QURE Jun 19, 2026QUREPhases
uniQure Announces Preliminary Data on the First Cohort in the Phase I/IIa Clinical Trial of AMT-260 in Refractory Mesial Temporal Lobe Epilepsy
uniQure has announced preliminary data from its Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy. The initial results show that three out of six patients experienced significant reductions in seizures, while the remaining patients had variable responses. The treatment was well-tolerated with no serious adverse events reported, supporting further evaluation of AMT-260.
Read more →
QURE Jun 17, 2026QUREFDA Updates
uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease
uniQure announced plans to submit a Biologics License Application (BLA) for AMT-130, a gene therapy for Huntington's disease, based on a 3-year analysis from its Phase I/II study. The FDA has indicated this data is acceptable for accelerated approval. The company aims to submit the BLA by Q3 2026 and is aligning on the confirmatory study design with the FDA.
Read more →
QURE May 5, 2026QUREFDA Updates
uniQure Announces First Quarter 2026 Financial Results and Provides Recent Company Updates
uniQure reported its Q1 2026 financial results, highlighting advancements in its gene therapy pipeline, particularly AMT-130 for Huntington's disease and AMT-191 for Fabry disease. The company has scheduled a Type B meeting with the FDA and expects to submit a Marketing Authorization Application in the UK. Despite a strong cash position, the company faces challenges with regulatory feedback and safety concerns in ongoing trials.
Read more →
QURE Apr 30, 2026QUREFDA Updates
uniQure Announces Successful UK MHRA Pre-Submission Meeting for AMT-130 in Huntington’s Disease
uniQure announced a successful Pre-Submission Meeting with the UK's MHRA regarding AMT-130 for Huntington's disease. The company plans to submit a Marketing Authorization Application in Q3 2026, supported by promising trial data showing a 75% slowing of disease progression. Additionally, a Type B meeting with the U.S. FDA is scheduled to discuss Phase III trial design.
Read more →
QURE Apr 28, 2026QUREConferences/Events
uniQure to Announce First Quarter 2026 Financial Results
uniQure N.V. will announce its first quarter 2026 financial results on May 5, 2026. The company will host a conference call at 8:30 a.m. ET to discuss these results. The event will be available via webcast, and a replay will be accessible for 90 days. Analysts can join the call by dialing in with a specific conference ID.
Read more →
QURE Mar 2, 2026QUREFDA Updates
uniQure Announces 2025 Financial Results and Provides Recent Company Updates
uniQure reported its 2025 financial results and highlighted advancements in its gene therapy programs. The company is evaluating Phase III development for AMT-130, which has shown significant efficacy in Huntington's disease. Additionally, AMT-260 and AMT-191 are progressing with promising early data. However, regulatory challenges remain, particularly with the FDA's recommendations for AMT-130.
Read more →
QURE Mar 2, 2026QUREFDA Updates
uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease
uniQure announced a regulatory update regarding AMT-130, its investigational gene therapy for Huntington's disease. The FDA indicated that the existing Phase I/II data is insufficient for a marketing application and recommended a new randomized study. uniQure plans to continue discussions with the FDA and request a Type B meeting to explore potential study designs.
Read more →
QURE Feb 23, 2026QUREConferences/Events
uniQure to Announce 2025 Financial Results
uniQure N.V. will announce its fourth quarter and full year 2025 financial results on March 2, 2026. Following the announcement, a conference call will be held at 8:00 a.m. ET, accessible via a live webcast. Analysts can participate in a Q&A session by calling in with a provided conference ID.
Read more →
QURE Feb 6, 2026QUREPhases
uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease
uniQure has released updated preliminary data from its Phase I/IIa trial of AMT-191 for Fabry disease, showing sustained increases in α-Gal A enzyme activity in all patients. Six out of eleven patients have successfully discontinued enzyme replacement therapy. The therapy demonstrated a manageable safety profile, although some patients experienced liver enzyme elevations, leading to a pause in higher dose cohorts for further assessment.
Read more →
QURE Jan 9, 2026QUREFDA Updates
uniQure Announces Type A Meeting Scheduled with FDA
uniQure has announced a Type A meeting with the FDA to discuss the Biologics License Application for AMT-130, a gene therapy aimed at treating Huntington's disease. The company emphasizes the urgent need for effective therapies in this area and is committed to working closely with the FDA. A regulatory update will follow after the meeting.
Read more →
QURE Dec 8, 2025QUREPhases
Data Published in the New England Journal of Medicine Confirm the Long-term Durability and Safety of HEMGENIX® (etranacogene dezaparvovec-drlb) Over Five Years
The five-year results from the Phase 3 HOPE-B study confirm the long-term durability and safety of HEMGENIX (etranacogene dezaparvovec-drlb) for adults with hemophilia B. Published in the NEJM, the data show sustained therapeutic benefits, including a 94% rate of freedom from prophylaxis treatment and strong factor IX activity levels. HEMGENIX remains the only gene therapy available for this condition.
Read more →
QURE Nov 10, 2025QUREFDA Updates
uniQure Announces Third Quarter 2025 Financial Results and Provides Corporate Update
uniQure reported its Q3 2025 financial results, highlighting significant progress in its clinical programs, particularly with AMT-130 for Huntington's disease, which met key endpoints. The company also received unexpected feedback from the FDA regarding its BLA submission, prompting a need for urgent discussions. Financially, uniQure raised $323.7 million, enhancing its cash reserves to $694.2 million.
Read more →
QURE Nov 6, 2025QUREConferences/Events
uniQure to Announce Third Quarter 2025 Financial Results
uniQure N.V. will announce its third quarter 2025 financial results on November 10, 2025, before market open. Following the announcement, a conference call will be held at 8:30 a.m. ET, which will also be available via webcast. Analysts can participate in the Q&A session by calling in with a passcode.
Read more →
QURE Sep 29, 2025QUREGeneral
uniQure Announces Closing of Upsized Public Offering and Full Exercise by Underwriters of Option to Purchase Additional Shares
uniQure N.V. has successfully closed an upsized public offering, selling 6,736,841 ordinary shares at $47.50 each, raising around $345 million. The offering included additional shares purchased by underwriters and pre-funded warrants. The funds will support commercialization readiness for AMT-130 and other clinical developments. The offering was managed by several financial institutions.
Read more →
QURE Sep 26, 2025QUREGeneral
uniQure Announces Pricing of Upsized $300 Million Public Offering
uniQure N.V. has announced the pricing of its public offering of 5,789,473 ordinary shares at $47.50 each, aiming to raise around $300 million. The offering is set to close on or about September 29, 2025, pending customary conditions. The company continues to advance its gene therapy pipeline for various severe diseases.
Read more →
QURE Sep 24, 2025QUREGeneral
uniQure Announces $200 Million Proposed Public Offering
uniQure N.V. has announced a proposed public offering of $200 million in ordinary shares and pre-funded warrants. The offering is underwritten and includes a 30-day option for underwriters to purchase additional shares. The completion of the offering is subject to market conditions, and the final terms will be disclosed in a prospectus supplement.
Read more →
QURE Sep 24, 2025QUREGeneral
uniQure Announces Refinancing of Existing $50 Million Debt and Securing Up to an Additional $125 Million in Non-Dilutive Funding
uniQure N.V. has announced a $175 million non-dilutive senior secured term loan facility with Hercules Capital. This refinancing extends the term of existing debt to October 2030 and provides access to additional funds to support the potential commercial launch of AMT-130 for Huntington's disease. The transaction aims to enhance the company's financial flexibility and reduce capital costs.
Read more →
QURE Sep 24, 2025QUREPhases
uniQure Announces Positive Topline Results from Pivotal Phase I/II Study of AMT-130 in Patients with Huntington’s Disease
uniQure announced positive topline results from its pivotal Phase I/II study of AMT-130 for Huntington's disease. The study met its primary endpoint, showing a significant 75% slowing of disease progression at 36 months. Additionally, AMT-130 demonstrated favorable trends in secondary endpoints and was well-tolerated. The company plans to submit a Biologics License Application in early 2026.
Read more →
QURE Sep 5, 2025QUREPhases
uniQure Announces Initial AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease
uniQure has announced promising initial data from its Phase I/IIa trial of AMT-191 for Fabry disease, showing significant increases in α-Gal A enzyme activity among all patients in the first cohort. All patients were able to discontinue enzyme replacement therapy, indicating the potential effectiveness of AMT-191. The treatment exhibited a manageable safety profile, with further results expected in 2026.
Read more →