Top Companies and Products in the RNA/DNA Therapy Products Market

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Publication Date: 2007-07-01

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Increasingly large pharmaceutical companies are pumping money and ambition into the DNA/RNA therapy market. Whether its through forming collaboration/licensing agreements, or acquiring smaller companies they can see the financial opportunities. In particular the large pharmaceutical companies Pfizer, sanofi-aventis, Eli Lilly & Company and Merck & Co all have substantial investment in the RNA/DNA therapy products market. With Merck & Co in particularly showing its faith in the future potential of this market by taking the gamble of acquiring the specialist RNAi company Sirna, towards the end of 2006.

This interest reflects the advances being made in this sector and implies that it is eventually on the verge of success. The investment of these larger companies makes that success both more likely to occur and more likely to occur quickly.

This report focuses on the commercial and strategic opportunities within the human genome project. It examines the companies utilizing novel molecular biological techniques to try to exploit this knowledge to produce RNA/DNA products. The genetic characterisation of many viruses is also leading to DNA and RNA products being used to treat infectious disease, a therapeutic area for which traditional drug development is difficult, as with the recent announcement regarding AIDS research.

The report authors have identified 68 companies actively involved in this field, 35 of these companies are primarily focused on the development of RNA/DNA therapy products and 33 companies are involved in collaborations. This represents a substantial investment in the future of the RNA/DNA therapy products market and over the forecast period visiongain believes this investment will begin to pay off. There are 2 RNA/DNA therapy products due to submit licence applications in 2007, with a further 67 products in clinical trials and a further 45 products in pre-clinical development. Of these products visiongain has identified 14 that it believes have the greatest potential both to be launched before 2012 and to make a contribution to this market. This would result in a substantial increase in the RNA/DNA therapy product market over the forecast period and imply that this sector is eventually achieving some of its anticipated potential.

Why you must buy the RNA/DNA Therapy Products Market Forecasts report:

There are many companies using established and novel molecular biological techniques to try to produce successful RNA/DNA products for a variety of indications. However, even with substantial investment, the RNA/DNA therapy products market is still in its infancy, only 3 products have obtained a licence and only Macugen, for wet AMD, is actively available on the worldwide markets. RNA/DNA therapy products have faced problems, principally due to immunogenicity and in proving effective enough, primarily due to inefficient delivery systems.

This exclusive management report delivers:

In the short term the leading potential therapeutic area for RNA/DNA therapy products is ocular disease. Macugen, an aptamer for the treatment of wet AMD, is the only RNA/DNA product currently available worldwide. Visiongain predicts that Macugen will lead the RNA/DNA therapy products market between 2007 to 2012, generating in the region of $1.7bn in cumulative sales over this period. However, it is the cardiovascular sector, with 5 products predicted to launch before 2012, where visiongain anticipates RNA/DNA therapy products will ultimately have the greatest impact.

DNA therapy products is the sector that has the greatest investment, with the most specialist companies and the greatest number of products predicted to launch prior to 2012. However, antisense is the sector with the most collaborating companies and it also has the product Genasense, which has filed for an EU and US license, although it is encountering problems in obtaining these licenses and its future is by no means certain.

RNAi technology undoubtedly has huge potential for developing products in many therapeutic areas. The investment in novel, often nanoparticle, delivery systems for RNAi products may ensure that this potential is ultimately realised. If solutions for efficient delivery of RNAi can be solved early on in RNAi development the RNA/DNA therapy market may see a future expansion due to successful RNAi therapies. RNAi has the potential to emerge as a dominant section of the RNA/DNA therapy products market, but only time will tell if it can ultimately fulfil this potential.

Table of Contents

  • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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. Asklpios Biopharmaceuticals Inc anticipates the launch of Biostrophin in
    • 5.2.11.Neurologix and NLX-E201 for Parkinson's disease
    • 5.2.13. Oxford BioMedica hope to register TroVax in
    • 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-
    • 5.4.1. Ten DNA therapy products could potentially be launched by
    • 5.4.2. Cancer is the leading indication for DNA therapy products likely to launch prior to
    • 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
  • Chapter 6. Top companies and products in the RNA therapy products market
    • 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
    • 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
    • 8.2.1. Macugen is the leading RNA/DNA therapy product
    • 8.2.2.Genasense and Advexin are due to submit license applications in
    • 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
    • 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
  • List of Tables
    • 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
    • Table 3.5. Comparison of Macugen, Lucentis and Retaane
    • Table 3.6. Sales revenue forecast for Macugen 2007-
    • 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
  • List of Figures
    • 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
    • Figure 3.5. Sales forecast for Macugen
    • Figure 3.6. Sales growth rate of Macugen
    • Figure 3.7. Predicted market share of the wet AMD market in
    • 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
    • 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
    • Figure 8.5. Leading indications for the development of RNA/DNA therapy products
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