Business
uniQure Announces 2022 Financial Results and Highlights Recent Company Progress
~ Achieved U.S. and European Commission marketing approvals of first and only gene therapyfor adults with hemophilia B ~ ~ Advancing development of AMT-130
About this update from Uniqure N.v.
[{"type":"text","content":"~ Achieved U.S. and European Commission marketing approvals of first and only gene therapyfor adults with hemophilia B ~ ~ Advancing development of AMT-130 for patients with Huntington’s disease – clinical update from U.S. Phase I/II trial expected in second quarter 2023 ~ ~Hosted virtual investor event featuring AMT-260 in refractory temporal lobe epilepsy - expected to enter clinical development in second half of 2023 ~ ~Expanded pipeline with in-licensing of AMT-162 for SOD1 ALS - plans to initiate a Phase I/II clinical trial in the second half of 2023 ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Feb. 27, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for 2022 and highlighted recent progress across its business. “We had a strong ending to 2022 with the historic approval of the world’s first gene therapy for hemophilia B for which we led the multi-year research and clinical development, and through our partnership with CSL Behring, HEMGENIX® will now be available to patients in the United States and European Union,” stated Matt Kapusta, chief executive officer of uniQure. “We are immensely proud of our efforts that led to this product approval, and of the innovation and leadership in genomic medicine that it represents. At the same time, we are working with urgency to advance AMT-130, the first AAV gene therapy being clinically investigated in Huntington’s disease, and are on track to provide a clinical update from our U.S. Phase I/II study in the second quarter of 2023. We are also preparing for the initiation of two new Phase I/II studies in refractory temporal lobe epilepsy and SOD1-ALS in the second half of this year, and readying for the submission of an investigational new drug application in Fabry disease in 2023.” “Throughout this progress, we have established cutting-edge capabilities in the field of genetic medicines – from how we discover and develop our gene therapies, to how we successfully navigate complex clinical and regulatory pathways, to how we manufacture these novel gene therapies globally for clinical and commercial usage,” said Mr. Kapusta. “We believe these capabilities position us well for continued growth and success as we look forward to developin...