The Platform Technology Approach to mRNA Product Development and Regulation

mRNA-lipid nanoparticle (LNP) medicinal products can be considered a platform technology because the development process is similar for different diseases and conditions, with similar noncoding mRNA sequences and lipid nanoparticles and essentially unchanged manufacturing and analytical methods ofte...

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Bibliographic Details
Published in:Vaccines (Basel) 2024-05, Vol.12 (5), p.528
Main Authors: Skerritt, John H, Tucek-Szabo, Carolyn, Sutton, Brett, Nolan, Terry
Format: Article
Language:English
Subjects:
Biological products
COVID-19 vaccines
Drug development
Gene sequencing
Genomes
Health aspects
Homeopathy
Infectious diseases
Lipids
Manufacturing
Materia medica and therapeutics
Messenger RNA
mRNA
mRNA vaccines
Nanoparticles
Oncology
Pandemics
Pathogens
Product development
Product quality
Quality of life
regulation
Regulatory compliance
Severe acute respiratory syndrome coronavirus 2
Technology
Therapeutics
vaccine development
Vaccines
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Summary:mRNA-lipid nanoparticle (LNP) medicinal products can be considered a platform technology because the development process is similar for different diseases and conditions, with similar noncoding mRNA sequences and lipid nanoparticles and essentially unchanged manufacturing and analytical methods often utilised for different products. It is critical not to lose the momentum built using the platform approach during the development, regulatory approval and rollout of vaccines for SARS-CoV-2 and its variants. This review proposes a set of modifications to existing regulatory requirements for mRNA products, based on a platform perspective for quality, manufacturing, preclinical, and clinical data. For the first time, we address development and potential regulatory requirements when the mRNA sequences and LNP composition vary in different products as well. In addition, we propose considerations for self-amplifying mRNA, individualised oncology mRNA products, and mRNA therapeutics. Providing a predictable development pathway for academic and commercial groups so that they can know in detail what product characterisation and data are required to develop a dossier for regulatory submission has many potential benefits. These include: reduced development and regulatory costs; faster consumer/patient access and more agile development of products in the face of pandemics; and for rare diseases where alternatives may not exist or to increase survival and the quality of life in cancer patients. Therefore, achieving consensus around platform approaches is both urgent and important. This approach with mRNA can be a template for similar platform frameworks for other therapeutics and vaccines to enable more efficient development and regulatory review.
ISSN:2076-393X
2076-393X
DOI:10.3390/vaccines12050528