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Market Research Report

mRNA Vaccines & Therapeutics 2017: An Industry Analysis of Technologies, Pipelines, Stakeholders and Deals

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mRNA Vaccines & Therapeutics 2017: An Industry Analysis of Technologies, Pipelines, Stakeholders and Deals
Published: June 22, 2017 Content info: 260 Pages
Description

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 VaccinesInfectious Disease VaccinesIn vivo TherapeuticsGene Editing
  • Czech Republic
  • 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

Companies mentioned in the report:

  • Acuitas Therapeutics
  • Alexion Pharmaceuticals
  • Arbutus Biopharma
  • Arcturus Therapeutics
  • Argos Therapeutics
  • AstraZeneca
  • Bayer
  • BioNTech
  • Boehringer Ingelheim
  • CRISPR Therapeutics
  • CureVac
  • eTheRNA immunotherapies
  • Ethris
  • GlaxoSmithKline Vaccines
  • In-Cell-Art
  • Intellia Therapeutics
  • Janssen
  • Kernal Biologics
  • MaxCyte
  • Moderna Therapeutics
  • Novartis
  • PhaseRx
  • Precision NanoSystems
  • RaNa Therapeutics
  • Regeneron Pharmaceuticals
  • Roche
  • Sangamo Therapeutics
  • Sanofi
  • Siemens
  • Silence Therapeutics
  • Synthetic Genomics
  • Takeda Pharmaceutical Co
  • Tiba Biotechnology
  • TriLink BioTechnologies
  • Ultragenyx Pharmaceutical
  • Vertex Pharmaceuticals
  • ZIOPHARM Oncology
Table of Contents
Product Code: LMFR0023

Table of Contents

1. Executive Summary

2. Introduction & Overview

3. Profiles of mRNA-based Vaccines & Therapeutics

  • 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. Pipeline Analysis

  • 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. Profiles of Selected mRNA & Delivery Technologies

  • 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. Technology Analysis

  • 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. Company Profiles

  • 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. Stakeholder Analysis

  • 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. mRNA Manufacturing

  • 9.1. In-house mRNA Manufacturing
  • 9.2. Out-sourced mRNA Manufacturing

10. Financial Perspective on mRNA

  • 10.1. mRNA Partnering Deals
  • 10.2. Funded mRNA Programs

11. Outlook

12. References

List of Tables:

  • Table 1: mRNA Modalities and Number of Programs per R&D Phase
  • Table 2: Pipeline of Standardized mRNA Cancer Vaccines
  • Table 3: Pipeline of Individualized mRNA Cancer Vaccines
  • Table 4: Pipeline of Therapeutic mRNA Infectious Disease Vaccines
  • Table 5: Pipeline of Prophylactic mRNA Infectious Disease Vaccines
  • Table 6: Pipeline of Replicon RNA Infectious Disease Vaccines
  • Table 7: Pipeline of mRNA Protein Therapeutics for Cancer and CV Diseases
  • Table 8: Pipeline of mRNA Therapeutics for OTC Deficiency
  • Table 9: Pipeline of mRNA Therapeutics for Cystic Fibrosis
  • Table 10: Pipeline of mRNA Protein Therapeutics for Other Genetic Diseases
  • Table 11: Pipeline of mRNA Antibody Therapeutics
  • Table 12: Pipeline of mRNA for ex vivo Gene Editing
  • Table 13: Pipeline of mRNA for in vivo Gene Editing
  • Table 14: Pipeline of mRNA for ex vivo T-Cell Engineering
  • Table 15: Examples of mRNA Modifications
  • Table 16: Efficiency of mRNA +/- Carrier in Preclinical Models
  • Table 17: Adjuvant/Stimulant & Carrier Technologies for mRNA-based Vaccines
  • Table 18: Adjuvant/Stimulant & Carrier Technologies for mRNA-based Vaccines
  • Table 19: mRNA Therapeutics Pipeline of Arcturus Therapeutics
  • Table 20: BioNTech's Pipeline of mRNA-based Vaccines & Therapeutics
  • Table 21: CureVac's Pipeline of mRNA-Based Vaccines and Therapeutics
  • Table 22: Moderna's R&D Pipeline of Infectious Disease mRNA Vaccines
  • Table 23: Moderna Therapeutics R&D Pipeline in Immuno-Oncology
  • Table 24: Moderna Therapeutics R&D Pipeline of mRNA Therapeutics
  • Table 25: Ex vivo Gene Editing Programs of CRISPR Therapeutics
  • Table 26: In vivo Gene Editing Programs of CRISPR Therapeutics
  • Table 27: Alexion Pharmaceuticals' mRNA Therapeutics Pipeline
  • Table 28: mRNA-based R&D Programs of Vertex Pharmaceuticals
  • Table 29: Summary Profiles of mRNA Vaccines & Therapeutics Companies
  • Table 30: R&D Focus of Major mRNA Players
  • Table 31: Technology Profiles of Small mRNA Therapeutics Companies
  • Table 32: mRNA Companies as Participants in Consortium R&D
  • Table 33: Biopharmaceutical Companies and Partnered mRNA Programs
  • Table 34: Big Pharma Companies and Partnered mRNA Programs
  • Table 35: mRNA Companies and Manufacturing Solutions
  • Table 36: Financial Terms of Partnering Deals with mRNA Companies
  • Table 37: Funded mRNA Vaccines & Therapeutics Programs
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