Enabling mRNA Therapeutics: Current Landscape and Challenges in Manufacturing
Recent advances and discoveries in the structure and role of mRNA as well as novel lipid-based delivery modalities have enabled the advancement of mRNA therapeutics into the clinical trial space. The manufacturing of these products is relatively simple and eliminates many of the challenges associate...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-10-01
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| Series: | Biomolecules |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2218-273X/13/10/1497 |
| _version_ | 1827721556249280512 |
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| author | Maryam Youssef Cynthia Hitti Julia Puppin Chaves Fulber Amine A. Kamen |
| author_facet | Maryam Youssef Cynthia Hitti Julia Puppin Chaves Fulber Amine A. Kamen |
| author_sort | Maryam Youssef |
| collection | DOAJ |
| description | Recent advances and discoveries in the structure and role of mRNA as well as novel lipid-based delivery modalities have enabled the advancement of mRNA therapeutics into the clinical trial space. The manufacturing of these products is relatively simple and eliminates many of the challenges associated with cell culture production of viral delivery systems for gene and cell therapy applications, allowing rapid production of mRNA for personalized treatments, cancer therapies, protein replacement and gene editing. The success of mRNA vaccines during the COVID-19 pandemic highlighted the immense potential of this technology as a vaccination platform, but there are still particular challenges to establish mRNA as a widespread therapeutic tool. Immunostimulatory byproducts can pose a barrier for chronic treatments and different production scales may need to be considered for these applications. Moreover, long-term storage of mRNA products is notoriously difficult. This review provides a detailed overview of the manufacturing steps for mRNA therapeutics, including sequence design, DNA template preparation, mRNA production and formulation, while identifying the challenges remaining in the dose requirements, long-term storage and immunotolerance of the product. |
| first_indexed | 2024-03-10T21:25:01Z |
| format | Article |
| id | doaj.art-f55c759081f847afb40be39cb730ebaf |
| institution | Directory Open Access Journal |
| issn | 2218-273X |
| language | English |
| last_indexed | 2024-03-10T21:25:01Z |
| publishDate | 2023-10-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Biomolecules |
| spelling | doaj.art-f55c759081f847afb40be39cb730ebaf2023-11-19T15:50:05ZengMDPI AGBiomolecules2218-273X2023-10-011310149710.3390/biom13101497Enabling mRNA Therapeutics: Current Landscape and Challenges in ManufacturingMaryam Youssef0Cynthia Hitti1Julia Puppin Chaves Fulber2Amine A. Kamen3Department of Bioengineering, McGill University, Montreal, QC H3A 0G4, CanadaDepartment of Bioengineering, McGill University, Montreal, QC H3A 0G4, CanadaDepartment of Bioengineering, McGill University, Montreal, QC H3A 0G4, CanadaDepartment of Bioengineering, McGill University, Montreal, QC H3A 0G4, CanadaRecent advances and discoveries in the structure and role of mRNA as well as novel lipid-based delivery modalities have enabled the advancement of mRNA therapeutics into the clinical trial space. The manufacturing of these products is relatively simple and eliminates many of the challenges associated with cell culture production of viral delivery systems for gene and cell therapy applications, allowing rapid production of mRNA for personalized treatments, cancer therapies, protein replacement and gene editing. The success of mRNA vaccines during the COVID-19 pandemic highlighted the immense potential of this technology as a vaccination platform, but there are still particular challenges to establish mRNA as a widespread therapeutic tool. Immunostimulatory byproducts can pose a barrier for chronic treatments and different production scales may need to be considered for these applications. Moreover, long-term storage of mRNA products is notoriously difficult. This review provides a detailed overview of the manufacturing steps for mRNA therapeutics, including sequence design, DNA template preparation, mRNA production and formulation, while identifying the challenges remaining in the dose requirements, long-term storage and immunotolerance of the product.https://www.mdpi.com/2218-273X/13/10/1497mRNA therapeuticsmRNA manufacturingin vitro transcriptionlipid nanoparticles |
| spellingShingle | Maryam Youssef Cynthia Hitti Julia Puppin Chaves Fulber Amine A. Kamen Enabling mRNA Therapeutics: Current Landscape and Challenges in Manufacturing Biomolecules mRNA therapeutics mRNA manufacturing in vitro transcription lipid nanoparticles |
| title | Enabling mRNA Therapeutics: Current Landscape and Challenges in Manufacturing |
| title_full | Enabling mRNA Therapeutics: Current Landscape and Challenges in Manufacturing |
| title_fullStr | Enabling mRNA Therapeutics: Current Landscape and Challenges in Manufacturing |
| title_full_unstemmed | Enabling mRNA Therapeutics: Current Landscape and Challenges in Manufacturing |
| title_short | Enabling mRNA Therapeutics: Current Landscape and Challenges in Manufacturing |
| title_sort | enabling mrna therapeutics current landscape and challenges in manufacturing |
| topic | mRNA therapeutics mRNA manufacturing in vitro transcription lipid nanoparticles |
| url | https://www.mdpi.com/2218-273X/13/10/1497 |
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