Messenger RNA Therapeutics

ISBN-13: 9783031084171 / Angielski / Miękka / 2023

Opis

This book focuses on the fundamentals and applications of messenger RNA (mRNA)-based therapeutics and discusses the strengths and key challenges of this emerging class of drugs. 

In the past 30 years, extensive research and technological development in many areas have contributed to the emergence of in vitro transcribed mRNA as a therapeutic that has now reached clinical testing. Formulations that protect the mRNA from nucleases and accelerate its cellular uptake, combined with improvements to the mRNA molecules themselves, have been critical advancements for mRNAs to become viable therapeutics. Though once regarded as a serious impediment, the transient nature of mRNA technology is now considered a major advantage in making mRNA therapies safe and, ultimately, a potential game changer in the field of medicine. 

This new book in the RNA Technologies series provides a state-of-the-art overview on the emerging field of mRNA therapeutics covering essential strategies for formulation, delivery, and application. It also reviews the promising role in cancer immunotherapy, respiratory diseases, and chronic HBV infection and discusses RNA vaccines in light of the current COVID-19 pandemic. mRNA-based approaches have great potential to revolutionize molecular biology, cell biology, biomedical research, and medicine. Thus, this handbook is an essential resource for researchers in academia and industry contributing to the development of this new area of therapeutics.

Spis treści

1. The Democratization of RNA Therapeutics

Cooke JP, Yang C, Sukhovershin R.

RNA Therapeutics Program, Houston Methodist Research Institute, Houston TX

John P. Cooke, - jpcooke@houstonmethodist.org

2. Supramolecular Strategies for mRNA Delivery

Horacio Cabral - horacio@bmw.t.u-tokyo.ac.jp

Department of Bioengineering, Graduate School of Engineering, The University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-8656, Japan

3. In Vitro-Transcribed mRNAs as a new generation of therapeutics in the dawn of 21st century: Exploitation of peptides as carriers for their intracellular delivery

Miliotou A.N., Pappas I.S., Vizirianakis I.S., Papadopoulou L.C.

Lefkothea C. Papadopoulou - lefkotea@pharm.auth.gr

4. Effective Delivery of mRNA Therapeutics and Vaccines using Lipid Nanoparticles

Peter Lutwyche - pete.lutwyche@genevant.com

Genevant Sciences Corp., 155 - 887 Great Northern Way, Vancouver, British Columbia V5T 4T5, Canada

5. Messenger RNA therapeutics 

Yingjie Xu - xuyingjie@shsmu.edu.cn

Shanghai Key Laboratory for Tumor Microenvironment and Inflammation, Department of Biochemistry and Molecular Cell Biology, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China

6. Formulation, delivery and application of mRNA therapeutics in respiratory diseases

Jenny Ka Wing LAM - jkwlam@hku.hk

Department of Pharmacology & Pharmacy, The University of Hong Kong

2/F, Laboratory Block, 21 Sassoon Road, Pokfulam, Hong Kong

7. Clinical applications of mRNA formulated in Lipid Nanoparticles

Marian Solinis - marian.solinis@ehu.eus

Laboratorio de Farmacia y Tecnología Farmacéutica, Paseo de la Universidad, 7 | 01006 Vitoria-Gasteiz

8. Preparation of messenger RNA loaded nanomedicine applied on tissue engineering and regenerative medicine

Chin-Yu Lin - geant@mail.cmu.edu.tw

Institute of New Drug Development, College of Medicine, China Medical University, Taichung, 40402, Taiwan

9. Lipid nanoparticle-mediated delivery of mRNA therapeutics: Immune activation by ionizable cationic lipids 

Aneesh Thakur - aneesh.thakur@sund.ku.dk

University of Copenhagen, Department of Pharmacy, Vaccine Design and Delivery Group, Universitetsparken 2, 2100 Copenhagen Ø, Denmark

10. Adjuvants, the elephant in the room for RNA vaccines

Joeri Aerts - joeri.aerts@vub.be

Neuro-Aging & Viro-Immunotherapy, Laarbeeklaan 103, 1090 Brussels

11. Addressing skin with modified mRNA constructs for novel therapies

Dirk Strunk - dirk.strunk@pmu.ac.at

Cell Therapy Institute, Spinal Cord Injury and Tissue Regeneration Center Salzburg (SCI-TReCS), Paracelsus Medical University (PMU), 5020 Salzburg, Austria

12. mRNA-Based Cancer Immunotherapies

Jessica Foster, Peter Madsen

Jessica Foster - fosterjb@chop.edu

Division of Oncology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA 

13. From bench to bedside: the journey of an mRNA drug candidate into a medicine

Neil Henderson - Neil.Henderson@astrazeneca.com

Translational Biomarkers & Bioanalysis, Clinical Pharmacology Biologics & Bioanalytics, Clinical Pharmacology & Safety Sciences, BioPharmaceuticals R&D, AstraZeneca, Gothenburg, Sweden

14. Synthetic mRNA gene therapies for chronic HBV infection

Kristie Bloom, Abdullah Ely, Patrick Arbuthnot

Kristie Bloom - Kristie.bloom@wits.ac.za

Wits/SAMRC Antiviral Gene Therapy Research Unit, Department of Molecular Medicine and Haematology, University of the Witwatersrand, Medical School, 7 York Road, Parktown

15. Delivery Vehicles for Self-Amplifying RNA

Anna K. Blakney, Nuthan Bathula, Petya Popova

Anna Blakney - anna.blakney@msl.ubc.ca

Michael Smith Laboratories, School of Biomedical Engineering, University of British Columbia, Vancouver, BC V6T 1Z4, Canada

16. Delivery technologies of mRNA vaccines

Yuanyu Huang - yyhuang@bit.edu.cn

School of Life Science, Advanced Research Institute of Multidisciplinary Science, Key Laboratory of Molecular Medicine and Biotherapy, Institute of Engineering Medicine, Beijing Institute of Technology, Beijing 100081, PR China

17. Nanosystems: The Key in Formulations of mRNA for Several Pathologies

Ana Santos, Ivana Jarak, Francisco Veiga, Ana Figueiras

Ana Figueiras - rfigueiras@ff.uc.pt

Faculty of Pharmacy, University of Coimbra, Azinhaga de Santa Comba, 3000-548, Coimbra, Portugal

18. Messenger RNA nanoformulation for cancer vaccine

Xiaojun XIA - xiaxj@sysucc.org.cn;  xjxia@hotmail.com

Sun Yat-sen University Cancer Center, Guangzhou, China, 510060

19. Preparation of synthetic mRNAs for in vivo applications – overview and considerations 

Siu-Hong Chan and Bijoyita Roy

Bijoyita Roy - broy@neb.com

RNA and Genome Editing, New England Biolabs Inc. Ipswich, MA 01938

20. Messenger RNA for Prophylaxis

Nick Jackson - nick.jackson@cepi.net

Coalition for Epidemic Preparedness Innovations (CEPI), Gibbs building, 215 Euston Road, Bloomsbury, London, NW1 2BE, UK

21. Nuclear export of mRNA with disease pathogenesis and therapeutic potentials

Sukesh Bhaumik - sbhaumik@siumed.edu

Department of Biochemistry and Molecular Biology, Southern Illinois University School of Medicine, Carbondale, IL 62901, USA

22. Messenger RNA Therapeutics: Start of a New Era in Medicine

Vandana Gupta - vandanagupta72@rediffmail.com

Ram Lal Anand College, University of Delhi, New Delhi 110021

23. mRNA delivery technologies for therapeutic applications

Stefano Persano 

Stefano Persano - Stefano.Persano@iit.it

Nanomaterials for Biomedical Applications, Istituto Italiano di Tecnologia (IIT), Genova, Italy 

O autorze

Professor Stefan Jurga conducts interdisciplinary scientific research in the field of nanoscience and nanotechnology, covering the physical, chemical, biological and medical sciences. He has authored over 270 publications in the interdisciplinary database Journal Citation Reports. He has served as Visiting Professor at, e.g., Cornell University, North Carolina State University, and the University of South Africa in Pretoria. He was a laureate of the Alexander von Humboldt Foundation and a scholarship holder at the Max Planck Institute for Polymer Research in Mainz (Germany). He has also worked at the University of Illinois at Urbana Champaign, at the Józef Stefan Institute in Ljubljana, and at many other international scientific institutions. He has served as Vice-Rector and Rector of the Adam Mickiewicz University Poznań (AMU) and also created the interdisciplinary NanoBioMedical Center at the AMU, which he has been the director of since 2010. From 2005 to 2007, he was appointed as the Under Secretary and Secretary of State for research and higher education at the Ministry of Education and Science and the Ministry of Science and Higher Education.

Jan Barciszewski is Professor at Adam Mickiewicz University (AMU) in Poznań, Poland and at the Institute of Bioorganic Chemistry of the Polish Academy of Sciences, Poznań, Poland where he has worked since 1974. He studied organic chemistry at the AMU. During his Ph.D. studies, he worked on the structure and function of modified bases and nucleoside sequences of plant phenylalanine specific transfer ribonucleic acid (tRNA), including cytokinins. He was subsequently granted Doctor of Science degree for his work on the properties of plant tRNAs and aminoacyl-tRNA synthetases. In the 1990s, he began working on the diagnosis and therapy of brain tumors. He developed a new method for the transformation of plant mitochondria based on catalytic RNAs and is currently involved in studies on a new type of catalytic RNAs (enantiomeric ribozymes) for efficient RNA target cleavage in vivo, as well as the search for new anti-aging agents.