uniQure N.V. (QURE) surged 76.5% on Jul 9 after it announced updated interim data, including up to 24 months of follow-up data, from 29 treated patients enrolled in the ongoing early to mid-stage studies of AMT130 for Huntington's disease in the United States and EU. Results demonstrated a potential long-term, durable clinical benefit and reduction of a key marker of neurodegeneration upon treatment with the candidate. uniQure is simultaneously conducting two phase I/II studies of AMT-130, with 26 participa ...

This human gene therapy company is expected to post quarterly loss of $1.24 per share in its upcoming report, which represents a year-over-year change of +13.9%. Revenues are expected to be $3.47 million, up 43.2% from the year-ago quarter. Adaptive Biotechnologies' consensus EPS estimate for the upcoming report has remained unchanged over the past month at -$0.32. Compared to the company's year-ago EPS, this represents a change of +3%. Adaptive Biotechnologies currently boasts a Zacks Rank of #2 (Buy). For ...

~ 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 ~ ~ Investor conference call and webcast today at 8:30 a.m. ET ~ "These updated results are exciting and provide compelling evidence of potential therapeutic benefit," stated Victor Sung ...

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, Gene ...

~ Genezen to manufacture global commercial supply of HEMGENIX® and support uniQure's pipeline programs as a preferred customer ~ Genezen agreed to acquire uniQure's commercial-scale gene-therapy manufacturing facility for total consideration of $25 million, comprised of $12.5 million of newly issued Series C Preferred Stock and a $12.5 million convertible note. In addition, Matt Kapusta, chief executive officer of uniQure, will join the Board of Genezen upon the closing of the transaction, which is expected ...

~ Genezen to manufacture global commercial supply of HEMGENIX® and support uniQure's pipeline programs as a preferred customer ~ ~ uniQure's comprehensive review of operations and options to reduce expenses is ongoing and expected to be complete in the third quarter of 2024 ~ Under the proposed transaction, Genezen will be responsible for manufacturing global commercial supply of HEMGENIX® for CSL and providing development and manufacturing services to support uniQure's investigational gene therapies. uniQu ...

~ Designation based on 24-month interim Phase I/II clinical data for AMT-130 announced in December 2023 ~ LEXINGTON, Mass. and AMSTERDAM, June 03, 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 Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational gene therapy AMT-130 for the treatment of Hunti ...

Exhibit 99.1 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 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 ~ ~ Comprehensive review of operations and options to reduce expenses ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands Not ...

Exhibit 99.1 uniQure Announces 2023 Financial Results and Highlights Recent Company Progress ~ 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 ~ ~ Announced FDA clearance of two Investigational New Drug (IND) applications; Initiation of Phase I/II clinical trials in mesial tempor ...