| Date | Title | Description |
| 06.03.2026 | FDA reversals leave investors worrying about the fates of other experimental drugs | The FDA in the past year has denied or discouraged applications of at least eight new drugs, according to RTW Investments. The agency initially refused to review Moderna’s flu shot before reversing course.
Companies have accused the FDA of ... |
| 05.03.2026 | FDA official calls UniQure’s gene therapy a ‘failed’ treatment for Huntington’s disease | UniQure needs to run another study to prove its gene therapy “actually helps people with Huntington’s disease,” a senior FDA official said.
The official said UniQure is the “latest company to make a failed therapy for Huntington’s patients.... |
| 31.01.2026 | Eli Lilly Forges $1.12 Billion Gene Editing Alliance for Hearing Loss | Eli Lilly sealed a $1.12 billion deal with Germany's Seamless Therapeutics. This major collaboration aims to develop advanced gene editing treatments for hearing loss. Lilly gains access to Seamless's precision recombinase platform. The tec... |
| 28.01.2026 | Lilly signs $1.12B deal with Germany-based firm to develop hearing loss treatments | Please subscribe to IBJ to decode this article.
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| 03.10.2025 |
Cramer’s Lightning Round: Broadcom over Ambiq Micro | Monday - Friday, 6:00 - 7:00 PM ET
It’s that time again! “Mad Money” host Jim Cramer rings the lightning round bell, which means he’s giving his answers to callers’ stock questions at rapid speed.
Ambiq Micro: “We got to be careful of that.... |
| 29.05.2025 | uniQure Presents Clinical Case Study of First Patient Dosed with AMT-260 in Refractory Mesial Temporal Lobe Epilepsy (MTLE) | ~ No serious adverse events and 92% reduction in seizure frequency observed in the first trial participant through first five months of follow up ~
~ Data to be presented today at Epilepsy Therapies & Diagnostics Development Symposium (... |
| 05.05.2025 | uniQure to Announce First Quarter 2025 Financial Results | ~ uniQure to host inaugural earnings call on Friday, May 9, 2025 at 8:30 a.m. ET ~
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 05, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing trans... |
| 27.02.2025 | uniQure Announces 2024 Financial Results and Highlights Recent Company Progress | ~ 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) submi... |
| 30.01.2025 | uniQure Announces Favorable Recommendation from Independent Data Monitoring Committee for its Phase I/II EPISOD1 Clinical Trial of AMT-162 for the Treatment of SOD1-ALS | ~ 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 ~
LEXINGTO... |
| 30.01.2025 | uniQure Announces Favorable Recommendation from Independent Data Monitoring Committee for its Phase I/II EPISOD1 Clinical Trial of AMT-162 for the Treatment of SOD1-ALS | ~ 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 ~
LEXINGTO... |
| 08.01.2025 | uniQure Announces Pricing of its Public Offering | 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 unde... |
| 07.01.2025 | uniQure Announces Proposed Public Offering | 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 a... |
| 10.12.2024 | uniQure Announces Alignment with FDA on Key Elements of Accelerated Approval Pathway for AMT-130 in Huntington’s Disease | ~ 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 ~
~ ... |
| 10.12.2024 | uniQure Announces Alignment with FDA on Key Elements of Accelerated Approval Pathway for AMT-130 in Huntington’s Disease | ~ 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 ~
~ ... |
| 14.11.2024 | Navigating the Future of Biopharma: Sobi's Meeting and TRexBio's Funding Surge | In the ever-evolving landscape of biopharmaceuticals, two significant events recently captured the industry's attention. Swedish Orphan Biovitrum AB (Sobi) announced an Extraordinary General Meeting, while TRexBio secured a hefty $84 millio... |
| 11.11.2024 | Notice of Extraordinary General Meeting in Swedish Orphan Biovitrum AB (publ) | Notice of Extraordinary General Meeting in Swedish Orphan Biovitrum AB (publ)
Mon, Nov 11, 2024 08:00 CET Report this content
The shareholders in Swedish Orphan Biovitrum AB (publ) (Sobi®) Reg. No. 556038-9321, are hereby summoned to the Ex... |
| 05.11.2024 | Sobi's New Leadership: A Strategic Shift in the Biopharmaceutical Landscape | In the ever-evolving world of biopharmaceuticals, leadership changes can signal new directions and strategies. Recently, Swedish Orphan Biovitrum AB (Sobi) announced the nomination of David Meek as the new Chair of the Board of Directors. T... |
| 05.11.2024 | Navigating Change in Biopharma: Leadership Shifts at Senhwa and Sobi | The biopharmaceutical landscape is a dynamic arena, where leadership changes can signal new directions and strategies. Recently, two companies, Senhwa Biosciences and Swedish Orphan Biovitrum AB (Sobi), have made significant appointments th... |
| 05.11.2024 | uniQure Announces Third Quarter 2024 Financial Results and Highlights Recent Company Progress | ~ 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/... |
| 04.11.2024 | David Meek is proposed as new Chair of the Board of Directors of Sobi | David Meek is proposed as new Chair of the Board of Directors of Sobi
Mon, Nov 04, 2024 21:00 CET Report this content
The Nomination Committee of Swedish Orphan Biovitrum AB (publ) (Sobi®) proposes that David Meek is elected as new Chair of... |
| 15.10.2024 | uniQure Announces Dosing of First Patient in Phase I/II Clinical Trial of AMT-162 for the Treatment of SOD1-ALS | 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 ... |
| 10.10.2024 | Kurma Partners: First Closing Of €250 Million Biofund IV | Kurma Partners, an asset manager focused on improving human health, announced that it has raised EUR 140 million, completing the first closing of Biofund IV. By the final closing in 2025, Kurma aims to have raised a total of €250 million in... |
| 04.10.2024 | Igniting Futures: The Power of STEM Exploration Day | In a world where technology reigns supreme, the future hinges on innovation. The Children's Museum of Indianapolis recognized this truth by hosting STEM Exploration Day, a vibrant event designed to inspire young girls to embrace careers in ... |
| 03.10.2024 | Kurma Partners raises €140 million first close of new €250 million Biofund IV | Paris-based Kurma Partners, an asset manager focused on improving human health, announced that it has raised €140 million, successfully completing the first closing of Biofund IV. Kurma aims to have raised a total of €250 million in its new... |
| 23.09.2024 | uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease | 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... |
| 23.09.2024 | uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease | 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... |
| 15.08.2024 | uniQure Announces Dosing of First Patient in Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease | 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 ... |
| 01.08.2024 | uniQure Announces Second Quarter 2024 Financial Results and Provides Company Update | ~ 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 ... |
| 23.07.2024 | uniQure Announces Closing of Sale of Manufacturing Facility to Genezen | ~ 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 AM... |
| 09.07.2024 | uniQure Announces Positive Interim Data Update Demonstrating Slowing of Disease Progression in Phase I/II Trials of AMT-130 for Huntington’s Disease | ~ 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... |
| 01.07.2024 | uniQure Announces Sale of Commercial Manufacturing Facility to Genezen | ~ 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... |
| 01.07.2024 | Genezen to Acquire uniQure's Commercial Gene Therapy Manufacturing Operations in Lexington, MA | 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... |
| 07.05.2024 | uniQure Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress | ~ On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024 ~
~ Clinical trial initiation for Fabry disease on track to begin in second quarter of 2... |
| 08.12.2023 | uniQure Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) | LEXINGTON, Mass. and AMSTERDAM, Dec. 08, 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 grant of inducement... |
| 26.06.2023 | uniQure Announces Leadership Addition to Support Advancement of Clinical Programs | ~ Walid Abi-Saab, M.D., appointed Chief Medical Officer, will bring extensive track record in leading drug development and execution ~
/EIN News/ -- LEXINGTON, Mass. and AMSTERDAM, June 26, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QUR... |
| 20.06.2023 | uniQure announces achievement of $100 million milestone related to hemophilia B gene therapy | - |
| 15.05.2023 | uniQure Announces Sale of Royalty Interest in HEMGENIX® for Up To $400 Million | - |
| 28.02.2023 | Life changing treatments for rare disease – a beacon of hope | This article appeared in Labiotech on Rare Disease Day 28 February 2023. Click here to read the online version. The text from the article follows below. |
| 31.01.2023 | uniQure and Apic Bio enter into global licensing agreement for APB-102, a clinical stage gene therapy for patients with ALS caused by mutations in SOD1 | /EIN News/ -- ~ 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 ... |
| 20.12.2022 | uniQure Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) | /EIN News/ -- LEXINGTON, Mass. and AMSTERDAM, Dec. 20, 2022 (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 ... |
| 29.11.2022 | uniQure Highlights Therapeutic Potential of AMT-260 in Refractory Temporal Lobe Epilepsy (rTLE) at Virtual Research & Development Event | /EIN News/ -- ~ IND Submission and Clinical Development Expected to Begin in 2023 ~
~ Advancements in Manufacturing and Technology Platform Also Highlighted ~
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 29, 2022 (GLOBE NEWSWIRE) -... |
| 11.10.2022 | uniQure Announces Multiple Presentations at the Upcoming Annual Meeting of The European Society of Gene and Cell Therapy (ESGCT) | LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 11, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that e... |
| 03.10.2022 | uniQure to Participate in Upcoming Industry Conferences in October | LEXINGTON, Mass. and AMSTERDAM, Oct. 03, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced its participation in th... |
| 06.09.2022 | uniQure to Participate in Upcoming Industry Conferences in September | LEXINGTON, Mass. and AMSTERDAM, Sept. 06, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced its participation in t... |
| 08.08.2022 | uniQure Announces Second Quarter 2022 Financial Results and Highlights Recent Company Progress | ~ 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) observe... |
| 23.06.2022 | uniQure Announces Update on Low-Dose Cohort in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease | ~ 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 mon... |
| 22.06.2022 | CORRECTION -- uniQure Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) | LEXINGTON, Mass. and AMSTERDAM, the Netherlands, June 22, 2022 (GLOBE NEWSWIRE) -- In a release issued under the same headline earlier today by uniQure N.V. (NASDAQ: QURE), please note that all the numbers have been updated. The corrected r... |
| 22.06.2022 | uniQure Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) | LEXINGTON, Mass. and AMSTERDAM, the Netherlands, June 22, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that t... |
| 24.05.2022 | uniQure Announces FDA Acceptance of Biologics License Application for Etranacogene Dezaparvovec under Priority Review | LEXINGTON, Mass. and AMSTERDAM, May 24, 2022 (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 D... |
| 11.05.2022 | uniQure Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) | LEXINGTON, Mass. and AMSTERDAM, May 11, 2022 (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 granted... |
| 02.05.2022 | uniQure Announces Multiple Presentations at Upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting | LEXINGTON, Mass. and AMSTERDAM, May 02, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that six data presentati... |
| 02.05.2022 | uniQure Announces First Quarter 2022 Financial Results and Highlights Recent Company Progress | ~ Partner CSL Behring submitted Marketing Authorization Application (MAA) in Europe for
etranacogene dezaparvovec in hemophilia B; MAA validated by the European Medicines Agency (EMA)
and granted accelerated assessment ~
~ Received $55 mill... |
| 21.03.2022 | uniQure Announces Completion of Patient Enrollment in the First Two Cohorts of its Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease | LEXINGTON, Mass. and AMSTERDAM, March 21, 2022 (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 pati... |
| 25.02.2022 | uniQure Announces 2021 Financial Results and Highlights Recent Company Progress | ~ Data from largest gene therapy study in hemophilia B showed sustained therapeutic effect at 18-months and statistical superiority in reducing annualized bleeding rate compared to baseline FIX prophylactic therapy; Submissions of marketing... |
| 07.02.2022 | uniQure Announces Dosing of First Patients in European Open-Label Clinical Trial of AMT-130 Gene Therapy in Huntington’s Disease | LEXINGTON, Mass. and AMSTERDAM, Feb. 07, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the dosing of the first... |
| 04.02.2022 | EAHAD 2022 - Final Analysis from the Pivotal Phase 3 HOPE-B Gene Therapy Trial: Stable Steady-State Efficacy and Safety of Etranacogene Dezaparvovec in Adults with Sever or Moderately Severe Hemophili... | FINAL ANALYSIS FROM THE PIVOTAL PHASE 3 HOPE-B GENE THERAPY TRIAL: STABLE STEADY-STATE EFFICACY AND SAFETY OF ETRANACOGENE DEZAPARVOVEC IN ADULTS WITH SEVERE OR MODERATELY SEVERE HAEMOPHILIA B
Wolfgang Miesbach
University Hospital Frankfurt... |
| 16.12.2021 | uniQure Announces Clinical Update on First Patients in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease | ~ 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 ... |
| 16.12.2021 | uniQure Announces Clinical Update on First Patients in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington's Disease | ~ 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 ... |
| 09.12.2021 | uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B | ~ Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec ~
~ Et... |
| 09.12.2021 | uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B | ~ Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec ~
~ Et... |
| 02.11.2021 | uniQure Announces Latest Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease | ~ No Significant Safety Concerns Observed in First Four Patients Enrolled in Higher-dose Cohort ~
~ Enrollment Expected to be Completed by Mid-2022 ~
LEXINGTON, Mass. and AMSTERDAM, Nov. 02, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QU... |
| 01.11.2021 | uniQure to Participate in Upcoming Industry Conferences in November | LEXINGTON, Mass. and AMSTERDAM, Nov. 01, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced its participation in th... |
| 25.10.2021 | uniQure Announces Third Quarter 2021 Financial Results and Highlights Recent Company Progress | ~ 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 ... |
| 21.10.2021 | uniQure Announces the Appointment of Rachelle Jacques to its Board of Directors | 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 Rach... |
| 19.10.2021 | uniQure Announces Presentations at the Annual Meeting of The European Society of Gene and Cell Therapy (ESGCT) | LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 19, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that f... |
| 16.07.2021 | ISTH 2021 - 52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults with Severe or Moderate-Severe Hemophilia B: Data from the Phase 3 HOPE-B Gene Therapy Trial | 52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults with Severe or Moderate-
Severe Hemophilia B: Data from the Phase 3 HOPE-B Gene Therapy Trial
Steven W. Pipe1, Frank W.G. Leebeek2, Michael Recht3, Nigel S. Key4, Susan Latt... |
| 14.07.2021 | ISTH 2021 - Clinical Outcomes in Adults with Hemophilia B With Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data from the HOPE-B Trial | Clinical Outcomes in Adults with
Hemophilia B With and Without Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data from the Phase 3 Etranacogene Dezaparvovec HOPE-B Gene Therapy Trial
Frank W.G. Leebeek1, Wolfgang Miesbach2, Michael ... |
| 02.07.2021 | uniQure Announces Presentations at Upcoming International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress | LEXINGTON, Mass. and AMSTERDAM, The Netherlands, July 02, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that e... |
| 22.06.2021 | uniQure updates lead gene therapy’s timeline; acquires Corlieve to feed pipeline | A hemophilia B gene therapy that uniQure is testing in a pivotal clinical trial is on track for a filing with the FDA early next year, an updated timeline that follows discussions with the agency about the data needed to support a submissio... |
| 22.06.2021 | uniQure Announces Positive 52-Week Clinical Data from HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B and Provides Regulatory Update | ~ Sustained increases in Factor IX (FIX) levels with mean FIX activity of 41.5 percent of normal in full study population one year following a single administration of etranacogene dezaparvovec ~
~ Held pre-BLA submission meeting with FDA a... |
| 22.06.2021 | uniQure to Acquire Corlieve Therapeutics and Advance its Gene Therapy Program to Treat Temporal Lobe Epilepsy (TLE) | ~ Expands uniQure’s Pipeline of Innovative Gene Therapies to Treat Neurological Disorders ~
~ Strengthens uniQure’s Global Leadership in the Development of Gene Therapies that Employ miRNA Silencing Technology ~
~ Large Opportunity Targetin... |
| 16.06.2021 | UNIQURE N.V.
uniQure Announces Enrollment of First Two Patients in Second Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease | LEXINGTON, Mass. and AMSTERDAM, The Netherlands, June 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that t... |
| 27.05.2021 | UNIQURE N.V.
uniQure Announces Positive Recommendation to Advance Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease | LEXINGTON, Mass. and AMSTERDAM, The Netherlands, May 27, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that th... |
| 13.05.2021 | UNIQURE N.V.
uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-existing Antibodies to AAV5 Vector | ~ Data Show No Clinically Significant Correlation Between Pre-Existing NAbs and FIX Activity ~
~ Further Supports Potential for AAV5 Gene Therapies to be Viable Treatments in Nearly All Patients --
~ Data Presented at the American Society o... |
| 13.05.2021 | UNIQURE N.V.
ASGCT 2021 - Clinical Outcomes in Patients With and Without Pre-existing Neutralizing Antibodies to the Vector: 6 Month Data from the Phase 3 HOPE-B Gene Therapy Trial of Etranacogene De... | Clinical Outcomes in Patients With and Without Pre-existing
Neutralizing Antibodies to the Vector:
6 Month Data from the Phase 3 HOPE-B Gene Therapy Trial of
Etranacogene Dezaparvovec
Michael Recht1, Frank W.G. Leebeek2, Wolfgang Miesbach3,... |
| 06.05.2021 | UNIQURE N.V.
uniQure announces closing of commercialization and license agreement with CSL Behring | LEXINGTON, Mass. and AMSTERDAM, May 06, 2021 (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 waiting period ... |
| 28.04.2021 | UNIQURE N.V.
uniQure Announces Presentations at Upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting | LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 28, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that ... |
| 26.04.2021 | UNIQURE N.V.
uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program | LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 26, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced today that ... |
| 13.04.2021 | Huntington’s community grieves not just one therapeutic setback, but two | For the community of people affected by Huntington’s disease — a group that is no stranger to disappointment — the back-to-back announcements last month still hit like a double whammy.
First, Roche announced it was cutting off the dosing of... |
| 08.04.2021 | UNIQURE N.V.
uniQure Announces Publications of Preclinical Data for AMT-130 in Huntington's Disease Showing Safety of Administration in NHPs and Widespread Long-Term HTT-Lowering in the Brain | LEXINGTON, Mass. and AMSTERDAM, April 08, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that three manuscripts... |
| 05.04.2021 | UNIQURE N.V.
uniQure Announces Completion of Enrollment in First Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease | ~ Enrollment of second dose cohort expected to begin in 3Q 2021 ~
~ Company announces plans to initiate a second clinical study of AMT-130
in Europe in the second half of 2021 ~
LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 05, 202... |
| 29.03.2021 | UNIQURE N.V.
uniQure Announces Findings from Reported Case of Hepatocellular Carcinoma (HCC) in Hemophilia B Gene Therapy Program | ~ Independent investigation shows that etranacogene dezaparvovec is highly unlikely to be the cause
of HCC in HOPE-B pivotal trial ~
~ Data from investigation submitted to U.S Food and Drug Administration (FDA) on March 26, 2021 ~
LEXINGTON... |
| 01.03.2021 | uniQure Announces 2020 Financial Results and Highlights Recent Company Progress | ~ Six patient procedures now completed in Phase I/II for Huntington’s disease, including two procedures conducted in February 2021, with full enrollment of first cohort expected mid-2021 ~
~ Last patient in HOPE-B pivotal study to complete ... |
| 26.02.2021 | uniQure to Participate in Multiple Upcoming Industry Conferences in March | LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Feb. 26, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced its pa... |
| 25.06.2020 | CSL Behring, uniQure in $2B deal to in-license hemophilia gene therapy | A Dutch biotech company developing a gene therapy for hemophilia has made a deal with the U.S.-based division of an Australian company that will enabled it to be marketed around the world.
Amsterdam-based uniQure said Wednesday that King of... |
| 04.12.2019 | Top 8 Predictions That Will Disrupt Healthcare in 2020 | Every year, our team of futurists, analysts, and consultants at Frost & Sullivan's Transformational Healthcare Group comes together to brainstorm and predict the themes, technologies, and global forces that will define the next 12 to 18... |
| 31.07.2017 | Even With High Costs & Uncertainty, Gene Therapy Finds Funding And Warm IPO Waters | 8 Shares Email Facebook Twitter LinkedIn
This is the first entry of the Homo Posterius (“the Next Human”) column on investments in future-setting technologies. This week, we will dive into gene therapy startups and the money that goes into ... |
| 30.01.2017 | uniQure wins a BTD for hemophilia B therapy; Allergan scores sNDA for Avycaz | • The Dutch biotech uniQure has picked up a breakthrough drug designation from the FDA for AMT-060, its gene therapy for hemophilia B. Spark Therapeutics and its partners at Pfizer have a rival gene therapy in the clinic f... |
| 12.09.2016 | With small RetroSense deal, Allergan hoping for big win | RP is a group of congenital retinal diseases driven by mutations in the rhodopsin gene. The disease initially impacts retinal cells called rods, which provide peripheral and night vision. As the rods degenerate, neighboring cones, which all... |
| 29.06.2016 | The biotech IPO feast flags as investors lose their appetite for R&D risk | There was a time after the 2008 crash that biotech IPOs were about as popular as snow in March. But from 2013 through 2015 generalist investors got over their fears and jumped into one of the hottest stock games on the planet. Fund... |
| 08.06.2015 | Five (non-cancer) areas Bristol-Myers Squibb is pursuing hard | The pharma giant has indeed found a sweet spot in immuno-oncology. Take a look at the recent success of Opdivo, which was recently forecasted to have a 2019 revenue of $5.68 billion. Beyond that, BMS is interested in pursuing the following ... |
| 13.11.2013 | GeNO, uniQure look to leap through biotech IPO window in Q4 rush | Waltham, MA-based GeNO is throwing its hat into a crowded biotech IPO ring, looking to raise $50 million to back its work on a new, patient-friendly approach for delivering inhaled nitric oxide on the go. And a Dutch biotech, uniQure, is al... |
| 27.07.2012 | Fundraising: Gene therapy firm targeting sickle cell anemia raises $60M | Product: Gene therapy to target sickle cell anemia, beta-thalassemia and childhood cerebral adrenoleukodystrophy
Money raised: $60 million in series D; at least $76 million in previous rounds based on press releases and U.S. Securities and ... |
| - | With small RetroSense deal, Allergan hoping for big win | Allergan’s recent deal for gene therapy company RetroSense Therapeutics, based in Ann Arbor, Michigan, could be a nice win for the big biotech.
For $60 million, and potential milestone payments down the line, Allergan picks up the developme... |
| - | uniQure updates lead gene therapy’s timeline; acquires Corlieve to feed pipeline | A hemophilia B gene therapy that uniQure is testing in a pivotal clinical trial is on track for a filing with the FDA early next year, an updated timeline that follows discussions with the agency about the data needed to support a submissio... |
| - | CSL Behring, uniQure in $2B deal to in-license hemophilia gene therapy | A Dutch biotech company developing a gene therapy for hemophilia has made a deal with the U.S.-based division of an Australian company that will enabled it to be marketed around the world.
Amsterdam-based uniQure said Wednesday that King of... |
| - | Fundraising: Gene therapy firm targeting sickle cell anemia raises $60M | Company name: bluebird bio.
Industry: Biotechnology.
Location: Cambridge, Massachusetts.
Product: Gene therapy to target sickle cell anemia, beta-thalassemia and childhood cerebral adrenoleukodystrophy
Money raised: $60 million in series D;... |
| - | Five (non-cancer) areas Bristol-Myers Squibb is pursuing hard | Outside of cancer immunotherapy, Bristol-Myers Squibb is interested in pursuing five areas: Heart failure, virology, rare disease, immunoscience and fibrosis.
Donnie McGrath, a vp at Bristol-Myers Squibb and head of search and evaluation, d... |
