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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

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    Report

  • 265 Pages
  • August 2021
  • Region: Global
  • Roots Analysis
  • ID: 5450294

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 naïve 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.


Scope of the Report

The “RNA Targeting Small Molecules Therapeutics Market by Type of Target Molecule (Cap Binding Complex, CDKL5, Dystrophin Protein, Erα, G542X, MNK1/MNK2, PAX6 gene, SMN2), Type of Approach (Indirect RNA Targeting- Epitranscriptomics, mRNA Translation Modulation, RNA Splicing Modification), Target Indication (Aniridia, Breast Cancer, Castrate-resistant Prostate Cancer, Colorectal Cancer, Crohn Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Dravet Syndrome, Epilepsy, HIV Infections, Impaired Renal Function, Non-Small Cell Lung Cancer, Rheumatoid Arthritis, Spinal Muscular Atrophy, Ulcerative Colitis), Target Therapeutic Area (Autoimmune Disorders, Genetic Disorders, Infectious Disorders, Inflammatory Disorders, Neurological Disorders, Oncological Disorders, Ophthalmic Disorders, Rare Disorders, Renal Disorders, Respiratory Disorders), Route of Administration (Oral and Subcutaneous Injection) and Region (North America, Europe, Asia, Middle East and North Africa, Latin America and Rest of the World): Industry Trends and Global Forecasts, 2021-2030” 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. 


Amongst other elements, the report includes:

  • A detailed overview of the current market landscape of RNA targeting small molecule therapeutics, including information on phase of development (clinical and preclinical), target disease indication(s), therapeutic area (non-oncological disorders and oncological disorders), target molecule (BMI1, cap binding complex, eLF4A, MNK1/MNK2, VEGF, others), type of approach (indirect RNA targeting – epitranscriptomics, mRNA translation modulation, RNA splicing modification) and route of administration (intravenous, subcutaneous, and oral). In addition, it highlights the developers of these novel therapeutics, along with information on their year of establishment, company size (in terms of employee count), location of headquarters and number of proprietary drug candidates.
  • An in-depth analysis of the company competitiveness and investment landscape, including a bubble analysis comparing the key players engaged in the domain of RNA targeting small molecules, based on several relevant parameters (such as company experience, pipeline strength, partnership activity, funding activity, patents filed and company size) and a multivariate representation illustrating the start-ups and investment activity based on various multiple parameters (such as funding type, therapeutic area and amount invested).
  • Elaborate profiles of key players engaged in the development of RNA targeting small molecule therapeutics. Each profile features a brief overview of the company, recent developments and an informed future outlook.
  • An analysis of the recent collaborations and partnerships that have been inked between various stakeholders in this domain, during the period between 2017 and 2021, covering R&D agreements, clinical trial agreements, acquisitions, licensing agreements and product development agreements.
  • An analysis of the investments made, during the period between 2015 and 2021, including grant, seed financing, venture capital financing, initial public offering (IPO), debt financing and other equity, in companies that are engaged in the development of RNA targeting small molecule therapeutics.

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. The report also provides details on the likely distribution of the current and forecasted opportunity across [A] type of target molecule (cap binding complex, CDKL5, dystrophin protein, Erα, G542X, MNK1/MNK2, PAX6 gene, SMN2) [B] type of approach (indirect RNA targeting- epitranscriptomics, mRNA translation modulation, RNA splicing modification) [C] target indication (aniridia, breast cancer, castrate-resistant prostate cancer, colorectal cancer, Crohn disease, cystic fibrosis, Duchenne muscular dystrophy, Dravet syndrome, epilepsy, HIV infections, impaired renal function, non-small cell lung cancer, rheumatoid arthritis, spinal muscular atrophy, ulcerative colitis) [D] target therapeutic area (autoimmune disorders, genetic disorders, infectious disorders, inflammatory disorders, neurological disorders, oncological disorders, ophthalmic disorders, rare disorders, renal disorders, respiratory disorders), [E] route of administration (oral and subcutaneous injection) and [E] region (North America, Europe, Asia, Middle East and North Africa, Latin America and Rest of the World). In order to account for future uncertainties and to add robustness to our forecast model, we have provided three forecast scenarios, portraying the conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.


The opinions and insights presented in the report were also influenced by discussions held with multiple stakeholders in this domain. 


The report features detailed transcripts of interviews held with the following individuals (in alphabetical order):

  • Clara Assouline (Business Development, Anima Biotech)
  • Dominique Cheneval (President/Co-Founder, Novation Pharmaceuticals)

All actual figures have been sourced and analyzed from publicly available information forums. Financial figures mentioned in this report are in USD, unless otherwise specified.


Key Questions Answered

  • Who are the key players engaged in the development of RNA targeting small molecule therapeutics?
  • What are the current investment trends in this field?
  • Which are the key therapeutic areas for which RNA targeting small molecule therapies are being developed?
  • What are the different technological approaches adopted by various players for the development of RNA targeting small molecule therapeutics?
  • What type of partnership models are most commonly adopted by stakeholders engaged in this domain?
  • What is the relative competitiveness of different RNA targeting small molecule therapeutics developers engaged in this market?
  • How is the current and future market opportunity likely to be distributed across key market segments?

Table of Contents

1. PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Key Questions Answered
1.4. Chapter Outlines


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
3.4.1. mRNA Translation Regulation
3.4.2. RNA Splicing Modification
3.4.3. RNA Targeting
3.4.3.1. Direct RNA Targeting
3.4.3.2 .Indirect RNA Targeting (Epitranscriptomics)
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
4.2.1. Analysis by Phase of Development
4.2.2. Analysis by Target Disease Indication(s)
4.2.3. Analysis by Phase of Development and Target Disease Indication(s)
4.2.4. Analysis by Therapeutic Area
4.2.5. Analysis by Phase of Development and Therapeutic Area
4.2.6. Analysis by Target Molecule
4.2.7. Analysis by Phase of Development and Target Molecule
4.2.8. Analysis by Type of Approach
4.2.9. Analysis by Phase of Development and Type of Approach
4.2.10. Analysis by Route of Administration
4.3. RNA Targeting Small Molecule Therapeutics: Preclinical-stage Drug Candidates
4.3.1. Analysis by Phase of Development
4.3.2. Analysis by Target Disease Indication(s)
4.3.3. Analysis by Phase of Development and Target Disease Indication(s)
4.3.4. Analysis by Therapeutic Area
4.3.5. Analysis by Phase of Development and Therapeutic Area
4.3.6. Analysis by Target Molecule
4.3.7. Analysis by Phase of Development and Target Molecule
4.3.8. Analysis by Type of Approach
4.3.9. Analysis by Phase of Development and Type of Approach
4.4. RNA Targeting Small Molecule Therapeutics: Developer Landscape
4.4.1. Analysis by Year of Establishment
4.4.2. Analysis by Company Size
4.4.3. Analysis by Location of Headquarters
4.4.4. Most Active Players: Analysis by Number of Proprietary Drug Candidates


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
6.3.1. Company Overview
6.3.2. Recent Developments and Future Outlook
6.4. Arrakis Therapeutics
6.4.1. Company Overview
6.4.2. Recent Developments and Future Outlook
6.5. eFFECTOR Therapeutics
6.5.1. Company Overview
6.5.2. Recent Developments and Future Outlook
6.6. Eloxx Pharmaceuticals
6.6.1. Company Overview
6.6.2. Recent Developments and Future Outlook
6.7. H3 Biomedicine
6.7.1. Company Overview
6.7.2. Recent Developments and Future Outlook
6.8. PTC Therapeutics
6.8.1. Company Overview
6.8.2. Recent Developments and Future Outlook
6.9. Ribometrix
6.9.1. Company Overview
6.9.2. Recent Developments and Future Outlook
6.10. Skyhawk Therapeutics
6.10.1. Company Overview
6.10.2. Recent Developments and Future Outlook
6.11. STORM Therapeutics
6.11.1. Company Overview
6.11.2. Recent Developments and Future Outlook


7. RECENT COLLABORATIONS AND PARTNERSHIPS
7.1. Chapter Overview
7.2. Partnership Models
7.3. RNA Targeting Small Molecule Therapeutics: Recent Partnerships
7.3.1. Analysis by Year of Partnership
7.3.2. Analysis by Type of Partnership
7.3.3. Analysis by Year and Type of Partnership
7.3.4. Analysis by Target Indication(s)
7.3.5. Analysis by Type of Partnership and Target Indication(s)
7.3.6. Analysis by Therapeutic Area
7.3.7. Analysis by Type of Partnership and Therapeutic Area
7.3.8. Most Active Players: Analysis by Number of Partnerships
7.3.9. Regional Analysis
7.3.9.1. Country-wise Distribution
7.3.9.2. Region-wise Distribution


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
8.3.1. Cumulative Year-wise Trend, 2015-2021
8.3.2. Analysis by Amount Invested
8.3.3. Analysis by Type of Funding
8.3.4. Analysis by Phase of Development
8.3.5. Analysis by Therapeutic Area
8.3.6. Analysis by Region
8.3.6.1. Most Active Players: Analysis by Number of Funding Instances
8.3.6.2. Most Active Players: Analysis by Amount of Funding
8.3.7. Most Active Investors: Distribution by Number of Funding Instances
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
9.4.1. RNA Targeting Small Molecule Therapeutics Market: Analysis by Target Disease Indication, 2021 and 2030
9.4.1.1. RNA Targeting Small Molecule Therapeutics Market for Aniridia, 2021-2030
9.4.1.2. RNA Targeting Small Molecule Therapeutics Market for Breast Cancer, 2021-2030
9.4.1.3. RNA Targeting Small Molecule Therapeutics Market for Castrate-resistant Prostate Cancer, 2021-2030
9.4.1.4. RNA Targeting Small Molecule Therapeutics Market for Colorectal Cancer, 2021-2030
9.4 1.5. RNA Targeting Small Molecule Therapeutics Market for Crohn Disease, 2021-2030
9.4.1.6. RNA Targeting Small Molecule Therapeutics Market for Cystic Fibrosis, 2021-2030
9.4.1.7. RNA Targeting Small Molecule Therapeutics Market for Duchenne Muscular Dystrophy, 2021-2030
9.4 1.8. RNA Targeting Small Molecule Therapeutics Market for Dravet Syndrome, 2021-2030
9.4.1.9. RNA Targeting Small Molecule Therapeutics Market for Epilepsy, 2021-2030
9.4.1.10 RNA Targeting Small Molecule Therapeutics Market for HIV Infections, 2021-2030
9.4.1.11 RNA Targeting Small Molecule Therapeutics Market for Impaired Renal Function, 2021-2030
9.4.1.12. RNA Targeting Small Molecule Therapeutics Market for Non-Small Cell Lung Cancer, 2021-2030
9.4 1.13 RNA Targeting Small Molecule Therapeutics Market for Rheumatoid Arthritis, 2021-2030
9.4 1.14 RNA Targeting Small Molecule Therapeutics Market for Spinal Muscular Atrophy, 2021-2030
9.4 1.15 RNA Targeting Small Molecule Therapeutics Market for Ulcerative Colitis, 2021-2030
9.4.2. RNA Targeting Small Molecule Therapeutics Market: Analysis by Key Therapeutic Areas, 2021 and 2030
9.4.2.1. RNA Targeting Small Molecule Therapeutics Market for Autoimmune Disorders, 2021-2030
9.4.2.2. RNA Targeting Small Molecule Therapeutics Market for Genetic Disorders, 2021-2030
9.4.2.3. RNA Targeting Small Molecule Therapeutics Market for Inflammatory Disorders, 2021-2030
9.4.2.4. RNA Targeting Small Molecule Therapeutics Market for Infectious Disorders, 2021-2030
9.4.2.5. RNA Targeting Small Molecule Therapeutics Market for Neurological Disorders, 2021-2030
9.4.2.6. RNA Targeting Small Molecule Therapeutics Market for Oncological Disorders, 2021-2030
9.4.2.7. RNA Targeting Small Molecule Therapeutics Market for Ophthalmic Disorders, 2021-2030
9.4.2.8. RNA Targeting Small Molecule Therapeutics Market for Rare Disorders, 2021-2030
9.4.2.9. RNA Targeting Small Molecule Therapeutics Market for Renal Disorders, 2021-2030
9.4.2.10. RNA Targeting Small Molecule Therapeutics Market for Respiratory Disorders, 2021-2030
9.4.3. RNA Targeting Small Molecule Therapeutics Market: Analysis by Target Molecule, 2021 and 2030
9.4.3.1. RNA Targeting Small Molecule Therapeutics Market for CDKL5, 2021-2030
9.4.3.2. RNA Targeting Small Molecule Therapeutics Market for Cap Binding Complex, 2021-2030
9.4.3.3. RNA Targeting Small Molecule Therapeutics Market for Dystrophin Protein, 2021-2030
9.4.3.4. RNA Targeting Small Molecule Therapeutics Market for Estrogen Receptor Alpha, 2021-2030
9.4.3.5. RNA Targeting Small Molecule Therapeutics Market for G542X, 2021-2030
9.4.3.6. RNA Targeting Small Molecule Therapeutics Market for MNK1/MNK2, 2021-2030
9.4.3.7. RNA Targeting Small Molecule Therapeutics Market for PAX6 Gene, 2021-2030
9.4.3.8. RNA Targeting Small Molecule Therapeutics Market for SMN2, 2021-2030
9.4.4. RNA Targeting Small Molecule Therapeutics Market: Analysis by Route of Administration, 2021 and 2030
9.4.4.1. RNA Targeting Small Molecule Therapeutics Market for Oral Drugs, 2021-2030
9.4.4.2. RNA Targeting Small Molecule Therapeutics Market for Subcutaneous Injections, 2021-2030
9.4.5. RNA Targeting Small Molecule Therapeutics Market: Analysis by Geography, 2021 and 2030
9.4.5.1. RNA Targeting Small Molecule Therapeutics Market in North America, 2021-2030
9.4.5.2. RNA Targeting Small Molecule Therapeutics Market in Europe, 2021-2030
9.4.5.3. RNA Targeting Small Molecule Therapeutics Market in Asia, 2021-2030
9.4.5.4. RNA Targeting Small Molecule Therapeutics Market in MENA, 2021-2030
9.4.5.5. RNA Targeting Small Molecule Therapeutics Market in Latin America, 2021-2030
9.4.5.6. RNA Targeting Small Molecule Therapeutics Market in Rest of the World, 2021-2030
9.4.6. RNA Targeting Small Molecule Therapeutics Market: Drug-wise Sales Forecast
9.4.6.1. Translarna (Ataluren, PTC127), PTC Therapeutics
9.4.6.1.1. Target Patient Population
9.4.6.1.2. Sales Forecast
9.4.6.1.3. Net Present Value
9.4.6.1.4. Value Creation Analysis
9.4.6.2 Risdiplam, PTC Therapeutics
9.4.6.2.1. Target Patient Population
9.4.6.2.2. Sales Forecast
9.4.6.2.3. Net Present Value
9.4.6.4.4. Value Creation Analysis
9.4.6.3. Tomivosertib (eFT508), eFFECTOR Therapeutics
9.4.6.3.1. Target Patient Population
9.4.6.3.2. Sales Forecast
9.4.6.3.3. Net Present Value
9.4.6.3.4. Value Creation Analysis
9.4.6.4. ELX-02, Eloxx Pharmaceuticals
9.4.6.4.1. Target Patient Population
9.4.6.4.2. Sales Forecast
9.4.6.4.3. Net Present Value
9.4.6.4.4. Value Creation Analysis
9.4.6.5. H3B-6545, H3 Biomedicine
9.4.6.5.1. Target Patient Population
9.4.6.5.2. Sales Forecast
9.4.6.5.3. Net Present Value
9.4.6.5.4. Value Creation Analysis
9.4.6.6. Translarna (Ataluren, PTC124), PTC Therapeutics
9.4.6.6.1. Target Patient Population
9.4.6.6.2. Sales Forecast
9.4.6.6.3. Net Present Value
9.4.6.6.4. Value Creation Analysis
9.4.6.7. ABX464 (Monotherapy), Abivax
9.4.6.7.1. Target Patient Population
9.4.6.7.2. Sales Forecast
9.4.6.7.3. Net Present Value
9.4.6.7.4. Value Creation Analysis
9.4.6.8. ABX464 and Methotrexate, Abivax
9.4.6.8.1. Target Patient Population
9.4.6.8.2. Sales Forecast
9.4.6.8.3. Net Present Value
9.4.6.8.4. Value Creation Analysis
9.5. Concluding Remarks


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 DATA13. APPENDIX II: LIST OF COMPANIES AND ORGANIZATION

Companies Mentioned

  • AbbVie Ventures
  • Abingworth
  • Abivax
  • AC Immune
  • Accel-Rx Health Sciences Accelerator
  • Accent Therapeutics
  • Advent Life Sciences
  • Agent Capital
  • Alexandria Venture Investments
  • ALS Society of Canada
  • Altitude Life Science Ventures
  • Amgen Ventures
  • Anima Biotech
  • Arrakis Therapeutics
  • Astellas Venture Management
  • AstraZeneca
  • Atlas Venture
  • AviTx
  • BDC Venture Capital
  • Biogen
  • BioMed Ventures
  • Brain Canada
  • Bristol-Myers Squibb
  • Cambridge Innovation Capital 
  • Canaan Partners
  • Catalyst Investors
  • Celgene
  • CHDI Foundation
  • Cystic Fibrosis Foundation
  • Droia Ventures
  • DSC Investment 
  • EcoR1 Capital
  • eFFECTOR Therapeutics
  • Eli Lilly
  • Eloxx Pharmaceuticals
  • Evotec
  • Flatiron Health
  • Forbion Capital Partners
  • Foundation Medicine
  • FutureNeuro
  • Genentech
  • Good Growth Capital
  • Gotham Therapeutics
  • GreatPoint Ventures
  • GV
  • H3 Biomedicine
  • Hatteras Venture Partners
  • HBM Healthcare Investments
  • Illumina Ventures
  • Imperial Innovations
  • ImStar Therapeutics
  • Innovate UK
  • IP Group
  • Johnson & Johnson Innovation
  • Korea Investment Partners
  • Kreos Capital
  • Longwood Fund
  • Life Sciences Partners
  • M Ventures
  • Massachusetts General Hospital
  • Merck
  • Mission Bay Capital
  • Mitsubishi UFJ Capital
  • MP Healthcare Venture Management
  • MPM Capital
  • Nextech Invest
  • Novartis Venture Fund
  • Novation Pharmaceuticals
  • NS Investment
  • Omega Funds
  • OPKO Health
  • Origenis
  • Osage University Partners
  • Pappas Capital
  • PCAS
  • Pfizer
  • Pontifax Venture Capital
  • Precipio
  • Reaction Biology
  • PTC Therapeutics
  • Quark Venture
  • Reborna Biosciences
  • Reily and Associates
  • Ribometrix
  • Roche
  • Saverna Therapeutics
  • Sectoral Asset Management
  • Selvita
  • Sevion Therapeutics
  • ShangPharma Innovation
  • Skyhawk Therapeutics
  • Sofinnova Partners
  • Spinal Muscular Atrophy Foundation
  • SR One Capital Management
  • STORM Therapeutics
  • SV Health Investors
  • Symeres
  • Taiho Ventures
  • Takeda Pharmaceutical
  • The Column Group
  • The Dementia Discovery Fund (DDF)
  • The Mark Foundation for Cancer Research
  • Touchstone Innovations
  • Twentyeight-Seven Therapeutics
  • U.S. Venture Partners
  • University of Pennsylvania
  • venBio
  • Versant Ventures
  • Vertex Pharmaceuticals
  • Virtusa
  • WuXi AppTec Venture Fund
  • Zikani Therapeutics
  • ZoBio

Methodology

 

 

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