STAT+：FDA发布关于个性化基因疗法更快审批途径的新细节
摘要
华盛顿——美国食品药品监督管理局周二发布了草案指南，旨在简化某些个性化基因疗法的审批流程，这对于批评该机构对待这些疗法方式的罕见病倡导者来说是一个胜利。“这有点像在地里插上一面旗帜，并说‘这很重要’，”PKD基金会社区项目与教育副总裁保罗·斯克里布纳在周二的一场罕见病活动上谈及该草案指南时表示。基因疗法，即通过改变人体细胞内缺陷基因来治疗或治愈疾病的疗法，其突破性进展激励了研究人员和公司，他们认为许多携带罕见突变的患者可以受益于这些个性化药物。继续阅读STAT+以获取完整报道……
English
WASHINGTON – The Food and Drug Administration published draft guidance on Tuesday that would streamline approvals of some individualized gene therapies, a win for rare disease advocates who have criticized the agency’s approach to these treatments. “It’s sort of putting a flag in the ground and saying, ‘This is important,’” Paul Scribner, vice president of community programs and education at the PKD Foundation, said of the draft guidance at a rare disease event on Tuesday. Breakthroughs in gene therapies, or treatments that treat or cure diseases by altering faulty genes inside a person’s cells, have energized researchers and companies who believe many patients with rare mutations could benefit from these personalized medicines. Continue to STAT+ to read the full story…