Use of RNAi, rnai therapeutics market

RITMIR006: RNAi – A Market Insight Report, July 2013
© RI Technologies - www.researchimpact.com Sample Page - 1
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 –

 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

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

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 double-
stranded 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,

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.

Segmentation of RNAi
Exhibit 1. Segmentation of Global RNAi Market by Application and Tools/Products
Application Tools/Products
Research Design/Software
Therapeutics Synthesis
Drug Development Delivery
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

Exhibit 2. Segmentation by Application
Research Therapeutics Drug Development
Functional Genomics Oncology Target Identification
Metabolism Neurology Target Validation
Protein Chemistry Ophthalmology Drug Mechanism
Other (1) Virology 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 Synthesis Delivery Monitoring/Optimizing Libraries
miRNA Target
Scan
Expression
Vectors
Vector Based Primers for RTqPCR Human
miRNA Analysis
Software
Chemical Transfection
Reagents
Controls Mouse
Design Software miRNA Isolation Electroporation miRNA Detection Tools Rat
Other (1) Dicer/RNAseIII Other (3) Other (4) Other (5)
In Vitro
Other (2)
1. Includes
Software for
Imaging and
Documentation,
etc.
2. Includes
miRNA Synthesis
Kits, Synthetic
shRNA, etc.
3. Includes
Nanoparticle
Mediated,
Aptamer
Mediated, etc.
4. Includes Gel
Electrophoresis,
Phenotypic Assays, etc.
5. Includes
Drosophila,
Ceanorhabditis,
Arabidopsis, etc.
© RIT, 2013

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

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%.

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

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

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….

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.

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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 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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Use of RNAi, rnai therapeutics market

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