RNA Targeting Small Molecules Therapeutics Market, 2030 – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “RNA Targeting Small Molecules Therapeutics Market by Type of Target Molecule, Type of Approach, Target Indication, Target Therapeutic Area, Route of Administration, and Key Geographical Region: Industry Trends and Global Forecasts, 2021-2030” report has been added to ResearchAndMarkets.com’s offering.

The “RNA Targeting Small Molecules Therapeutics Market” report features an extensive study of the current landscape, offering an informed opinion on the likely adoption of RNA targeting small molecule therapeutics in the pharmaceutical industry, over the next decade. The report features an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain.

One of the key objectives of the report was to estimate the existing size and the future opportunity associated with the RNA targeting small molecules therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2021-2030.

Overview

Small molecule products presently dominate the overall pharmaceutical market, representing around 80% of the total pipeline candidates. In fact, in 2020, the USFDA approved 55 drugs, of which, over 65% were small molecule therapeutics. It is worth noting that majority of these drugs were conventionally developed to target proteins.

However, reports suggest that merely 2% of the human genome is responsible for coding for proteins. Hence, the pharmaceutical industry is now focused on evaluating previously untapped targets, such as non-coding DNA and RNA; the latter molecules are expected to correspond to ~70% of the unknown portion of the human genome.

In recent past, RNA’s limitless potential as a target for small molecules, against a wide range of indications (both oncological and non-oncological), has been recognized. Specifically, a variety of small molecules that affect RNA function have been discovered.

Further, many RNA riboswitch regulatory elements (capable of binding to a variety of small molecule metabolites and control gene expression) and small molecule antibiotics (having the ability to bind to ribosomal RNA and interfere with translation) have been identified. Consequently, drug developers are experimenting with RNA modification, RNA translation, RNA splicing, indirect RNA targeting (epitranscriptomics), and other ways to target RNA with small molecules.

Currently, over 100 small molecule therapeutics, targeting naive RNA, are under development. Several experiments and clinical trials focused on RNA targeting antisense oligonucleotides (many of which have received regulatory approval), as well as preclinical studies for synthetic RNAs, which can redirect cellular RNA interference (RNAi) machinery or activate CRISPR-based systems, have demonstrated strong proof of principle for RNA targeting drugs.

Moreover, a number of industry players (mostly small firms and start-ups) have developed their proprietary technologies for direct and indirect RNA targeting. Additionally, in the past five years, more than USD 800 million was invested in companies engaged in the development of RNA targeting small molecule therapeutics (primarily those developing candidates against oncological disorders).

Many big pharma players, such as Pfizer, Novartis and Takeda Pharmaceutical, have also actively supported the initiatives taken by such drug developers, by making lucrative investments. Driven by encouraging clinical trial results, strategic collaborations and initiatives undertaken to expand expanding intellectual capital, this emerging market is expected to grow at a steady pace in the foreseen future.

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

3.1. Chapter Overview

3.2. RNA Targeting Therapeutics

3.3. RNA Targets for Small Molecules

3.4. Approaches Used to Target RNA

3.5. Emerging Technologies

3.6. Future Perspective

4. MARKET OVERVIEW

4.1. Chapter Overview

4.2. RNA Targeting Small Molecule Therapeutics: Clinical-stage and Marketed Drug Candidates

4.3. RNA Targeting Small Molecule Therapeutics: Preclinical-stage Drug Candidates

4.4. RNA Targeting Small Molecule Therapeutics: Developer Landscape

5. COMPANY COMPETITIVENESS AND INVESTMENTS LANDSCAPE

5.1. Chapter Overview

5.2. Assumptions and Key Parameters

5.3. RNA Targeting Small Molecule Therapeutics Developers: Competitiveness Analysis

5.4. RNA Targeting Small Molecule Therapeutics Developers: Investment Analysis

5.5. Short Profiles of Top Five Players

5.5.1. PTC Therapeutics

5.5.2. AC Immune

5.5.3. Skyhawk Therapeutics

5.5.4. Abivax

5.5.5. eFFECTOR Therapeutics

6. COMPANY PROFILES

6.1. Chapter Overview

6.2. Abivax

6.2.1. Company Overview

6.2.2. Recent Developments and Future Outlook

6.3. AC immune

6.4. Arrakis Therapeutics

6.5. eFFECTOR Therapeutics

6.6. Eloxx Pharmaceuticals

6.7. H3 Biomedicine

6.8. PTC Therapeutics

6.9. Ribometrix

6.10. Skyhawk Therapeutics

6.11. STORM Therapeutics

7. RECENT COLLABORATIONS AND PARTNERSHIPS

7.1. Chapter Overview

7.2. Partnership Models

7.3. RNA Targeting Small Molecule Therapeutics: Recent Partnerships

8. FUNDING AND INVESTMENT ANALYSIS

8.1. Chapter Overview

8.2. Type of Funding

8.3. RNA Targeting Small Molecule Therapeutics: Funding and Investment Analysis

8.4. Concluding Remarks

9. MARKET SIZING AND OPPORTUNITY ANALYSIS

9.1. Chapter Overview

9.2. Scope and Limitations

9.3. Key Assumptions and Forecast Methodology

9.4. Global RNA Targeting Small Molecule Therapeutics Market, 2021-2030

• Translarna (Ataluren, PTC127), PTC Therapeutics
• Risdiplam, PTC Therapeutics
• Tomivosertib (eFT508), eFFECTOR Therapeutics
• ELX-02, Eloxx Pharmaceuticals
• H3B-6545, H3 Biomedicine
• Translarna (Ataluren, PTC124), PTC Therapeutics
• ABX464 (Monotherapy), Abivax
• ABX464 and Methotrexate, Abivax

10. CONCLUSION

11. EXECUTIVE INSIGHTS

11.1. Chapter Overview

11.2. Novation Pharmaceutials

11.2.1. Company Overview

11.2.2. Dominique Cheneval, President/Co-Founder

11.3. Anima Biotech

11.3.1. Company Overview

11.3.2. Clara Assouline, Business Development

12. APPENDIX 1: TABULATED DATA

13. APPENDIX II: LIST OF COMPANIES AND ORGANIZATION

For more information about this report visit https://www.researchandmarkets.com/r/44e6ng

Contacts

ResearchAndMarkets.com

Laura Wood, Senior Press Manager

[email protected]

For E.S.T Office Hours Call 1-917-300-0470

For U.S./CAN Toll Free Call 1-800-526-8630

For GMT Office Hours Call +353-1-416-8900