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Antisense Oligonucleotide Therapeutics Market by Target Indication, Type of Therapy, Type of Molecule Type of Generation and Key Geographies: Industry Trends and Global Forecasts, 2020-2030

  • ID: 5306286
  • Report
  • January 2021
  • Region: Global
  • 252 Pages
  • Roots Analysis

FEATURED COMPANIES

  • Abbott
  • Biolegio
  • EB Research Partnership
  • Genuity Science
  • Nippon Shinyaku
  • Secarna Pharmaceuticals

Overview

Since the approval of Vitravene™ (for the treatment of cytomegalovirus retinitis) in 1998, antisense oligonucleotide (ASO) therapies have evolved into a prominent class of therapeutics. In addition to the seven drugs, based on such molecules, that are commercially available, around 160 candidates are under development. Examples of recently approved antisense therapeutics include (in reverse chronological order) Viltepso™ (duchenne muscular dystrophy, March 2020), Vyondys 53® (duchenne muscular dystrophy, December 2019) and Waylivra® (hereditary transthyretin-mediated (hATTR) amyloidosis, May 2019). Given their ability to target the root cause of diseases, at the protein expression level, these disease-modifying interventions have potential applications across a wide range of therapeutic areas (including but not limited to oncological disorders, neurodegenerative disorders, respiratory disorders, and even certain rare genetic disorders). Further, recent advances in antisense oligonucleotide chemistry have enabled the development and synthesis of specialized oligonucleotides, having improved safety profiles and better cell targeting capabilities. In fact, these advanced variants of antisense oligonucleotide-based therapeutic candidates are deemed to possess the potential to cater to the unmet need for effective treatment options for diseases that were previously considered difficult to treat.

Presently, several drug developers, along with ASO technology providers, are actively engaged in the development of various antisense therapy candidates. Innovation in this field of research is mostly focused on improving cell targeting ability, target affinity, nuclease resistance, and optimizing toxicity profiles of antisense oligonucleotide candidates. Several big pharma players have also demonstrated interest in antisense oligonucleotide therapeutics and are investing both time and capital in this domain. The market has witnessed substantial partnership activity over the last few years. The activity in this segment of the industry has also attracted the attention of both private and public sector investors/investment funds, which have extended financial support to the initiatives of capable developer companies. Driven by encouraging clinical trial results, the antisense oligonucleotide therapies market is poised to witness healthy growth as multiple late stage drug candidates are approved and marketed over the coming decade.

Scope of the Report

The “Antisense Oligonucleotide Market, 2020-2030” report features an extensive study on the current market landscape, offering an informed opinion on the likely adoption of these therapies over the next ten years. The study underlines an in-depth analysis, highlighting the diverse capabilities of stakeholders engaged in this domain. 

In addition to other elements, the study includes:

  • A detailed assessment of the current market landscape of antisense oligonucleotide therapeutics, providing information on type of antisense molecule (RNA molecule and DNA molecule), ASO generation (first-generation, second-generation, third-generation and next-generation), phase of development (commercial, clinical, preclinical, and discovery stage) of lead candidates, target genes, target disease indications, target therapeutic areas, route of administration (subcutaneous, intravenous, intrathecal, intravitreal and others) and type of therapy (monotherapy, combination therapy and both). In addition, it provides details on drug developer(s), including year of establishment, company size, and location of headquarters.
  • Tabulated profiles of prominent antisense oligonucleotide therapeutic developers. Each profile features a brief overview of the company, its financial information (if available), product portfolio, recent developments and an informed future outlook.
  • An in-depth analysis of completed, ongoing and planned clinical studies of various antisense oligonucleotide therapeutics, based on the various relevant parameters, such as trial registration year, trial phase, trial recruitment status, enrolled patient population, study design, leading industry sponsors/collaborators (in terms of number of trials conducted), trial focus, target therapeutic area and target genes, highlighting popular indications, popular interventions and regional distribution of trials.
  • A detailed analysis of grants that have been awarded to research institutes for antisense oligonucleotide therapeutic projects, in the period between 2017 and 2020 (till September), on the basis of important parameters, such as year of grant award, amount awarded, administering institute center, support period, type of grant application, purpose of grant award, activity code, study section involved, type of recipient organizations and focus area. In addition, it highlights geographical distribution of recipient organizations, popular therapeutic areas, popular funding institute centers, prominent program officers, and popular recipient organizations. 
  • An analysis of the partnerships that have been established in the recent past (2016-2020), covering acquisitions and mergers, licensing agreements, product development agreements, research agreements, joint venture agreements and other relevant types of deals.
  • A case study on the oligonucleotide contract manufacturers and purification service providers, providing information on the year of establishment, company size, scale of operation, location of headquarters and type of purification method used.

One of the key objectives of the report was to estimate the existing market size and the future opportunity for antisense oligonucleotide therapeutics, over the next ten years. Based on multiple parameters, such as target patient population, likely adoption rate and the annual treatment cost, we have provided informed estimates on the evolution of antisense oligonucleotide therapeutics market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] type of antisense molecule (RNA and DNA molecule) [B] different target indications (duchenne muscular dystrophy, spinal muscular atrophy, hereditary transthyretin-mediated (hATTR) amyloidosis, familial chylomicronemia syndrome, familial partial lipodystrophy, pouchitis, leber's congenital amaurosis, huntington’s disease and amyotrophic lateral sclerosis) [C] ASO generations (first-generation, second-generation and third-generation) [D] route of administration (intrathecal, intravenous, intravitreal, subcutaneous, and topical) [E] type of therapy (combination therapy and monotherapy) and [F] key geographical regions (US, UK, EU4, Asia-Pacific and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth. 

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

Key Questions Answered

  • Who are the leading industry and non-industry players in this market?
  • What are the key therapeutic areas for which antisense oligonucleotide therapeutics are being/have been developed?
  • What are the prevalent trends within competitive landscape of antisense oligonucleotide therapeutics?
  • Which geographies are the most active in conducting clinical trials on antisense oligonucleotide therapeutics?
  • What kind of partnership models are commonly adopted by industry stakeholders? 
  • How is the current and future market opportunity likely to be distributed across key market segments?
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • Abbott
  • Biolegio
  • EB Research Partnership
  • Genuity Science
  • Nippon Shinyaku
  • Secarna Pharmaceuticals

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. Overview on Oligonucleotides and Affiliated Medical Products
3.2.1. Types of Oligonucleotides
3.2.1.1. Antisense Oligonucleotides (ASOs)
3.2.1.2. Aptamers
3.2.1.3. miRNA
3.2.1.4. shRNA
3.2.1.5. siRNA
3.2.1.6. Other Oligonucleotides
3.3. Oligonucleotide Therapeutics
3.3.1. RNA-Interference Therapeutics
3.3.1.1. Components of RNA-Interference Therapeutics
3.3.1.2. Mechanism of RNA-Interference Therapeutics
3.3.2. Antisense Oligonucleotide Therapeutics
3.3.2.1. Mechanism of Antisense Oligonucleotide Therapeutics
3.3.3.2. Types of Antisense Oligonucleotide Therapeutics
3.4. Future Perspectives

4. ANTISENSE OLIGONUCLEOTIDE THERAPEUTICS: MARKET LANDSCAPE
4.1. Chapter Overview
4.2. Antisense Oligonucleotide Therapeutics: Pipeline Review
4.2.1. Analysis by Type of Antisense Molecule 
4.2.2. Analysis by ASO Generation
4.2.3. Analysis by Phase of Development
4.2.4. Analysis by Target Genes
4.2.5. Analysis by Target Indications
4.2.6. Analysis by Therapeutic Areas
4.2.7. Analysis by Route of Administration
4.2.8. Analysis by Type of Therapy
4.3. Antisense Oligonucleotide Therapeutics: List of Developers
4.3.1. Analysis by Year of Establishment
4.3.2. Analysis by Company Size
4.3.3. Analysis by Location of Headquarters
4.4. Grid Analysis: Distribution by Phase of Development, Company Size and Location of Headquarters

5. COMPANY PROFILES
5.1. Chapter Overview
5.2. Antisense Therapeutics
5.2.1. Company Overview
5.2.2. Antisense Oligonucleotide Therapeutics Portfolio
5.2.3. Recent Developments and Future Outlook
5.3. Biogen
5.3.1. Company Overview
5.3.2. Financial Information 
5.3.3. Antisense Oligonucleotide Therapeutics Portfolio
5.3.4. Recent Developments and Future Outlook
5.4. Bio-Path Holdings
5.4.1. Company Overview
5.4.2. Antisense Oligonucleotide Therapeutics Portfolio
5.4.3. Recent Developments and Future Outlook
5.5. Ionis Pharmaceuticals
5.5.1. Company Overview
5.5.2. Financial Information
5.5.3. Antisense Oligonucleotide Therapeutics Portfolio
5.5.4. Recent Developments and Future Outlook
5.6. ProQR Therapeutics
5.6.1. Company Overview
5.6.2. Antisense Oligonucleotide Therapeutics Portfolio
5.6.3. Recent Developments and Future Outlook
5.7. Sarepta Therapeutics
5.7.1. Company Overview
5.7.2. Financial Information
5.7.3. Antisense Oligonucleotide Therapeutics Portfolio
5.7.4. Recent Developments and Future Outlook
5.8. Sterna Biologicals
5.8.1. Company Overview
5.8.2. Antisense Oligonucleotide Therapeutics Portfolio
5.8.3. Recent Developments and Future Outlook
5.9. Wave Life Sciences
5.9.1. Company Overview
5.9.2. Financial Information
5.9.3. Antisense Oligonucleotide Therapeutics Portfolio
5.9.4. Recent Developments and Future Outlook

6. CLINICAL TRIAL ANALYSIS
6.1. Chapter Overview
6.2. Scope and Methodology
6.3. Antisense Oligonucleotide Therapeutics: Clinical Trial Analysis
6.3.1. Analysis by Trial Registration Year
6.3.2. Analysis by Trial Phase
6.3.3. Analysis by Trial Recruitment Status
6.3.4. Analysis by Trial Registration Year and Number of Patients Enrolled
6.3.5. Analysis by Study Design
6.3.6. Analysis by Type of Sponsor / Collaborator
6.3.7. Leading Players: Analysis by Number of Registered Trials
6.3.8. Word Cloud: Key Focus Areas
6.3.9. Analysis by Target Therapeutic Area
6.3.10. Analysis by Trial Registration Year and Target Gene
6.3.11. Popular Indications: Analysis by Number of Registered Trials
6.3.12. Popular Interventions: Analysis by Number of Registered Trials
6.3.13. Geographical Analysis by Number of Registered Trials
6.3.14. Geographical Analysis by Number of Patients Enrolled

7. ACADEMIC GRANTS ANALYSIS
7.1. Chapter Overview
7.2. Scope and Methodology
7.3. Antisense Oligonucleotide Therapeutics: Analysis of Academic Grants
7.3.1. Analysis by Year of Grant Award
7.3.2. Analysis by Amount Awarded
7.3.3. Analysis by Administering Institute Center
7.3.4. Analysis by Support Period
7.3.5. Analysis by Administering Institute Center and Support Period
7.3.6. Analysis by Type of Grant Application
7.3.7. Analysis by Purpose of Grant Award
7.3.8. Analysis by Activity Code
7.3.9. Analysis by Study Section Involved
7.3.10. Analysis by Type of Recipient Organization
7.3.11. Word Cloud Analysis: Emerging Focus Areas
7.3.12. Geographical Distribution of Recipient Organizations
7.3.13. Popular Therapeutic Areas: Analysis by Number of Grants
7.3.14. Popular NIH Departments: Analysis by Number of Grants
7.3.15. Prominent Program Officers: Analysis by Number of Grants
7.3.16. Popular Recipient Organizations: Analysis by Number of Grants

8. PARTNERSHIPS AND COLLABORATIONS
8.1. Chapter Overview
8.2. Partnership Models
8.3. Antisense Oligonucleotide Therapeutics: List of Partnerships and Collaborations
8.3.1. Analysis by Year of Partnership
8.3.2. Analysis by Type of Partnership
8.3.3. Analysis by Type of Partnership and Generation of Antisense Molecule Involved
8.3.4. Analysis by Type of Partnership and Target Therapeutic Area
8.3.5. Analysis by Year of Partnership and Type of Partner
8.3.6. Analysis by Type of Partnership and Type of Partner
8.3.7. Most Active Players: Analysis by Number of Partnerships
8.3.8. Regional Analysis
8.3.8.1. Intercontinental and Intracontinental Agreements

9. MARKET FORECAST AND OPPORTUNITY ANALYSIS
9.1. Chapter Overview
9.2. Forecast Methodology and Key Assumptions
9.3. Global Antisense Oligonucleotide Therapeutics Market, 2020-2030
9.4. Global Antisense Oligonucleotide Therapeutics Market, 2020-2030: Individual Product Sales Forecasts
9.4.1. Alicaforsen (Atlantic Healthcare)
9.4.1.1. Target Patient Population
9.4.1.2. Sales Forecast
9.4.2. Eteplirsen (Sarepta Therapeutics)
9.4.2.1. Target Patient Population
9.4.2.2. Sales Forecast
9.4.3. Golodirsen (Sarepta Therapeutics)
9.4.3.1. Target Patient Population
9.4.3.2. Sales Forecast
9.4.4. Inotersen (Ionis Pharmaceuticals)
9.4.4.1. Target Patient Population
9.4.4.2. Sales Forecast
9.4.5. Sepofarsen (ProQR Therapeutics)
9.4.5.1. Target Patient Population
9.4.5.2. Sales Forecast
9.4.6. Tofersen (Biogen)
9.4.6.1. Target Patient Population
9.4.6.2. Sales Forecast
9.4.7. Tominersen (Roche)
9.4.7.1. Target Patient Population
9.4.7.2. Sales Forecast
9.4.8. Viltolarsen (Nippon Shinyaku)
9.4.8.1. Target Patient Population
9.4.8.2. Sales Forecast
9.4.9. Volanesorsen (Ionis Pharmaceuticals)
9.4.9.1. Target Patient Population
9.4.9.2. Sales Forecast
9.5. Global Antisense Oligonucleotide Therapeutics Market, 2020-2030: Distribution by Type of Antisense Molecule
9.5.1. Global Antisense Oligonucleotide Therapeutics Market for RNA Molecules, 2020-2030
9.5.2. Global Antisense Oligonucleotide Therapeutics Market for DNA Molecules, 2020-2030
9.6. Global Antisense Oligonucleotide Therapeutics Market, 2020-2030: Distribution by ASO Generation
9.6.1. Global Antisense Oligonucleotide Therapeutics Market for First-generation Products, 2021-2030
9.6.2. Global Antisense Oligonucleotide Therapeutics Market for Second-generation Products, 2020-2030
9.6.3. Global Antisense Oligonucleotide Therapeutics Market for Third-generation Products, 2020-2030
9.7. Global Antisense Oligonucleotide Therapeutics Market, 2020-2030: Distribution by Target Disease Indication
9.7.1. Global Antisense Oligonucleotide Therapeutics Market for Amyotrophic Lateral Sclerosis, 2023-2030
9.7.2. Global Antisense Oligonucleotide Therapeutics Market for Duchenne Muscular Dystrophy, 2020-2030
9.7.3. Global Antisense Oligonucleotide Therapeutics Market for Familial Chylomicronemia Syndrome, 2020-2030
9.7.4. Global Antisense Oligonucleotide Therapeutics Market for Familial Partial Lipodystrophy, 2024-2030
9.7.5. Global Antisense Oligonucleotide Therapeutics Market for Hereditary Transthyretin-Mediated (hATTR) Amyloidosis, 2020-2030
9.7.6. Global Antisense Oligonucleotide Therapeutics Market for Huntington’s Disease, 2024-2030
9.7.7. Global Antisense Oligonucleotide Therapeutics Market for Leber's Congenital Amaurosis, 2023-2030
9.7.8. Global Antisense Oligonucleotide Therapeutics Market for Pouchitis, 2021-2030
9.7.9. Global Antisense Oligonucleotide Therapeutics Market for Spinal Muscular Atrophy, 2020-2030
9.8. Global Antisense Oligonucleotide Therapeutics Market, 2020-2030: Distribution by Route of Administration
9.8.1. Global Antisense Oligonucleotide Therapeutics Market for Intrathecal Therapies, 2020-2030
9.8.2. Global Antisense Oligonucleotide Therapeutics Market for Intravenous Therapies, 2020-2030
9.8.3. Global Antisense Oligonucleotide Therapeutics Market for Intravitreal Therapies, 2023-2030
9.8.4. Global Antisense Oligonucleotide Therapeutics Market for Subcutaneous Therapies, 2020-2030
9.8.5. Global Antisense Oligonucleotide Therapeutics Market for Intraorifice Therapies, 2021-2030
9.9. Global Antisense Oligonucleotide Therapeutics Market, 2020-2030: Distribution by Type of Therapy
9.9.1. Global Antisense Oligonucleotide Therapeutics Market for Combination Therapies 2020-2030
9.9.2. Global Antisense Oligonucleotide Therapeutics Market for Monotherapies, 2020-2030
9.10. Global Antisense Oligonucleotide Market, 2020-2030: Geographical Distribution
9.10.1. Antisense Oligonucleotide Market in the US, 2020-2030
9.10.2. Antisense Oligonucleotide Market in Canada, 2020-2030
9.10.3. Antisense Oligonucleotide Market in the UK, 2020-2030
9.10.4. Antisense Oligonucleotide Market in Germany, 2020-2030
9.10.5. Antisense Oligonucleotide Market in France, 2020-2030
9.10.6. Antisense Oligonucleotide Market in Italy, 2020-2030
9.10.7. Antisense Oligonucleotide Market in Spain, 2020-2030
9.10.8. Antisense Oligonucleotide Market in Australia, 2025-2030
9.10.9. Antisense Oligonucleotide Market in Japan, 2020-2030
9.10.10. Antisense Oligonucleotide Market in Korea, 2020-2030
9.10.11. Antisense Oligonucleotide Market in Brazil, 2023-2030
9.10.12. Antisense Oligonucleotide Market in Israel, 2023-2030

10. CASE STUDY: OLIGONUCLEOTIDE MANUFACTURERS AND PURIFICATION SERVICES
10.1. Chapter Overview
10.2. List of Oligonucleotide Manufacturers Focused on Research and Diagnostic Applications
10.2.1. Analysis by Year of Establishment
10.2.2. Analysis by Company Size
10.2.3. Analysis by Scale of Operation
10.2.4. Analysis by Location of Headquarters
10.2.5. Analysis by Type of Purification Method Used
10.3. List of Oligonucleotide Manufacturers Focused on Therapeutic Applications
10.3.1. Analysis by Year of Establishment
10.3.2. Analysis by Company Size
10.3.3. Analysis by Scale of Operation
10.3.4. Analysis by Location of Headquarters
10.3.5. Analysis by Type of Purification Method Used

11. CONCLUDING REMARKS

12. APPENDIX 1: TABULATED DATA

13. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

Note: Product cover images may vary from those shown
  • Abbott
  • AcedrA BioPharmaceutical 
  • ACGT
  • Achieve Life Sciences
  • Agilent Technologies
  • Ajinomoto Bio-Pharma Services
  • Akcea Therapeutics
  • Alcyone Lifesciences
  • A Biotechnology & Pharmaceuticals
  • Allianz BioInnovation
  • Alta Bioscience
  • AM Chemicals
  • AmpTec
  • Amylon Therapeutics
  • Antisense Therapeutics
  • Applied Biological Materials
  • Aro Biotherapeutics
  • AstraZeneca
  • ATDBio
  • Atlantic Healthcare
  • ATZ Labs
  • Axolabs
  • Bachem
  • Bayer
  • BianoScience
  • Bio Basic
  • Biogen
  • Biolegio
  • BioMarin Pharmaceutical
  • Bioneer
  • Bio-Path Holdings
  • Biosearch Technologies
  • BioServe Biotechnologies
  • BioSpring
  • Bio-Synthesis
  • Boston Children’s Hospital
  • BR Biochem Life Sciences
  • Bridgen
  • Catabasis Pharmaceuticals
  • ChemGenes
  • Codiak BioSciences
  • Cold Spring Harbor Laboratory
  • CordenPharma
  • Creative Biogene
  • CUSABIO TECHNOLOGY
  • CyberGene
  • Daiichi Sankyo
  • Dalton Pharma Services
  • Deep Genomics
  • Denali Therapeutics
  • Dharmacon
  • Duke University
  • Dynacure
  • EB Research Partnership
  • Eli Lilly and Company
  • Elim Biopharmaceuticals
  • Empirico
  • Eton Bioscience
  • Eurofins Genomics
  • Evotec
  • Evox Therapeutics
  • F. Hoffmn-La-Roche
  • Firebrand Therapeutics
  • F-Star Therapeutics
  • FUTUREsynthesis
  • GCC Biotech
  • Genbiotech
  • Gene Signal
  • Gene Universal
  • GeneDesign
  • Genei Laboratories
  • GenePharma
  • General Biosystems
  • GENERI BIOTECH
  • GeneTx Biotherapeutics
  • GENEWIZ
  • GenScript
  • Genuity Science
  • GlaxoSmithKline
  • Golden Mountain Partners
  • Hanugen Therapeutics
  • IBA Lifesciences
  • inqaba biotec™
  • Integrated DNA Technologies
  • Ionis Pharmaceuticals
  • Isarna Therapeutics
  • Janssen Biotech
  • Johns Hopkins University
  • Johnson Matthey
  • Kaneka Eurogentec
  • KareBay Biochem
  • Lions Eye Institute
  • Lipigon Pharmaceuticals
  • Lonza
  • Mateon Therapeutics
  • Medical Need
  • Medigene
  • Microsynth
  • Midland Certified Reagent 
  • Moligo Technologies
  • National Cancer Institute
  • National Institute of Allergy and Infectious Diseases
  • NeuBase Therapeutics
  • Nippon Shinyaku
  • Nitto Denko Avecia
  • Novartis
  • Novatia
  • NZYTech
  • Oligomer
  • OncoGenex Pharmaceuticals
  • Oncoltelic
  • Oregon Health & Science University
  • Parexel
  • Pfizer
  • Primetech
  • Prometheus Research
  • ProQR Therapeutics
  • PTC Therapeutics
  • PureTech Health
  • PYC Therapeutics
  • Quintara Biosciences
  • ReadCoor
  • Rena Therapeutics
  • Rexahn Pharmaceuticals
  • Rockland Immunochemicals
  • RogCon Biosciences
  • Ruibo Bio-Technology
  • Sarepta Therapeutics
  • Scandinavian Gene Synthesis
  • Secarna Pharmaceuticals
  • Severn Biotech
  • Sigma Aldrich
  • Spring Bank Pharmaceuticals
  • SRI International
  • ST Pharm
  • STA Pharmaceuticals
  • Stanford University
  • Sterna Biologicals
  • Stoke Therapeutics
  • Sumitomo Chemical
  • Sun Yat-sen University
  • Sunomix Biosciences
  • Suzhou Ribo Life Science
  • Synbio Technologies
  • Syngene International
  • TAG Copenhagen
  • Takeda Pharmaceutical
  • The University of Texas MD Anderson Cancer Center
  • The University of Texas Southwestern Medical Center
  • Thermo Fisher Scientific
  • Thomas Jefferson University
  • TriLink Biotechnologies
  • Ultragenyx Pharmaceutical
  • United States Army Medical Research Institute of Infectious Diseases
  • United States Department of Defense
  • University of California
  • University of Florida
  • University of Massachusetts Medical School
  • University of Rochester
  • Vallentech
  • Vivantis Technologies
  • Wave Life Sciences
  • Wings Therapeutics
  • Xcelris
  • Zaphyr Pharmaceuticals
  • Zhejiang Haichang Biotechnology
Note: Product cover images may vary from those shown

 

 

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