uniQure
NEWS
Gene therapy company uniQure announced Tuesday “ongoing evidence” of a clinical benefit for its Huntington’s disease treatment. However, investors were not impressed as the stock dropped nearly 20%.
The Huntington’s disease space saw a flurry of activity Wednesday as PTC and uniQure released data from their respective mid-phase trials.
Clinical results for experimental treatments in Alzheimer’s, ALS and more are expected over the coming months. BioSpace highlights a few of the more highly anticipated datasets.
In its 2022 financial report Monday, uniQure provided updates on the progress of its Huntington’s disease and ALS programs.
uniQure inked a deal with Apic Bio to gain development and commercialization rights to APB-102, a gene therapy for a rare, genetic form of ALS.
BMS is terminating a seven-year-old gene therapy collaboration to develop potential treatments for congestive heart failure with uniQure N.V. valued at $1 billion.
The FDA approved CSL Behring and uniQure’s Hemgenix, a one-time gene therapy developed for adults with Hemophilia B,the companies announced Tuesday afternoon.
A Data Safety Monitoring Board overseeing the Phase Ib/II trial assessing UniQure’s gene therapy for Huntington’s disease recommended that enrollment in the higher-dose cohort could resume.
uniQure gave a 12-month update on AMT-130, the first-ever AAV gene therapy for HD to enter clinical trials. BioSpace spoke with the company’s president of R&D, Dr. Ricardo Dolmetsch.
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