Technology growth

Global RNA Therapy Market 2021 – Opportunities for Technology Growth –

DUBLIN–(BUSINESS WIRE)–The report “RNA Therapeutics: Global Technology Growth Opportunities, 2021” has been added to from offer.

RNA therapeutics is a rapidly expanding field of next-generation biologic drugs. RNA is a versatile molecule and naturally regulates gene expression, either through upregulation or downregulation. This function is used to design new RNA-based therapies. The fundamental principles of RNA therapeutics are to deliver functional RNA to a target; however, this is limited due to its fragile nature and lack of appropriate delivery systems.

This research aims to answer the following questions:

  • What are the determining factors for RNA-based therapies?

  • What are the remaining challenges and barriers to the adoption of RNA-based therapies?

  • What initiatives are industry players undertaking to accelerate adoption?

  • What are the business outcomes desired by the RNA-based therapeutics industry compared to traditional drugs?

Advances in RNA stability, chemical modification, and delivery systems have driven the commercial translation of RNA-based therapies over the past two decades. Additionally, RNAs are relatively easy to manufacture, are cost effective, and can act on non-drug targets. Traditionally, only university laboratories and small biotech companies focus on RNA-based therapeutic research.

Previously, RNA therapeutics R&D focused on rare diseases and cancer. However, recent advances in chemical modification, improved stability, formulation, and nucleic acid delivery systems have improved the prospects for RNA therapeutics, and pharmaceutical and biotechnological interest is increasing. .

mRNA-based expression of CRISPR/Cas 9 gene-editing tool and monoclonal antibodies, self-amplified RNA and activator small RNA are among the new concepts in therapeutic research on the RNA.

The initial commercialization of antisense oligonucleotides (ASO) and short interfering RNA (siRNA) therapies is very promising. Applications of mRNA in expression of missing proteins or mRNA-encoding antigens are the next area to be explored for applications in genetic diseases and expression of immunomodulatory molecules in cancer and vaccinology.

RNA therapeutics is widely applicable to tumors, neurological diseases, cardiovascular diseases, rare genetic diseases and infectious diseases. Although they have high potential to treat disease by altering gene regulation, RNAs are fragile in nature, large in size, and negatively charged require modification and formulation to improve stability, targeting, and uptake cellular. Therefore, efficient delivery of therapeutic RNA molecules to targeted cells, activation of RNA molecules and specificity without stimulating the immune response are of paramount importance for the success of RNA-based therapies.

RNA therapies consist of several types of drugs, some of which are commercially available, while most are in development. Despite a wide variety, messenger RNA therapies and RNA interference are the most studied approaches among RNA therapies. The following figure shows a variety of RNA-based therapies divided into 4 main types, then further categorizes RNA interferences and messenger RNAs.

Main topics covered:

1. Strategic imperatives

1.1 Why is it increasingly difficult to grow taller? Strategic imperative 8: Factors creating pressure on growth

1.2 The strategic imperative 8

1.3 The impact of the top three strategic imperatives on the RNA therapeutics industry

1.4 About the Growth Pipeline Engine

1.5 Growth Opportunities Fuel the Growth Pipeline Engine

2. Research Background and Summary of Findings

2.1 Research Context

2.2 Scope of the research: key questions the research will answer

2.3 Research methodology

2.4 Main Results of RNA Therapy

3. Technology Overview

3.1 RNA-based therapeutics: new emerging class of drugs

3.2 RNA-based therapeutics: a more simplified approach to standard drugs

3.3 Four main types of RNA therapies are pursued

3.4 COVID-19 pandemic: Momentum for mRNA vaccines

3.5 Key Trends in RNA Therapeutics

3.6 Stability, delivery and immunogenicity are the main technical challenges of RNA therapeutics

4. Non-coding RNA therapy: RNA interference and RNA activation

4.1 RNA interference inhibits gene expression

4.2 Late Stage Clinical Trials of Therapeutic siRNAs

4.3 Activator small RNA enhances gene expression

4.4 Trends in siRNA and small activating RNA therapeutics

4.5 microRNA: another approach to RNA interference

4.6 Opportunities in miRNA therapeutics

5. RNA antisense oligonucleotide

5.1 Presentation of the antisense oligonucleotide

5.2 Prospects in antisense oligonucleotide therapeutics

6. RNA aptamers

6.1 Aptamers as targeted therapy

6.2 Advances in RNA Aptamer Therapeutics

6.3 Prospects of RNA aptamers as therapeutics

7. mRNA therapeutics

7.1 Treatment of Single Gene Disorders with mRNA Therapeutics

7.2 mRNA-based vaccines

7.3 mRNA Vaccines in Late Stage Clinical Trials

7.4 Advances in mRNA Vaccines

7.5 Self-Amplified RNAs to Overcome mRNA Challenges

7.6 mRNA: a new way to deliver CRISPR/Cas9

7.7 Insights into self-amplified RNAs and CRISPR mRNA delivery

8. Supports Stability and Delivery of RNA Therapeutics

8.1 RNA modifications: a way to improve RNA stability

8.2 Categories of delivery formulations

9. RNA Therapeutics: Intellectual Property, Commercialization, Funding and Regional Trends

9.1 RNA Therapeutic IP

9.2 RNA Therapeutics Available in the Market

9.3 Investments and companies to watch in RNA therapy

9.4 Funding Trends and Regional Adoption of RNA Therapeutics

10. Key Business Entities in RNA Therapeutics

10.1 Alnylam Pharmaceuticals Inc. USA

10.2 Ionis Pharmaceuticals Inc. USA

10.3 BioNTech SE, Germany

10.4 Moderna Therapeutics, USA

10.5 CureVac NV, Germany

10.6 Translate biography, United States

10.7 Additional Companies to Watch in siRNA Therapeutics

10.8 Nucleic Acid Carriers Based in Canada

10.9 United States-Based Nucleic Acid Carriers

11. Growth Opportunities

11.1 Growth Opportunity: Formulation and Delivery of RNA Therapeutics, 2021

11.2 Growth Opportunity: Stability of mRNA Vaccines and RNA Therapeutics at Room Temperature, 2021

11.3 Growth Opportunity: mRNA Vaccines and Active Involvement of Big Pharma, 2021

12. Key Industry Influences

13. Next steps

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