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4 - uniQure N.V. (0001590560) (Issuer)

4 - uniQure N.V. (0001590560) (Issuer)

4 - uniQure N.V. (0001590560) (Issuer)

4 - uniQure N.V. (0001590560) (Issuer)

LEXINGTON, Mass. and AMSTERDAM, Nov. 21, 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 first patient has been dosed in the GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE). "The dosing of the first patient in our Phase I/II trial in temporal lobe epilepsy represents an important milestone for uniQure and our third clinical trial initiation over the past six months," stated Walid Abi-Saab, M.D., chief medical officer of uniQure. "Nearly one-third of people experiencing focal onset seizures

~ Type B meeting scheduled with the FDA in the fourth quarter of 2024 to initiate discussions regarding a potential expedited clinical development pathway for AMT-130 in Huntington's disease ~ ~ Presented positive interim data from Phase I/II trial of AMT-130 demonstrating slowing of Huntington's disease progression and reductions in key biomarker of neurodegeneration ~ ~ Initiated patient dosing in new Phase I/II studies of AMT-162 in SOD1-ALS and AMT-191 in Fabry disease; First patient enrolled in observational stage of Phase I/II study of AMT-260 in mTLE ~ ~ Completed sale of Lexington manufacturing facility and announced organizational restructuring expected to significantly reduce op

LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 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 first patient has been dosed in the Phase I/II clinical trial of AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1), a rare, inherited and progressive motor neuron disease. EPISOD1 is a Phase I/II multi-center, open-label trial being conducted in the United States with three dose-escalating cohorts assessing the safety, tolerability and exploratory signs of efficacy of AMT-162 in individuals with SOD1-A

LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 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 U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure's investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease. In August 2024, uniQure announced the dosing of the first patient in its U.S., multi-center, open-label Phase I/IIa trial of AMT-191. "This important designation highlights the need for new gene therapies like AMT-191 for patients with Fabry disease with the potential of

LEXINGTON, Mass. and AMSTERDAM, Aug. 15, 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 first patient has been dosed in a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease, a rare, inherited genetic disease. The Phase I/IIa study is a multi-center, open-label trial being conducted in the United States with two dose-escalating cohorts assessing the safety, tolerability and early signs of efficacy of AMT-191 in individuals with Fabry disease. "We are very pleased to begin patient dosing for AMT-191 in Fabry disease, marking a sig

~ Announced RMAT designation for AMT-130 in Huntington's disease and positive interim Phase I/II data demonstrating the slowing of disease progression and reductions in a key biomarker of neurodegeneration; Meeting with FDA expected in the second half of 2024 to discuss potential for expedited clinical development ~ ~ Initiated patient screening for three additional Phase I/II studies in mesial temporal lobe epilepsy, SOD1 ALS, and Fabry disease ~ ~ Today, announced an organizational restructuring intended to streamline operations; Together with the recent sale of the Lexington manufacturing facility, these changes are expected to reduce headcount by 65% and lower recurring cash burn by $7

~ uniQure maintains preferential access to industry-leading manufacturing capabilities to support its pipeline of gene therapy candidates ~ ~ Immediate reduction in cash burn, projected to save $40 million annually ~ LEXINGTON, Mass. and AMSTERDAM, July 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the closing of the sale of its global manufacturing facility in Lexington, Massachusetts to Genezen, a leading contract development and manufacturing organization specializing in the supply of retroviral vectors, lentiviral vectors, and adeno-associated virus (AA

~ 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

Strategic acquisition will enable Genezen to deliver late-phase and commercial gene therapy development and manufacturing services to customers around the world. BOSTON and INDIANAPOLIS, July 1, 2024 /PRNewswire/ -- Genezen, a best-in-class gene therapy CDMO, today announced an agreement to acquire uniQure's (NASDAQ:QURE) commercial gene therapy operations in Lexington, MA, bringing a strategic manufacturing facility and a world-class team of employees to the company. Under the terms of the agreement, Genezen will enter into strategic supply agreements for uniQure's clinical portfolio and CSL's commercial HEMGENIX® product, the first one-time gene therapy approved in the U.S. and Europe for

~ Proposed divestiture streamlines uniQure's focus and represents an important milestone in its ongoing effort to significantly reduce operating expenses ~ ~ Genezen to manufacture global commercial supply of HEMGENIX® and support uniQure's pipeline programs as a preferred customer ~ ~ uniQure to reduce cash burn by $40 million per year and receive total consideration of $25 million ~ ~ uniQure's comprehensive review of operations and options to reduce expenses is ongoing and expected to be complete in the third quarter of 2024 ~ LEXINGTON, Mass. and AMSTERDAM, July 01, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company

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

~ 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

Cantor Fitzgerald analyst Kristen Kluska reiterates uniQure (NASDAQ:QURE) with a Overweight and maintains $24 price target.

Shares of Conagra Brands, Inc. (NYSE:CAG) fell during Thursday's session after the company reported worse-than-expected fourth-quarter sales results and issued FY25 adjusted EPS guidance below estimates. The company reported adjusted earnings per share of 61 cents, beating the analyst consensus estimate of 57 cents. Quarterly revenues of $2.905 billion missed the street view of $2.930 billion, according to data from Benzinga Pro. Conagra Brands shares declined 3.9% to $27.70 on Thursday. Here are some other stocks moving in today’s mid-day session. Gainers Kazia Therapeutics Limited (NASDAQ:KZIA) shares surged 88.6% to $1.26. Kazia Therapeutics, on Wednesday, announced result

UniQure N.V. (NASDAQ:QURE) shares are skyrocketing Wednesday following the release of promising interim data from its Phase I/II clinical trials of AMT-130, a gene therapy targeting Huntington’s disease. What’s Going On: The latest results, which include up to 24 months of follow-up data from 29 patients across the U.S. and Europe, demonstrated a significant slowing of disease progression. Patients treated with AMT-130 exhibited an impressive 80% reduction in disease progression compared to a control group, as measured by the Unified Huntington’s Disease Rating Scale (cUHDRS). Additionally, levels of neurofilament light protein (NfL) in cerebrospinal fluid, a key biomarker of neurodeg

Shares of SeaStar Medical Holding Corporation (NASDAQ:ICU) fell sharply during Wednesday's session after the company entered into a $10 million registered direct offering of 947,868 shares at $10.55 per share. SeaStar Medical shares dipped 34% to $8.55 on Wednesday. Here are some other stocks moving in today’s mid-day session. Gainers Virpax Pharmaceuticals, Inc. (NASDAQ:VRPX) shares jumped 139% to $2.78 after the company announced results for a Swine Model pilot study for Probudur. Kazia Therapeutics Limited (NASDAQ:KZIA) gained 129% to $0.4325 after the company announced results from GBM-AGILE, a phase II/III study that included an evaluation of paxalisib versus standard of

U.S. stocks were mostly higher, with the Nasdaq Composite gaining around 0.4% on Wednesday. Shares of SMART Global Holdings, Inc. (NASDAQ:SGH) rose sharply during Wednesday's session after the company reported better-than-expected third-quarter financial results. SMART Global reported quarterly earnings of 37 cents per share, which beat the analyst consensus estimate of 30 cents. Quarterly sales came in at $300.6 million, which beat the analyst consensus estimate of $300 million, according to data from Benzinga Pro. SMART Global shares jumped 17.4% to $27.27 on Wednesday. Here are some other big stocks recording gains in today’s session. Solaris Oilfield Infrastructure, Inc.

U.S. stock futures were higher this morning, with the Nasdaq futures gaining around 50 points on Wednesday. Shares of Kura Sushi USA, Inc. (NASDAQ:KRUS) fell sharply in today's pre-market trading after the company posted downbeat results for its third quarter on Tuesday. Kura Sushi reported quarterly earnings of $0.00 per share which missed the analyst consensus estimate of $0.01. The company reported quarterly sales of $63.082 million which missed the analyst consensus estimate of $63.101 million, according to data from Benzinga Pro. Indivior shares fell 4.2% to $56.00 in the pre-market trading session. Here are some big stocks recording losses in today's pre-market trading sess

U.S. stocks traded mixed toward the end of trading, with the S&P 500 gaining around 0.1% on Tuesday. The Dow traded down 0.05% to 39,323.7 while the NASDAQ rose 0.10% to 18,422.24. The S&P 500 also rose, gaining, 0.12% to 5,579.76. Check This Out: Nvidia To Rally Over 40%? Here Are 10 Top Analyst Forecasts For Tuesday Leading and Lagging SectorsFinancials shares jumped by 1.1% on Tuesday. In trading on Tuesday, materials shares fell by 0.6%. Top Headline Helen of Troy Limited (NASDAQ:HELE) reported soft first-quarter earnings and lowered its FY25 guidance. Equities Trading UP                        Soligenix, Inc. (NASDAQ:SNGX) shares shot up 379% to $9.59 after the

Shares of Helen of Troy Limited (NASDAQ:HELE) fell sharply during Tuesday's session after the company reported soft first-quarter earnings and lowered its FY25 guidance. Helen of Troy reported quarterly earnings of 99 cents per share which missed the analyst consensus estimate of $1.59. The company reported quarterly sales of $416.85 million which missed the analyst consensus estimate of $446.22 million, according to data from Benzinga Pro. Helen of Troy shares dipped 30.3% to $62.06 on Tuesday. Here are some other stocks moving in today’s mid-day session. Gainers Soligenix, Inc. (NASDAQ:SNGX) shares jumped 195% to $5.90 after the company announced interim results for extende

U.S. stocks traded mixed midway through trading, with the Nasdaq Composite gaining more than 50 points on Tuesday. The Dow traded down 0.41% to 39,182.94 while the NASDAQ rose 0.28% to 18,455.86. The S&P 500 also rose, gaining, 0.10% to 5,578.67. Check This Out: Nvidia To Rally Over 40%? Here Are 10 Top Analyst Forecasts For Tuesday Leading and Lagging SectorsInformation technology shares jumped by 0.8% on Tuesday. In trading on Tuesday, real estate shares fell by 0.9%. Top Headline Helen of Troy Limited (NASDAQ:HELE) reported soft first-quarter earnings and lowered its FY25 guidance. Equities Trading UP                        Soligenix, Inc. (NASDAQ:SNGX) shares sho