Advances in RNA Modifications, Formulation, and Stabilization to Drive Growth of RNA Therapeutics
This research is intended to answer the following questions:
- What are the driving factors for RNA therapeutics?
- What challenges and impediments remain to the adoption of RNA therapeutics?
- What initiatives are industry participants undertaking to accelerate adoption?
- What are the RNA therapeutics industry-desired business outcomes compared to traditional drugs?
RNA therapeutics is a rapidly expanding area of next-generation biological drugs. RNA is a versatile molecule and naturally regulates gene expression, either by upregulation or downregulation. This function is employed to design novel RNA therapeutics. The fundamentals of RNA therapeutics consists of delivering functional RNA at a target; however, this is restricted due to its fragile nature and lack of appropriate delivery systems.
Advances in RNA stability, chemical modification, and delivery systems have led to the commercial translation of RNA therapeutics during the past two decades. Moreover, RNAs are relatively easy to manufacture, are cost-effective, and can act on undruggable targets. Conventionally, only academic labs and small biotech companies focus on RNA therapeutics research.
Previously, RNA therapeutics R&D was focused on rare diseases and cancer. However, recent advances in chemical modification, improved stability, formulation, and nucleic acid delivery systems have improved the prospects of RNA therapeutics, and pharmaceutical and biotechnology interest are on the rise.
mRNA-based expression of CRISPR/Cas 9 gene editing tool and monoclonal antibodies, self-amplifying RNA, and small activating RNA are some of the new concepts in RNA therapeutic research.
Initial commercialization of antisense oligonucleotides (ASOs) and short interfering RNA (siRNA)-based therapeutics shows great promise. mRNA applications in the expression of missing proteins or mRNA encoding antigens is the next area to be explored for applications into 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. Despite having high potential for treating disease through altering gene regulation, RNAs are fragile in nature, large in size, and negative charge needs modification and formulation to improve stability, targeting, and cell uptake. Therefore, efficient delivery of therapeutic RNA molecules to targeted cells, activation of RNA molecules, and specificity without stimulating immune response are of paramount importance for the success of RNA therapeutics.
RNA therapeutics consist of several types of drugs, some of which are commercially available, while most are in the development phase. Despite a wide variety, messenger RNA therapeutics and RNA interference are the most widely studied approaches among RNA therapeutics. The following figure shows a variety of RNA therapeutics divided into 4 major types, and then it further classifies RNA interference and messenger RNAs.
Table of Contents
1. Strategic Imperatives
1.1 Why Is It Increasingly Difficult to Grow? The 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 Context and Summary of Findings
2.1 Research Context
2.2 Research Scope: Key Questions the Research Will Answer
2.3 Research Methodology
2.4 Key Findings in RNA Therapeutics
3. Technology Snapshot
3.1 RNA Therapeutics: Emerging Novel Class of Drugs
3.2 RNA Therapeutics: A More Simplified Approach to Standard Drugs
3.3 Four Major Types of RNA Therapeutics Are Being 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 Key Technical Challenges in RNA Therapeutics
4. Non-coding RNA Therapeutics: RNA Interference and RNA Activation
4.1 RNA Interference Inhibits Gene Expression
4.2 siRNA Therapeutics Late-stage Clinical Trials
4.3 Small Activating RNA Enhances Gene Expression
4.4 Trends in siRNA and Small Activating RNA Therapeutics
4.5 microRNA: Another Approach of RNA Interference
4.6 Opportunities in miRNA Therapeutics
5. RNA Antisense Oligonucleotide
5.1 Overview of Antisense Oligonucleotide
5.2 Prospects in Antisense Oligonucleotide Therapeutics
6. RNA Aptamers
6.1 Aptamers as Targeted Therapeutics
6.2 Advances in RNA Aptamer Therapeutics
6.3 Outlook of RNA Aptamers as Therapeutics
7. mRNA Therapeutics
7.1 Treating Monogenic Disorders Through mRNA Therapeutics
7.2 mRNA-based Vaccines
7.3 mRNA Vaccines in Late-stage Clinical Trials
7.4 Advancements in mRNA Vaccines
7.5 Self-amplifying RNAs to Overcome Challenges of mRNA
7.6 mRNA: A Novel Way to Deliver CRISPR/Cas9
7.7 Insights in Self-amplifying RNAs and CRISPR-mRNA Delivery
8. RNA Therapeutics Stability & Delivery Carriers
8.1 RNA Modifications: A Way to Improve RNA Stability
8.2 Categories of Delivery Formulations
9. RNA Therapeutics: IP, Commercialization, Funding, and Regional Trends
9.1 RNA Therapeutics IP
9.2 RNA Therapeutics Available on Market
9.3 Investments and Companies to Watch In RNA Therapeutics
9.4 Funding Trends and Regional Adoption of RNA Therapeutics
10. Key Commercial Entities in RNA Therapeutics
10.1 Alnylam Pharmaceuticals Inc. US
10.2 Ionis Pharmaceuticals Inc. US
10.3 BioNTech SE, Germany
10.4 Moderna Therapeutics, US
10.5 CureVac NV, Germany
10.6 Translate Bio, US
10.7 Additional Companies to Watch in siRNA Therapeutics
10.8 Canada-based Nucleic Acid Delivery Carriers
10.9 US-based Nucleic Acid Delivery 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 Ambient Temperature, 2021
11.3 Growth Opportunity: mRNA Vaccines and Active Participation of Big Pharmaceutical Companies, 2021
12. Key Industry Influencers
12.1 Industry Interactions
13. Next Steps
13.1 Your Next Steps
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Alnylam Pharmaceuticals Inc.
- BioNTech SE
- CureVac N.V.
- Ionis Pharmaceuticals Inc.
- Moderna Therapeutics
- Translate Bio
