44.51

US$69.38

US$47.05

US$90.71

Thu, 13 Aug 26

Fair ValueUS$69.38

Share Pricen/a

QURE Community Narratives

AnalystConsensusTarget's

Fair Value

Fair Value US$69.38 35.8% undervalued

QURE: Pivotal Three-Year Huntington’s Data Will Drive Approval and Market Uptake

AnalystLowTarget's

Fair Value

Fair Value US$47.05 5.4% undervalued

Tighter Gene Therapy Regulations And Safety Risks Will Challenge Neurology Pipeline Yet Offer Long-Term Promise

AnalystHighTarget's

Fair Value

Fair Value US$90.71 50.9% undervalued

Huntington’s Gene Therapy And Rare Disease Pipeline Will Reshape Long Term Outcomes

About QURE

Founded: 1998
Employees: 221
CEO: Matthew Kapusta
WebsiteView website: www.uniqure.com

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in phase I/II clinical study for the treatment of Huntington’s disease. The company also develops AMT-260, which is in phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

Recent QURE News & Updates

Announcement • Sep 03

Uniqure Submits Biologics License Application for Ifezuntirgene Inilparvovec (Amt-130) in Huntington’S Disease

uniQure N.V. has submitted a Biologics License Application (BLA) to the United States Food and Drug Administration for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for the treatment of Huntington’s disease. The Company also announced that its Marketing Authorisation Application (MAA) for ifezuntirgene inilparvovec has been submitted to the United Kingdom’s Medicines and Healthcare products Regulatory Agency. The Company has requested priority review for the BLA. If granted, priority review would shorten the FDA review cycle to six months following the FDA’s 60-day BLA filing review period. The BLA and MAA are supported by the previously announced three-year data analysis from the Phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database. The Company intends to present a four-year data analysis from the ongoing Phase I/II clinical studies before the end of the current third quarter. Ifezuntirgene inilparvovec is the first investigational therapy for Huntington’s disease to have received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Ifezuntirgene inilparvovec also holds Fast Track designation from the FDA. Ifezuntirgene inilparvovec is a novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes a proprietary, gene-silencing miQURE platform and incorporates a miRNA, specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment. Treated patients receive a single administration through targeted, MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). uniQure is conducting two multi-center Phase I/II clinical studies evaluating the safety, tolerability, and efficacy of ifezuntirgene inilparvovec for the treatment of Huntington’s disease. The U.S. randomized study enrolled 26 patients who received either a single administration of ifezuntirgene inilparvovec (n=6 low dose; n=10 high dose) or a sham procedure (n=10); four control patients subsequently crossed over to treatment after approximately 12 months. The European open-label study enrolled 13 patients (n=6 low dose; n=7 high dose). A third cohort of 12 patients explored both doses in combination with immunosuppression, and a fourth cohort of six U.S. patients is evaluating the high dose in patients with lower striatal volumes compared to those of patients enrolled in previous cohorts. Additional details are available on clinicaltrials.gov (NCT05243017, NCT04120493). 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., EU, and the UK, 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 updates

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uniQure N.V. Stock Price

QURE Share Price Performance

QURE Community Narratives

QURE: Pivotal Three-Year Huntington’s Data Will Drive Approval and Market Uptake

Tighter Gene Therapy Regulations And Safety Risks Will Challenge Neurology Pipeline Yet Offer Long-Term Promise

Huntington’s Gene Therapy And Rare Disease Pipeline Will Reshape Long Term Outcomes

Tighter Gene Therapy Regulations And Safety Risks Will Challenge Neurology Pipeline Yet Offer Long-Term Promise

QURE: Pivotal Three-Year Huntington’s Data Will Drive Approval and Market Uptake

Huntington’s Gene Therapy And Rare Disease Pipeline Will Reshape Long Term Outcomes

Trending Discussion

Recently Updated Narratives

QURE: Accelerated Huntington Filing Plans Balance Single Program And Execution Risks

QURE: Accelerated Approval Path And 2026 BLA Filing Will Drive Upside

Huntington’s Gene Therapy And Rare Disease Pipeline Will Reshape Long Term Outcomes

Snowflake Analysis

uniQure N.V. Key Details

About QURE

Recent QURE News & Updates

uniQure Seeks Accelerated US Approval for Huntington’s Gene Therapy After Phase I/II Data

Uniqure Submits Biologics License Application for Ifezuntirgene Inilparvovec (Amt-130) in Huntington’S Disease

QURE: Accelerated Huntington Filing Plans Balance Single Program And Execution Risks

Recent updates

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