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Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine
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 technol...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7744278/ https://www.ncbi.nlm.nih.gov/pubmed/31024072 http://dx.doi.org/10.1038/s41434-019-0074-7 |
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author | Elverum, Kris Whitman, Maria |
author_facet | Elverum, Kris Whitman, Maria |
author_sort | Elverum, Kris |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-7744278 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | Nature Publishing Group UK |
record_format | MEDLINE/PubMed |
spelling | pubmed-77442782020-12-28 Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine Elverum, Kris Whitman, Maria Gene Ther Review Article 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. Nature Publishing Group UK 2019-04-25 2020 /pmc/articles/PMC7744278/ /pubmed/31024072 http://dx.doi.org/10.1038/s41434-019-0074-7 Text en © The Author(s) 2019 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/. |
spellingShingle | Review Article Elverum, Kris Whitman, Maria Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine |
title | Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine |
title_full | Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine |
title_fullStr | Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine |
title_full_unstemmed | Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine |
title_short | Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine |
title_sort | delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine |
topic | Review Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7744278/ https://www.ncbi.nlm.nih.gov/pubmed/31024072 http://dx.doi.org/10.1038/s41434-019-0074-7 |
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