~ 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 ...
~ 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 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 c...
~ 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 ~
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 ...
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.
Amid the valuations of large-cap stocks, notably tech stocks in the Magnificent 7 declining, this has led to this list of micro-cap stocks. Not only are investors expected to rotate into smaller and under-capitalized companies, but these companies also have unique theses that should keep investors interested.
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