2023年10月31日，美国食品和药物管理局（FDA）召开细胞、组织和基因治疗咨询委员会会议，讨论Vertex Pharmaceuticals 与CRISPR Therapeutics 合作开发的治疗镰状细胞病（SCD）的基因治疗药物：Exagamglogene autotemcel（Exa-cel）。经过长达8个小时针对药物的临床结果、安全性担忧等问题的数据展示、问询，顾问委员会深度讨论了Exa‑cel基因编辑脱靶的风险。FDA将在12 月 8 日之前决定是否批准Exa-cel的上市申请。CRISPR Therapeutics Announces Completion of FDA Advisory Committee Meeting for Exagamglogene Autotemcel (exa-cel) for Severe Sickle Cell Disease-Exa-cel PDUFA target action date is December 8, 2023 for severe sickle cell disease (SCD)-ZUG, Switzerland and BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today announced the completion of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Commi
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