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4D分子治疗学称4D-710囊性纤维化肺病1期AEROW试验的中期数据显示有意义的肺功能活动

2025-12-17 20:03

  • Clinically meaningful lung function activity, measured by ppFEV1 and LCI2.5, with follow-up through 1 year at dose selected for Phase 2 
  • Durable CFTR transgene expression within target therapeutic range with follow-up through at least 1 year
  • Data support 4D-710's potential to be a durable, redosable, variant-agnostic, disease-modifying treatment for people with cystic fibrosis lung disease with high unmet need

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