Financial HealthUniQure's healthy balance sheet and cash runway into the second half of 2027, along with multiple clinical data readouts expected, suggest the company is significantly undervalued.
Pipeline DevelopmentPrograms for epilepsy, Fabry, and ALS are progressing in the clinic, suggesting a robust pipeline beyond AMT-130.
Regulatory ApprovalThe FDA granted Breakthrough Therapy designation to uniQure's lead gene therapy program AMT-130 for the treatment of Huntington’s disease, supporting an accelerated pathway to approval.