Clinical AdvancementManagement remains optimistic about AMT-130's success due to fundamental differences in their program, such as the use of a clinical endpoint for efficacy and clean safety data in patients dosed.
Financial PositionBacked by $377M in cash and funding into the second half of 2027, uniQure remains an HCW Top Pick for 2025 in the coverage universe.
Regulatory AlignmentUniQure has reached regulatory alignment with the FDA on their statistical analysis plan and CMC requirements for AMT-130, viewed as a significant de-risking event.