Welcome to our dedicated page for Uniqure news (Ticker: QURE), a resource for investors and traders seeking the latest updates and insights on Uniqure stock.
uniQure N.V. (NASDAQ: QURE) is a gene therapy company that regularly issues news on its clinical programs, regulatory interactions, and financing activities. The company describes itself as delivering single‑treatment gene therapies with potentially curative results and has reported that the approvals of its gene therapy for hemophilia B mark a major milestone in genomic medicine. News about uniQure often highlights progress in its pipeline for Huntington’s disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe conditions.
A major focus of QURE news coverage is AMT‑130, uniQure’s investigational gene therapy for Huntington’s disease. Press releases and related SEC filings detail pivotal Phase I/II trial results, including statistically significant slowing of disease progression at 36 months in high‑dose cohorts compared to a propensity score‑matched external control, as well as trends in motor and cognitive endpoints and biomarker changes. Investors also see updates on the company’s regulatory dialogue with the U.S. Food and Drug Administration, including pre‑BLA and Type A meetings and feedback on the adequacy of Phase I/II data for a potential Biologics License Application.
Beyond AMT‑130, uniQure news includes early‑stage data and enrollment updates for AMT‑260 in refractory mesial temporal lobe epilepsy, AMT‑191 in Fabry disease, and AMT‑162 in SOD1 ALS. Corporate news items cover public offerings of ordinary shares and pre‑funded warrants, amendments to loan facilities, and quarterly financial results. This QURE news page aggregates such disclosures so readers can follow clinical milestones, regulatory developments, and capital markets events that shape uniQure’s gene therapy business over time.
uniQure (NASDAQ: QURE) reported 2025 results and program updates on March 2, 2026. Key highlights include $622.5 million in cash and investments as of Dec 31, 2025, expected to fund operations into H2 2029, and $16.1 million revenue for 2025 with a $199.0 million net loss.
Clinical updates: AMT-130 showed a 75% slowing on cUHDRS at 36 months but FDA recommended a prospective randomized, sham-controlled study; AMT-260 completed first cohort enrollment; AMT-191 showed durable, dose-dependent α-Gal A increases with dosing pauses for asymptomatic Grade 3 liver enzyme elevations; AMT-162 remains on voluntary pause.
uniQure (NASDAQ: QURE) received final FDA minutes from a Jan 30, 2026 Type A meeting about AMT-130 for Huntington’s disease. The FDA said Phase I/II data versus an external control are not sufficient to support a marketing application and strongly recommended a prospective, randomized, double-blind, sham surgery-controlled study.
uniQure plans further engagement and intends to request a Type B meeting in Q2 2026 to discuss Phase III design options.
uniQure (NASDAQ: QURE) will report fourth-quarter and full-year 2025 financial results before market open on Monday, March 2, 2026. Management will host a live conference call at 8:00 a.m. ET the same day.
The event will be webcast via the company’s Events & Presentations page and a replay will be archived for 90 days. Analysts may join the live Q&A by phone using conference ID 4607289; attendees are asked to dial in 15 minutes early.
uniQure (NASDAQ: QURE) reported updated preliminary Phase I/IIa AMT-191 data in 11 Fabry disease patients showing dose-dependent, sustained elevations in α-Gal A activity across three dose cohorts through a cutoff of Jan 8, 2026.
α-Gal A increases ranged from 0.34- to 312.52-fold versus mean normal by dose; six of 11 patients stopped enzyme replacement therapy, and plasma lyso-Gb3 remained stable. Safety showed manageable events, with protocol pause of additional mid/high dosing after two Grade 3 liver enzyme elevations at the mid dose.
uniQure (NASDAQ: QURE) announced a Type A meeting with the FDA to discuss the Biologics License Application (BLA) data package seeking accelerated approval for AMT-130, its investigational gene therapy for Huntington's disease. The company said it expects to provide a regulatory update after receipt of official meeting minutes and emphasized continued collaboration with the FDA and commitment to patient access.
This meeting represents a formal regulatory engagement on the pathway to potential accelerated approval but does not guarantee a regulatory decision.
uniQure (Nasdaq: QURE) five-year HOPE-B results published in NEJM on Dec 7, 2025 confirm long-term durability and safety of a single HEMGENIX infusion for adults with hemophilia B.
Key outcomes at year five: mean factor IX activity ~36.1%, adjusted annualized bleeding rate (ABR) for all bleeds reduced ~90% versus lead-in, joint bleeds reduced 93%, spontaneous bleeds reduced 94%, and 94% of patients remained off continuous prophylaxis. No serious adverse events were attributed to treatment; most treatment-related adverse events occurred in the first four months. Over 75 individuals across eight countries have received HEMGENIX in real-world use.
uniQure (NASDAQ: QURE) received final meeting minutes from the FDA following an Oct 29, 2025 pre-BLA meeting about AMT-130 for Huntington’s disease. The FDA conveyed that Phase I/II data are currently unlikely to provide the primary evidence to support a BLA. uniQure said it is evaluating the feedback and plans to urgently request a follow-up meeting in Q1 2026. Management reaffirmed commitment to working with regulators and noted community support from patients, families, clinicians and advocates.
The update signals additional regulatory engagement is needed before a BLA pathway can be confirmed.
uniQure (NASDAQ: QURE) reported Q3 2025 results and program updates on Nov 10, 2025. Key clinical highlights include AMT-130 Phase I/II topline 36-month data showing a 75% slowing in disease progression on the cUHDRS (p=0.003) and 60% slowing on TFC (p=0.033) versus an external control; safety remained manageable through 36 months. The company said preliminary FDA pre-BLA feedback introduced uncertainty about using Phase I/II versus external control as primary evidence and plans urgent follow-up.
Other updates: AMT-260 and AMT-191 have additional data expected H1 2026; AMT-162 enrollment paused after a dose-limiting toxicity. Financially, uniQure raised ~$323.7M net proceeds and held $694.2M in cash and investments as of Sept 30, 2025.
uniQure (NASDAQ: QURE) will report third quarter 2025 financial results before market open on Monday, November 10, 2025. Management will host a conference call at 8:30 a.m. ET the same day that will be webcast under the Events & Presentations section of uniQure’s investor website.
Analysts may join the live Q&A by dialing (646) 307-1963 or toll-free (800) 715-9871 with passcode 2196195. Attendees are asked to join 15 minutes before the start time. A replay of the webcast will be archived for 90 days.
uniQure (NASDAQ: QURE) on Nov. 3, 2025 announced FDA feedback from a pre-BLA meeting for AMT-130, its investigational gene therapy for Huntington’s disease.
uniQure said the FDA now appears to disagree that Phase I/II data versus an external control—as prespecified—may be adequate as primary evidence for a BLA, a material shift from prior Type B meeting guidance. As a result, the BLA timing is now unclear. uniQure expects final meeting minutes within 30 days and plans urgent interactions with the FDA while also engaging regulators in the EU and UK. AMT-130 holds Breakthrough Therapy (Apr 2025) and RMAT (May 2024) designations.