RNAi therapies – new study showing you trends, R&D progress, and predicted revenues When will RNAi therapies reach the market? Visiongain’s brand new report shows you potential revenues to 2025, assessing data, trends, opportunities and prospects there.
Our 180-page report provides 95 tables, charts, and graphs. Discover the most lucrative areas in the industry and the future market prospects. Our new study lets you assess forecasted sales at overall world market and regional level. You will see financial results, interviews, trends, opportunities, and revenue predictions. There is much opportunity in this nascent pharmaceutical market.
Forecasts from 2015-2025 and other analyses show you commercial prospects Besides revenue forecasting to 2025, our new study provides you with recent results, growth rates, and market shares. Discover qualitative analyses (including SWOT and STEP analysis), product profiles and commercial developments. Read the full transcripts of two exclusive expert opinion interviews from industry specialists informing your understanding and allowing you to assess prospects for investments and sales, including: • Joseph Payne, CEO, Arcturus Therapeutics • J. Michael French, President and CEO, Marina Biotech • Dr. Ana I. Jiménez, COO and R&D Director, Sylentis
See revenue forecasts for the most promising products in development How will leading RNAi therapies perform to 2025 at world level? Our study forecasts sales of 11 products, including: • ALN-TTR02 (patisiran) • Vigil immunotherapy • ALN-TTRsc (revusiran) • ARC-520 • Atu027 • PF-655 • QPI-1002 • RXI-109 • siG12D-LODER • SPC3649 (miravirsen) • SYL040012
Discover how high revenues can go. Our analysis also breaks the main world forecast into geographical markets.
What are the prospects in the leading regions and countries? In addition to analyses of the overall world market, you discover individual revenue forecasts for 4 regional markets plus the Rest of the World to 2025: • US • EU5 • Asia-Pacific • Latin America • Rest of the World
There will be growth in established pharma markets and in developing countries. Our analyses show that the US, in particular, will achieve high revenue growth to 2025. Developments worldwide in biotechnology infrastructure and RNAi therapy development will influence the market.
Leading companies and potential for market growth Overall world revenue for RNAi therapies will reach $391m in 2018, our work forecasts. We predict strong revenue growth through to 2025. Market entry of novel therapies, investment from Big Pharma and high unmet medical needs in many markets will increase sales to 2025.
Our work shows you what organisations hold greatest potential. See Visiongain’s analysis of 11 leading companies, including these: • Alnylam Pharmaceuticals • Arrowhead Research • Benitec Biopharma • Gradalis Incorporated • Quark Pharmaceuticals • Roche Innovation Center Copenhagen (formerly Santaris Pharma) • RXi Pharmaceuticals • Silence Therapeutics • Silenseed • Sylentis • Tekmira Pharmaceuticals.
A company profile gives you the following information where available: • Historical revenues from 2010 to 2014 • Discussion of a company’s activities and outlook • Recent financial results • Assessment of recent developments – mergers and acquisitions (M&A), new products, and collaborations, including alliances, partnerships and joint ventures
Discover capabilities, progress, and commercial prospects, helping you stay ahead.
Research and development – assess innovation, trends and possibilities What about R&D – the pipeline for new drugs? You see trends for these areas and technologies: • Small interfering RNA (siRNA) therapies • Short hairpin RNA (shRNA) therapies • DNA-directed RNAi (ddRNAi) therapies • Delivery technologies such as lipid nanoparticles and viral vectors • Other progress in RNAi therapy development.
RNAi therapy R&D is strong in siRNA-based therapies. Novel compounds and new delivery vectors with high safety, efficacy and affordability are in great demand. Our work explains, discussing many issues.
What issues will affect the RNAi therapies industry? Our new report discusses issues and events affecting the RNAi therapies market. You will find discussions, including qualitative analyses: • Approval of other oligonucleotide products indicates a path to market acceptance • Challenges exist with delivery of current RNAi therapies • Big Pharma is investing in RNAi therapy developers • Alternative approaches to gene silencing are advancing rapidly
You will see discussions of technological, commercial, and economic matters, with emphasis on the competitive landscape and business outlooks.
How the RNAi for Therapeutic Applications: Technology and Market Forecast 2015-2025 report helps you In summary, our 180-page report gives you the following knowledge: • Revenue forecasts to 2025 for the world RNAi therapies market – discover the industry’s prospects, finding promising places for investments and revenues • Revenue forecasts to 2025 for the leading 5 regional markets – US, EU5, Asia-Pacific, Latin America and Rest of the World • Predicted revenues of 11 leading drugs to 2025 – see potentials of top products • Assessment of 11 leading companies – hear about products, results and strategies including pipeline analysis and recent developments • Review of R&D pipelines – investigate developmental trends and progress • Discussion of what stimulates and restrains companies and the market • Prospects for established firms and those seeking to enter the market • View opinions from our survey, seeing interviews with authorities
You will find quantitative and qualitative analyses with independent predictions. You will receive information that only our report contains, staying informed with this invaluable business intelligence.
Information found nowhere else With our survey you are less likely to fall behind in knowledge or miss opportunity. See how you could benefit your research, analyses, and decisions. Also see how you can save time and receive recognition for commercial insight.
Visiongain’s study is for everybody needing commercial analyses for the RNAi therapies market and leading companies. You find data, trends and predictions. Please order our report now.
Visiongain is a trading partner with the US Federal Government CCR Ref number: KD4R6
1. Report Overview 1.1 Global RNAi Therapies Market Overview 1.2 Why You Should Read This Report 1.3 How This Report Delivers 1.4 Key Questions Answered by This Analytical Report 1.5 Who is This Report For? 1.6 Methodology 1.7 Frequently Asked Questions (FAQ) 1.8 Associated Visiongain Reports 1.9 About Visiongain
2. Introduction to RNAi Therapies 2.1 The Pharmaceutical Industry: a Very Brief Overview 2.2 Overview of RNA: From DNA to Proteins, and Beyond 2.2.1 The Functions of RNA 2.2.2 The Discovery of RNA Interference 2.3 RNAi Timeline: From Discovery to Human Trials 2.4 RNAi: Repurposing a Natural Cellular Gene-Silencing Process 2.4.1 siRNA: Application in Genetic Diseases 2.4.2 miRNA: Natural Gene Expression Regulators 2.5 Development of RNAi Therapies 2.5.1 The Delivery Approach: Exogenous Introduction of siRNA 2.5.2 ddRNAi: Stable Expression of RNAi Inducers 2.5.2.1 shRNA: Employing Viral Vectors 2.6 Ribopharma AG: The First Company to Specialise in RNAi Therapeutics 2.7 Phases of Clinical Trials 2.8 Scope of This Report 2.8.1 Products Excluded from This Report 2.9 Currency Exchange Rates in This Report
3. Leading Therapeutic Applications for RNAi 3.1 Therapeutic Areas Driving the RNAi Therapeutics Market to 2025 3.2 Infectious Diseases and Cancer Account for Most Pipeline Therapies in 2015 3.3 Infectious Diseases – Protecting and Saving Lives through Medical Progress 3.3.1 Viral Hepatitis 3.3.1.1 Hepatitis B and C 3.3.1.2 Prospects for RNAi in Hepatitis B and C 3.3.2 Prospects for RNAi in HIV Infection 3.4 Cancer: Great Scope for New Therapies 3.5 Ophthalmic Diseases – Limited Opportunity for Developers? 3.5.1 RNAi for AMD: No Clinical Candidates in 2015 3.5.2 RNAi for Glaucoma: Limited Role in Treatment? 3.6 Lung Diseases and Respiratory Disorders – Life-Improving Treatments 3.6.1 Respiratory Syncytial Virus Infection 3.7 Kidney Disease – Investigations to Treat the Urinary System 3.7.1 Delayed Graft Function (DGF)
4. RNAi Therapies: World Market 2015-2025 4.1 The World RNAi Therapies Market in 2014 4.2 RNAi Therapies Market Segmentation, 2014 4.3 World RNAi Therapies Market: Sales Forecast 2015-2025 4.4 World RNAi Therapies Market: Drivers and Restraints 2015-2025
5. Leading Regional Markets 2015-2025 5.1 Scope and Limitations 5.2 World RNAi Therapies Market: Regional Forecast 2015-2025 5.3 How Will Regional Market Shares Change to 2025? 5.4 US RNAi Therapies Market 2015-2025 5.4.1 FDA Approval of Kynamro Improves RNAi Prospects 5.5 EU5 RNAi Therapies Market 2015-2025 5.5.1 RNAi Prospects in Leading EU Markets 5.6 Asia-Pacific RNAi Therapies Market 2015-2025 5.6.1 Prospects for RNAi Therapies in Japan 5.6.2 China Steps Up Efforts in RNAi Drug Development 5.6.3 India: Company Collaborations to Stimulate Interest in RNAi 5.7 Latin American RNAi Therapies Market 2015-2025 5.8 The RNAi Therapies Market in the Rest of the World 2015-2025
6. RNAi Therapies: Leading Companies 2015-2025 6.1 Alnylam Pharmaceuticals 6.1.1 RNAi Therapeutic Pipeline: Strong Portfolio 6.1.1.1 Genetic Medicine STAr 6.1.1.2 Cardio-metabolic Disease STAr 6.1.1.3 Hepatic Infectious Disease STAr 6.1.1.4 Other Pipeline Products: Development on Hold 6.1.1.5 Other Pipeline Products: ALN-RSV01 for RSV Infection 6.1.1.6 Other Pipeline Products: ALN-VSP02 for Liver Cancers 6.1.2 Sales and Recent Performance 6.1.3 Recent Developments 6.1.3.1 Alnylam Files Trade Secret Misappropriation Suit Against Dicerna 6.1.3.2 Alnylam Enters Strategic Alliance with Genzyme (Sanofi) 6.1.3.3 Other Alliances with Leading Companies 6.1.3.4 Acquisition of Sirna Therapeutics 6.1.3.5 Contract Manufacturing of siRNAs by Tekmira Pharmaceuticals 6.2 Arrowhead Research 6.2.1 RNAi Therapeutic Pipeline: Targeting Hepatitis B and Alpha-1 Antitrypsin Deficiency 6.2.2 Sales and Recent Performance 6.2.3 Recent Developments 6.2.3.1 Roche Acquisition Strengthens Company’s Market Position 6.2.3.2 Arrowhead Acquires Novartis’ RNAi Assets 6.3 Benitec Biopharma 6.3.1 RNAi Therapeutic Pipeline 6.3.2 Sales and Recent Performance 6.3.3 Recent Developments 6.3.3.1 ddRNAi Patent Maintained by Australia’s CSIRO 6.3.3.2 Agreement with Omnia Biologics 6.4 Gradalis Incorporated 6.4.1 RNAi Therapeutic Pipeline: Advancing Gradalis for Cancer 6.5 Quark Pharmaceuticals 6.5.1 RNAi Therapeutic Pipeline: A Broad siRNA Portfolio in Clinical Development 6.5.1.1 Preclinical Pipeline: Collaboration with Nitto Denko 6.5.2 Recent Developments 6.5.2.1 Quark Partners with Big Pharma to Develop RNAi Therapies 6.5.2.2 RiboQuark: Joint Venture with Suzhou Ribo Life Science 6.6 Roche Innovation Center Copenhagen 6.6.1 RNAi Therapeutic Pipeline: Potential in Locked Nucleic Acid Platform? 6.6.1.1 Former Santaris Pipeline Candidates 6.6.2 Recent Developments 6.6.2.1 Targeting Rare Genetic Diseases through Alliance with Shire 6.6.2.2 Roche and Isis Pharmaceuticals to Settle Patent Lawsuit? 6.7 RXi Pharmaceuticals 6.7.1 RNAi Therapeutic Pipeline: Lead Candidate RXI-109 Prevents Scar Formation 6.7.2 Sales and Recent Performance 6.7.3 Recent Developments 6.7.3.1 Early Distribution Agreement with Ethicor 6.7.3.2 Acquisition of RNAi Assets from OPKO Health 6.8 Silence Therapeutics 6.8.1 RNAi Therapeutic Pipeline: Lead Candidate Atu027 Completed Phase 2 6.8.1.1 Multiple Delivery Technologies Offers Promise 6.8.2 Sales and Recent Performance 6.8.3 Recent Developments 6.8.3.1 Partnerships for Silence Therapeutics’ RNAi Platform 6.8.3.2 Silence Therapeutics Wins European Patent Office Appeal Covering RNAi Trigger Modification 6.9 Silenseed 6.9.1 RNAi Therapeutic Pipeline 6.9.2 Recent Developments 6.9.2.1 Silenseed IPO Filed in 2014 6.10 Sylentis 6.10.1 RNAi Therapeutic Pipeline: First Spanish Company to Conduct Clinical Trials of an RNAi Drug 6.10.2 Recent Developments 6.10.2.1 Zeltia Subsidiary PharmaMar May Merge with Its Parent Company 6.11 Tekmira Pharmaceuticals 6.11.1 RNAi Therapeutic Pipeline: Focusing on Cancer and Ebola 6.11.2 Sales and Recent Performance 6.11.3 Recent Developments 6.11.3.1 Out-Licensing of LNP Technology Adding Extra Revenue 6.11.3.2 Tekmira Merges with OnCore 6.12 Other Companies of Interest
8. Qualitative Analysis of the RNAi Therapies Market 2015-2025 8.1 SWOT Analysis of the RNAi Therapies Market 8.2 Strengths 8.2.1 A Highly Specific and Selective Therapy 8.2.2 Lower Therapeutic Dosages 8.2.3 Shorter Lead Identification Times 8.2.4 Manufacturing Synthetic RNAs: Cost-Effective and Easier Than for Other Biologics 8.3 Weaknesses 8.3.1 Challenges of Systemic Delivery of RNAi 8.3.1.1 Poor Cellular Uptake and Low Stability of siRNAs in Blood Serum 8.3.1.2 Systemic and Targeted Delivery of RNAi-based Drug Candidates 8.3.1.3 Industry’s Approach to Overcome the Introduction Challenges 8.3.2 Off-target Binding, Immune Response and Oversaturation Elevates Risk of Side Effects 8.4 Opportunities 8.4.1 Treating Multifactorial Diseases: A Revolutionary Therapeutic? 8.4.2 Combination Therapy with Antisense Technology or Aptamers? 8.4.2.1 Antisense Technology: First-to-Market Advantage 8.4.2.2 Aptamers: Offering Advantages over Antibodies 8.4.3 The Untapped Potential of mRNA 8.5 Threats 8.5.1 Novel Class of Treatment May Have Prolonged Approval Process 8.5.2 Large Scale Production of RNAs: A Potential Threat to Commercialisation? 8.5.3 Advances in Alternative Gene-Silencing Approaches Such As CRISPRs 8.6 STEP Analysis of the RNAi Therapies Market 8.6.1 Social Factors 8.6.2 Technological Factors 8.6.3 Economic Factors 8.6.4 Political Factors
9. Research Interviews 9.1 Interview with Mr Joseph Payne, CEO of Arcturus Therapeutics, San Diego, California 9.1.1 Arcturus Therapeutics’ RNAi Therapies Pipeline 9.1.2The Outlook for TTR-Mediated Disease RNAi Therapies? 9.1.3 Barriers to Market Entry 9.1.4 Opportunities for RNAi Developers 9.1.5 Interest from Big Pharma and Investors 9.1.6 Therapeutic Areas and Targets for RNAi Developers 9.1.7 Other Trends and Developments in RNAi Therapeutics 9.2 Interview with Mr J. Michael French, President and CEO of Marina Biotech, Bothell, Washington 9.2.1 Marina Biotech’s RNAi Pipeline and Platform 9.2.2 Barriers to Market Entry 9.2.3 Opportunities for RNAi Developers 9.2.4 Interest from Big Pharma and Investors 9.2.5 Therapeutic Areas and Targets for RNAi Developers 9.2.6 Other Trends and Developments in RNAi Therapeutics 9.3 Interview with Dr. Ana I. Jiménez, COO and R&D Director, Sylentis, Madrid, Spain 9.3.1 Sylentis’ RNAi Therapies Pipeline 9.3.2 Barriers to Market Entry 9.3.3 Opportunities for RNAi Developers 9.3.4 Interest from Big Pharma and Investors 9.3.5 Therapeutic Areas and Targets for RNAi Developers 9.3.6 The Intellectual Property Landscape of RNAi Therapies 9.3.7 Other Trends and Developments in RNAi Therapeutics
10. Conclusions 10.1 Market Entry of RNAi Therapies Expected in 2015-2025 10.2 The World RNAi Therapies Market in 2014 10.3 Leading RNAi Therapies Companies 10.4 Leading Regional Markets 10.5 World RNAi Therapies Market Forecast 2015-2025 10.6 RNAi Therapies Late-Stage Pipeline 10.7 The Future of the RNAi Therapies Market: Time for a RNAissance?
Acuity Pharmaceuticals (now OPKO Health) Advirna Aegera Therapeutics Agilent Technologies Alcon (subsidiary of Novartis) Alexion Allele Biotechnology Allergan Almirall Alnylam Altogen Labs AmpliPhi Biosciences Antisense Therapeutics AptaBio Aptose Biosciences Arcturus Therapeutics ArmaGen Technologies Arrowhead Research Ascletis Pharmaceuticals AstraZeneca Atugen AG (acquired by Silence Therapeutics) Avecia OligoMedicines Bayer Benitec Biopharma Biocon Biogen Idec Biomatik Corporation Biomics Biotechnologies Biomics Biotechnology Bionexus BioNTech BioSpring BioSpring GmbH Bio-Synthesis Blueberry Therapeutics Bristol-Myers Squibb Calando Pharmaceuticals (subsidiary of Arrowhead) Celsion CRISPR Therapeutics Cubist Pharmaceuticals (acquired by Merck) CureVac Dainippon Sumitomo Dendreon Dharmacon RNAi Technologies (part of GE Healthcare) Dicerna DuPont Pharmaceuticals Eli Lilly Entelos, Inc Enzo Therapeutics Enzon Pharmaceuticals Ethicor Ethris Eurofins MWG-Biotech Eurogentec Express Scripts Eyetech Pharmaceuticals EZBiolab GE Healthcare Gene Link Gene Signal GeneCare Research Institute GeneDesign Genzyme (part of Sanofi) Gilead Sciences Girindus America (part of Solvay Group) Gradalis Incorporated Grifols GSK Halo-Bio RNAi Therapeutics Integrated DNA Technologies Intradigm Corporation (merged into Silence Therapeutics) Isarna Therapeutics Isis Pharmaceuticals Kalypsys Kunshan RiboQuark Pharmaceutical Technology (RiboQuark) Kyowa Hakko Kirin Life Technologies Life Technologies (part of Thermo Fisher Scientific) Marina Biotech MedImmune (subsidiary of AstraZeneca) Medtronic Merck & Co. (Merck) Mirna Therapeutics Moderna Therapeutics Monsanto Nitto Denko Novartis NovaRx Noxxon Pharma Oligoengine (division of Halo-Bio RNAi Therapeutics) Omnia Biologics OncoGenex OnCore Biopharma (merged into Tekmira) Ophthotech OPKO Health Otsuka Pfizer PharmaMar (subsidiary of Zeltia) Pharmasset (part of Gilead Sciences) PhaseRx ProNAi Therapeutics Protiva Biotherapeutics Qiagen Quark Pharmaceuticals Regeneron Regulus Therapeutics Ribomic Ribopharma AG (now part of Alnylam) RiboTask Roche Roche Innovation Center Copenhagen (part of Roche) RXi Pharmaceuticals SABiosciences (part of Qiagen) Samyang Corporation Sanofi Santaris Pharma (acquired by Roche) Santen Sarepta Therapeutics Sigma-Aldrich Silence Therapeutics Silenseed Sirna Therapeutics (assets acquired by Alnylam) Sirnaomics Solvay Group SomaGenics Suzhou Ribo Life Science Sylentis (subsidiary of Zeltia) Synteract Inc. Tacere Therapeutics (acquired by Benitec) Takeda Tekmira The Medicines Company (MDCO) Thermo Fisher Scientific Traversa Therapeutics UniQure Zeltia Group
Other Organisations Mentioned in this Report American Cancer Society (ACS) American Society of Clinical Oncology (ASCO) American Society of Gene and Cellular Therapy (ASGCT) Brazilian Industrial Biotech Association Brigham Young University Carnegie Institution for Science Commonwealth Scientific and Industrial Research Organisation (CSIRO) (Australia) European Medicines Agency (EMA) European Patent Office (EPO) Georgetown University Hannover Medical School Kunshan RNAi Institute Mary Crowley Cancer Research Centre Max Planck Institute MD Anderson Cancer Center Ministry of Science and Technology (India) National Institute for Health and Care Excellence (NICE) (UK) National Institutes of Health (NIH) (US) Ohio State University (OSU) Pachyonychia Congenita Project United States Food and Drug Administration (FDA) University of Bayreuth University of Calgary University of Helsinki University of Massachusetts Medical School University of Oxford University of Pittsburgh US Department of Defense Wellcome Trust World Health Organization (WHO)
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