FDA 就个体化基因疗法加快审批路径发布指南草案
STAT+: FDA offers new details on quicker approval pathway for personalized gene therapies
FDA 于周二发布指南草案，拟简化部分个体化基因疗法的审批流程。PKD Foundation 社区项目与教育副总裁 Paul Scribner 在当天的罕见病活动上称，这份草案相当于立下一面旗帜，表明这件事很重要。报道指出，基因疗法领域的进展让研究人员和公司相信，许多携带罕见突变的患者可能从这类个体化药物中获益。
WASHINGTON – The Food and Drug Administration published draft guidance on Tuesday that would streamline approvals of some individualized gene therapies, a win for rare disease advocates who have criticized the agency’s approach to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is important,’” Paul Scribner, vice president of community programs and education at the PKD Foundation, said of the draft guidance at a rare disease event on Tuesday.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.
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来源：STAT News · statnews.com