Contents:

Overview Catalyst Summary Executive Summary Scope of the analysis Datamonitor insight into the gene therapy market Related reports Upcoming reports 1. Market Overview Key findings Market definition for this report Clinical trial trends 1989-2009 A timeline of clinical gene therapy Geographic distribution of gene therapy clinical trials, 1989-2009 Current market Gendicine (SiBiono, a subsidiary of Benda Pharmaceuticals, China) Factors leading Gendicine to become the first commercially available gene therapy Commercial success of Gendicine Clinical efficacy Safety of Gendicine Lessons from Gendicine Rexin-G (Epeius Biotechnologies, Philippines) Current pipeline Phase III and pre-approval projects Collategene (AnGes and Daiichi Sankyo) Glybera (Amsterdam Molecular Therapeutics) Cerepro (Ark Therapeutics) Generx (Cardium Therapeutics) TNFerade (GenVec) TK007 (MolMed) XRP0038 (Sanofi-Aventis) Allovectin-7 (Vical) Broad applicability across disease types Key therapy areas Most frequently targeted indications Cancer Cancer types targeted by gene therapy Pancreatic cancer Head and neck cancer Breast cancer Prostate cancer Central nervous system Parkinson's disease Leber congenital amaurosis (retinal disorder) Multi-indication potential of nerve growth factor Cardiovascular and circulatory system Peripheral arterial disease Key companies 2. Technology Review Key findings Gene therapy methods Ex vivo gene therapy In vivo gene therapy Delivery of genes Different delivery mechanisms for different situations Viral vectors Movement towards adeno-associated viruses vectors Adenovirus Retrovirus Adeno-associated viruses Mechanical delivery Gene regulation Regulatable vectors 3. Commercial Considerations Key findings Key drivers to commercial success Endless lucrative targets for companies mastering delivery Access to previously untapped markets Key inhibitors to commercial success Safety Immune response Mutagenesis Germline transmission Funding and Big Pharma involvement Academic funding Big Pharma involvement Reliable delivery and regulatable expression Challenging to treat multifactorial diseases Access to vectors Stigma associated with gene therapy Market entry strategies Pricing Regulation US FDA - Center for Biologics Evaluation and Research (CBER) NIH - Recombinant DNA Advisory Committee (RAC) Europe European Medicines Agency (EMEA) Germany UK France Bibliography Journals Websites Appendix A Raw data Appendix B Contributing experts Report methodology About the healthcare research & analysis team Disclaimer List of Tables Table 1: Gendicine efficacy in head and neck squamous cell carcinoma Table 2: Gendicine follow-up survival data in head and neck squamous cell carcinoma Table 3: Phase III and pre-approval gene therapy projects, 2009 Table 4: Gene therapy methods for the treatment of cancer Table 5: Gene therapies in development for pancreatic cancer, 2009 Table 6: Gene therapies in development for head and neck cancer, 2009 Table 7: Gene therapies in development for breast cancer, 2009 Table 8: Gene therapies in development for prostate cancer, 2009 Table 9: Gene therapies in development for Parkinson's disease, 2009 Table 10: Gene therapies in development for Leber congenital amaurosis (retinal disorder), 2009 Table 11: Gene therapies in development for peripheral arterial disease, 2009 Table 12: Vector system properties Table 13: Advantages and disadvantages of the adenoviral vector in gene therapy Table 14: Advantages and disadvantages of the lentiviral vector in gene therapy Table 15: Advantages and disadvantages of the adeno-associated viral (AAV) vector in gene therapy Table 16: Clinical programs in development using gene therapy, 2009 List of Figures Figure 1: Timeline of gene therapy clinical development Figure 2: Number of gene therapy clinical trials approved worldwide, 1989-2008 Figure 3: Geographic distribution of gene therapy clinical trials, 1989-March 2009 Figure 4: Number of clinical trials for gene therapy at each stage of development, 2009 Figure 5: Phase II results investigating high-dose Allovectin-7 in recurrent or unresponsive Stage III/IV melanoma Figure 6: Number of gene therapy clinical projects underway for each therapy area, 2009 Figure 7: Number of genes in clinical trials for each therapy area, 2009 Figure 8: Number of gene therapy clinical projects underway by stage for key therapy areas, 2009 Figure 9: Indications with three or more clinical projects in development, 2009 Figure 10: Number and stage of development for cancer gene therapy programs, 2009 Figure 11: Number and stage of development for CNS gene therapy programs, 2009 Figure 12: Number and stage of development for cardiovascular and circulatory system gene therapy projects, 2009 Figure 13: Key companies with gene products in clinical development, 2009 Figure 14: In vivo versus ex vivo gene therapy Figure 15: Vectors used in gene therapy clinical trials, 1989-March 2009 Figure 16: Vectors used in gene therapy clinical trials, 2009 Figure 17: A typical virus replication cycle Figure 18: Virus types used as gene therapy vectors in clinical trials, 1989-March 2009 Figure 19: Virus types used as gene therapy vectors in clinical trials, 2009