AMT-260

About uniQure

uniQure's mission is to reimagine the future of medicine by delivering innovative, potentially curative gene therapies for severe genetic and neurodegenerative diseases. The company's strategic foundation is its industry-leading, modular AAV5 platform, which has demonstrated a validated safety profile and holds exclusive rights for therapeutic delivery to the brain and liver. Its most advanced program, AMT-130 for Huntington's disease, represents a potential first-in-class treatment, positioning uniQure at the forefront of CNS gene therapy with a pipeline targeting high-unmet-need indications.

Therapeutic Areas