Company Financial PositionWith $694.2 million in cash extending runway into 2029 and multiple regulatory and data catalysts ahead, uniQure remains well positioned to reestablish the U.S. path for AMT-130.
Gene Therapy DataThe strength, duration, and consistency of the 3-year Phase 1/2 data for AMT-130 in Huntington's disease should overcome FDA pre-specification concerns.
Regulatory PathwaysThere is a better than 50/50 chance uniQure can find a regulatory path forward for AMT-130 in Huntington's disease given the company's multi-pronged approach.