uniQure N.V. - QURE STOCK NEWS

uniQure N.V. has appointed Rachelle Jacques to its Board of Directors, enhancing its expertise in gene therapy development. With over 25 years in the biotech sector, including leadership roles in major companies, Jacques brings valuable experience in launching rare disease therapies. She will serve on the Audit Committee and assist in advancing uniQure’s clinical programs, including a BLA submission for hemophilia B. Jacques's past achievements highlight her capability to bolster uniQure's pipeline, which addresses severe genetic diseases through innovative gene therapies.

uniQure N.V. (NASDAQ: QURE) announced its participation in several upcoming investor and scientific conferences. Key events include Chardan’s 5th Annual Genetic Medicines Conference on October 4-5, 2021, featuring a fireside chat with CEO Matt Kapusta. Other conferences include the Cell & Gene Meeting on the Mesa (October 12-14), where Joshua Leeman will present, and the ESGCT meeting (October 19-22) for preclinical data presentations. The Jefferies Gene Therapy/Editing Summit will take place on October 27-28, with another fireside chat featuring Kapusta.

uniQure N.V. (NASDAQ: QURE) announced its participation in several investor and scientific conferences from September 8 to September 30, 2021. Key events include Citi’s 16th Annual BioPharma Virtual Conference, the Wells Fargo 2021 Virtual Healthcare Conference with a fireside chat featuring CEO Matt Kapusta, and the H.C. Wainwright 23rd Annual Global Investment Conference. Additionally, Ricardo Dolmetsch will participate in discussions at the SVB Leerink CybeRx Series and the 2021 Cantor Virtual Global Healthcare Conference. uniQure aims to advance gene therapies for severe genetic diseases.

uniQure has completed two additional patient procedures in the higher-dose cohort of its Phase I/II clinical trial for AMT-130, targeting Huntington’s disease. With a total of 14 procedures now done, the independent Data Safety Monitoring Board gave a positive recommendation. The company anticipates completing full study enrollment by mid-2022 and plans to share preliminary imaging and biomarker data later this year. The trial aims to explore the safety and efficacy of AMT-130 in treating early manifest Huntington’s disease.

uniQure N.V. (QURE) reported significant milestones in its recent press release, including a strong financial performance with revenues of $463.9 million for Q2 2021, largely due to a licensing agreement with CSL Behring. The company reported a net income of $399.5 million and strong clinical outcomes in the HOPE-B study, showing a mean FIX activity of 41.5% of normal and substantial reductions in bleeding events. Additionally, uniQure expanded its gene therapy pipeline with four new research programs and plans to acquire Corlieve Therapeutics, enhancing its focus on neurological diseases.

uniQure N.V. (NASDAQ: QURE) announced it will present eight studies at the ISTH Virtual Congress from July 17-21, 2021, focusing on gene therapy for hemophilia B. Highlights include five-year data on FIX expression stability and outcomes from the Phase 3 HOPE-B trial. Notable presentations include the clinical outcomes of patients with pre-existing AAV5 antibodies and a case report on liver safety. The company continues to advance gene therapies for severe conditions, emphasizing its commitment to innovative treatment solutions.

uniQure (NASDAQ: QURE) has announced a definitive agreement to acquire Corlieve Therapeutics for €46.3 million. This acquisition expands uniQure's gene therapy pipeline targeting temporal lobe epilepsy (TLE), which affects 1.3 million individuals in the U.S. and Europe, including 800,000 drug-resistant patients. The lead program, AMT-260, leverages miRNA silencing technology to address chronic spontaneous seizures. The deal may involve additional milestone payments totaling up to €203.7 million, pending clinical progression and regulatory approvals.

uniQure N.V. (QURE) announced promising results from its Phase III HOPE-B trial of etranacogene dezaparvovec, showing sustained Factor IX (FIX) activity at an average of 41.5% of normal after one year. The treatment reduced bleeding episodes by 80% and allowed 96% of patients to discontinue FIX replacement therapy. No severe adverse events were reported. Following a meeting with the FDA, uniQure will submit a BLA by Q1 2022, focusing on non-inferiority in annualized bleeding rates.