Uniqure Plummets More Than 40% On Another Twist In Its Gene Therapy Saga

Biotech firm uniQure’s stock crashed over 40% after the FDA raised new concerns about its Huntington’s disease gene therapy, AMT-130, during a Phase 3 trial review. The setback marks another delay in the decade-long development of AMT-130, which uses a one-time AAV-based gene therapy to silence the mutant huntingtin protein causing the fatal neurodegenerative disorder. Regulators flagged potential safety risks, including off-target effects and long-term efficacy questions, despite earlier Phase 1/2 data showing promising biomarker reductions in patients. The company must now submit additional preclinical data, pushing potential approval timelines further into 2027 and raising doubts about its lead program’s viability. Investors reacted sharply, wiping hundreds of millions in market cap, as uniQure’s broader gene therapy pipeline faces renewed scrutiny over clinical and regulatory hurdles.