Uniqure is a biotechnology company focusing on the development of gene therapies for the treatment of genetic diseases. The company is dedicated to creating innovative and transformative therapies that target various conditions, particularly those related to genetic disorders and rare diseases. By leveraging advanced gene-editing technologies, Uniqure aims to provide long-lasting solutions that address the underlying causes of these ailments. Their pipeline includes research and development efforts centered on delivering effective treatments to improve patients' lives through the unique potential of gene therapy. Read More
~ Achieved alignment with the FDA on the AMT-130 statistical analysis plan and CMC requirements to support a planned BLA submission in the first quarter of 2026;
Looking for insights into the US markets one hour before the close of the markets on Thursday? Delve into the top gainers and losers of today's session and gain valuable market intelligence.
The new designation is in addition to the existing Regenerative Medicine Advanced Therapy, Orphan Drug, and Fast Track designations granted to AMT-130.
Let's have a look at what is happening on the US markets before the opening bell on Thursday. Below you can find the top gainers and losers in today's pre-market session.
~ Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in Huntington’s disease; Initiated preparations for a potential Biologics License Application (BLA) submission ~
