Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine.,Gene Therapy

当前位置： X-MOL 学术 › Gene Ther. › 论文详情

Our official English website, www.x-mol.net, welcomes your feedback! (Note: you will need to create a separate account there.)

Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine.
Gene Therapy ( IF 4.6 ) Pub Date : 2019-04-25 , DOI: 10.1038/s41434-019-0074-7
Kris Elverum ₁ , Maria Whitman ₂

Affiliation

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.

中文翻译：

为患者提供细胞和基因疗法：实现下一代医学潜力的解决方案。

药物从小分子到蛋白质的演变推动了治疗益处的增加，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批准了美国首个基因疗法诺华的 tisagenlecleucel，而 CRISPR-Cas9 等技术有望创造新药浪潮。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% 的人仅通过商业交付获得药物，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。本文回顾了提供细胞和基因疗法的现有系统，并概述了使患者可以使用它们的要求。通过专家访谈得知，

更新日期：2019-11-18

点击分享查看原文

点击收藏

公开下载

阅读更多本刊最新论文本刊介绍/投稿指南11