Commercial uptake and reimbursement challenges have pushed several hemophilia gene therapies into underperformance, product withdrawals and market reassessment, according to industry commentary and payer feedback. Companies including BioMarin and Pfizer have struggled with pricing, access and adoption despite clinical promise; long‑term value and payer turnover concerns remain front‑and‑center. In parallel, UniQure warned that FDA feedback had complicated its planned filing for the Huntington’s gene therapy AMT‑130, prompting a sharp sell‑off and the prospect of longer or additional trials. Together these developments signal that scientific success in gene editing and delivery is colliding with real‑world commercial and regulatory constraints.
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