Main description:

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.

Contents:

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 Tecnologia Farmaceutica, 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 O, 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