2026年，多起基因治疗临床试验受试者死亡事件相继披露，其中包括2名儿童，在医疗界引发广泛关注1。这些事件涉及研究者发起的临床研究项目，反映了当前基因治疗在安全性上存在的突出问题1。面对这一局面，国家相关部门迅速采取行动。9月4日，国家医保局副局长李滔宣布将把更多符合条件的罕见病创新药纳入医保报销范围1。9月14日，国家卫生健康委等多部门联合发布《关于进一步加强罕见病防治工作的通知》1。
罕见病防治中基因治疗的地位至关重要，因为约八成罕见病由基因缺陷导致，相当一部分在儿童时期发病1。业界专家正在探讨如何在确保科学严谨的前提下推进基因治疗的临床应用1。专家认为，基因治疗的关键难点在于"怎么将基因安全地递送到患者体内"1。
Several deaths among clinical trial participants in gene therapy studies for rare diseases have prompted heightened regulatory attention in 2026.1 The incidents, which included two child participants, have raised concerns about the safety and oversight of investigator-initiated trials in this emerging field.1
In response to these developments, Chinese health authorities have intensified their focus on rare disease prevention and treatment. On September 4, Vice Director Li Tao of the National Healthcare Security Administration announced plans to expand medical insurance coverage for more innovative rare disease drugs that meet eligibility criteria.1 Subsequently, on September 14, the National Health Commission and multiple other government departments issued a notice to strengthen rare disease prevention and treatment efforts.1
The scientific challenge underlying gene therapy development remains complex. Approximately 80 percent of rare diseases stem from genetic defects, with a significant portion manifesting during childhood.1 Industry experts emphasize that the critical obstacle in advancing gene therapy is determining how to safely deliver genetic material into patients' bodies.1
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