Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine.
Journal
Gene therapy
ISSN: 1476-5462
Titre abrégé: Gene Ther
Pays: England
ID NLM: 9421525
Informations de publication
Date de publication:
12 2020
12 2020
Historique:
received:
20
04
2018
accepted:
20
03
2019
revised:
18
03
2019
pubmed:
27
4
2019
medline:
19
8
2021
entrez:
27
4
2019
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 31024072
doi: 10.1038/s41434-019-0074-7
pii: 10.1038/s41434-019-0074-7
pmc: PMC7744278
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
537-544Références
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