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~ AMT-130 granted Breakthrough Therapy designation by FDA ~ ~ Initial safety data from third cohort of Phase I/II study show AMT-130 continues to be generally well-tolerated, with no treatment-related serious adverse events ~ ~ Held Type B FDA meetings in the first and second quarters of 2025 to advance BLA preparations for AMT-130; regulatory update expected in the second quarter of 2025 ~ ~ AMT-260 clinical data from first patient to be presented at Epilepsy Therapies & Diagnostics Development Symposium on May 29, 2025 ~ ~ Cash, cash equivalents and current investment securities of approximately $409.0 million as of March 31, 2025 expected to fund operations into second half o

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 05, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report first quarter 2025 financial results before market open on Friday, May 9, 2025. Management will then host a conference call at 8:30 a.m. ET. As the Company advances its pipeline and AMT-130 toward a BLA submission and potential commercialization, uniQure is initiating quarterly earnings calls to provide regular updates on progress and to enhance engagement with the investment community. The event will be webcast under the Events & Presentations section of uniQure's

~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory update and guidance on the Biologics License Application submission expected in the second quarter of 2025 ~ LEXINGTON, Mass. and AMSTERDAM, April 17, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington's disease, a rare, inherited neurodegenerative disorder for which the

~ Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in Huntington's disease; Initiated preparations for a potential Biologics License Application (BLA) submission ~ ~ Completed patient enrollment in the third cohort of the Phase I/II study of AMT-130 ~ ~ Initiated dosing of the Phase I/II study of AMT-260 in mesial temporal lobe epilepsy (mTLE); Implementing protocol changes to expand study inclusion criteria ~ ~ Completed enrollment of the first cohorts in the Phase I/II studies of AMT-191 in Fabry disease and AMT-162 in SOD1-ALS; Received favorable recommendations

94 percent of patients eliminated factor IX prophylaxis and remained free of continuous prophylaxis through four years post-treatmentMean factor IX activity levels were sustained at near normal levels of 37% through four years post-treatment, reinforcing the efficacy of HEMGENIX in the treatment of hemophilia BPhase 3 HOPE-B data showed that a one-time treatment with HEMGENIX provided long-term bleed protection as mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by approximately 90% from lead-in as compared to year fourKING OF PRUSSIA, Pa., Feb. 7, 2025 /PRNewswire/ -- Global biotechnology leader CSL ((ASX:CSL, OTC:CSLLY) today announced the four-year results from the

~ Independent Data Monitoring Committee recommends proceeding with dosing of second cohort after planned safety assessment ~ ~ Company expects to initiate enrollment of second dose cohort in the first quarter of 2025 ~ LEXINGTON, Mass. and AMSTERDAM, Feb. 03, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the completion of enrollment in the first cohort of the Phase I/IIa trial of AMT-191, an investigational gene therapy for the treatment of Fabry disease. Additionally, the Independent Data Monitoring Committee (IDMC) reviewed safety data from the initia

~ Independent Data Monitoring Committee Recommends Proceeding with Dose Escalation After Planned Safety Assessment of First Dose Cohort ~ ~ Company Expects to Initiate Enrollment of Second Dose Cohort in the First Quarter of 2025 ~ LEXINGTON, Mass. and AMSTERDAM, Jan. 30, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the Independent Data Monitoring Committee (IDMC) for EPISOD1, uniQure's Phase I/II clinical trial of AMT-162, an investigational gene therapy for amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene

LEXINGTON, Mass. and AMSTERDAM, Jan. 08, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the pricing of its underwritten public offering of 4,411,764 of its ordinary shares at a public offering price of $17.00 per share. The aggregate gross proceeds to uniQure from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by uniQure, are expected to be approximately $75 million. All securities to be sold in the offering are being sold by uniQure. In addition, uniQure has granted to the underwriters a 30-day option to pu

LEXINGTON, Mass. and AMSTERDAM, Jan. 07, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it has commenced an underwritten public offering of its ordinary shares and pre-funded warrants to purchase its ordinary shares. All securities to be sold in the offering will be offered by uniQure. In addition, uniQure intends to grant the underwriters a 30-day option to purchase up to 15% additional ordinary shares at the public offering price, less underwriting discounts and commissions. The offering is subject to market and other conditions, and there can be no assuran

~ U.S. Food and Drug Administration (FDA) agrees that data from ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a Biologics License Application (BLA) for Accelerated Approval ~ ~ FDA agrees that the composite Unified Huntington's Disease Rating Scale (cUHDRS) may serve as an intermediate clinical endpoint for Accelerated Approval ~         ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, Dec. 10, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the company reached agreement

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LEXINGTON, Mass. and AMSTERDAM, June 26, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Walid Abi-Saab, M.D., as Chief Medical Officer, effective immediately. He will report to Matt Kapusta, Chief Executive Officer of uniQure, and will be based in the Company's Basel, Switzerland office. Dr. Abi-Saab will be responsible for leading all clinical research and development, regulatory affairs, medical affairs, and program management at uniQure. With Dr. Abi-Saab's appointment, Dr. Ricardo Dolmetsch becomes President and Chief Scientific Officer. He

Sander van Deventer is co-founder and former Chief Technology Officer of the Company Elena Ritsou assumes role of Chief Corporate Officer Barbara Sanders is promoted to Chief Technology Officer Industry veteran Frank Walsh joins as an Independent Board Member VectorY Therapeutics, a biotech company developing innovative vectorized antibody approaches for the treatment of neurodegenerative diseases, today announces the appointment of Sander van Deventer, co-founder and formerly Chief Technology Officer, as Chief Executive Officer. The Company has also made key appointments to its leadership team. Sander van Deventer has 25 years of experience in drug development and a longstanding

Genespire appoints Dr. Sabah Sallah as Chief Medical Officer Accomplished executive with extensive preclinical and clinical gene therapy experience joins Genespire to accelerate pipeline to the clinic Milan, Italy, 7 July 2022: Genespire, a next generation gene therapy company developing first-in-class transformative therapies with advanced lentiviral vectors, today announces the appointment of Dr. Sabah Sallah MD, PhD as Chief Medical Officer. Dr. Sallah will lead the Company's clinical strategy, development and operations as Genespire progresses its novel advanced lentiviral gene therapy platforms towards the clinic. Dr. Sallah is a board-certified hematologist who has spent more than

NEW YORK and LONDON, March 02, 2022 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (NASDAQ:AKTX), a late-stage biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement (C5) and/or leukotriene (LBT4) systems are implicated, today announced the appointment of Rachelle Jacques as President and Chief Executive Officer of Akari Therapeutics. Ms. Jacques will also join the company's Board of Directors. Her appointment begins at the end of March 2022. Outgoing Chief Executive Officer, Clive Richardson, will continue to serve the Company, supporting Rachelle to accelerate business development and ensuring a smooth transition."

~ Completed 78-week follow-up for hemophilia B pivotal study, with top-line data expected by year-end 2021 ~ ~ Enrolled 14 patients to date in Phase 1/2 clinical trial of AMT-130 in Huntington's Disease, with no significant safety concerns observed ~ ~ Preliminary data on first four patients in ongoing Phase I/II clinical trial of AMT-130 and initiation of European Phase Ib/II clinical trial expected by year-end 2021 ~ ~ Closed acquisition of Corlieve Therapeutics adding preclinical program in temporal lobe epilepsy to proprietary research pipeline ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 25, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy com

LEXINGTON, Mass. and AMSTERDAM, Oct. 21, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Rachelle Jacques to its Board of Directors. Ms. Jacques, who will serve on the Board's Audit Committee, has more than 25 years of industry experience in strategic, cross-functional executive roles spanning finance, business operations, manufacturing, and commercial. She has led successful launches of several rare disease therapies including the recent approval of a Regenerative Medicine Advanced Therapy (RMAT)-designated product, one of only three since the p

Chardan Capital Markets resumed coverage of uniQure with a rating of Buy and set a new price target of $38.00

Raymond James upgraded uniQure from Outperform to Strong Buy

Raymond James resumed coverage of uniQure with a rating of Outperform and set a new price target of $20.00

Goldman downgraded uniQure from Buy to Neutral and set a new price target of $8.00

Mizuho downgraded uniQure from Buy to Neutral and set a new price target of $10.00 from $52.00 previously

UBS upgraded uniQure from Neutral to Buy and set a new price target of $40.00

Raymond James downgraded uniQure from Strong Buy to Outperform and set a new price target of $58.00 from $75.00 previously

William Blair initiated coverage of uniQure with a rating of Outperform and set a new price target of $66.00

Credit Suisse reiterated coverage of uniQure with a rating of Outperform and set a new price target of $81.00 from $78.00 previously

BTIG Research initiated coverage of uniQure with a rating of Buy and set a new price target of $46.00

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 05, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report first quarter 2025 financial results before market open on Friday, May 9, 2025. Management will then host a conference call at 8:30 a.m. ET. As the Company advances its pipeline and AMT-130 toward a BLA submission and potential commercialization, uniQure is initiating quarterly earnings calls to provide regular updates on progress and to enhance engagement with the investment community. The event will be webcast under the Events & Presentations section of uniQure's

~ U.S. Food and Drug Administration (FDA) agrees that data from ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a Biologics License Application (BLA) for Accelerated Approval ~ ~ FDA agrees that the composite Unified Huntington's Disease Rating Scale (cUHDRS) may serve as an intermediate clinical endpoint for Accelerated Approval ~         ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, Dec. 10, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the company reached agreement

~ Achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit; Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 24 months compared to a propensity score-weighted external control ~ ~ Achieved statistically significant lowering of CSF neurofilament light protein (NfL) compared to baseline at 24 months in patients treated with AMT-130; Mean CSF NfL levels for both doses were below baseline at 24 months ~ ~ Granted first-ever Regenerative Medicine Advanced Therapy (RMAT) designation in Huntington's disease; uniQure expects to meet with the FDA in t

~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~ ~ Mean CSF NfL continue to demonstrate favorable trends with low-dose patients below baseline at 30 months and high-dose patients near baseline at 18 months ~ ~ AMT-130 continues to be generally well-tolerated across both doses ~ ~ Data support continuing clinical development of AMT-130 and pursuing regulatory interactions to discuss potential strategies for ongoing development ~ ~ Investor conference call and webcast today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, D

~ AMT-130 continues to be generally well-tolerated across both dose cohorts ~ ~ Patients treated with AMT-130 show preserved function compared to baseline and clinical benefits relative to natural history of the disease ~ ~ Neurofilament Light Chain (NfL) in cerebrospinal fluid (CSF) was below baseline at 24 months in patients treated with the low-dose of AMT-130 and declining towards baseline at 12 months in patients treated with the high-dose of AMT-130 ~ ~ Suppression of CSF mHTT in low-dose cohort supports AMT-130 target engagement; Greater variability observed in high-dose cohort ~ ~ Promising data support continuing clinical development of AMT-130 and pursuing regulatory interacti

 ~ License of APB-102 further strengthens uniQure's pipeline of innovative gene therapies to treat neurological disorders and miRNA-based gene silencing programs ~ ~ APB-102 and uniQure's c9orf72-ALS program have the potential to address most inherited forms of ALS ~~ uniQure plans to initiate a Phase I/II trial of APB-102 in the second half of 2023 ~ LEXINGTON, Mass. and AMSTERDAM and CAMBRIDGE, Mass., Jan. 31, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, and Apic Bio, an innovative gene therapy company developing novel treatment options for patients with rare genetic diseas

~ Announced 12-month data on the lower-dose cohort of AMT-130 in Huntington's disease showed the investigative gene therapy was generally well tolerated at this dose with a mean reduction of 53.8% of mutant Huntingtin protein (mHTT) observed in cerebral spinal fluid (CSF) ~ ~ Announced postponement of AMT-130 higher-dose procedures due to recent suspected unexpected severe adverse reactions at this dose; Lower-dose procedures are not affected and no impact is expected on anticipated data readouts in 2023 ~ ~ Advancing regulatory reviews of the U.S. and European marketing applications for etranacogene dezaparvovec in hemophilia B ~ ~ Investor conference call and webcast today at 8:30 a.m.

~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean reduction of 53.8% of mutant HTT (mHTT) observed in cerebral spinal fluid (CSF) at 12 months in evaluable patients treated with AMT-130 ~ ~ Neurofilament Light Chain (NfL) in CSF near baseline at 12 months in patients treated with AMT-130 ~ ~ Biomarker and clinical data including 24-month follow-up in the low-dose U.S. cohort and 12-month follow up in the high-dose U.S. cohort expected in first half of 2023 ~ ~ Investor conference call and webcast today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, June 23, 2022 (GLOBE NEWSWIRE) --

~ Treatment was well tolerated with no significant safety issues related to AMT-130 in first two treated patients through one year of follow-up ~ ~ Neurofilament Light Chain (NfL) rose as expected immediately following surgery and returned to baseline in treated patients ~ ~ A total of 19 patient procedures have been performed in the U.S. Phase I/II clinical trial, with higher-dose cohort enrollment expected to be completed by mid-2022 ~ ~ Screening initiated in European open-label Phase I/II study ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Dec. 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company ad

~ Strong Presence at ASH Featuring Five Presentations, Including Late-BreakingOral Presentation on HOPE-B Pivotal Trial ~ ~ uniQure to Host Investor Webcast Tuesday, December 8, 2020 at 5:00 p.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, Nov. 30, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan, will present clinical data from the HOPE-B pivotal trial of etranacogene dezaparvovec in hemophilia B