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
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 |
- Prophylactic 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.
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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
mRNA Vaccines & Therapeutics 2017: an industry analysis of technologies, pipelines, stakeholders and deals
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
mRNA Vaccines & Therapeutics 2017: an industry analysis of technologies, pipelines, stakeholders and deals
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
mRNA Vaccines & Therapeutics 2017: an industry analysis of technologies, pipelines, stakeholders and deals
TABLES in the Text
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