Top Companies and Products in the RNA/DNA Therapy Products Market Forecasts: The challenge of turning technology into therapy

Chapter 1. Executive Summary

Chapter 2. Introduction to RNA and DNA products

• 2.1.The biology of DNA, RNA, transcription and translation
  • 2.1.1. DNA and RNA
  • 2.1.2. Translation
  • 2.1.3. Transcription
• 2.2. The genetic basis of disease
  • 2.2.1. The genetic basis of inherited diseases
  • 2.2.2.The genetic basis of cancer
  • 2.2.3.The genetic basis of immune and autoimmune disease
• 2.3. The use of RNA and DNA products to silence genes
  • 2.3.1. DNA gene therapy
  • 2.3.2. Antisense
  • 2.3.3. Interfering RNA
  • 2.3.4. Chimera based gene therapy
  • 2.3.3. Aptamers
  • 2.3.6. Ribozymes and DNAzymes
  • 2.3.7.DNA Vaccines
• 2.4. Mode of delivery of DNA and RNA therapy
  • 2.4.1. Viral vectors
  • 2.4.2. Non-viral vectors
• 2.5. A brief history of gene therapy
  • 2.5.1. Gene therapy in infectious disease
  • 2.5.2. Gene therapy in inherited disease
  • 2.5.3. Gene therapy in cancer
  • 2.5.4. Gene therapy in immune and autoimmune disease
• 2.6. Summary

Chapter 3. Top companies and products in the Aptamers market 2007-2012

• 3.1. Leading companies and products in the aptamer market
  • 3.1.1. OSI Pharmaceuticals and the continuing story of the ownership of Macugen
  • 3.1.2. Archemix hold the keys to therapeutic aptamer development
  • 3.1.3. SomaLogic Inc. and diagnostic aptamers
  • 3.1.4. Spiegelmers, second generation aptamers developed by NOXXON Pharma
  • 3.1.5.Nuvelo are developing anticoagulant aptamers with Archemix
  • 3.1.6. Aptamera is acquired by Antisoma
  • 3.1.7. Regado Biosciences are developing antidote controlled anticoagulants
• 3.2. Summary of the top 5 aptamer companies, Archemix leads the way
• 3.3. Summary of the leading developmental aptamer products and their potential markets
• 3.4. Macugen, the leading aptamer product
  • 3.4.1. Age-related macular degeneration
  • 3.4.2.Macugen- effectiveness, current licenses and future applications
  • 3.4.3. The ocular disease market 2006
  • 3.4.4. Future competitors in the AMD market, Evizon and Bevsiranib due to start phase III trials
  • 3.4.5. Comparison of the leading AMD therapies, Lucentis is the market leader
  • 3.4.6. The AMD market 2007-2012 and revenue forecast for Macugen
• 3.5. Summary

Chapter 4. Top companies and products in the antisense market 2007-2012

• 4.1. Phase III failures in antisense
• 4.2. Key companies and products in the antisense market
  • 4.2.1. Vitravene, the first antisense product to obtain a license
  • 4.2.2. Isis Pharmaceuticals is the leading antisense company
    • 4.2.2.1. Isis' s development and licensee partners
    • 4.2.2.2. Isis' s strong antisense pipeline
    • 4.2.2.3. Alicaforsen, Isis' s leading product
    • 4.2.2.4. Isis' s cholesterol lowering, cancer, diabetes and multiple sclerosis antisense products in phase II trials
    • 4.2.2.5. Isis' s pre-clinical product ISIS 369645 for asthma
  • 4.2.3. Is Genasense on the verge of success or failure?
  • 4.2.4. AVI BioPharma plan to launch Resten-CP for retenosis within 5 years
    • 4.2.4.1. AVI BioPharma and Ercole Biotech are collaborating on developing splice-altering technologies
    • 4.2.4.2. AVI BioPharma have collaborations with Eleos and the Defence Threat Reduction Agency
  • 4.2.5. OncoGenex and antisense drugs to reduce cancer drug resistance
  • 4.2.6. Antisense Therapeutics are developing antisense for autoimmune disease
  • 4.2.7. Ester Neurosciences and antisense for neurological disorders
  • 4.2.8. Santaris Pharma and RNA antagonists
  • 4.2.9. Lorus Therapeutics and GTI-2040 for cancer
  • 4.2.10 Enzo Biochem and HGTV43, anti-HIV antisense
  • 4.2.11. Isis is the leading antisense company, who else is in the top 5?
• 4.3. The antisense market 2007-2012, products likely to succeed
  • 4.3.1. The potential market for Genasense is not substantial
  • 4.3.2. Alicaforsen could take a share of the $800m UC market
  • 4.3.3. The considerable potential of the Resten family of antisense products
  • 4.3.4. ISIS 301012 could enhance the effects of statins
• 4.4. Summary

Chapter 5. Top companies and products in the DNA therapy products market 2007-2012

• 5.1. Gendicine is licensed in China
• 5.2. Key companies and products in the DNA therapy product market
  • 5.2.1.Introgen' s large gene therapy product pipeline
    • 5.2.1.2. NGN 241 for head and neck cancer
    • 5.2.1.1. Introgen' s lead product Advexin could be launched in 2008
    • 5.2.1.3.INGN 234, a novel product for prevention of oral cancer and the cancer vaccine INGN 234, are in phase II trials
    • 5.2.1.4. Introgen' s early stage products
  • 5.2.2. Cardium therapeutics and Generx for recurrent angina
  • 5.2.3 Gencell Aventis have NVIFGF in phase III trials
  • 5.2.4. GenVec and TNFerade for pancreatic cancer
  • 5.2.5. AnGes is developing cardiovascular and autoimmune gene therapies
  • 5.2.6. VIRxSYS is producing a lentivector for HIV treatment
  • 5.2.7. Applied Genetic Technologies Corporation hope to launch AGTC-0106 in 2009
  • 5.2.8. Amsterdam Molecular Therapeutics is developing gene therapy for metabolic and ocular disease
  • 5.2.9. Epeius Biotechnologies are developing Rexin- G and other targeted gene therapy products
  • 5.2.10. Asklepios Biopharmaceuticals Inc anticipates the launch of Biostrophin in 2010
  • 5.2.11.Neurologix and NLX-E201 for Parkinson' s disease
  • 5.2.13. Oxford BioMedica hope to register TroVax in 2009
  • 5.2.14. Vical, has patented plasmid DNA delivery technology
    • 5.2.14.1. Vical has Allovectin-7 in phase III trials
  • 5.2.15. Transgene
    • 5.2.15.1. Roche obtain worldwide license for TG 4001 cervical cancer vaccine
    • 5.2.15.2. TG 4010, TG 1042 and MyoSys are in development
  • 5.2.16. Coley and CpG oligonucleotides
  • 5.2.17. Bavarian Nordic is branching out into therapeutic vaccines
  • 5.2.18. Virax and VIR201 HIV DNA vaccine
• 5.3. Who are the top 5 companies in the DNA therapy products market?
• 5.4. The DNA therapy products market 2007- 2012
  • 5.4.1. Ten DNA therapy products could potentially be launched by 2012
  • 5.4.2. Cancer is the leading indication for DNA therapy products likely to launch prior to 2012
  • 5.4.3. The immediate potential market for Advexin is not substantial
  • 5.4.4. Generex and NVIFGF could become leading products in the cardiovascular and RNA/DNA therapy market
  • 5.4.5. Can the cancer vaccines, TroVax, Allovectin-7, PF-351267 and TG4001 make their mark on the cancer therapies market?
  • 5.4.6. Biostrophin or AGTC-0106 could be the first gene therapy products for inherited disease
  • 5.4.7. INGN 234, a product to watch
• 5.5. Summary of the DNA therapy products market 2007-2012

Chapter 6. Top companies and products in the RNA therapy products market 2007-2012

• 6.1. Where is the RZ market in 2007?
• 6.2. The promise of RNAi
• 6.3. Key companies and products in the RNA therapies market
  • 6.3.1. Sirna acquired by Merck & Co
  • 6.3.2. The newly formed company Opko Corporation, has the leading RNAi product Bevasiranib
  • 6.3.3. Gene Shears' s hammerhead RZ for HIV
  • 6.3.4. Alnylam Pharmaceuticals hold patents on RNAi technology
  • 6.3.5. CytRx subsidiary RXi is dedicated to the development of RNAi products
  • 6.3.6. Silence Therapeutics and RTP801i for AMD
  • 6.3.7. Intradigm Corporation and ICS-283 cancer therapy
  • 6.3.8. Nucleonics Inc. and NUC B100 for hepatitis B infection
  • 6.3.9. Nastech Pharmaceutical Company is developing inhaled RNAi products
  • 6.3.10. Calando Pharmaceuticals and RONDEL
  • 6.3.11. The antisense company Lorus Therapeutics is also developing an RNAi product
  • 6.3.12. Who is the leading RNA therapy product company?
• 6.4. The RNA therapy market 2007-2012
  • 6.4.1. The potential market for Bevasiranib
  • 6.4.2. The potential market for HIV hammerhead RZ therapy
  • 6.4.3. Infectious disease is the most promising potential market for RNA therapy products
• 6.5. Summary and outlook for the RNA therapy products market

Chapter 7. Issues affecting the RNA/DNA therapy products market

• 7.1. Principle drivers of the RNA/DNA therapy market
• 7.2. Principle restraints on the RNA/DNA therapy market
• 7.3. Summary

Chapter 8. Conclusion

• 8.1. The top 20 companies in the RNA/DNA therapy products market
• 8.2. Summary of the RNA/DNA therapy market 2007-2012
  • 8.2.1. Macugen is the leading RNA/DNA therapy product
  • 8.2.2.Genasense and Advexin are due to submit license applications in 2007
  • 8.2.3. The RNA/DNA therapy products pipeline is sizeable
  • 8.2.4 Fourteen RNA/DNA therapy products could potentially be launched prior to 2012
  • 8.2.5. Cancer is the leading indication for the development of RNA/DNA therapy products
  • 8.2.6. Cardiovascular disease is the sector that may ultimately gain the most from RNA/DNA therapy products
• 8.3. Summary

• Table 2.1. Types of RNA and DNA products in development
• Table 2.2. Types of delivery systems used for transfer of RNA and DNA products
• Table 3.1 Companies involved in aptamer development
• Table 3.2. Aptamers in development
• Table 3.3. Aptamers being developed by NOXXON
• Table 3.4. The ocular disease market 2006
• Table 3.5. Comparison of Macugen, Lucentis and Retaane
• Table 3.6. Sales revenue forecast for Macugen 2007- 2012
• Table 4.1 Key companies involved in antisense development
• Table 4.2. Antisense products currently in clinical trials
• Table 4.3. ISIS' s antisense products, pipeline and collaborations
• Table 4.4. AVI BioPharma antisense products in development
• Table 5.1. Companies involved in the development of DNA therapy products
• Table 5.2. Products involved in gene therapy clinical trials
• Table 5.3. DNA vaccines in clinical trials
• Table 5.4. Introgen' s DNA therapy pipeline
• Table 6.1. Companies involved in RNA therapy product development
• Table 6.2. RNA therapeutic products in development
• Table 7.1. A SWOT chart for the RNA/DNA therapy products market

• Figure 2.1. Transcription and translation of DNA
• Figure 3.1. Number of aptamers each company has in development, including in collaboration with others.
• Figure 3.2. Number of aptamers in development for different therapeutic area
• Figure 3.3 Number of aptamers in different stages of development
• Figure 3.4. The market share for wet AMD therapies in 2006
• Figure 3.5. Sales forecast for Macugen 2007-2012
• Figure 3.6. Sales growth rate of Macugen 2007-2012
• Figure 3.7. Predicted market share of the wet AMD market in 2012
• Figure 4.1. Number of antisense products ISIS has in different stages of development
• Figure 4.2. ISIS' s therapeutic areas of interest
• Figure 4.3. Number of antisense products, in clinical trials, that companies are involved in developing
• Figure 4.4. Number of antisense products in different phase clinical trials
• Figure 4.5. Distribution of indications with antisense products in development
• Figure 5.1. DNA product companies main therapeutic areas of interest
• Figure 5.2. Number of DNA therapy products in development for different indications
• Figure 5.3. Number of DNA therapy products at different stages of clinical development
• Figure 5.4. The number of products being developed by leading DNA therapy companies
• Figure 5.5. Predicted launch dates of DNA therapy products
• Figure 5.6. Indications likely to benefit from the DNA therapy products by 2012
• Figure 6.1. RNA therapy product companies therapeutic areas of interest
• Figure 6.2. The number of products being developed by leading RNA therapy companies
• Figure 6.3. Number of RNA therapy products at different stages of development
• Figure 6.4. Leading therapeutic areas for which RNA therapy products are being developed
• Figure 8.1. Number of specialist and collaborating companies involved in each sector of the RNA/DNA therapy products market
• Figure 8.2. The number of RNA/DNA therapy products at different stages of development
• Figure 8.3. The distribution of active developmental sectors within the RNA/DNA products market
• Figure 8.4. Predicted potential launch dates of RNA/DNA therapy products 2007-2012
• Figure 8.5. Leading indications for the development of RNA/DNA therapy products

• Acuity Pharmaceuticals
• Advaxis
• Alcon (Nestle)
• Alfresa Pharma Corporation
• Allergan
• Alnylam Pharmaceuticals
• ALSA
• Amsterdam Molecular Therapeutics
• AnGen
• Antisense Therapeutics
• Antisoma
• Applied Genetic Technologies
• Aptamera
• Archemix
• Arrowhead Research
• Asklepios Biopharmaceuticals
• Atlantic Healthcare
• AVI BioPharma
• Bavarian Nordic
• Bayer Schering
• BioAsia
• Biogen Idec.
• Calando Pharmaceuticals
• Cardium
• Colegate Palmolive
• Coley
• Cook Group
• CytRx/Rxi
• Daiichi Pharmaceutical
• Digna Biotech
• Elan
• Eleos
• Eli Lilly
• Enzon Pharmaceuticals
• Epeius Biotechnologies
• Ercole Biotech
• Ester Neurosciences
• eXegenics
• Exiqon
• Eytech
• Froptic Corporation
• Genaera
• Gencell Aventis
• Gene Shears
• Genentech
• Genta
• GenVec
• GlaxoSmithKline
• iCo
• IDIS
• Inex Pharmaceuticals
• Intradigm Corporation
• Introgen
• ISIS Pharmaceuticals
• Johnson & Johnson Pharmaceutical
• Lorus Therapeutics
• Medtronic
• Merck & Co.
• Merck KaA
• Nastech Pharmaceutical
• Neurologix
• Norvartis Ophthalmics
• Novarits
• NOXXON Pharma
• Nucleonics
• Nuvelo
• OncoGenex
• Opko Corporation
• OSI Pharmaceuticals
• Oxford BioMedica
• Oxxon Therapetics
• Pfizer
• Pharmexa
• Proyecto de Biomedicina CIMA
• Quark Biotech
• Quest Diagnostics
• Regado Biosciences
• Roche
• Rosetta Genomics
• S.L.
• Sanofi Aventis
• Santaris Pharma
• Sarissa
• Silence Therapeutics
• Sirna
• Smitomo Pharmaceuticals Co.
• SomaLogic
• SR Pharam
• Therion Biologics
• Transgene
• UTE CIMA
• Vical
• VIRxSYS