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RNAi Therapeutics: Challenges in Drug Development and DeliveryPublished by: CHI Insight Pharma Reports Published: Oct. 1, 2005 - 120 Pages Table of ContentsChapter 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 Alnylam Pharmaceuticals Atugen Benitec Calando Pharmaceuticals CytRx Corporation Devgen Galenea Intradigm Corporation Nastech Pharmaceutical Company Nucleonics Inc. Santaris Pharma Sirna Therapeutics Chapter 7. Profiles of Companies in Discovery/Research/Reagents/Services in the RNAi Market 7.1. Background 7.2. Company Profiles Ambion Cenix Bioscience CombiMatrix Dharmacon Imgenex Corporation Invitrogen InvivoGen Mirus Bio Qiagen SomaGenics 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 Glossary Index List of Figures Figure 1.1. Detailed Diagrammatic Representation of Small Interfering RNA (siRNA) Pathway Figure 1.2. Diagrammatic Presentations of Small Interfering RNA (siRNA) and MicroRNA (miRNA) Action Figure 1.3.Timeline of Significant Events in the Discovery and Development of RNAi Figure 1.4. Gene Therapy in Clinical Trials Figure 4.1. Alnylam’s Systemic RNAi Delivery Technology that Is Clinically Relevant Figure 2.1.The Normal Macula Compared to Wet and Dry Macular Degeneration Figure 4.2. RNAi-Mediated Gene Silencing in Mammals Using Short Hairpin RNAs Figure 4.3. Inhibition of Hepatitis C Virus (HCV) Internal Ribosomal Entry Site-Mediated Reporter Gene Expression in Mice Figure 5.1. Key Attributes of RNAi Drugs for Use in Humans Figure 5.2. Possible Risks Before Launching a Successful RNAi Drug Figure 6.1. RNAi Drugs in Development (Alnylam) Figure 6.2. RNAi Drugs in Development (CytRx) Figure 6.3. RNAi Drugs in Development (Intradigm) Figure 6.4. RNAi Drugs in Development (Sirna) List of Tables Table 1.1. Summary of RNAi Results Presented at the 2005 American Society of Gene Therapy Conference, St. Louis, Missouri Table 4.1. Pros and Cons of Electroporetic Delivery Table 4.2. Pros and Cons of Viral Delivery Table 4.3. Pros and Cons of Lipid Delivery Table 6.1. Companies with Plans of Launching RNAi Drugs for One or More Indications AbstractInterfering RNA (RNAi) offers tremendous therapeutic promise. This report:
RNAi Therapeutics: Challenges in Drug Development and Delivery thoroughly evaluates the field of RNAi therapeutics, with particular attention to the prospects and challenges faced in RNAi delivery. This report is a comprehensive blueprint for moving RNAi from the bench to the bedside. RNAi research is accelerating rapidly. Two clinical trials are in progress to determine RNAi’s effectiveness for treating age-related macular degeneration, and many products have already reached the market to test the promise that RNAi holds. Before RNAi, finding gene function by knockdown or knockout technology was a laborious and time-consuming process. As RNAi technology progresses, many companies are now trying to increase their shares in the research market by shifting some of their resources to in vitro and in vivo work. The real prize, however, is in therapeutics, and a few companies have been started solely with this objective.To establish priority, patents that stake claims in many possible (as well as speculative) applications of RNAi are not only being filed but disputed. RNAi Therapeutics: Challenges in Drug Development and Delivery analyzes these and other important developments in this dynamic field. Key points include the following:
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