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Delivering RNAi-Based Therapeutics & Diagnostics: Challenges & Opportunities Product Image

Delivering RNAi-Based Therapeutics & Diagnostics: Challenges & Opportunities

  • Published: July 2009
  • 225 Pages
  • PharmaVision

FEATURED COMPANIES

  • AiRNA Pharmaceuticals
  • Calando
  • Genetics Corporation
  • Mirna
  • Pharmaceuticals
  • Senesco Technologies
  • MORE

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.

“Several novel RNAi-based therapeutics are expected to be launched in the next ten years as companies have made significant advances READ MORE >

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)

RNAi therapeutic companies:
- AiRNA Pharmaceuticals
- Allergan
- Alnylam
- Pharmaceuticals
- Benitec
- BioCandell Therapeutics
- Calando
- Pharmaceuticals
- Cenix BioScience
- Cequent Pharmaceuticals
- Copernicus
- Therapeutics
- Dicerna Pharmaceuticals
- Expression Genetics Corporation,
- General Research Laboratory
- Intradigm Corporation
- MDRNA
- Mirna
- Therapeutics
- MiRagen Therapeutics
- Nitto Denko Technical Corporation,
- Opko Health
- Oxford BioMedica
- Polyplus Transfection
- Quark
- Pharmaceuticals
- Regulus Therapeutics
- RXi Therapeutics
- Santaris
- Pharma
- Senesco Technologies
- Silence Therapeutics
- Siranoamics,
- siRNAsense A/S
- Sylentis Pharmaceuticals
- Tacere Therapeutics
- Targeted
- Genetics Corporation
- Tekmira Pharmaceuticals Corporation.

Antisense therapeutic companies:
- Isis Pharmaceuticals
- Lorus
- Therapeutics & Santaris Pharma.

Pharmaceuticals & biotechnology companies:
- AstraZeneca
- Bristol-
- Myers Squibb
- Biogen Idec
- Cubist Pharmaceuticals
- Eli Lilly
- Enzon,
- Genzyme
- Kyowa Hakko Kirin
- Merck & Co.
- Novartis
- Oncolys BioPharma,
- Pfizer
- Roche & Takeda.

RNAi diagnostic companies:
- Asuragen Diagnostics
- Exiqon Diagnostics
- Rosetta Genomics

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