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Global market report for RNAi Drug Development Scope of the Study RNAi report covers the areas of Application of RNAi and the various Tools/Products in the market catering to RNAi based applications. The Application areas covered include Research, Therapeutics and Drug Development. Research applications include Functional Genomics, Protein Chemistry, Metabolism, and Other Applications (such as Cell Biology, Apoptosis); Therapeutic applications include Oncology, Neurology, Ophthalmology, Virology and Other (such as Diabetes, High Cholesterol, Obesity) and Drug Development applications include Target Identification, Target Validation, Drug Mechanism and Other. The Tools/Products sector is segmented into Design/Software which includes Design Algorithms, miRNA Analysis Software, miRNA Target Scan, and Other (such as software for imaging, documentation), Synthesis which include Chemical, In Vitro, Dicer RNAseIII, Expression Vectors, miRNA Isolation and Other (such as miRNA Synthesis Kits, Synthetic shRNA), Delivery which includes Vector Based, Electroporation, Transfection Reagents, and Other (such as Nanoparticle Mediated, Aptamer Mediated), Monitoring/Optimizing which include Primers for RTqPCR, Controls, miRNA Detection Tools, and Other (such as Gel Electrophoresis, Phenotypic Assays) and Libraries which include Human, Mouse, Rat, and Other (such as Drosophila, Ceanorhabditis, Arabidopsis). The report provides an in depth analysis of each of the application and product areas by their respective categories. The study includes estimates and projections for the total global RNAi market and also key regional markets that include The United States, Europe, Asia-Pacific and Rest of World. Estimates and projections are illustrated graphically. Business profiles of 34 major companies are discussed in the report. The report serves as a guide to global RNAi industry, as it covers more than 281 companies that are engaged in RNAi R&D, discovery, testing and supply of products and services. Major Contract Research Organizations serving RNAi industry are also covered in the Corporate Directory section of this report. Information related to recent product releases, product developments, partnerships, collaborations, and mergers and acquisitions is also covered in the report. RNAi report is an ideal research tool providing strategic business intelligence to the corporate sector. This report may help strategists, investors, laboratories, pharmaceutical companies, contract research organizations, biotechnology companies and drug approval authorities in –

RITMIR006: RNAi – A Market Insight Report, July 2013 © RI Technologies -

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 Gauging Competitive Intelligence  Identifying Key Growth Areas and Opportunities  Understanding Geographic Relevance to Product  Knowing Regional Market Sizes and Growth Opportunities and Restraints  Keeping Tab on Emerging Technologies  Equity Analysis  Tapping New Markets

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Analytics and Data Presented in this Report Pertain to Several Parameters Such as –

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Global And Regional Market Sizes, Market Shares, Market Trends Product (Global And Regional) Market Sizes, Market Shares, Market Trends Technology Trends Corporate Intelligence Key Companies By Sales, Brands, Products Consumer Behavioral Patterns Other Strategic Business Affecting Data

Research Methodology RI Technologies publishes business intelligence reports by going through a cycle of diligent research and analysis activity. Research is done using both online and offline resources. The study outline of this report is sketched on the following lines – global market analysis, regional market analysis, product segmentation, global and regional market analysis by product segment, market trends, M&A, R&D, competitive landscape, technology trends, and other key drivers. Current data helps in analyzing the future of the industry and is also helpful for doing market evaluations, and estimating the market size for the future. This report is uniquely researched and the methodology includes:

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Need and Scope of Study Product Definitions Segmental Analysis Regional Analysis Exclusive Data Analytics Corporate Intelligence Feedback

Right from concept to final compilation of this report, both primary and secondary research methods are applied. We have provided exclusive feedback forms/pre-release questionnaires for this report to use the information for authentication of our own findings. Secondary research includes government publications, investment research reports, web based surveys, website information of both companies and markets, and other offline resources such as print publications and CDs.

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II. REPORT SYNOPSIS Introduction to RNAi RNA interference, a term coined by Andy Fire and Craig Mello for their discovery which won them a Nobel Prize in 2006, is abbreviated as RNAi. It stands for the regulation mechanism of gene expression by RNA in living organisms. Andy Fire and Craig Mello worked on Ceonorhabditis elegans.

RNAi - Nature’s Way of Silencing Gene Gene silencing has been a fascination for scientists as it gives them a tool to guide gene expression. They have tried their hands on gene silencing by introducing molecules into cells such as Antisense RNA, Ribozymes etc. RNAi discovery is a boon to the scientists working on gene silencing as it is the natural way of control of gene expression and opens gates to non-immunogenic gene silencing. RNAi was discovered in C. elegans, a nematode, and its presence was cited in other living organisms with some difference in the mode of mechanism but basically with the same frame work. RNAi is induced by miRNA/siRNA. These molecules, in association with RNA induced silencing complexes (RISC), bind to target mRNA and degrade it. There are other proteins involved in the accomplishment of this mechanism, which may differ in different organisms. This innate cell mechanism of gene expression regulation has gained importance since it will not elicit any immune response in the organism, thus making it an ideal therapeutic reaction mechanism.

Definition RNAi is the mechanism of regulating gene expression by double stranded RNA (dsRNA). This mechanism occurs as cell degrades dsRNA and silences the gene with sequence homology to dsRNA (either complete or a part). RNA interference (RNAi) is a process of molecular biology in which the available fragments of doublestranded RNA (dsRNA) impede with the expression of a particular gene that contribute to a homologous sequence with the dsRNA. RNAi is different from other gene silencing processes in the aspect that it can reach from cell to cell and produce heritable phenotypes in first generation progeny when it is applied in Caenorhabditis elegans. Prior to characterization RNAi was known by other names like silencing gene silencing and post transcriptional transgene. It was only after the characterization at the molecular level it was recognized as the same phenomenon. RNA finds wide application in plants to minimize expression and this has become a common practice from many years. This practice started with the introduction of Single-stranded antisense RNA into plant cells,

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which hybridized to form the cognate, single-stranded, sense messenger RNA. Initially the scientists were of the opinion that the resulting dsRNA helix cannot be transformed into a protein, but it has been proved comprehensively that dsRNA was responsible for triggering the RNAi response. The application of dsRNA has increased enormously after the discovery of the RNAi technology, initially in petunias and later in roundworms (C.elegans).

Summary RNAi can be considered as a highly potential and an unambiguous process, which is keenly carried out in the cell by special systems called as the RNA interference machinery. Even though the exact procedure followed by this machinery is not clear it is believed that the system when finds a double-stranded RNA molecule, slices it up with an enzyme called Dicer, and it is at this point the short dsRNA interacts with the RNA-induced silencing complex (RISC). This is followed by RNA unwinding to an ssRNA form, probably by a helicase, and this is considered to be the activation point of the RISC. Further the ssRNA will hybridize with mRNA and if the base pairing is ideal or near to ideal the argonaute protein component of the RISC will cleave mRNA. This phenomenon usually takes place in plants. If the trend takes a reverse swing and base pairing between the siRNA and mRNA remains imperfect then RISC down regulates the genes mainly by the translation inhibition. This is a phenomenon usually observed in animals. The life cycle and reproduction phases of many viruses contain a double-stranded RNA stage, and hence the RNA interference machinery proves as an ideal defense mechanism against such viruses. The system is also made use of by the cell itself to regulate gene activity. Some parts of the genome set down into microRNA, short RNA molecules which in turn fold itself to attain the shapes of hairpin resulting in creating a double strand. On detection of the double strands the RNA interference system destroys all mRNAs that match the microRNA. This prevents the translation and lowers the activity of numerous other genes, which was first depicted in the JAW microRNA of Arabidopsis. This is believed to be responsible for regulation of numerous genes, which controls the plant shape. This system has been identified in several eukaryotes and around 330 microRNAs have been detected in humans. RNAi is considered to be responsible for a number of cellular processes like gene regulation through microRNAs, formation of centromeric structure and heterochromatin formation.

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Segmentation of RNAi Exhibit 1. Segmentation of Global RNAi Market by Application and Tools/Products Application






Drug Development


Other (1)

Monitoring/Optimizing Libraries Other (2)

1. Includes Bioinformatics, Genomics, etc.

2. Includes Cell Lines for Vectors, mRNA Catcher, RNA Isolation Kits, etc.

© RIT, 2013

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Exhibit 2. Segmentation by Application Research


Drug Development

Functional Genomics


Target Identification



Target Validation

Protein Chemistry


Drug Mechanism

Other (1)


Other (3)

Other (2) 1. Includes Cell Biology, Apoptosis, etc.

2. Includes Diabetes, High Cholesterol, Obesity, etc.

3. Includes Other Drug Development Procedures

© RIT, 2013

Exhibit 3. Segmentation by Tools/Products Design/Software





miRNA Target Scan

Expression Vectors

Vector Based

Primers for RTqPCR


miRNA Analysis Software


Transfection Reagents



Design Software

miRNA Isolation


miRNA Detection Tools


Other (1)


Other (3)

Other (4)

Other (5)

3. Includes Nanoparticle Mediated, Aptamer Mediated, etc.

4. Includes Gel Electrophoresis, Phenotypic Assays, etc.

5. Includes Drosophila, Ceanorhabditis, Arabidopsis, etc.

In Vitro Other (2) 1. Includes Software for Imaging and Documentation, etc.

2. Includes miRNA Synthesis Kits, Synthetic shRNA, etc.

© RIT, 2013

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Analysis by Segmentation Application Worldwide RNAi Application market was estimated at US$XX.XX billion in 2012 and is expected to touch US$ XX.XX billion for 2015. Growing at a CAGR of XX.XX % during the period 2005-2020, the market is further projected to reach US$ XX.XX billion by 2020. The Drug Development Application is estimated as the largest market with a share of about XX.XX% followed by Research and Therapeutics with XX.XX% and XX.XX% respectively in 2012. Other Applications are projected to be the fastest growing market for various RNAi applications with a CAGR of XX.XX%, closely followed by Therapeutics and Research growing at XX.XX% and XX.XX% respectively. Exhibit 4. RNAi Applications - Global Value Market Estimations & Predictions (2005-2020) in US$ Million for Research, Therapeutics, Drug Development and Other

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Application Analysis by Geographic Region Research Global market for Research for RNAi application is projected to reach US$ XX.XX billion by 2020 from an estimated US$ XX.XX million in 2012, registering a CAGR of XX.XX% during the analysis period 2005-2020. The United States market for Research (for RNAi Application) was estimated at US$ XX.XX million in 2012 and is estimated to reach about US$ XX.XX million to represent the largest market with a share of XX.XX% in 2020. Growing at a CAGR of XX.XX% during the period 2005-2020, the US is further projected to retain the leading position with market worth US$ XX.XX million in the year 2020. Europe represents the second largest market for Research for RNAi Application with an estimated US$ XX.XX million in 2012 and is projected to attain the value of US$ XX.XX million by 2020. Rest of World is projected to be the fastest growing market during the analysis period with a CAGR of XX.XX%.

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III. MARKET DYNAMICS Market Overview RNAi - A Breakthrough in Molecular Biology The most popular subject in molecular biology and a new regulatory pattern in biology is ‘Gene Silencing’ which is obtained by RNA interference (RNAi) or by DNA methylation process. Naturally gene silencing acts as a gene regulator in plants and animals and pathologically inhibits genes involved in tumor suppression in cancer... Gene silencing is a way of accomplishing selective interference of gene function and loss of function screens on a genomic wide scale in functional genomics. In the mechanism of RNA interference (RNAi), tiny small interference RNA (siRNA) are formed by the splitting of the double stranded RNA (dsRNA) the which are sequence specific mediators of gene silencing. Other major topic is accomplishing gene silencing in the chromatin of mammalian cells by DNA methylation. Few papers analyze the role of gene silencing as a gene regulator in plants and the counter mechanism by which viruses suppress post-transcriptional gene silencing. The role of DNA hypermethylation in the inactivation of suppressing tumor in cancer and the use of gene silencing interfering selectively in functional genomics in majority of organisms has been discussed by other papers. The next step in molecular revolution is the RNA interference technology, which has redefined drug discovery and target ratification, and can bring paradigm shift in the drug discovery and development process. RNA interference is an extremely powerful, post transcriptional gene silencing mechanism and uses short double stranded RNA to mark messenger RNA transcripts for degradation. By using new industry alert from script reports to find out the benefits of RNAi over antisense technology, Its emerging role in gene functions and medicinal developments and its therapeutic applications including in-depth examination of the market potential and a review of intellectual property issues of RNAi, will help in finding out how RNAi is beneficial for your organization. Gene silencing mediated by RNA depicts a number of related processes which use 21- to 25- RNA nucleotides to suppress the specific target genes from expressing. These processes comprise of post-transcriptional regulation of mRNA by either RNAi or internally encoded micro RNAs (miRNAs) and the regulation of transcription of mRNA by mediating RNAi chromatin silencing. Bidirectional transcription or an inverted repeat produces dsRNA, which generally triggers RNAi. dsRNA is broken down into small interfering RNAs (siRNA) of around 21 nucleotide

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which are then placed in the RNA- induced silencing complex (RISC), which splits mRNA sequences fully complementary to the siRNA sequences.miRNAs principally differs from siRNAs in their synthesis, and are similar to siRNAs as functions in RISC- like complexes.miRNAs genes encode precursors with complex hairpin structures which are processed by endonucleolytic cleavage to form mature miRNAs. Animal miRNAs usually form imperfect base pairing to the 3`-untranslated region (3`-UTR) Thereby inhibiting target mRNA translations, whereas a majority of plant miRNAs unlike animal miRNAs show nearly exact complementary coding regions to target mRNA coding regions and resemble siRNAs by triggering mRNA degradation. Finally, siRNAs produced from repeated transcripts of elements are ingrained into the nuclear RNAi-induced initiator of transcriptional silencing complex (RITS) in the RNAi-mediated chromatin silencing. This (RITS) guides histone methylation, which is a modified chromatin and this in turn leads to silencing of the transcription process.

Global Market Analysis

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RNAI - THE OUTLOOK Global RNAi market was estimated at US$2.8 billion in 2010, and with a compound annual growth rate of 27.30% during analysis period 2005-2020, the market is projected to reach US$10.45 billion by 2015. Ambion, Dharmacon, Invitrogen and Qiagen continue to dominate the market for RNAi products. However their positions vary across market segments and geographic regions.Ambion, Invitrogen and Qiagen Products are used equally in academia and industry, while Dharmacon is cited as a supplier far more frequently by industrial researchers than by academic researchers. RNAi’s discovery has led to better understanding of gene control. The benefits of RNAi studies are huge and help regulating both the double-stranded RNA (already present and those produced). The development of an organism and its internal functions depend on its RNAi mechanism. RNAi can also block infected RNA viruses. Foreign elements in the genome can also be mutated. Hence, RNAi has revolutionized not only the study of genes but also its applications in the medical field. RNAi is already proven as a valuable tool in studying gene functions, in target identification and validation….

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IV. PRODUCT/TECHNOLOGY RESEARCH The RNA-mediated gene silencing pathways gained wide awareness and appreciation in the biotechnology industry. The double-stranded RNA (dsRNA) is chopped into small RNAs by an enzyme called Dicer. The small RNAs with the help of silencing effector (RISC) compound bind to homologous target sequences (RNA or DNA) through base pairing, and result in effective stoppage of protein synthesis either at post transcriptional or transcriptional level or both. RNA-mediated gene silencing pathways in plants, animals, and many fungi are a key for the development, chromosome structure, and virus resistance. RNAi provides effective equipment for researchers engaged in the study of gene function. In addition, RNAi generates several novel platforms for the pharmaceutical companies to develop new drugs through target validation and RNA-based therapeutics for human beings. In 1998, Andy Fire and Craig Mello carried out studies on RNA-mediated gene silencing in the nematode worm Caenorhabditis elegans via RNAi, The process in which dsRNA generates sequence-specific mRNA degradation. The RNA-mediated silencing was discovered in few botanical labs, which were engaged in the research of gene silencing in transgenic plants. RNA interference (RNAi) is an age-old defense mechanism used against dsRNA. RNA’s containing 22 nucleotides in length are termed as siRNAs. These are sliced from bigger dsRNA chains using an enzyme called Dicer. The antisense strand of the siRNA is made use of by an RNA-induced silencing complex (RISC) to guide messenger RNA (mRNA) cleavage, which prompts mRNA degradation.

Introduction to RNA RNA can be defined as a group of nucleotides consisting of a nitrogen base, a 5-carbon sugar (ribose), and phosphate groups. The primary function is to transfer genetic information in DNA during the process of protein synthesis. RNA is a nucleic acid polymer containing nucleotide monomers. It consists of a ribose rings and uracil as an exception to DNA which primarily contain deoxyribose and thymine. This gets transcribed from DNA using enzymes called RNA polymerases and further processed using other enzymes. RNA can be considered as a master control responsible for controlling translation of genes into proteins, carrying amino acid to ribosome to form proteins and also converts the transcript into proteins. Gene expression process is essential for all the living organisms. Majority of the genes are found in the chromosomes present in the nucleus of the cell.

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These genes are expressed by the protein synthesis in the cytoplasm. The genetic material deoxyribonucleic acid (DNA) was identified in the year 1944 and the double-helical behavior of DNA was discovered by Crick, James Watson and Maurice Wilkins in the year 1953. These scientists were awarded the Nobel Prize in Physiology or Medicine in the year 1962. However, the DNA in the cell nucleus governing the protein synthesis in the cytoplasm was a mystery. Then a conclusion was drawn that another single-stranded ribonucleic acid (RNA) was present that initiated the protein synthesis in the process. This resulted in the formulation of Central Dogma that indicated that the genetic information is transcribed from DNA to RNA and then translated into protein. Initially, the RNA carrying the genetic information was believed to be present in ribosome’s and based on that a hypothesis called “one gene-one ribosome-one protein was formulated. Francois Jacob and Jacques Monod discovered a visionary gene control model in the year 1961. They shared the Nobel Prize in Physiology or Medicine with André Lwoff in the year 1965. The model suggested that the gene gets transcribed to a specific RNA species called messengerRNA (mRNA). Later, it was proved that the synthesis of proteins was caused by a short-lived, non-ribosomal RNA. Soon the genetic code was broken by the scientists Marshall Nirenberg and Gobind Khorana and the code words (codons; triplets of nucleotides) were assigned to the twenty amino acids. They shared the Nobel Prize in Physiology or Medicine with Robert Holleyin the year 1968. According to Francis Crick, an RNA molecule plays a role of adaptor among mRNA and the amino acid, and this led to the identification of a short, stable RNA called transfer RNA (tRNA).

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About RI Technologies RI Technologies is a premier source of market research on the Biotechnology & Healthcare sector. We emphasize on factual insights and forecasts with maximum global coverage. RI Technologies is constantly monitoring the biotechnology & Healthcare industry, tracking market trends, and forecasting industry based on specialized analysis. The life sciences sector is an ever growing marketplace with emerging technologies in areas of discovery, design and development.

Research – As Good as the Methodology is! 

Gauging Competitive Intelligence

Identifying Key Growth Areas and Opportunities

Understanding Geographic Relevance to Product

Knowing Regional Market Sizes and Growth Opportunities and Restraints

Keeping Tab on Emerging Technologies

Equity Analysis

Tapping New Markets

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