Stakeholder Opinions: Gene Therapy - Gene therapy to deliver after 20 years of clinical development

Introduction

Gene therapy looks to be coming of age, with seven projects in Phase III clinical development and one project currently under regulatory review. A shift in development from pure gene replacement to using gene therapy as a therapeutic delivery tool has enhanced the technology's commercial potential over the last 10 years.

Scope

*In-depth analysis of the current gene therapy clinical pipeline.

*Analysis of key target indications for gene therapies and key companies active in the filed.

*Key opinion leader review of technologies driving clinical gene therapy.

*Identification of drivers and inhibitors to the commercial success of gene therapy.

Highlights

Most of the indications being targeted in the current gene therapy pipeline are multifactorial rather than monogenic in etiology. Reflecting this trend, cancer has become the leading therapy area for gene therapy development, constituting nearly half of the clinical projects under development.

The development of highly engineered recombinant viruses has increased the safety of gene therapy. As clinical evidence for the enhanced safety of these vectors gathers over the next few years, the perceived risk/reward profile for gene therapy will change in some disease areas.

With endless lucrative targets for companies developing safe and efficient delivery of genes, the commercial potential of gene therapy remains high.

Reasons to Purchase

*Quantify the gene therapy pipeline.

*Assess the changing vectors and target indications for gene therapy.

*Identify the leading companies involved in gene therapy development.


Report Highlights

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Overview 1
Catalyst 1
Summary 1
About Datamonitor healthcare 2
About the healthcare research & analysis team 2
Executive Summary 3
Scope of the analysis 3
Datamonitor insight into the gene therapy market 3
Related reports 4
Upcoming reports 4
Table of Contents 5
1. Market Overview 6
Key findings 6
Market definition for this report 7
Clinical trial trends 1989-2009 7
A timeline of clinical gene therapy 7
Geographic distribution of gene therapy clinical trials, 1989-2009 11
Current market 12
Gendicine (SiBiono, a subsidiary of Benda Pharmaceuticals, China) 12
Factors leading Gendicine to become the first commercially available gene therapy 13
Commercial success of Gendicine 13
Clinical efficacy 14
Safety of Gendicine 15
Lessons from Gendicine 16
Rexin-G (Epeius Biotechnologies, Philippines) 16
Current pipeline 16
Phase III and pre-approval projects 17
Collategene (AnGes and Daiichi Sankyo) 18
Glybera (Amsterdam Molecular Therapeutics) 18
Cerepro (Ark Therapeutics) 19
Generx (Cardium Therapeutics) 19
TNFerade (GenVec) 20
TK007 (MolMed) 20
XRP0038 (Sanofi-Aventis) 20
Allovectin-7 (Vical) 21
Broad applicability across disease types 22
Key therapy areas 23
Most frequently targeted indications 25
Cancer 26
Cancer types targeted by gene therapy 27
Pancreatic cancer 29
Head and neck cancer 30
Breast cancer 31
Prostate cancer 32
Central nervous system 32
Parkinson's disease 33
Leber congenital amaurosis (retinal disorder) 35
Multi-indication potential of nerve growth factor 35
Cardiovascular and circulatory system 36
Peripheral arterial disease 37
Key companies 39
2. Technology Review 41
Key findings 41
Gene therapy methods 42
Ex vivo gene therapy 42
In vivo gene therapy 43
Delivery of genes 43
Different delivery mechanisms for different situations 45
Viral vectors 46
Movement towards adeno-associated viruses vectors 47
Adenovirus 49
Retrovirus 50
Adeno-associated viruses 53
Mechanical delivery 56
Gene regulation 58
Regulatable vectors 58
3. Commercial Considerations 60
Key findings 60
Key drivers to commercial success 61
Endless lucrative targets for companies mastering delivery 61
Access to previously untapped markets 61
Key inhibitors to commercial success 62
Safety 62
Immune response 63
Mutagenesis 64
Germline transmission 64
Funding and Big Pharma involvement 64
Academic funding 64
Big Pharma involvement 65
Reliable delivery and regulatable expression 67
Challenging to treat multifactorial diseases 68
Access to vectors 68
Stigma associated with gene therapy 69
Market entry strategies 69
Pricing 71
Regulation 72
US 72
FDA - Center for Biologics Evaluation and Research (CBER) 72
NIH - Recombinant DNA Advisory Committee (RAC) 73
Europe 73
European Medicines Agency (EMEA) 73
Germany 74
UK 74
France 74
Bibliography 76
Journals 76
Websites 78
Datamonitor reports 80
Appendix A 82
Raw data 82
Appendix B 86
Contributing experts 86
Report methodology 86
About Datamonitor 87
About Datamonitor Healthcare 87
About the healthcare research & analysis team 88
Disclaimer 90
List of Tables
Table 1: Gendicine efficacy in head and neck squamous cell carcinoma 15
Table 2: Gendicine follow-up survival data in head and neck squamous cell carcinoma 15
Table 3: Phase III and pre-approval gene therapy projects, 2009 18
Table 4: Gene therapy methods for the treatment of cancer 27
Table 5: Gene therapies in development for pancreatic cancer, 2009 29
Table 6: Gene therapies in development for head and neck cancer, 2009 30
Table 7: Gene therapies in development for breast cancer, 2009 31
Table 8: Gene therapies in development for prostate cancer, 2009 32
Table 9: Gene therapies in development for Parkinson's disease, 2009 34
Table 10: Gene therapies in development for Leber congenital amaurosis (retinal disorder), 2009 35
Table 11: Gene therapies in development for peripheral arterial disease, 2009 38
Table 12: Vector system properties 46
Table 13: Advantages and disadvantages of the adenoviral vector in gene therapy 49
Table 14: Advantages and disadvantages of the lentiviral vector in gene therapy 51
Table 15: Advantages and disadvantages of the adeno-associated viral (AAV) vector in gene therapy 53
Table 16: Clinical programs in development using gene therapy, 2009 83
List of Figures
Figure 1: Timeline of gene therapy clinical development 10
Figure 2: Number of gene therapy clinical trials approved worldwide, 1989-2008 11
Figure 3: Geographic distribution of gene therapy clinical trials, 1989-March 2009 12
Figure 4: Number of clinical trials for gene therapy at each stage of development, 2009 17
Figure 5: Phase II results investigating high-dose Allovectin-7 in recurrent or unresponsive Stage III/IV melanoma 21
Figure 6: Number of gene therapy clinical projects underway for each therapy area, 2009 23
Figure 7: Number of genes in clinical trials for each therapy area, 2009 24
Figure 8: Number of gene therapy clinical projects underway by stage for key therapy areas, 2009 25
Figure 9: Indications with three or more clinical projects in development, 2009 26
Figure 10: Number and stage of development for cancer gene therapy programs, 2009 28
Figure 11: Number and stage of development for CNS gene therapy programs, 2009 33
Figure 12: Number and stage of development for cardiovascular and circulatory system gene therapy projects, 2009 37
Figure 13: Key companies with gene products in clinical development, 2009 40
Figure 14: In vivo versus ex vivo gene therapy 42
Figure 15: Vectors used in gene therapy clinical trials, 1989-March 2009 44
Figure 16: Vectors used in gene therapy clinical trials, 2009 45
Figure 17: A typical virus replication cycle 47
Figure 18: Virus types used as gene therapy vectors in clinical trials, 1989-March 2009 48
Figure 19: Virus types used as gene therapy vectors in clinical trials, 2009 49

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List of Tables
Table 1: Gendicine efficacy in head and neck squamous cell carcinoma 15
Table 2: Gendicine follow-up survival data in head and neck squamous cell carcinoma 15
Table 3: Phase III and pre-approval gene therapy projects, 2009 18
Table 4: Gene therapy methods for the treatment of cancer 27
Table 5: Gene therapies in development for pancreatic cancer, 2009 29
Table 6: Gene therapies in development for head and neck cancer, 2009 30
Table 7: Gene therapies in development for breast cancer, 2009 31
Table 8: Gene therapies in development for prostate cancer, 2009 32
Table 9: Gene therapies in development for Parkinson's disease, 2009 34
Table 10: Gene therapies in development for Leber congenital amaurosis (retinal disorder), 2009 35
Table 11: Gene therapies in development for peripheral arterial disease, 2009 38
Table 12: Vector system properties 46
Table 13: Advantages and disadvantages of the adenoviral vector in gene therapy 49
Table 14: Advantages and disadvantages of the lentiviral vector in gene therapy 51
Table 15: Advantages and disadvantages of the adeno-associated viral (AAV) vector in gene therapy 53
Table 16: Clinical programs in development using gene therapy, 2009 83

List of Figures
Figure 1: Timeline of gene therapy clinical development 10
Figure 2: Number of gene therapy clinical trials approved worldwide, 1989-2008 11
Figure 3: Geographic distribution of gene therapy clinical trials, 1989-March 2009 12
Figure 4: Number of clinical trials for gene therapy at each stage of development, 2009 17
Figure 5: Phase II results investigating high-dose Allovectin-7 in recurrent or unresponsive Stage III/IV melanoma 21
Figure 6: Number of gene therapy clinical projects underway for each therapy area, 2009 23
Figure 7: Number of genes in clinical trials for each therapy area, 2009 24
Figure 8: Number of gene therapy clinical projects underway by stage for key therapy areas, 2009 25
Figure 9: Indications with three or more clinical projects in development, 2009 26
Figure 10: Number and stage of development for cancer gene therapy programs, 2009 28
Figure 11: Number and stage of development for CNS gene therapy programs, 2009 33
Figure 12: Number and stage of development for cardiovascular and circulatory system gene therapy projects, 2009 37
Figure 13: Key companies with gene products in clinical development, 2009 40
Figure 14: In vivo versus ex vivo gene therapy 42
Figure 15: Vectors used in gene therapy clinical trials, 1989-March 2009 44
Figure 16: Vectors used in gene therapy clinical trials, 2009 45
Figure 17: A typical virus replication cycle 47
Figure 18: Virus types used as gene therapy vectors in clinical trials, 1989-March 2009 48
Figure 19: Virus types used as gene therapy vectors in clinical trials, 2009 49
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