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Delivering RNAi-Based Therapeutics & Diagnostics: Challenges & Opportunities

PharmaVision
July 1, 2009
225 Pages - SKU: PBA2492828

Abstract Table of Contents Search inside this report Related Reports

This cutting edge & insightful report can be used to interpret & assess the potential of RNAi-based medicines. It provides opinions & market projections to:

assess the commercial potential of RNAi-based therapies in all of the key therapeutic areas: cancer, CNS disorders, viral infectious diseases, ocular diseases, cardiovascular, metabolic and other conditions;
identify key pharma & delivery specialists advancing RNAi-based therapeutics & diagnostics;
gain an in-depth understanding of the technological & delivery issues which face companies developing RNAi-based therapies;
evaluate the options available for chemically and structurally modifying RNAi silencing agents and delivering them to target tissues now & in the future;
discover which companies are tackling efficacy and safety issues & are in a prime position to exploit new opportunities;
analyze how the market will evolve over the next decade highlighting key trends, opportunities & challenges.

Chapter 1 RNA Interference & Delivery: Key Findings; Introduction; RNAi a Natural Phenomenon; Antisense technologies; RNAi silencing agents; RNA silencing pathways; microRNAs and antagomirs; miRNA gene silencing; Pros & Cons of RNAi Silencing Agents; RNAi R&D; High throughput in vitro screening; RNAi in vivo screening; RNAi diagnostic makers; RNAi Therapeutics; RNAi Diagnostics; RNAi Therapy - A Discovery Timeline; Major Scientific Challenges for the Development of RNAi-Based; Diagnostics & Therapies; Report Structure
Chapter 2 RNAi-Based Therapeutics: Pipeline Analysis: Key Points; Key Therapy Areas Covered by RNAi-Based Therapies; Product pipelines; RNAi for Diagnostics; RNAi Companies Analyzed in This Report
Chapter 3 RNAi-Based Therapies for Cancer: Key Findings; Introduction; RNAi delivery in cancer; RNAi-Based Therapies -siRNA; Case study: ALN-VSP (Alnylam Pharmaceuticals); Case study: CALAA-01 (Calando Pharmaceuticals); Case study: Dicer substrate RNA (Dicerna Pharmaceuticals); Case study: Intratumoral siRNA (Genesis Research & Development; Corporation); Case study: NPX delivery technology (Intradigm Corporation); Case study: SNS-01 (Senesco Technologies); Case study: Atu027 (Silence Therapeutics); Case study: STP702 (Sirnaomics); Case study: PLK SNALP (Tekmira Pharmaceutical Corporation); RNAi-Based Therapies -shRNA; Case study: CEQ501 (Cequent Pharmaceuticals); Alternative RNA Approaches to Develop Cancer Therapies; miRNA in Cancer; Case study: miRNAs (Mirna Therapeutics); Case study: miRNAs (Regulus Therapeutics); Case study: SPC2996 & EZN3042 (Santaris Pharma A/S/Enzon; Pharmaceuticals); MiRNA-Based Diagnostics; mRNA Antagonists; Case study: LOR-2040 (Lorus Therapeutics); Antisense; Case study: OGX-011 (Isis Pharmaceuticals/OncoGenex) &; LY2181308 (Isis Pharmaceuticals/Eli Lilly); Case study: TF siRNA (siRNAsense A/S/Polyplus Transfection); Conclusions; Our Opinion on RNAi-Based Therapies & Diagnostics for Cancer; Where the technology is now, its evolution, achievements and pitfalls; Competition; Potential future applications; Activity in the market, major players and winners
Chapter 4 RNAi-Based Therapies for CNS Disorders: Key Findings; Introduction; Neurodegenerative Diseases; Amyotrophic lateral sclerosis; Case study: SOD1 siRNA (RXi Therapeutics); Case study: SOD1 shRNA (Oxford BioMedica); Huntington’s Disease; Case study: ALN-HTT (Alnylam Pharmaceuticals/Medtronic); Case study: AVV-HTT (Targeted Genetics Corporation); Parkinson’s Disease; Case study: SNCA-siRNA (Alnylam Pharmaceuticals); Case study: biotinylated siRNA (ArmaGen Technologies); Other CNS conditions; Conclusions; Our Opinion on RNAi-Based Therapies for CNS disorders; Where the technology is now, its evolution, achievements and pitfalls; Competition; Potential future applications; Activity in the market, major players and winners
Chapter 5 RNAi-Based Therapies for Viral Infectious Diseases: Key Findings; Introduction; Respiratory Syncytial Virus; Case study: ALN-RSV01 (Alnylam Pharmaceuticals); Hepatitis C; Case study:TT-033 (Oncolys BioPharma/Pfizer/Tacere Therapeutics); Case study: miR-122 (Regulus Therapeutics); Case study: SPC3649 (Santaris Pharma A/S); Human Immunodeficiency Virus; Case study: rHIV7-shl-TAR-CCR5RZ (Benitec Ltd/City of Hope); Pandemic Influenza; Bioterrorism; Other Infectious Diseases; Conclusions; Our Opinion on RNAi-Based Therapies for Viral Infectious Diseases; Where the technology is now, its evolution, achievements and pitfalls; Competition; Potential future applications; Activity in the market
Chapter 6 RNAi-Based Therapies for Ocular Diseases: Key Findings; Introduction; Age-Related Macular Degeneration; Case study: AGN-745 (Allergan/Sirna Therapeutics); Case study: VEGF-A165b sparing siRNA & Bevasiranib (Opko Health); Case study: PF-4523655 (Quark Pharmaceuticals/Pfizer); Diabetic Retinopathy & Diabetic Macular Edema; Glaucoma; Conclusions; Our Opinion on RNAi-Based Therapies for Ocular Diseases; Where the technology is now, its evolution, achievements and pitfalls; Competition; Potential future applications; Activity in the market
Chapter 7 RNAi-Based Therapies for Cardiovascular, Metabolic & Other Conditions: Key Points; Introduction; RNAi-Based Therapeutics for Cardiovascular Diseases; Case study: miR-21 (Regulus Therapeutics); Case Study: MHC miRNA inhibitors (MiRagen Therapeutics Inc); RNAi-Based Therapeutics for Metabolic Disorders; Case Study: ApoB SNALP (Tekmira Pharmaceutical Corporation); Case study: MDR-04227 (MDRNA); Case Study: PCSK9 siRNA (Alnylam Pharmaceuticals); Case study: ApoB rxRNA and GeRP delivery (RXi Therapeutics); RNAi-Based Therapeutics in Other Therapeutic Areas; Case study: ALN-TTR siRNA (Alnylam Pharmaceuticals); Case study: HSP47 siRNA (Nitto Denko Technical Corporation); Case study: QPI-1002 (Silence Therapeutics/Quark Pharmaceuticals); Conclusion; Our Opinion on RNAi-Based Therapies for Cardiovascular, Metabolic; & Other Diseases; Where the technology is now, its evolution, achievements and pitfalls; Competition; Potential future applications; Activity in the market, major players and winners
Chapter 8 Challenges & Opportunities for RNAi-Based Therapeutics & Diagnostics: Key Points; Introduction; Stability & Efficacy Issues for RNAi Research; Stability; Efficacy; Safety Issues for RNAi Research; Intellectual Property; Delivering RNAi-Based Therapies; Challenges; Opportunities
Chapter 9 The Future of RNAi-Based Therapeutics & Diagnostics: Market Trends: Key Points; Key RNAi Companies; Leading RNAi-Based Therapeutic Companies; Leading miRNA-Based Therapeutic Companies; Leading RNAi-Based Diagnostic Companies; Recent Deals & Alliances; The Global RNAi-Based Therapy Market 2008-2014; Analysis parameters; Market forecast 2008-2014; Global RNAi-Based Therapy Market in 2020; Drivers and market trends; Summary & Conclusions; Acknowledgements; Bibliography & Endnotes
List of Figures: Figure 1: siRNA - RISC mediated gene silencing; Figure 2: miRNA - RISC mediated gene silencing; Figure 3: High throughput RNAi screening for functional genomics and target validation; Figure 4: Rapid lead generation; Figure 5: ArteMice© RNAi model; Figure 6: RNAi therapeutics applications; Figure 7: Stages of pharmaceutical development; Figure 8: RNAi research: a timeline; Figure 9: RNAi-based therapeutics: defining therapeutic areas; Figure 10: Number of projects in the different stages of pharmaceutical development; Figure 11: Number of projects in each therapeutic area; Figure 12: Proportions of naked and targeted/vehicle assisted RNAi species; Figure 13: Delivery routes for RNAi therapeutics; Figure 14: Current approaches for cancer treatment; Figure 15: Potential RNAi approaches for cancer treatment; Figure 16: RONDEL delivery platform; Figure 17: In vivo efficacy studies with CALAA-01; Figure 18: Dicer substrate processing; Figure 19: Intratumoral siRNA delivery mediated with A) liposome and B) nanoparticles; Figure 20: RNAi NPX delivery platform; Figure 21: SNS-01 in mouse lung model; Figure 22: AtuPlex proprietary delivery system; Figure 23: SNALP delivery technology; Figure 24: Cequent’s tkRNAi delivery system; Figure 25: LNA monomer structure; Figure 26: Polyplus transfection In-vivo-jetPEI system -RNAi delivery to the lung; Figure 27: Photochemical Internalization enhances siRNA’s gene silencing effect; Figure 28: RNAi applications in CNS disorders; Figure 29: Medtronic’s SynchroMed II implantable infusion pump; Figure 30: Alzet osmotic pump for brain infusion; Figure 31: Diffusion profile of pressure and diffusion delivery; Figure 32: Molecular schematic of ArmaGen’s molecular Trojan horse; Figure 33: RNAi viral infectious disease targets; Figure 34: PARI Pharma’s eFlow nebuliser; Figure 35: Tacere’s shRNA construct to target HCV; Figure 36: Benitec’s RNAi approach to HIV; Figure 37: Proof of concept Ebola siRNAs; Figure 38: RNAi ocular disease targets; Figure 39: RNAi applications in CV, metabolic & other disorders; Figure 40: miR-21 mechanism of action in myocardial infarction; Figure 41: ApoB SNALP preclinical data in mouse model; Figure 42: ApoB nanotransporter in mouse model; Figure 43: GeRP mechanism of delivery; Figure 44: NDT’s siRNA delivery platform; Figure 45: Mechanism of action of siRNA HSP47 in liver cirrhosis; Figure 46: Challenges and opportunities for RNAi research; Figure 47: Safety issues; Figure 48: RNAi-based therapeutics: market drivers
List of Tables: Table 1: Antisense drugs in clinical development; Table 2: Pros and cons of RNAi silencing agents; Table 3: Leading RNAi diagnostic companies; Table 4: Novel non-coding RNA; Table 5: Diseases targeted by RNAi-based therapeutics; Table 6: RNAi and RNA therapeutic & diagnostic companies discussed in this report; Table 7: Leading companies evaluating RNAi therapies for cancer; Table 8: Academic institutions with an interest in RNAi based cancer research; Table 9: Potentially pro-oncogenic and tumor suppressor miRNAs; Table 10: Identification of key tumor suppressor miRNAs; Table 11: Leading companies evaluating RNAi-based therapies for CNS disorders; Table 12: Academic institutions with an interest in RNAi for the treatment CNS disorders; Table 13: Leading companies evaluating RNAi-based therapies for viral infectious diseases; Table 14: Academic institutions with an interest in RNAi for the treatment of viral infectious diseases; Table 15: Leading companies evaluating RNAi-based therapies for ocular diseasess; Table 16: Academic institutions with an interest in RNAi for the treatment of ocular diseases; Table 17: Leading companies evaluating RNAi-based therapies for cardiovascular disease; Table 18: Academic institutions with an interest in RNAi for the treatment of cardiovascular disease, metabolic & other conditions; Table 19: Leading companies evaluating RNAi-based therapies for metabolic disorders; Table 20: Academic institutions with an interest in RNAi for the treatment of metabolic disorders; Table 21: Leading companies evaluating RNAi-based therapies for other conditions; Table 22: A seminal RNAi patents; Table 23: Summary of leading RNAi-based therapeutic companies; Table 24: Recent RNAi acquisitions & licensing deals; Table 25: Recent RNAi alliances; Table 26: RNAi-based therapies included in the market forecast; Table 27: Forecast of pipeline RNAi therapy products 2008-2014 (US$m); Table 28: Forecast of pipeline RNAi therapy products 2015-2020 (US$m)

Delivering RNAi-Based Therapeutics & Diagnostics: Challenges & Opportunities

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