uniQure is a clinical-stage gene therapy company developing one-time treatments for rare diseases. The company has no commercial products today — its only approved therapy, HEMGENIX (a gene therapy for hemophilia B), was out-licensed to CSL Behring in 2020 in exchange for royalties and manufacturing fees. uniQure's lead program is AMT-130, a gene therapy for Huntington's disease, a fatal neurodegenerative disorder with no approved disease-modifying treatment. AMT-130 uses uniQure's proprietary miQURE gene-silencing platform and is delivered via a one-time, MRI-guided direct injection into the brain. Phase I/II data showed a 75% slowing of disease progression at 36 months in the high-dose group, and the FDA granted Breakthrough Therapy designation in 2025. However, the FDA has indicated that Phase I/II data vs. an external control are insufficient to support a BLA, and is requiring a randomized controlled Phase III study — a material setback that extends the timeline and increases costs. uniQure's pipeline also includes AMT-260 for refractory epilepsy, AMT-191 for Fabry disease, and AMT-162 for SOD1-ALS, all in Phase I/II. uniQure funds operations primarily through equity raises, and held roughly $649M in cash as of September 2025, which it expects to fund operations into 2029. If AMT-130 is ultimately approved, uniQure plans a direct U.S. commercial launch through roughly 50 neurosurgical centers capable of performing the procedure.