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mRNA Vaccines & Therapeutics 2017: an industry analysis of technologies, pipelines, stakeholders and deals

June 2017 | 260 pages | ID: MB07AC7B4ABEN
La Merie Publishing

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mRNA Vaccines & Therapeutics 2017: an industry analysis of technologies, pipelines, stakeholders and deals

For a long time, messenger RNA (mRNA) was thought to be a poor choice for a therapeutic agent given its relatively short half-life and its immunogenicity. But mRNA is rather versatile and offers a range of advantages. mRNA lacks genomic integration and its use results in transient expression of the encoded protein. This favorable safety profile makes mRNA especially attractive for vaccines and gene editing. mRNA is well defined chemically which ensures reproducible manufacturing at high yield, purity and activity. Improvements of lipid nanoparticle formulations as a vehicle for in vivo systemic delivery of mRNA has greatly favored the development of in vivo transfection strategies.

Range of clinical mRNA applications
  • Cancer Vaccines
  • Infectious Disease Vaccines
  • In vivo Therapeutics
  • Gene Editing
  • Standardized preselected
  • Individualized shared antigens
  • Individualized neoantigens
  • Prophylactic vaccines
  • Therapeutic vaccines
  • Synthetic self-amplifying mRNA vaccines for pandemic outbreaks
  • Therapeutic proteins
  • Therapeutic antibodies
  • Ex vivo gene editing of gene defects
  • In vivo gene editing of gene defects
  • Ex vivo gene editing of autologous and allogeneic T-cells
mRNA Technologies have attracted more than US$ 3.4 bln in equity financing and frontloaded partnership payments. Further billions of US$ have been committed to mRNA R&D funding and potential milestone payments. A select group of major pharmaceutical and rare disease specialist biopharmaceutical companies have partnered with mRNA technology companies. At least 16 mRNA vaccines and therapeutics are in clinical stages and a considerable number is in IND or pre-IND stage.

This report mRNA Vaccines & Therapeutics 2017: an industry analysis of technologies, pipelines, stakeholders and deals as of June 2017 brings you up-to-date regarding key mRNA players, key mRNA technologies and applications, mRNA vaccines & therapeutics, business projects, business deals and funding opportunities. The report analyzes the mRNA vaccine and therapeutic pipelines and stakeholders in the field, especially technology companies and rare disease biopharma and major pharmaceutical companies. The report highlights the value of mRNA vaccines and therapeutics in terms of partnering economic condition and equity financing rounds.

What will you find in the report?
  • Profiles of standardized therapeutic cancer mRNA vaccines
  • Profiles of individualized therapeutic cancer mRNA vaccines
  • Profiles of therapeutic infectious disease mRNA vaccines
  • Profiles of prophylactic infectious disease mRNA vaccines
  • Profiles of mRNA protein therapeutics for immuno-oncology
  • Profiles mRNA gene editing products
  • Profiles of mRNA protein therapeutics for monogenetic rare diseases
  • Pipelines of cancer and infectious disease mRNA vaccines
  • Pipelines of mRNA Therapeutics for OTC deficiency and cystic fibrosis
  • mRNA antibody therapeutics
  • Therapeutic mRNA gene editing
  • Ex vivo mRNA T-cell engineering
  • Profiles of mRNA and delivery technologies
  • mRNA technology analysis
  • Profiles of mRNA stakeholder companies
  • mRNA stakeholder analysis
  • mRNA manufacturing
  • Financial perspective on mRNA



3.1 Standardized Therapeutic Cancer mRNA Vaccines&
  3.1.1 BI-1361849; CV9202 & CV9201&
  3.1.2 CV9104 & CV9103&
  3.1.3 Tetravalent Lipo-MERIT Vaccine&
  3.1.4 TriMixDC-Mel&
3.2 Individualized Therapeutic Cancer mRNA Vaccines&
  3.2.1 IVAC Mutanome&
  3.2.2 IVAC Warehouse&
  3.2.3 mRNA-4157&
  3.2.4 Rocapuldencel-T; AGS-003&
3.3 Therapeutic Infectious Disease mRNA Vaccines&
  3.3.1 AGS-004&
3.4 Prophylactic Infectious Disease mRNA Vaccines & Adjuvants&
  3.4.1 CV7201&
  3.4.2 MRK-1777&
  3.4.3 mRNA-1325&
  3.4.4 mRNA-1388&
  3.4.5 mRNA-1440; VAL-506440&
  3.4.6 mRNA-1647&
  3.4.7 mRNA-1653&
  3.4.8 mRNA-1706&
  3.4.9 mRNA-1851; VAL-339851&
  3.4.10 RNAdjuvant; CV8102&
3.5 mRNA in Immuno-Oncology&
  3.5.1 CARMA-hMeso&
  3.5.2 CTX101&
  3.5.3 mRNA-2416&
  3.5.4 mRNA-2905&
3.6 mRNA Therapeutic for Monogenetic Diseases&
  3.6.1 CRISPR/Cas9 TTR mRNA-LNP&
  3.6.2 PRX-ASL&
  3.6.3 PRX-ASS1&
  3.6.4 PRX-OTC&
3.7 mRNA Therapeutic for Other Diseases&
  3.7.1 AZD8601&


4.1 Standardized mRNA Cancer Vaccines&
4.2 Individualized mRNA Cancer Vaccines&
4.3 Therapeutic mRNA Infectious Disease Vaccines&
4.4 Prophylactic mRNA Infectious Disease Vaccines&
4.5 Replicon RNA Infectious Disease Vaccines&
4.6 mRNA Protein Therapeutics for Cancer and Cardiovascular Diseases&
4.7 mRNA Protein Therapeutics for Ornithine TransCarbomylase (OTC) Deficiency&
4.8 mRNA Protein Therapeutics for Cystic Fibrosis&
4.9 mRNA Protein Therapeutics for Other Genetic Diseases&
4.10 mRNA Antibody Therapeutics&
4.11 Therapeutic mRNA-based Gene Editing&
4.12 Ex vivo mRNA-based T-Cell Engineering&


5.1 Ex vivo mRNA Loading&
  5.1.1 Arcelis Technology&
  5.1.2 Flow Electroporation Technology&
5.2 In vivo Carrier & mRNA&
  5.2.1 Hybrid mRNA Technology&
  5.2.2 Lipid Nanoparticle (LNP) mRNA Technology&
  5.2.3 Nanotaxi Technology&
  5.2.4 RNArt & RNAntibody Technology&
  5.2.5 UNA & LUNAR Technologies&
5.3 mRNA Vaccines & Stimulants / Adjuvants&
  5.3.1 RNActive Technology&
  5.3.2 Self-Amplifying mRNA (SAM) Technology&
  5.3.3 TriMix Technology&


6.1 mRNA Modifications&
6.2 mRNA Efficiencies&
6.3 Adjuvant/Stimulant & Carrier Technologies for mRNA-based Vaccines&
6.4 Carriers for mRNA-based Protein & Antibody Therapeutics&


7.1 Companies focused on mRNA Therapeutics and Vaccines&
  7.1.1 Arcturus Therapeutics&
  7.1.2 Argos Therapeutics&
  7.1.3 BioNTech&
  7.1.4 CureVac&
  7.1.5 eTheRNA immunotherapies&
  7.1.6 Ethris&
  7.1.7 In-Cell-Art&
  7.1.8 Kernal Biologics&
  7.1.9 Moderna Therapeutics&
  7.1.10 PhaseRx&
  7.1.11 RaNa Therapeutics&
7.2 Companies with a focus on self-amplifying mRNA (Replicon RNA)&
  7.2.1 GlaxoSmithKline Vaccines&
  7.2.2 Synthetic Genomics&
  7.2.3 Tiba Biotechnology&
7.3 Companies with a focus on mRNA Delivery&
  7.3.1 Acuitas Therapeutics&
  7.3.2 Arbutus Biopharma&
  7.3.3 Silence Therapeutics&
7.4 Companies with a focus on Gene Editing and CAR T-Cells&
  7.4.1 CRISPR Therapeutics & Casebia Therapeutics&
  7.4.2 Intellia Therapeutics&
  7.4.3 MaxCyte&
  7.4.4 Sangamo Therapeutics&
  7.4.5 ZIOPHARM Oncology&
7.5 Companies focused on Contract Manufacturing of mRNA&
  7.5.1 TriLink BioTechnologies&
  7.5.2 Precision NanoSystems&
7.6 Biopharmaceutical Companies with mRNA Programs&
  7.6.1 Alexion Pharmaceuticals&
  7.6.2 Regeneron Pharmaceuticals&
  7.6.3 Ultragenyx Pharmaceutical&
  7.6.4 Vertex Pharmaceuticals&
7.7 Major Pharmaceutical Companies with mRNA Programs&
  7.7.1 AstraZeneca&
  7.7.2 Bayer&
  7.7.3 Boehringer Ingelheim&
  7.7.4 Janssen&
  7.7.5 Merck&
  7.7.6 Roche&
  7.7.7 Sanofi&
  7.7.8 Takeda Pharmaceutical Co.&


8.1 mRNA Technology Companies&
  8.1.1 Companies Focused on mRNA Vaccines & Therapeutics&
  8.1.2 Companies Focused on Self-Amplifying mRNA&
  8.1.3 Companies Focused on mRNA Delivery&
  8.1.4 Companies Focused on Gene Editing & CAR T-Cells by Use of mRNA&
8.2 Pharmaceutical Companies with mRNA Programs&
  8.2.1 Rare Disease Biopharmaceutical Companies with mRNA Programs&
  8.2.2 Major Pharmaceutial Companies with mRNA Programs&


9.1 In-house mRNA Manufacturing&
9.2 Out-sourced mRNA Manufacturing&


10.1 mRNA Partnering Deals&
10.2 Funded mRNA Programs&



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