UniQure Biopharma B.V.

uniQure is a gene therapy company that develops one-time, potentially curative treatments for patients with severe medical needs, including rare and genetic diseases. It uses an AAV-based gene therapy platform and has its own manufacturing capabilities. Its products include HEMGENIX (etranacogene dezaparvovec) for hemophilia B, which is commercialized by CSL Behring, and AMT-130 for Huntington's disease, which is in clinical development.