RNA Interference Therapy in Oncology Drug Pipeline Update 2012

• Publication date: February 2012

Potentially any disease-causing gene, cell type or tissue can be targeted with RNAi, including those not ’druggable’ with small molecules or protein-based therapies. There are today 43 companies plus partners developing 58 RNA interference drugs in 75 developmental projects in cancer. In addition, the accumulated number of ceased drugs over the last years amount to another 23 drugs. Rna Interference Therapy In Oncology Drug Pipeline Update...

Potentially any disease-causing gene, cell type or tissue can be targeted with RNAi, including those not ’druggable’ with small molecules or protein-based therapies.
There are today 43 companies plus partners developing 58 RNA interference drugs in 75 developmental projects in cancer. In addition, the accumulated number of ceased drugs over the last years amount to another 23 drugs. Rna Interference Therapy In Oncology Drug Pipeline Update lists all drugs and gives you a progress analysis on each one of them. Identified drugs are linked to 44 different targets. These targets are further categorized on in the software application by 23 classifications of molecular function and with pathway referrals to BioCarta, KEGG and NetPath.

How May Drug Pipeline Update Be of Use?
* Show investors/board/management that you are right on top of drug development progress in your therapeutic area.
* Find competitors, collaborations partners, M&A candidates etc.
* Jump start competitive drug intelligence operations
* Excellent starting point for world wide benchmarking
* Compare portfolio and therapy focus with your peers
* Speed up pro-active in-/out licensing strategy work
* Fast and easy way of tracking drugs using search engines; just one click from inside the application and you may search the World Wide Web and PubMed for any drug.

Drug Pipeline Update is delivered to you as a downloadable application, which requires no installation on your computer. Please read more about application features and system requirements below.

Drug Pipeline Update at a Glance

Investigators
Includes more than 43 principal investigators plus their collaborators. There is direct access from inside the application to web pages of all principal investigators.

Note: You are able to sort and find drugs according to investigators and partners from drop-down menus in the application. You may also sort and find drugs according to country of investigator.

Drug name & Synonyms
Lists commercial, generic and code names for drugs.

Developmental stage
This Drug Pipeline Update contains 58 RNA interference drugs in development, which have a total of 75 developmental projects in cancer. In addition there are ceased drugs.

Pipeline Breakdown According to Number of Drugs
Phase I# 7
Preclinical# 59
Suspended# 0
Ceased# 23

Note: You are able to sort and find drugs according to developmental stage from drop-down menu in the application.

Indications
Included RNA interference drugs are also in development for 26 other indications, where of 22 are different cancer indications.

Note: You are able to find and sort drugs according to type of indication from drop-down menu in the application.

Targets
Identified drugs are linked to more than 44 different targets, divided into 23 classifications of molecular function:
- Cell adhesion molecule activity
- Cofactor binding
- Cytokine activity
- Growth factor activity
- GTPase activity
- Kinase activity
- Ligand-dependent nuclear receptor activity
- Metallopeptidase activity
- Molecular function unknown
- Motor activity
- Oxidoreductase activity
- Peroxidase activity
- Protease inhibitor activity
- Protein serine/threonine kinase activity
- Protein threonine/tyrosine kinase activity
- Receptor activity
- Receptor signaling complex scaffold activity
- RNA binding
- Structural molecule activity
- Transcription factor activity
- Translation regulator activity
- Transmembrane receptor protein tyrosine kinase activity
- Unknown

Identified targets are categorized into 15 different primary and alternate sub-cellular localizations:
- Cell junction
- Cell surface
- Centrosome
- Clathrin-coated vesicle
- Cytoplasm
- Cytoskeleton
- Endoplasmic reticulum
- Endosome
- Extracellular
- Golgi apparatus
- Microtubule
- Mitochondrion
- Nucleolus
- Nucleus
- Plasma membrane

Note: You are able to find and sort drugs according to target gene name, molecular function of target, and target localization from drop-down menus in the application.

Target Expression Profile
Direct links are provided from inside the application to 36 protein expression profiles of 25 drug targets in various human tissues and cancer types, cell lines and primary cells, including up to:
- 48 different normal tissue types
- 20 different types of cancer
- 47 cell lines
- 12 samples of primary blood cells

Pathway Referals
Identified targets have been cross referenced against their involvement in different cellular pathways, according to BioCarta, KEGG and NetPath.
- BioCarta# 104 Pathways
- KEGG# 65 Pathways
- NetPath# 19 Signaling Pathways

Note: You are able to find and sort drugs according to targeted pathways from drop-down menus in the application.

Mechanism
In total there are different drug mechanism of action represented in this Drug Pipeline Update.

Note: You are able to find and sort drugs according to mechanism of action from drop-down menu in the application.

Compound
Identified drug compounds are described by:

Compound type, Chemical name, CAS Number and molecular weight

Note: You are able to sort and find drugs according to compound type from drop-down menu in the application.

Drug Profile
Progress analysis and review of drug development. A typical drug profile reports on, depending on stage of development and available information:

Drug Name & Synonyms
Presentation of drug name and synonyms

Principal Investigator & Partners
Presentation of principal investigator and partners

Target and Molecular Function of Target
Described target(s) is/are presented with:
Official Gene Symbol – Official Gene Name [Species/Homo Sapiens] – Molecular Function

Target Localization
Described target(s) is/are presented with primary and alternate localizations.
Target Expression Profiles
Links to protein expression profile(s) of target(s) in various human tissues, cell lines and primary cells, including up to:
48 different normal tissue types
20 different types of cancer
47 cell lines
12 samples of primary blood cells

Targeted Pathways
Described target(s) is/are matched for the involvement in cellular pathways according to BioCarta, KEGG, and NetPath.

Mechanism
Drug mechanism of action

Developmental Projects
Summary field of developmental projects for the drug, including indication, developmental stage and status.
Example:
Cancer, myeloma – Phase II Clinical Trial – Active
Cancer, prostate – Phase III Clinical Trial – Ceased

Drug Description
Short introduction to drug

Compound Data
Compound type, Chemical name, CAS Number and molecular weight

Patent Data
Available patent information related to the drug is presented here.

Business & Markets
Collaborations and deals
Approvals and submissions
Analyst comments

Phase III Data
Available Phase III development data, developmental history and scientific data.

Phase II Data
Available Phase II development data, developmental history and scientific data.

Phase I Data
Available Phase I development data, developmental history and scientific data.

Preclinical Data
Available preclinical development data, developmental history and scientific data.

Licensing Information
Availability for licensing

Application Features
Use 13 different parameters, available in drop-down menus, to find and sort among drugs
- Drug status
- Clinical Trial Status
- Principle Investigator
- Partners
- Indication
- Developmental Stage
- Compound Type
- Mechanism
- Target
- Molecular Function of Target
- Target Localization
- Targeted Pathways
- Free text search of entire drug profile contents

Direct linkage from inside the application to related internet resources
- Drug data is linked to search engines like Google and PubMed
- Drug target data is linked directly to BioCarta, Human Protein Atlas, KEGG, and NetPath
- Direct links to company web pages of principal investigators

Dynamic Report Generator
Our dynamic report generator lets you with ease and speed generate html reports directly in your web browser (Internet Explorer and FireFox), whether it is a single drug profile or an entire search you want have a report of.

System Requirements
- Operating system: Windows (2000/XP/Vista/7) and Mac OS X 10.6 (Snow Leopard) and OS X 10.7 (Lion)
- Browser Application (Internet Explorer)
- Internet access (to access related internet resources)

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