STAT+: Makary, Prasad outline pathway for bespoke gene therapy
6.5
来源：
STAT
发布时间：
2025-11-13 12:00
摘要：
FDA has introduced a new regulatory pathway for personalized gene-editing therapies, which may significantly impact treatment for ultra-rare diseases. Additionally, Day One Pharmaceuticals is acquiring Mersana Therapeutics for $129 million, highlighting ongoing investment in the oncology sector. This acquisition could lead to advancements in antibody-drug conjugates for cancer treatment.
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关键证据
FDA outlines pathway for bespoke gene editing
Day One Pharmaceuticals is acquiring Mersana Therapeutics in a deal valued at $129 million
This shift could transform last year’s one-off miracle for 'Baby KJ' into a scalable model for ultra-rare diseases.
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AI评分总结
FDA has introduced a new regulatory pathway for personalized gene-editing therapies, which may significantly impact treatment for ultra-rare diseases. Additionally, Day One Pharmaceuticals is acquiring Mersana Therapeutics for $129 million, highlighting ongoing investment in the oncology sector. This acquisition could lead to advancements in antibody-drug conjugates for cancer treatment.