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RNAi: Broad Application, Expansive Opportunity, 2nd Edition

Published by: Drug and Market Development Publishing

Published: Jan. 5, 2005 - 212 Pages


Table of Contents



Chapter 1: Executive Summary

Chapter 2: Small Interfering RNAs (siRNAs)

Introduction

Background of Small Interfering RNAs2-3Discovery of RNAi

MicroRNAs

2-3Discovery of RNAi

2-5Current Frontier of Research in RNAi

2-8siRNA Vector Systems

2-10Viral Vectors

2-13Lentivirus Vectors

2-13Lentivirus Vectors

2-14Transfection

2-16In vivo siRNA Delivery

2-17Cholesterol Conjugation for

2-17Specificity of RNAi Molecule

2-18Stabilization of RNA Dimers For Therapeutic Treatments

2-18Duration of RNAi Activity

2-20RNAi Microarrays

2-20Pathway Analysis and Libraries

2-21RNAi Libraries

2-21Genome-Wide Screens and Signaling Pathway Analysis

2-21Patents

2-22References

Chapter 3: Markets

3-1Market Drivers

3-1Cost of Drug Developments

3-1Demand for High-Throughput Target Discovery

3-2Demand for Target Validation

3-2Demand For New Therapeutics

3-3Diseases Ideal for siRNA Therapeutic

3-3Diseases Ideal for siRNA Therapeutic

3-5Viral Infectious Disease

3-7Hepatitis C

3-8HIV

3-9Hepatitis B

3-10Influenza

3-10Severe Acute Respiratory Syndrome Virus (SARS)

3-11Polio Virus

3-11Respiratory Synctial Virus

3-11CNS Disease

3-11Cardiovascular Disease

3-13Other Indications

3-14References

Chapter 4: Other RNAi Technology in Target Validation and Therapeutics

Antisense

4-1Ribozyme

4-3Aptamers

4-4Peptide Nucleic Acids

4-5Morpholino Antisense Nucleotides

4-5References

Chapter 5: Other Rival Technologies In Target Validation

5-1Gene Knockouts

5-1Mice and Rats

5-1Zebra Fish

5-3References

Chapter: 6 Related Technologies Critical To High-Throughput Target Validation

6-1Protein Profiling Technology

6-1Cell Microarrays

6-1Phenotype Profiling Technology

6-2References

Chapter 7: Profiles of Companies Developing siRNA Therapeutics

7-1Acuity Pharmaceuticals

7-1AGY Therapeutics

7-3Alnylam Pharmaceuticals, Inc.

7-6Antisense Therapeutics Limited

7-11Benitec Australia Limited

7-14CytRx Corporation

7-17Genta Incorporated

7-19Hemispherx Biopharma, Inc.

7-23International Therapeutics

7-26Intradigm Corporation

7-28ISIS Pharmaceuticals

7-36Noxxon Pharma AG

7-36Nucleonics Inc.

7-39Sirna Therapeutics, Inc.

Chapter 8: Profiles of Companies Developing siRNA-mediated Target Validation

8-1AtugenAG

8-1Cenix Bioscience GmbH

8-4Compugen Ltd.

8-7deVGen nv

8-10Galapagos Genomics nv .

8-13Immusol Inc.

8-16Ingenium Pharmaceuticals AG

8-19Lexicon Genetics

8-22Phenomix Corporation Phenomix Australia Pty Ltd.
8-26Phylonix Pharmaceuticals, Inc

8-29RNAx GmbH

8-31Zygogen LLC

Chapter 9: Profiles of Companies Specialized in siRNA and siRNA-related Reagents

9-1Allele Biotechnology and Pharmaceuticals, Inc.

9-1amaxa GmbH .

9-3Ambion, Inc. …

9-5CombiMatrix Corporation

9-9Cyclacel

9-12Cyntellect, Inc.

9-15Dharmacon, Inc.

9-17Eurogentec

9-20ExpressOn BioSystems Ltd .

9-22Gene Therapy Systems, Inc.

9-24Imgenex Corp.

9-26Invitrogen Corporation

9-31 Mirus Bio Corporation

9-33Ocimum Biosolutions LLC…

9-37Oligoengine, Inc.

9-39Polyplus-transfection SAS

9-40Proligo

9-42Qiagen N.V

Chapter 10: Trends

10-1Strengths

Weaknesses

0-1Opportunities

0-3Threats

Abstract

Dr. Edward Weck Dr. Weck has extensive experience in industry, technology transfer, and as a consultant. Following work in Germany and California cloning starch control genes, he worked on vectors for corn transformation. This culminated in participation in the development transgenic Bt corn at Northrup King (now Syngenta Seeds). Dr. Weck has recently worked in assessing biotechnologies for transfer to client companies with Interlink Biotechnology. He has authored reports on Transgenic Plants, Transgenic Animals and Enzymes for IBCUSA. He played an important role in transferring plant breeding and genetics technology to developing countries for the FAO/IAEA Joint Division of Nuclear Techniques in Food and Agriculture in Vienna, Austria (1995-1997). Dr. Weck received his Ph.D. in Chemistry from the University of Arkansas in 1980, doing his dissertation work on small molecules involved in tumor regression in chickens. Dr Weck is currently a registered patent agent and Biotechnology consultant at BioTec Information in Northfield, MN and is inhis last year of law school at William Mitchell College of Law.

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