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Gene Therapy Market, 2015 - 2025

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    February 2015

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  1. Gendicine became the first approved gene therapy in China in 2004. So far, there have been three more marketed gene therapies: Rexin-G, Oncorine and Neovasculgen. All of these have been made commercially available in Asian countries. Glybera was the first gene therapy to be approved in the EU in 2012.
  2. We have identified a total of 483 gene therapy molecules in the marketed and the clinical pipeline which are designed for different therapeutic areas. Except a few which are in advanced stages of development, majority of the gene therapies are in early development stages.
  3. There are several challenges and ethical issues associated with this field; germline gene therapy is one of the important issues. The existing gene therapies use somatic cells. However, research is in progress to introduce germline gene therapy in the market since some of the serious inherited genetic diseases, such as Lecsh-Nyhan Syndrome, can only be treated by germline gene therapy.
  4. There has been a growing interest from Venture Capital firms in the gene therapy space. In fact, in 2013 / 2014, several companies were involved in investment in gene therapies; during January 2013 to April 2014, a total USD 600 million was raised by the US companies alone. This activity is likely to increase further and will help companies, especially start-ups, drive their respective products to commercialisation.
  5. We expect the gene therapy market to be worth USD 11 billion by 2025, representing a healthy annual growth rate of 48.9%; specific therapies such as Prostavac, ProstAtak and TroVax are likely to achieve blockbuster status.

Overview

Several disorders that arise inside the body are a result of either a direct genetic aberration or a dysfunctional/non-functional protein. The attempt to use nucleic acids to correct or delete the genes causing a particular disease is known as gene therapy. Although gene therapy has not contributed significantly to the global pharmaceutical market yet, it is anticipated to grow at a fast pace over the next decade.

Gendicine, developed by SiBiono GeneTech, was the foremost gene therapy that entered market in 2004 in China. Since then four more therapies have received approval in China, Philippines, Russia and the EU. This number for approved / marketed therapies seems weak at present; however, the strong and highly populated pipeline holds tremendous potential. There are 12 gene therapies in late stage of clinical development for the treatment of cancer, ocular and cardiovascular disorders.

There are several concerns that remain to be answered; examples include insertional mutagenesis, treatment of multigene disorders, curbing the risk of immune reactions, eugenics, high cost of therapy and ethical concerns related to making alterations at the genetic level. Despite this, gene therapy does offer a ray of hope for patients who either have no treatment options or show no benefits with drugs that are currently available. Such a benefit far outweighs any disadvantages that may be associated with this upcoming therapeutic field.

There is a lot of research underway for the first generation of controlled gene therapy, gene expression cassettes which deliver optimised gene expression and vectors which minimise the risk of immunogenicity. These research efforts have received strong support from various venture capital firms and institutional investors. With the expected launch of several new gene therapies in the US and EU, we expect the market to witness significant growth in the coming decade.

 

Scope of the Report

The "Gene Therapy Market, 2015-2025" report provides an extensive study on the marketed and pipeline gene therapies. A lot of research has been carried out in this field for over a decade but there are only five approved therapies (four available in Asian markets; one approved in the EU). There are many promising therapies which are currently being developed worldwide; the approach is likely to result in several commercial success stories in the foreseen future. The report covers various aspects, such as key players, marketed gene therapy products, products in clinical / pre-clinical research, associated ethical issues, likely future developments and upcoming opportunities for a variety of stakeholders.

As pharma companies continue to expand their research programs in this area, one of the key objectives outlined for this report is to understand the future potential of the market. This is done by analysing:

  • The gene therapy pipeline in terms of phase of development, therapeutic area, target indications, types of vectors, gene types, etc.
  • Interest of venture capital firms and recent funding rounds that have taken place in the gene therapy space.
  • Development and sales potential based on target consumer segments, likely adoption rate and expected pricing.
  • The constraints of gene therapy and future developments planned to overcome these.

The study provides an estimate of the short-midterm and long term market forecast for the period 2015 - 2025. The research, analysis and insights presented in this report include potential sales of the five approved gene therapies and 12 gene therapy products in late phases of development.

Owing to niche nature of the market, with most products in the pipeline, we have provided three market forecast scenarios to add robustness to our model. The conservative, base and optimistic scenarios represent three different tracks of industry evolution. All actual figures have been sourced and analysed from publicly available information and discussions with industry experts. The figures mentioned in this report are in USD, unless otherwise specified.

Contents

Chapter 2  presents an executive summary of the report. The summary offers key highlights of the gene therapy market and a high level view on where the market is headed in the mid-long term.

Chapter 3  provides a detailed introduction to the market. In this section, we have talked about theclassification and routes of administration of gene therapy. It also includes the advantages, challenges and ethical issues associated with gene therapy.

Chapter 4  provides a comprehensive view of the gene delivery systems. It includes details about the different viral and non-viral vectors associated with the process of gene therapy.

Chapter 5  includes information on over 483 molecules which are currently approved orin different stages of development (both clinical and pre-clinical / discovery). The detailed analysis of this pipeline covers information on drug developers, phases of development, therapeutic areas, target indications, vectors used, gene types and the genes involved.

Chapter 6  provides details on the marketed therapies.The chapter includes detailed profiles of marketed gene therapy products. We have presented information on several parameters such as history of development, clinical trial results, manufacturing, costs, collaborations and the likely sales forecast over the next ten years.

Chapter 7  includes detailed profiles of gene therapy products in late stage clinical trials. We have presented information on several parameters such as history of development, technology used, clinical trial results, manufacturing, costs, collaborations and the likely sales forecast over the next ten years.

Chapter 8  highlights the promising new therapeutic areas for gene therapies. It elaborates on the number of products being developed under each therapeutic area.

Chapter 9  includes details about the venture capital investments that have taken place in this field. It also highlights the contract manufacturers participating in development of gene therapy.

Chapter 10  summarises the overall report. In this chapter, we provide a recap of the current gaps in the industry and highlight the future scope of gene therapy.

Chapter 11  is a collection of interview transcripts of the discussions which were held during the course of this study.

Chapters 12 and 13  are appendices, which provide the list of companies and tabulated data for all the figures presented in the report.

Table of Contents

1. PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Chapter Outlines
 
2. EXECUTIVE SUMMARY
 
3. INTRODUCTION
3.1. Context and Background
3.2. Historical Evolution of Gene Therapy
3.3. Classification of Gene Therapy
3.3.1. Somatic vs. Germline
3.3.2. Ex-vivo vs. In-vivo
3.4. Route of Administration
3.5. How Does Gene Therapy Work?
3.6. Advantages and Disadvantages of Gene Therapy
3.7. Ethical and Social Concerns in Gene Therapy
3.7.1. Somatic Gene Therapy
3.7.2. Germ-line Gene Therapy
3.8. Future Constraints and Challenges
3.8.1 Manufacturing
3.8.2 Reimbursement
3.8.3 Commercial Viability
 
4. VIRAL AND NON-VIRAL VECTORS
4.1. Chapter Overview
4.2. Viral Methods of Gene Transfer
4.2.1. Retroviruses
4.2.2. Lentiviruses
4.2.3. Adenoviruses
4.2.4. Adeno Associated Virus
4.2.5. Herpes Simplex Virus
4.2.6. Alphavirus
4.2.7. Vaccinia Virus
4.2.8. Simian Virus
4.3. Non-Viral Vectors
4.3.1. Naked/Plasmid Vectors
4.3.2. Biolistic Method: Gene Gun
4.3.3. Electroporation
4.3.4. Receptor Mediated Gene Delivery Methods
4.3.5. Liposomes, Lipoplexes and Polyplexes
4.3.6. Gene Activated Matrix (GAM)
 
5. PIPELINE OF GENE THERAPY
5.1. Chapter Overview
5.2. Gene Therapy: Pipeline Analysis
5.3 Oncology: The Most Popular Therapeutic Area
5.4. Distribution of Gene Therapies by Phase of Development
5.5. Distribution of Gene Therapies by Type of Vector
5.6. Distribution of Gene Therapies by Type of Genes Targeted
5.7. Distribution of Gene Therapies by Type of Sponsor
 
6. MARKETED GENE THERAPIES AND APPLICATIONS
6.1. Chapter Overview
6.2. Gendicine (SiBionoGeneTech)
6.2.1. Company and Pipeline Overview
6.2.2. History of Approval
6.2.3. Mechanism of Action and Vectors Used
6.2.4. Target Indication
6.2.5. Development Status
6.2.6. Dosage, Sales and Manufacturing
6.2.7. Patent Portfolio
6.2.8. Gendicine Sales Forecast, 2015 – 2025
6.3. Oncorine (Shanghai Sunway Biotech)
6.3.1. Company and Pipeline Overview
6.3.2. History of Approval
6.3.3. Mechanism of Action and Vectors Used
6.3.4. Target Indication
6.3.5. Development Status
6.3.6. Dosage and Sales
6.3.7. Patent Portfolio
6.3.8. Oncorine Sales Forecast, 2015 – 2025
6.4. Rexin-G (Epeius Biotechnologies)
6.4.1. Company and Pipeline Overview
6.4.2. History of Approval
6.4.3. Mechanism of Action and Vector Used
6.4.4. Target Indication
6.4.5. Development Status
6.4.6. Dosage and Manufacturing
6.4.7. Patent Portfolio
6.4.8. Rexin-G Sales Forecast, 2015 – 2025
6.5. Neovasculgen (Human Stem Cell Institute)
6.5.1. Company and Pipeline Overview
6.5.2. History of Approval
6.5.3. Mechanism of Action and Vector Used
6.5.4. Target Indication
6.5.5. Development Status
6.5.6. Dosage, Sales and Manufacturing
6.5.7. Neovasculgen Sales Forecast, 2015 – 2025
6.6. Glybera (uniQure)
6.6.1. Company and Pipeline Overview
6.6.2. History of Approval
6.6.3. Target Indication
6.6.4. Technology
6.6.5. Development Status
6.6.6. Dosage and Manufacturing
6.6.7. Collaborations
6.6.8. Glybera Sales Forecast, 2015 – 2025
 
7. GENE THERAPY: PIPELINE PRODUCTS
7.1. Chapter Overview
7.2. Generx (Taxus Cardium)
7.2.1. Company and Pipeline Overview
7.2.2. History of Development
7.2.3. Target Indication
7.2.4. Technology
7.2.5. Development Status
7.2.6. Dosage and Manufacturing
7.2.7. Collaborations
7.2.8. Generx Sales Forecast, 2015 – 2025
7.3. TK (MolMedS.p.A)
7.3.1. Company and Pipeline Overview
7.3.2. History of Development
7.3.3. Target Indication
7.3.4. Technology
7.3.5. Development Status
7.3.6. Dosage and Manufacturing
7.3.7. Collaborations
7.3.8. TK Sales Forecast, 2015 – 2025
7.4. Collategene (AnGes MG)
7.4.1. Company and Pipeline Overview
7.4.2. History of Development
7.4.3. Target Indication
7.4.4. Technology
7.4.5. Development Status
7.4.6. Dosage and Manufacturing
7.4.7. Collaborations
7.4.8. Collategene Sales Forecast, 2015 – 2025
7.5. TissueGene-C (TissueGene Inc./Kolon Life Sciences)
7.5.1. Company and Pipeline Overview
7.5.2. History of Development
7.5.3. Target Indication
7.5.4. Technology
7.5.5. Development Status
7.5.6. Dosage and Manufacturing
7.5.7. Collaborations
7.5.8. TissueGene-C Sales Forecast, 2015 – 2025
7.6. SPK-RPE65 (Spark Therapeutics)
7.6.1. Company and Pipeline Overview
7.6.2. History of Development
7.6.3. Target Indication
7.6.4. Technology
7.6.5. Development Status
7.6.6. Dosage and Manufacturing
7.6.7. Collaborations
7.6.8. SPK-RPE65 Sales Forecast, 2015 – 2025
7.7. Prostvac (Bavarian Nordic)
7.7.1. Company and Pipeline Overview
7.7.2. History of Development
7.7.3. Target Indication
7.7.4. Technology
7.7.5. Development Status
7.7.6. Dosage and Manufacturing
7.7.7. Collaborations
7.7.8. Prostvac Sales Forecast, 2015 – 2025
7.8. T-VEC (Amgen)
7.8.1. Company and Pipeline Overview
7.8.2. History of Development
7.8.3. Target Indication
7.8.4. Technology
7.8.5. Development Status
7.8.6. Dosage and Manufacturing
7.8.7. Collaborations
7.8.8. T-Vec Sales Forecast, 2015 – 2025
7.9. ProstAtak (Advantagene)
7.9.1. Company and Pipeline Overview
7.9.2. History of Development
7.9.3. Target Indication
7.9.4. Technology
7.9.5. Development Status
7.9.6. Dosage and Manufacturing
7.9.7. Collaborations
7.9.8. ProstAtak Sales Forecast, 2015 – 2025
7.10. TroVax (Oxford BioMedica)
7.10.1. Company and Pipeline Overview
7.10.2. History of Development
7.10.3. Target Indication
7.10.4. Technology
7.10.5. Development Status
7.10.6. Dosage and Manufacturing
7.10.7. Collaborations
7.10.8. TroVax Sales Forecast, 2015 – 2025
7.11. Algenpantucel-L (Newlink Genetics Corporation)
7.11.1. Company and Pipeline Overview
7.11.2. History of Development
7.11.3. Target Indication
7.11.4. Technology
7.11.5. Development Status
7.11.6. Dosage and Manufacturing
7.11.7. Collaborations
7.11.8. Algenpantucel-L Sales Forecast, 2015 – 2025
7.12. ASP0113 (Vical/Astellas Pharma)
7.12.1. Company and Pipeline Overview
7.12.2. History of Development
7.12.3. Target Indication
7.12.4. Technology
7.12.5. Development Status
7.12.6. Dosage and Manufacturing
7.12.7. Collaborations
7.12.8. ASP0113 Sales Forecast, 2015 – 2025
7.13. E10A (Marsala Biotech)
7.13.1. Company and Pipeline Overview
7.13.2. History of Development
7.13.3. Target Indication
7.13.4. Technology
7.13.5. Development Status
7.13.6. Dosage and Manufacturing
7.13.7. Collaborations
7.13.8. E10A Sales Forecast, 2015 - 2025
7.14. Other Late Phase Gene Therapies
7.15. Overall Gene Therapy Market, 2015 – 2025
 
8. PROMISING THERAPEUTICS AREAS
8.1. Chapter Overview
8.2. Cancer
8.3. Neurological Disorders
8.3.1. Neurodegenerative Disorders
8.3.2. Lysosomal Storage Disorders (LSDs)
8.4. Ocular Diseases
8.5. Muscle Disorders
8.6. Blood Disorders (Anemia and Hemophilia)
8.7. Immunodeficiency Diseases
 
9. GENE THERAPY: ADDITIONAL CONSIDERATIONS
9.1. Chapter Overview
9.2 Venture Capital Investment in Gene Therapy
9.3. Conferences and Exhibitions on Gene Therapy
9.4. Contract Manufacturing in Gene Therapy
 
10. CONCLUSION
10.1. Move From Monogenic Diseases To Cancer
10.2. Controlled Gene Therapy for Optimised Gene Expression: Gradually Evolving
10.3. mRNA Mediated Gene Therapy: A Promising Approach to Improve Transfection Efficiency
10.4. Germline Gene Therapy: Potential yet to Unveil
10.5. A Strong Pipeline Likely To Result In A Multi-Billion Dollar Market
 
11. INTERVIEW TRANSCRIPTS
 
12. APPENDIX1: TABULATED DATA
 
13. APPENDIX 2: LIST OF COMPANIES AND ORGANISATIONS

List of Figures

Figure 3.1  History of Evolution: Timeline
Figure 3.2  Gene Transfer using Viral Vectors
Figure4.1  Gene Transfer: Viral and Non-Viral Methods
Figure 5.1  Pipeline Analysis: Distribution by Therapeutic Area
Figure 5.2  Pipeline Analysis: Distribution by Phase of Development
Figure 5.3 Pipeline Analysis: Distribution by Type of Vector
Figure 5.4  Pipeline Analysis: Distribution by Target Gene Type
Figure 5.5  Pipeline Analysis: Distribution by Drug Developer Type
Figure 6.1 Pipeline Overview: SiBionoGeneTech
Figure 6.2 Gendicine: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 6.3 Pipeline Overview: Shanghai Sunway Biotech
Figure 6.4 Adenovirus Construct in Oncorine
Figure 6.5 Oncorine: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 6.6 Pipeline Overview: Epeius Biotechnologies
Figure 6.7 Rexin-G: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 6.8 Pipeline Overview: Human Stem Cell Institute
Figure 6.9 Human Stem Cell Institute: Revenues (RUB’000)
Figure 6.10 Neovasculgen: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 6.11  Pipeline Overview: uniQure
Figure 6.12 Glybera: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure7.1 Pipeline Overview: TaxusCardium
Figure 7.2 Generx: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.3 Pipeline Overview: MolMedS.p.A.
Figure 7.4 TK: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.5 Pipeline Overview: AnGes
Figure 7.6 Collategene: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.7 Pipeline Overview: Kolon Life Science
Figure 7.8 TissueGene-C: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.9 Pipeline Overview: Spark Therapeutics
Figure 7.10 SPK-RPE65: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.11 Pipeline Overview: Bavarian Nordic
Figure 7.12 Incidence and Mortality 2012: Prostate Cancer (in ‘000)
Figure 7.13 Prostvac: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.14 Pipeline Overview: Amgen
Figure 7.15 Incidence and Mortality 2014: Melanoma (in ‘000)
Figure 7.16 Skin Cancer: Geographical Distribution of Death Rate (Cases per 100,000 People)
Figure 7.17 T-Vec: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.18 Pipeline Overview: Advantagene
Figure 7.19 Incidence and Mortality 2012: Prostate Cancer (in ‘000)
Figure 7.20 ProstAtak: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.21 Pipeline Overview: Oxford BioMedica
Figure 7.22 Incidence and Mortality 2012: Colorectal Cancer (in ‘000)
Figure 7.23 TroVax: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.24 Pipeline Overview: NewLink Genetics
Figure 7.25 Age-Standardised Rate 2012: Pancreatic Cancer
Figure7.26 Incidence and Mortality 2012: Pancreatic Cancer (in ‘000)
Figure 7.27 Algenpantucel-L: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.28 Pipeline Overview: Vical
Figure 7.29 ASP0113: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.30 Pipeline Overview: Marsala Biotech
Figure 7.31 E10A: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Figure 7.32 Overall Gene Therapy Market Outlook 2015-2025 (USD Million)
Figure 7.33 Contribution of Oncology to Gene Therapy Market
Figure 8.1 Number of Gene Therapies in Pre-clinical/Clinical Development for Cancer: By Disease Area
Figure 8.2 Number of Gene Therapies in Pre-clinical/Clinical Development for Cancer: By Transfer Vectors
Figure 8.3 Number of Gene Therapies in Pre-clinical/Clinical Development for Neurological Disorders: By Disease Area
Figure 8.4 Number of Gene Therapies in Pre-clinical/Clinical Development for Neurological Disorders: By Transfer Vectors
Figure 8.5 Number of Gene Therapies in Pre-clinical/Clinical Development for Lysosomal Storage Disorders: By Disease Area
Figure 8.6 Number of Gene Therapies in Pre-clinical/Clinical Development for Lysosomal Storage Disorders: By Transfer Vectors
Figure 8.7  Number of Gene Therapies in Pre-clinical/Clinical Development for Ocular Disorders: By Disease Area
Figure 8.8  Number of Gene Therapies in Pre-clinical/Clinical Development for Ocular Disorders: By Transfer Vectors
Figure 8.9  Number of Gene Therapies in Pre-clinical/Clinical Development for Muscle Disorders: By Disease Area
Figure 8.10  Number of Gene Therapies in Pre-clinical/Clinical Development for Muscle Disorders: By Transfer Vectors
Figure 8.11  Number of Gene Therapies in Pre-clinical/Clinical Development for Blood Disorders: By Disease Area
Figure 8.12 Number of Gene Therapies in Pre-clinical/Clinical Development for Blood Disorders: By Transfer Vectors
Figure 8.13  Number of Gene Therapies in Pre-clinical/Clinical Development for Immunodeficiency Diseases: By Disease Area
Figure 8.14  Number of Gene Therapies in Pre-clinical/Clinical Development for Immunodeficiency Diseases: By Transfer Vectors
Figure 9.1  Gene Therapy: Type of Investments in 2013 and 2014
Figure 9.2  Gene Therapy: Investments Made for Different Body Systems (USD Million)
Figure 9.3  Gene Therapy Conferences in 2015: Distribution by Month
Figure 9.4  Gene Transfer: Top Conference Sponsors
Figure 9.5  Contract Manufacturing in Gene Therapy: By Capability
Figure 9.6  Contract Manufacturing in Gene Therapy: By Location
Figure 10.1 Gene Therapy Market (USD Million), 2017, 2021 and 2025

List of Tables

Table 3.1 Differences between Ex vivo and In vivo Gene Therapy
Table 3.2 Price comparison of Marketed Gene Therapies
Table 3.3 Approved ATMPs in EU
Table 4.1 Features of Retrovirus
Table 4.2 Features of Lentivirus
Table 4.3 Features of Adenovirus
Table 4.4 Features of Adeno-associated Virus Vectors
Table 4.5 Features of Herpes Simplex Virus Vectors
Table 5.1 Pipeline: Approved/Marketed Gene Therapies
Table 5.2  Pipeline: Pre-registration/Phase III Gene Therapies
Table 5.3  Pipeline: Phase II/III Gene Therapies
Table 5.4  Pipeline: Phase II Gene Therapies
Table 5.5  Pipeline: Phase I/II Gene Therapies
Table 5.6  Pipeline: Phase I Gene Therapies
Table 5.7  Pipeline: Preclinical Stage
Table 5.8  Gene Therapy: University Spin-offs
Table 6.1  Marketed and Approved Gene Therapies
Table 6.2  Company Overview: SiBionoGeneTech
Table 6.3  Gendicine: Status of Development
Table 6.4  Gendicine: Patent Portfolio
Table 6.5  Company Overview: Shanghai Sunway Biotech
Table 6.6  H100 Series: Status of Development
Table 6.7  Company Overview: Epeius Biotechnologies
Table 6.8 Rexin-G: Status of Development
Table 6.9 Rexin G: Patent Portfolio
Table 6.10 Company Overview: Human Stem Cell Institute
Table 6.11  Neovasculgen: Status of Development
Table 6.12 Company Overview: uniQure
Table 6.13  Glybera: Status of Development
Table 7.1  Gene Therapy: Late Stage Development Products
Table 7.2 Company Overview: Taxus Cardium
Table 7.3 Generx: Status of Development
Table 7.4  Company Overview: MolMedS.p.A.
Table 7.5 TK: Status of Development
Table 7.6 Company Overview: AnGes MG
Table 7.7 Collategene: Status of Development
Table 7.8 Company Overview: Kolon Life Science
Table 7.9 TissueGene-C: Status of Development
Table 7.10 Company Overview: Spark Therapeutics
Table 7.11  SPK-RPE65: Status of Development
Table 7.12 Company Overview: Bavarian Nordic
Table 7.13 Prostvac: Status of Development
Table 7.14 Company Overview: Amgen
Table 7.15 T-Vec: Status of Development
Table 7.16 Company Overview: Advantagene
Table 7.17 ProstAtak: Status of Development
Table 7.18 Company Overview: Oxford BioMedica
Table 7.19 TroVax: Status of Development
Table 7.20 Company Overview: NewLink Genetics
Table 7.21 Algenpantucel-L: Status of Development
Table 7.22 Company Overview: Vical
Table 7.23 ASP0113: Status of Development
Table 7.24 Company Overview: Marsala Biotech
Table 7.25 E10A: Status of Development
Table 7.26 Gene Therapies in Phase II/III
Table 7.27 Important Highlights of Gene Therapies in Phase II/III
Table 7.28  Gene Therapy: Expected Years of Launch
Table 8.1  Gene Therapy for Cancer
Table 8.2  Gene Therapy for Neurological Disorders
Table 8.3 Classification of Lysosomal Storage Disorders
Table 8.4  Gene Therapy for Lysosomal Storage Disorders
Table 8.5  Gene Therapy for Ocular Disorders
Table 8.6  Gene Therapy for Muscle Disorders
Table 8.7  Gene Therapy for Blood Disorders
Table 8.8  Gene Therapy for Immunodeficiency Diseases
Table 9.1  Recent Investments in Gene Therapy
Table 9.2  Gene Transfer: Conferences 2015
Table 9.3 Contract Manufactures in Gene Therapy
Table 12.1  Pipeline Analysis: Distribution by Therapeutic Area
Table 12.2  Pipeline Analysis: Distribution by Phase of Development
Table 12.3  Pipeline Analysis: Distribution by Type Gene Delivery Methods
Table 12.4 Pipeline Analysis: Distribution by the Gene Type
Table 12.5  Pipeline Analysis: Distribution by Drug Developer Type
Table 12.6 Gendicine: Sales Forecast 2015 - 2025, Base Scenario (USD Million
Table 12.7  Gendicine: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.8  Gendicine: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.9  Oncorine: Sales Forecast 2015 - 2025, Base Scenario (USD Million
Table 12.10 Oncorine: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.11  Oncorine: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.12  Rexin-G: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.13  Rexin-G: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.14  Rexin-G: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.15  Human Stem Cell Institute: Revenues (RUB ’000)
Table 12.16  Neovasculgen: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.17  Neovasculgen: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.18  Neovasculgen: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.19  Glybera: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.20  Glybera: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.21  Glybera: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.22  Generx: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.23  Generx: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.24  Generx: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.25  TK: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.26  TK: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.27  TK: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.28  Collategene: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.29  Collategene: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.30  Collategene: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.31  TissueGene-C: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.32  TissueGene-C: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.33  TissueGene-C: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.34  SPK-RPE65: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.35  SPK-RPE65: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.36  SPK-RPE65: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.37  Incidence and Mortality Rate 2012: Prostate Cancer
Table 12.38  Prostvac: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.39  Prostvac: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.40  Prostvac: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.41  Incidence and Mortality Rate 2014: Melanoma
Table 12.42  Skin Cancer: Geographical Distribution of Death Rate (Cases per 100,000 People)
Table 12.43  T-Vec: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.44   T-Vec: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.45  T-Vec: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.46  Incidence and Mortality Rate 2012: Prostate Cancer
Table 12.47  ProstAtak: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.48  ProstAtak: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.49  ProstAtak: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.50  Incidence and Mortality Rate 2012: Colorectal Cancer
Table 12.51 TroVax: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.52  TroVax: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.53  TroVax: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.54  Age-Standardised Rate 2012: Pancreatic Cancer
Table 12.55  Incidence and Mortality Rate 2012: Pancreatic Cancer
Table 12.56  Algenpantucel-L: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.57  Algenpantucel-L: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.58  Algenpantucel-L: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.59  ASP0113: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.60  ASP0113: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.61  ASP0113: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.62  E10A: Sales Forecast 2015 - 2025, Base Scenario (USD Million)
Table 12.63  E10A: Sales Forecast 2015 - 2025, Conservative Scenario (USD Million)
Table 12.64  E10A: Sales Forecast 2015 - 2025, Optimistic Scenario (USD Million)
Table 12.65  Overall Gene Therapy Forecast 2015-2025: Base Scenario (USD Million)
Table 12.66  Overall Gene Therapy Forecast 2015-2025: Conservative Scenario (USD Million)
Table 12.67  Overall Gene Therapy Forecast 2015-2025: Optimistic Scenario (USD Million)
Table 12.68  Contribution of Oncology in Gene therapy market (USD Million)
Table 12.69  Number of Gene Therapies in Pre-clinical/Clinical Development for Cancer: By Disease Area
Table 12.70  Number of Gene Therapies in Pre-clinical/Clinical Development for Cancer: By Transfer Vectors
Table 12.71  Number of Gene Therapies in Pre-clinical/Clinical Development for Neurological Disorders: By Disease Area
Table 12.72  Number of Gene Therapies in Pre-clinical/Clinical Development for Neurological Disorders: By Transfer Vectors
Table 12.73  Number of Gene Therapies in Pre-clinical/Clinical Development for Lysosomal Storage Disorders: By Disease Area
Table 12.74  Number of Gene Therapies in Pre-clinical/Clinical Development for Lysosomal Storage Disorders: By Transfer Vectors
Table 12.75  Number of Gene Therapies in Pre-clinical/Clinical Development for Ocular Disorders: By Disease Area
Table 12.76  Number of Gene Therapies in Pre-clinical/Clinical Development for Ocular Disorders: By Transfer Vectors
Table 12.77 Number of Gene Therapies in Pre-clinical/Clinical Development for Muscle Disorders: By Disease Area
Table 12.78  Number of Gene Therapies in Pre-clinical/Clinical Development for Muscle Disorders: By Transfer Vectors
Table 12.79  Number of Gene Therapies in Pre-clinical/Clinical Development for Blood Disorders: By Disease Area
Table 12.80  Number of Gene Therapies in Pre-clinical/Clinical Development for Blood Disorders: By Transfer Vectors
Table 12.81  Number of Gene Therapies in Pre-clinical/Clinical Development for Immunodeficiency Diseases: By Disease Area
Table 12.83  Number of Gene Therapies in Pre-clinical/Clinical Development for Immunodeficiency Diseases: By Transfer Vectors
Table 12.83  Gene Therapy: Type of Investments in 2013 and 2014
Table 12.84  Gene Therapy: Investments made for different Body Systems
Table 12.85  Gene Therapy Conferences in 2015: Distribution by Month
Table 12.86  Contract Manufacturing in Gene Therapy: By Capability
Table 12.87  Contract Manufacturing in Gene Therapy: By Location
Table 12.88  Gene Therapy Market (USD Million), 2017, 2021 and 2025

Listed Companies

The following companies have been mentioned in this report.

  1. AAV Life
  2. Abeona Therapeutics
  3. ActoGenix
  4. Adaptimmune
  5. Aduro Biotech
  6. Advantagene
  7. Advaxis
  8. Aeras
  9. Alphavax
  10. Amgen
  11. AmphliPhi Biosciences
  12. Amsterdam Molecular Therapeutics
  13. Anaeropharma Science
  14. AnGes MG
  15. Applied Genetic Technologies Corp
  16. Asklepios BioPharmaceutical Inc.
  17. Astellas Pharma
  18. Audentes Therapeutics
  19. Avalanche Biotechnologies
  20. AveXis Inc
  21. Bavarian Nordic
  22. Baxter Healthcare Corporation
  23. Bellicum Pharmaceuticals
  24. Benitec BioPharma
  25. BioCancell
  26. Bioheart
  27. Biomics Biotechnologies
  28. Biontech
  29. Biovex
  30. Bluebird Bio
  31. BN ImmunoTherapeutics
  32. Calimmune
  33. Cardium Therapeutics
  34. Cell Therapy Catapult
  35. Celladon Corporation
  36. Ceregene
  37. ChiesiFarmaceutici
  38. ClinDatrix
  39. Cold Genesys, Inc.
  40. Crucell
  41. CytRx Corporation
  42. Daiichi Sankyo
  43. Diamyd Inc.
  44. Digna Biotech S.L.
  45. DNAtrix
  46. DNAVEC Corporation
  47. EGEN, Inc.
  48. Emergent Biosolutions
  49. Enzo Biochem
  50. Epeius Biotechnologies
  51. Errant Gene Therapeutics
  52. ESTEVE
  53. Etubics Corporation
  54. FKD Therapies Oy
  55. Fondazione Telethon
  56. Genable Technologies
  57. Gene Medicine Japan
  58. GeneCure Biotechnologies
  59. Genelux Corporation
  60. GeneOne Life Sciences
  61. GeneSight Biologics
  62. Genethon
  63. Genexine
  64. Genprex
  65. GenVec
  66. Genzyme 
  67. GeoVax Inc.
  68. GlaxoSmithKline
  69. GlobeImmune
  70. Guangzhou DoublleBioproducts
  71. Gradalis Inc.
  72. Heat Biologics
  73. Helica Labs
  74. Herantis Pharma
  75. Ichor Medical Systems
  76. Imaxio
  77. Immune Design
  78. Inovio Pharmaceuticals
  79. Invivodata
  80. INVIVOGEN
  81. Ion Channel Innovations
  82. IrsiCaixa
  83. Isis Innovation
  84. Jennerex Biotherapeutics
  85. Juventas Therapeutics, Inc.
  86. Kolon Life Science
  87. LYSOGENE
  88. Marsala Biotech
  89. Medison Pharma
  90. Medigen Biotechnology Corporation
  91. Memgen Bio
  92. Merck
  93. Milo Biotechnology
  94. MiroVAX
  95. Mitsubishi Tanabe
  96. MolMed S.p.A
  97. Mologen AG
  98. Momotaro-Gene Inc
  99. MultiGene Vascular Systems 
  100. NanoCor Therapeutics
  101. NEURALGENE
  102. Neurotech Pharmaceuticals
  103. Newlink Genetics Corporation
  104. Nightstar
  105. Novartis 
  106. Oncolys BioPharma
  107. OncoSec Medical Incorporated
  108. Oncos Therapeutics
  109. Orca Therapeutics
  110. Oxford BioMedica
  111. Quintiles
  112. Pacific-Link Consulting
  113. Paragon Biomedical
  114. Pfizer
  115. PNP Therapeutics
  116. Profectus Biosciences
  117. Regen BioPharma
  118. Regeneron
  119. RegenX Biosciences
  120. ReiThera Sri
  121. REPLIcor
  122. Roche
  123. SanBio
  124. Sangamo Biosciences
  125. Sanofi
  126. Scancell Ltd
  127. Schering AG
  128. Shanghai Sunway Biotech
  129. SiBiono GeneTech
  130. SillaJen
  131. SironRX Therapeutics
  132. Spark Therapeutics
  133. SynerGene Therapeutics
  134. Tacere Therapeutics
  135. Takara Bio
  136. Theradex
  137. Theravectys S.A.
  138. Tocagen
  139. Transgene
  140. Treeway
  141. uniQure
  142. UCSF
  143. Vascular Biogenics
  144. Vaximm GmbH
  145. VCN Biosciences
  146. Vical
  147. ViroMed
  148. VIRxSYS Corporation
  149. Voyager Therapeutics
  150. VGXI
  151. Virttu Biologics
  152. Ziopharm

 

The following organisations and universities have been mentioned in this report.

  1. Alpha-1 Foundation
  2. Assistance Publique - Hôpitaux de Paris; Paris 12 Val de Marne University; Université Paris VI
  3. ALS Therapy Development Institute
  4. Baylor College of Medicine 
  5. Centro de InvestigaciónMédicaAplicada (CIMA)
  6. Children's Hospital Boston
  7. Children's Hospital of Philadelphia (CHOP)
  8. Choroideremia Research Foundation
  9. Christie Hospital NHS Foundation Trust Erasmus Medical Center
  10. City of Hope Medical Center
  11. Duke University Medical Center
  12. Erasmus Medical Center
  13. European and Developing Countries Clinical Trials Partnership (EDCTP)
  14. European Commission
  15. European Organisation for Research and Treatment of Cancer (EORTC)
  16. Fred Hutchinson Cancer Research Center; National Heart, Lung and Blood Institute (NHLBI)
  17. Goethe University
  18. Great Ormond Street Hospital for Children NHS Foundation Trust
  19. H. Lee Moffitt Cancer Center and Research Institute
  20. Hadassah Medical Organization
  21. Health Innovation Challenge Fund;
  22. Henry Ford Health System
  23. HIV Vaccine Trials Network (HVTN)
  24. Hospital InfantilUniversitario Niño Jesús
  25. Huazhong University of Science and Technology
  26. Human Stem Cells Institute
  27. Ichor Medical Systems
  28. Institut National de la Santé Et de la RechercheMédicale, France
  29. International AIDS Vaccine Initiative
  30. Jiangsu Province Hospital
  31. Jonsson Comprehensive Cancer Center 
  32. King Khaled Eye Specialist Hospital
  33. Kuopio University Hospital
  34. Kyushu University Hospital
  35. Leukemia Lymphoma Research
  36. Loyola University
  37. M.D. Anderson Cancer Center
  38. Malaria Vectors Vaccines Consortium (MVVC)
  39. Massachusetts General Hospital
  40. Mayo Clinic
  41. Memorial Sloan-Kettering Cancer Center
  42. Mie University
  43. Milton S. Hershey Medical Center
  44. National Center for Research Resources 
  45. National Eye Institute
  46. National Institute for Longevity Sciences of Japan
  47. National Institute of Allergy and Infectious Diseases
  48. National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
  49. National Institute of Dental and Craniofacial Research (NIDCR)
  50. National Institute of Health (NIH) 
  51. National Institute of Infectious Diseases of Japan
  52. National Institutes of Health Clinical Center (CC)
  53. National Taiwan University Hospital
  54. Nationwide Children's Hospital
  55. Naval Medical Research Center (NMRC)
  56. Cancer Research UK
  57. Memorial Sloan-Kettering Cancer Center
  58. Weill Medical College of Cornell University
  59. Oregon Health and Science University
  60. Phase One Foundation
  61. Public Health Agency of Canada
  62. ReiTheraSrl
  63. Research Center for Hematology of the Russian Ministry of Healthcare
  64. Rockefeller University
  65. Royal Brompton &Harefield NHS Foundation Trust
  66. Sidney Kimmel Comprehensive Cancer Center
  67. Shanxi Taxus Pharmaceuticals
  68. St. Jude Children's Research Hospital
  69. Stanford University
  70. Stichting Het NederlandsKankerInstituut
  71. Sun Yat-Sen University Cancer Center
  72. Swedish Institute for Infectious Disease Control
  73. Texas Children's Hospital
  74. The Ellis Lab; University of Texas Southwestern
  75. The Methodist Hospital System
  76. The University of Kansas Medical Center
  77. Tianjin Medical University Cancer Institute and Hospital
  78. Tumor Hospital of Medical Sciences Academy of China
  79. United Kingdom Cystic Fibrosis Gene Therapy Consortium
  80. Universidad de Navarra
  81. University College London 
  82. University Hospital Birmingham
  83. University of Alabama at Birmingham
  84. University of Birmingham
  85. University of California, Berkeley
  86. University of Cambridge
  87. University of Connecticut
  88. University of Florida
  89. University of Miami
  90. University of Michigan
  91. University of Nairobi
  92. University of Nebraska Medical Center 
  93. University of New South Wales
  94. University of Oxford
  95. University of Pennsylvania
  96. University of Pittsburgh
  97. University of Southern California
  98. University of Tennessee
  99. University of Texas
  100. University of Tokyo Hospital
  101. University of Washington
  102. University of Wisconsin
  103. University of Zurich
  104. Uppsala University Hospital
  105. Wayne State University 
  106. West China Hospital
  107. WuxiAppTec

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