uniQure

uniQure NV engages in the discovery, development, and commercialization of innovative gene therapies. Its discoveries intend to treat hemophilia, Huntington's disease, glybera, and cardiovascular problems. The company was founded by Sander J. van Deventer in 1998 and is headquartered in Amsterdam, the Netherlands.

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~ Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington's disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory interactions and clarity on potential strategies for clinical development expected in 2024 ~

uniQure Announces 2023 Financial Results and Highlights Recent Company Progress

uniQure is valued here as a pre-revenue company assessing its liquidation value, value of potential cash flows from HEMGENIX sales and estimating the worth of its future R&D efforts. In 2023, uniQure achieved some major milestones – approval for its HEMGENIX treatment in US & EU, sale of its royalty interests in it, encouraging data from Huntington's disease trials. With the value for uniQure close to $33/share, it represents a very attractive bargain, trading at below $6 recently.

uniQure: The Valuation Got More Attractive

~ 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 ~

uniQure Announces Update on Phase I/II Clinical Trials of AMT-130 Gene Therapy for the Treatment of Huntington's Disease

CSL Ltd

HEMGENIX® is the first and only gene therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of hemophilia B and to show sustained efficacy and safety at three years post-treatment Long-term data continues to show a one-time infusion of HEMGENIX offers elevated and sustained factor IX activity levels for years and significantly reduces the rate of annual bleeds versus factor IX prophylaxis with a favorable safety profile At three years post-treatment with HEMGENIX, 94% of patients remained free of continuous prophylactic treatment KING OF PRUSSIA, Pa. , Dec. 11, 2023 /PRNewswire/ -- Global biotechnology leader CSL (ASX: CSL; USOTC: CSLLY) today announced the three-year results from the pivotal HOPE-B study confirming continued long-term durability and safety of HEMGENIX® (etranacogene dezaparvovec-drlb) following a one-time infusion in people living with hemophilia B.

CSL Behring's HEMGENIX® (etranacogene dezaparvovec-drlb) Demonstrates at Three Years Post-Treatment Long-Term Durability, Safety and Greater Bleed Protection Versus Prophylactic Treatment in People Living with Hemophilia B

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 equity awards to newly hired employees.

uniQure Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

LEXINGTON, Mass. and AMSTERDAM, Nov. 29, 2023 (GLOBE NEWSWIRE) -- uniQure N.V.

uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-191 Gene Therapy for Fabry Disease

KalVista Pharma

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The mean of analysts' price targets for uniQure (QURE) points to a 405.4% upside in the stock. While this highly sought-after metric has not proven reasonably effective, strong agreement among analysts in raising earnings estimates does indicate an upside in the stock.

Wall Street Analysts Believe uniQure (QURE) Could Rally 405.37%: Here's is How to Trade

~ Announced FDA clearance of Investigational New Drug (IND) application for AMT-260 in  refractory mesial temporal lobe epilepsy ~

uniQure Announces Third Quarter 2023 Financial Results and Highlights Recent Company Progress

HEMGENIX®, the first and only FDA-approved gene therapy for adults with hemophilia B, wins for Best Product for Rare/Orphan Diseases KING OF PRUSSIA, Pa. and LEXINGTON, Mass.

CSL and uniQure Win 2023 Prix Galien USA Award

UniQure (QURE) possesses the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.

UniQure (QURE) Expected to Beat Earnings Estimates: Should You Buy?

LEXINGTON, Mass. and AMSTERDAM, Oct. 24, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 12 data presentations, including two oral presentations, will be delivered at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting being held October 24-27 in Brussels, Belgium.

uniQure Announces Major Presence at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting

uniQure (QURE) gains on plans to discontinue investment in some projects and reduce 20% of its workforce.

uniQure (QURE) Up on Restructuring Plans, Workforce Reduction

uniQure said it is cutting back 28% of its workforce. The company is discontinuing much of its pipeline to focus on more promising therapies.

Why Shares of uniQure Are Up Thursday

Gene therapy-focused biotechnology firm uniQure announced it is slashing more than half of its research and technology projects as part of a strategic reorganization aimed at reducing its operating expenses while advancing a selection of its clinical-stage programs to proof-of-concept.  The company said it will no longer be investing in AMT-210 for the treatment of Parkinson's disease and multiple other undisclosed programs.

uniQure slashes more than half of its research programs to preserve cash

UniQure N.V. QURE, -4.74%, a gene therapy company focused on developing therapies for patients with severe medical needs, said Thursday it's planning a strategic overhaul that will cut 28% of its workforce as it strives to cut costs and advance several clinical-stage programs.

UniQure to cut 28% of workforce and scrap more than half its research projects in strategic overhaul

Dutch gene therapy maker uniQure said on Thursday it would cut about 20% of its total workforce, or about 114 jobs, and end investments in more than half of its research and technology projects to reduce costs.

Gene therapy maker UniQure to cut about 20% of workforce

~ Reduction of 28% of workforce not related to HEMGENIX ® manufacturing obligations; Total cost savings of $180 million to extend cash runway into second quarter of 2027 ~

uniQure Announces Strategic Reorganization to Reduce Operating Expenses and Support Advancement of Multiple Clinical-Stage Programs

The Food and Drug Administration has cleared uniQure's application for an epilepsy therapy. The company grew revenue in the second quarter by 380%, year over year.

Why Shares of uniQure Are Dropping Tuesday

uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-260 Gene Therapy for Refractory Mesial Temporal Lobe Epilepsy

Entravision Communications

Rush Street Interactive

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UniQure (QURE) came out with a quarterly loss of $1.44 per share versus the Zacks Consensus Estimate of $2.97. This compares to loss of $0.84 per share a year ago.

UniQure (QURE) Reports Q2 Loss, Misses Revenue Estimates

~ Announced encouraging interim analysis data from ongoing Phase I/II clinical trial of AMT-130 in Huntington's disease showing preservation of function compared to baseline and clinical benefits relative to natural history; both dose cohorts of AMT-130 continue to be generally well-tolerated ~

uniQure Announces Second Quarter 2023 Financial Results and Highlights Recent Company Progress

~ Walid Abi-Saab, M.D., appointed Chief Medical Officer, will bring extensive track record in leading drug development and execution ~ ~ Walid Abi-Saab, M.D., appointed Chief Medical Officer, will bring extensive track record in leading drug development and execution ~

uniQure Announces Leadership Addition to Support Advancement of Clinical Programs

LOS ANGELES, CA / ACCESSWIRE / June 23, 2023 / The Schall Law Firm, a national shareholder rights litigation firm, announces that it is investigating claims on behalf of investors of uniQure N.V. ("uniQure" or "the Company") (NASDAQ:QURE) for violations of the securities laws.

INVESTIGATION ACTION ALERT: The Schall Law Firm Announces it is Investigating Claims Against uniQure N.V. and Encourages Investors with Losses to Contact the Firm

News for uniQure Stock (QURE)