RNAi therapeutics have garnered significant attention post the 2006 Nobel Prize that was awarded to Andrew Fire and Craig Mello for the discovery of RNA interference (RNAi). Subsequently, upon the approval of the first RNAi therapeutic, ONPATTRO®, in 2018, the field has witnessed a further surge in activity. Since then, three more RNAi based therapies, namely (in chronological order of approval year) GIVLAARI® (November 2019), LEQVIO® (October 2020) and OXLUMO™ (November 2020), have been approved by regulatory authorities. In addition, over 200 RNAi drug candidates are being evaluated across several clinical trials for the treatment of a wide range of disease indications. The ongoing research and development efforts are driven by the various advantages offered by these molecules; these include higher efficacy and enhanced potency at reduced effector molecule concentrations, over other modalities based on the anti-sense approach. RNAi-based therapies have also been identified as potential candidates for the treatment of COVID-19.
However, despite the numerous benefits offered by RNAi therapies, developers often face concerns related to the stability and targeted delivery of their candidates. In order to mitigate the aforementioned challenges, several players have developed novel technologies and delivery systems to ensure target specific delivery of these molecules. Such advancements have allowed therapy developers to shift their focus towards the use of RNAi therapeutics. Initiatives are also being undertaken to explore the vast potential associated with such therapeutic modalities for the control of disease-associated gene expression. In addition to effective gene silencing, these candidates can be deployed for applications related to precision medicine. Studies have also demonstrated the safety of RNAi therapeutics in systemic delivery, paving the way for systemic applications of the therapy. The high specificity, efficiency, and long term and stable gene silencing capabilities of RNAi therapeutics are believed to offer the potential to revolutionize the biopharmaceutical industry. As a result, several big pharma players have also undertaken various initiatives in this field of research. In the last five years, USD 8.5 billion has been invested to enable the advancement of ongoing research efforts. Moreover, 3,000+ articles related to RNAi therapeutics and technologies have been published. It is also worth highlighting that over 2,100 patents have been filed / granted, highlighting the continuous pace of ongoing innovation in this field. Given the high research activity and ongoing technology advancements, the RNAi therapeutics market is poised to grow at a steady pace in the foreseen future.
One of the key objectives of the report was to estimate the existing market size and estimate the future size of the RNAi therapeutics market. The publisher has provided informed estimates on the evolution of the market for the period 2022-2035. The publisher's year-wise projections of the current and future opportunity have further been segmented on the basis of [A] key therapeutic areas (oncological disorders, genetic disorders, metabolic disorders, haematological disorders, ophthalmic disorders and other diosrders), [B] route of administration (subcutaneous, intravenous, topical and intradermal), [C] share of leading industry players, [D] type of RNAi molecule (siRNA and shRNA) and [E] key geographical regions (North America, Europe and Asia-Pacific). In order to account for future uncertainties and to add robustness to their model, the publisher has provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the market’s evolution.
All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.
However, despite the numerous benefits offered by RNAi therapies, developers often face concerns related to the stability and targeted delivery of their candidates. In order to mitigate the aforementioned challenges, several players have developed novel technologies and delivery systems to ensure target specific delivery of these molecules. Such advancements have allowed therapy developers to shift their focus towards the use of RNAi therapeutics. Initiatives are also being undertaken to explore the vast potential associated with such therapeutic modalities for the control of disease-associated gene expression. In addition to effective gene silencing, these candidates can be deployed for applications related to precision medicine. Studies have also demonstrated the safety of RNAi therapeutics in systemic delivery, paving the way for systemic applications of the therapy. The high specificity, efficiency, and long term and stable gene silencing capabilities of RNAi therapeutics are believed to offer the potential to revolutionize the biopharmaceutical industry. As a result, several big pharma players have also undertaken various initiatives in this field of research. In the last five years, USD 8.5 billion has been invested to enable the advancement of ongoing research efforts. Moreover, 3,000+ articles related to RNAi therapeutics and technologies have been published. It is also worth highlighting that over 2,100 patents have been filed / granted, highlighting the continuous pace of ongoing innovation in this field. Given the high research activity and ongoing technology advancements, the RNAi therapeutics market is poised to grow at a steady pace in the foreseen future.
Scope of the Report
The “RNAi Market: Therapeutics and Technologies (Focus on siRNA, miRNA, shRNA and DNA) (3rd Edition) by Key Therapeutic Areas (Oncological Disorders, Genetic Disorders, Metabolic Disorders, Hematological Disorders, Ophthalmic Disorders and Other Disorders), Route of Administration (Subcutaneous, Intravenous, Topical and Intradermal), Leading Industry Players, Type of RNAi Molecule (siRNA and shRNA) and Key Geographical Regions (North America, Europe, Asia-Pacific and Rest of the World): Industry Trends and Global Forecasts, 2022-2035” report features an extensive study of the current market landscape and the likely future potential; associated with the RNAi therapeutics market, over the next decade. The study also includes a detailed analysis of key drivers and trends within this evolving market. Amongst other elements, the report features:- A detailed overview of the overall market landscape of players engaged in the development of RNAi therapeutics, based on several relevant parameters, such as phase of development (marketed, clinical, and preclinical / discovery stage) of pipeline candidates, target disease indication(s), key therapeutic areas (oncological disorders, infectious diseases, genetic disorders, ophthalmic diseases, respiratory disorders, hepatic disorders, metabolic disorders, cardiovascular disorders, dermatological disorders and other disorders), type of RNAi molecule (siRNA, miRNA, shRNA, sshRNA and DNA), target gene, route of administration and special drug designations (if any).
- A detailed overview of the players engaged in the development of RNAi therapeutics, based on several relevant parameters, such as year of establishment, company size, location of headquarters and regional landscape.
- A competitiveness analysis of RNAi therapeutic developers, based on developer strength (in terms of employee count and years of experience) and product portfolio strength (in terms of number of RNAi molecules across different phases of development, type of RNAi molecule offered, purpose of technology and target therapeutic area).
- Elaborate profiles of drug candidates that are in the advanced stages of development (phase II / III and above). Each drug profile features information on the current development status of the drug, along with details on its developer, mechanism of action, route of administration, affiliated technology Platform (if available), dosage and recent clinical trial results.
- An overview of the current market landscape of technology platforms available for the development of RNAi therapeutics, along with information on their developer, purpose of technology, type of molecule (s) delivered and type of cell (s) / tissue (s) targeted. In addition, the chapter includes information on technology developer(s), highlighting the year of their establishment, company size and location of headquarters. The chapter also includes brief profiles of key RNAi-based drug discovery / development technology platforms and drug delivery technologies (shortlisted on the basis of competitiveness score). Each profile features details on the various pipeline molecules that have been / are being developed using the technology and insights from patents associated with the technology.
- An insightful technology competitiveness analysis, benchmarking RNAi technologies, based on supplier power (in terms of company size and years of experience) and key technology specifications. The analysis was designed to enable stakeholder companies to compare their existing capabilities within and beyond their respective peer groups and identify opportunities to achieve a competitive edge in the industry.
- A detailed analysis of the target indications (segregated by various therapeutic areas, including oncological disorders, infectious diseases, metabolic disorders, ophthalmic diseases and genetic disorders) that are currently the prime focus of companies engaged in the development of RNAi therapeutics.
- An analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, current status, phase of development, type of RNAi molecule, enrolled patient population and regional distribution of trials.
- An analysis highlighting the key opinion leaders (KOLs) in this domain. It features a 2X2 analysis to assess the relative experience of certain KOLs who were shortlisted based on their contributions (in terms of involvement in various clinical studies) in this field.
- An in-depth analysis of patents related to RNAi therapeutics, filed / granted, since 2016, based on type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry / non-industry players (in terms of the number of patents filed / granted), and patent valuation.
- A detailed review of around 3,000 peer-reviewed, scientific articles related to research on RNAi therapeutics, based on several relevant parameters, such as year of publication, type of publication, popular keywords, top journals, top publishers, key copyright holders and key funding institutes.
- An analysis of the various partnerships related to RNAi therapeutics inked by players engaged in this domain, based on various parameters, such as the type of partnership, year of partnership, target disease indication, therapeutic area, type of RNAi molecule, financial details (wherever applicable), focus area of collaboration and most active players (in terms of number of partnerships).
- A detailed analysis of various investments made by players engaged in this domain, during the period 2016-2021, based on several relevant parameters, such as year of funding, type of funding (seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other offerings), amount invested, type of RNAi molecule, most active players (in terms of number and amount of funding instances) and key investors (in terms of number of instances).
- An elaborate discussion on the various commercialization strategies that can be adopted by drug developers engaged in this domain, across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing of the drug.
- An elaborate discussion on the use of miRNA as a potential biomarker, along with a list of diagnostic kits that are either available in the market, or likely to be approved in the foreseen future.
- A detailed analysis of the companies that are actively supporting the development of RNAi therapeutics market, including contract manufacturers, contract researcher organizations and other service providers based on various parameters, such as type of service provider, location of their headquarters and type of RNAi molecule developed.
- An informed estimate of the annual clinical and commercial demand for RNAi therapeutics, taking into account the target patient population in ongoing and planned clinical trials of RNAi therapeutics, sponsored by both industry and non-industry players.
- A detailed analysis capturing affiliated trends, key drivers and challenges, under a comprehensive SWOT framework, featuring a Harvey ball analysis, highlighting the relative impact of each SWOT parameter on the RNAi therapeutics market.
One of the key objectives of the report was to estimate the existing market size and estimate the future size of the RNAi therapeutics market. The publisher has provided informed estimates on the evolution of the market for the period 2022-2035. The publisher's year-wise projections of the current and future opportunity have further been segmented on the basis of [A] key therapeutic areas (oncological disorders, genetic disorders, metabolic disorders, haematological disorders, ophthalmic disorders and other diosrders), [B] route of administration (subcutaneous, intravenous, topical and intradermal), [C] share of leading industry players, [D] type of RNAi molecule (siRNA and shRNA) and [E] key geographical regions (North America, Europe and Asia-Pacific). In order to account for future uncertainties and to add robustness to their model, the publisher has provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the market’s evolution.
All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. 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 RNAi therapeutics?
- Which drugs are being evaluated across early and late stages of pipeline?
- Which are the key therapeutic areas targeted by RNAi therapeutics?
- Which technology platforms/delivery systems are being used for the development and delivery of RNAi therapeutics?
- What are the most active trial sites (in terms of number of clinical studies being conducted) related to RNAi therapeutics?
- Who are the key opinion leaders/experts engaged in the field of RNAi therapeutics?
- Which companies are actively filing patents to drive innovation in the field of RNAi therapeutics?
- What type of partnership models are commonly adopted by industry stakeholders?
- Who are the key investors in this domain?
- Which commercialization strategies are most commonly adopted by RNAi therapy developers, across different stages of product development?
- Who are the key service providers engaged in the RNAi therapeutics domain?
- How is the current and future market opportunity likely to be distributed across key market segments?
Table of Contents
1. PREFACE
3. INTRODUCTION
4. MARKET OVERVIEW
5. COMPETITIVE LANDSCAPE
6. COMPANY COMPETITIVENESS ANALYSIS
7. APPROVED AND LATE STAGE RNAi THERAPEUTICS
8. TECHNOLOGY PLATFORMS AND DELIVERY SYSTEMS
9. TECHNOLOGY COMPETITIVENESS ANALYSIS
10. KEY THERAPEUTIC INDICATIONS
11. CLINICAL TRIAL ANALYSIS
12. KEY OPINION LEADERS
13. PATENT ANALYSIS
14. PUBLICATION ANALYSIS
15. RECENT PARTNERSHIPS
16. FUNDING AND INVESTMENT ANALYSIS
17. KEY COMMERCIALIZATION STRATEGIES
18. RNAi IN DIAGNOSTICS
19. SERVICE PROVIDERS FOR RNAi THERAPEUTICS
20. DEMAND ANALYSIS
21. MARKET SIZING AND OPPORTUNITY ANALYSIS
22. SWOT ANALYSIS
24. CONCLUSION
List of Figures
List of Tables
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- 1Globe Health Institute
- 20Med Therapeutics
- Adhera Therapeutics
- Advirna
- Ajinomoto Bio-Pharma Services
- Alexion Pharmaceuticals
- Allele Biotechnology
- Alnylam Pharmaceuticals
- Altogen Labs
- AM Chemicals
- Amgen
- AMSBIO
- Anjarium Biosciences
- Applied Biological Materials (abm)
- Arbutus Biopharma
- Arcturus Therapeutics
- ARIZ Precision Medicine
- Aro Biotherapeutics
- Arrowhead Pharmaceuticals
- Ascletis Pharma
- AstraZeneca
- Atlanta Therapeutics
- AUM LifeTech
- Avidity Biosciences
- Benitec Biopharma
- BianoScience
- Bioasis Technologies
- BioAxone BioSciences
- Biomics Biopharma
- Bioneer
- Biosettia
- BioSpring
- Bio-Synthesis
- bluebird bio
- Boehringer Ingelheim
- Boston Children’s Hospital
- BR Biochem Life Sciences
- Bristol-Myers Squibb
- Calimmune (Acquired by CSL Behring)
- Cell Signaling Technology
- Cellecta
- Celsion
- Certara
- Charles River Laboratories
- CMIC Group
- CordenPharma
- Creative Animodel
- Creative Biogene
- Creative Biolabs
- CUSABIO TECHNOLOGY
- Dharmacon (Acquired by Horizon Discovery)
- Dicerna Pharmaceuticals
- Eli Lilly
- Entos Pharmaceuticals
- Eurofins Genomics
- Eurogentec
- Evotec
- Exiqon
- GCC Biotech
- GeneCopoeia
- GeneCust
- GeneDesign (Acquired by Ajinomoto Bio-Pharma Services)
- Genel
- GenePharma
- Genevant Sciences
- GlaxoSmithKline
- Gradalis
- Halo-Bio RNAi Therapeutics
- Hansoh Pharma
- HISTALIM
- Horizon Therapeutics
- Hugel
- IIDong Pharmaceutical
- Integrated DNA Technologies
- InteRNA Technologies
- iTherapeutics
- Janssen Pharmaceuticals
- Kanglin Biotec
- LC Sciences
- Lemonex
- LGC, Biosearch Technologies
- Mallinckrodt Pharmaceuticals
- MD Anderson Cancer Center
- Microsynth
- miReven
- Mirimus
- Mirus Bio
- MultiplexDX
- NanoDe Therapeutics
- NantWorks
- National Cancer Institute (NCI)
- Netherlands Organisation for Health Research and Development (ZonMw)
- Nitto BioPharma
- Nitto Denko
- Nitto Denko Avecia
- Novartis (The Medicines Company)
- Novo Nordisk
- Nucleome Informatics
- Oligoengine
- OliX Pharmaceuticals
- OriGene Technologies
- OZ Biosciences
- PCI Biotech
- Phalanx Biotech
- Pharmaron
- Phio Pharmaceuticals (previously Rxi Pharmaceuticals)
- Phyzat
- Polyplus-transfection
- Promega
- QIAGEN
- Quark Pharmaceuticals
- Radboud University
- Regen BioPharma
- Regeneron
- Regulus Therapeutics
- RiboBio
- Roche
- Sanofi Genzyme
- Santa Cruz Biotechnology
- Shanghai Biotechnology
- Shanghai Public Health Clinical Center
- Sigma-Aldrich
- Silence Therapeutics
- Silenseed
- SIRION Biotech
- Sirnaomics
- siTools Biotech
- Soluventis
- SomaGenics
- Sumitomo Chemical
- Suzhou Ribo Life Science
- Sylentis
- Syngene
- Takara Bio
- Takeda Pharmaceuticals
- tebu-bio
- Théa Open Innovation
- Thermo Fisher Scientific
- Transgene Biotek
- Translate Bio (Acquired by Sanofi)
- Transomic Technologies
- TriPhos Therapeutics
- UCSF Diabetes Research Center (DRC) Lentiviral RNAi Core
- University of Virginia School of Medicine
- Vector Biolabs
- Vir Biotechnology
- Viridian Therapeutics
- ViThera
- Westen Biotechnology
Methodology
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