RNAi Therapeutics: Challeges in Drug Development and Delivery

Table of Contents

Chapter 1. Introduction

1.1. Promising Therapeutic Avenue

1.2. Discovery of RNAi

1.3. RNAi Mechanism

1.4. Gene-Silencing Technology

• Aptamers
• Ribozymes
• Antisense
• Difficulties Plaguing Antisense Clinical Trials

1.5. Gene Therapy

• Approaches to Gene Therapy
• Latest Advances in RNAi Applications to Gene Therapy

Chapter 2. RNAi in Discovery and Development

2.1. Criteria to be Satisfied before Moving into RNAi Clinical Trials

2.2. Targeting and Validation Using RNAi

2.3. RNAi in Clinical Trials for (Wet) Age-Related Macular Degeneration

• Acuity Pharmaceuticals
• Sirna Therapeutics

Chapter 3. Prospects for Curing Disease By RNAi Gene Silencing

3.1. RNAi in Viral and Infectious Disease States

• Human Papilloma Virus
• Human Immunodeficiency Virus Type I
• Rotavirus
• Malaria
• Influenza

3.2. RNAi and Diabetes

3.3. RNAi and Cancer

• Progress in In Vitro RNAi Work with Cultured Cells
• Progress in In Vivo Gene Silencing

3.4. Prospects of Improving Existing Treatment for Chronic Myelogenous Leukemia Patients by RNAi

3.5. RNAi as a New Kind of Gene Therapy: Therapeutic Potential and Specific Applications

• RNAi and Spinocerebellar Ataxia
• RNAi-Mediated Gene Therapy and Intracranial Tumor
• Transposon-Delivered RNAi and Huntington's Disease
• Advantages and Challenges of RNAi Therapy

Chapter 4. RNA Delivery

4.1. Challenges to Overcome in Delivering RNA

4.2. Standard Delivery Methods

• Electroporation
• Viral Delivery
• Lipid Delivery
• Systemic Delivery

4.3. Is Delivery of Stabilized RNAi Molecules Essential?

• Use of Plasmid Vectors

4.4. Detection/Proof of Knockdown Efficiency In Vivo

4.5. Specific Academic and Industry Concerns about RNAi Delivery

Chapter 5. Market and Market Potential

Chapter 6. Profiles of Companies with Primary Interest in RNAi Therapeutics

6.1. Background

6.2. Company Profiles

• Acuity Pharmaceuticals
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Alnylam Pharmaceuticals
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Atugen
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Benitec
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Calando Pharmaceuticals
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• CytRx Corporation
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Devgen
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Galenea
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Intradigm Corporation
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Nastech Pharmaceutical Company
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Nucleonics Inc.
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Santaris Pharma
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Sirna Therapeutics
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges

Chapter 7. Profiles of Companies in Discovery/Research/ Reagents/Services in the RNAi Market

7.1. Background

7.2. Company Profiles

• Ambion
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
• Cenix Bioscience
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• CombiMatrix
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• Dharmacon
• Activities
  • History
  • Research and Development/Products
  • Business Strategy
• Imgenex Corporation
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
• Invitrogen
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• InvivoGen
• Activities
  • History
  • Research and Development/Products
  • Business Strategy
• Mirus Bio
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
• Qiagen
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy
  • Challenges
• SomaGenics
  • Activities
  • History
  • Research and Development/Products
  • Business Strategy

Chapter 8. Expert Interviews

8.1. Background

8.2. Interviews/Summaries

• Dr. Mick W. Graham, Principal Scientist, Benitec
• Dr. Brian Johnston, President and CEO, SomaGenics Inc., and Consulting Associate Professor of Pediatrics, Stanford University School of Medicine
• Dr. Sujay Singh, President and CEO, Imgenex Corporation
• John Rossi, City of Hope
• Dr. Barry Polisky, Senior Vice President of Research and CSO, Sirna Therapeutics
• Dr. Jean-Paul Reynes, Chief Scientist, siRNA Project, InvivoGen
• Dr. Henrik Orum, Vice President and CSO, Santaris Pharma
• Dr. Sam Reich, Vice President of Research and Development, Acuity Pharmaceuticals

Chapter 9. Concluding Remarks

Acknowledgments

References

Suggested Reading

Glossary

Index