Turnstone Biologics, New York, NY, USA.
ZS Associates, Philadelphia, PA, USA.
Gene Ther. 2020 Dec;27(12):537-544. doi: 10.1038/s41434-019-0074-7. Epub 2019 Apr 25.
The evolution of medicines from small molecules to proteins drove increased therapeutic benefits, and the next generation of cell and gene therapies holds tremendous promise for patients. The Food and Drug Administration approved the U.S.'s first gene therapy, Novartis' tisagenlecleucel, and technologies like CRISPR-Cas9 are poised to create a wave of new medicines. Unfortunately, the vast majority of patients may not benefit from cell and gene therapies. At least 95% of people receive medicines only through commercial delivery, but stakeholders have struggled to develop and sustain successful business models for cell and gene therapies. This paper reviews the existing system to deliver cell and gene therapies and outlines the requirements to make them accessible to patients. Informed by interviews with experts, opportunities for improvement are identified along the patient and cell journeys, and a call to action is made for stakeholders to detail and implement change.
从小分子药物到蛋白质药物的发展带来了更高的治疗效益，下一代细胞和基因疗法为患者带来了巨大的希望。美国食品和药物管理局 (FDA) 批准了首个基因疗法，诺华公司的 tisagenlecleucel，而 CRISPR-Cas9 等技术也有望掀起一波新的药物浪潮。不幸的是，绝大多数患者可能无法从细胞和基因疗法中受益。至少 95%的人只能通过商业渠道获得药物，但利益相关者一直在努力开发和维持细胞和基因疗法的成功商业模式。本文回顾了现有的细胞和基因疗法的输送系统，并概述了使这些疗法能够惠及患者的要求。通过对专家的访谈，确定了在患者和细胞治疗过程中可以改进的机会，并呼吁利益相关者详细说明并实施变革。
