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市場調査レポート

遺伝子治療市場 2015-2025年

Gene Therapy Market, 2015 - 2025

発行 ROOTS ANALYSIS 商品コード 324981
出版日 ページ情報 英文 279 Pages
納期: 即日から翌営業日
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遺伝子治療市場 2015-2025年 Gene Therapy Market, 2015 - 2025
出版日: 2015年02月25日 ページ情報: 英文 279 Pages
概要

当レポートでは、遺伝子治療市場について取り上げ、主要企業、市販の遺伝子治療製品、臨床/前臨床研究中の製品、遺伝子治療関連の倫理的問題、将来の発展見込みおよびステークホルダーにとっての機会などを分析しており、市場の短期的・中期的展望をまとめ、お届け致します。

第1章 序論

第2章 エグゼクティブサマリー

第3章 イントロダクション

  • 事情・背景
  • 遺伝子治療の歴史的な発展経緯
  • 遺伝子治療の分類
  • 投与経路
  • 遺伝子治療の作用
  • 遺伝子治療のメリット・デメリット
  • 遺伝子治療の倫理的・社会的懸念事項
  • 将来の抑制因子・課題

第4章 ウィルス・非ウィルスベクター

  • 本章の概要
  • 遺伝子導入のベクター法
  • 非ウィルスベクター

第5章 遺伝子治療のパイプライン

  • 本章の概要
  • 遺伝子治療:パイプライン分析
  • 腫瘍:最も一般的な治療領域
  • 遺伝子治療の区分:開発段階別
  • 遺伝子治療の区分:ベクター種類別
  • 遺伝子治療の区分:標的遺伝子の種類別
  • 遺伝子治療の区分:スポンサーの種類別

第6章 市販の遺伝子治療薬・用途

  • 本章の概要
  • Gendicine (SiBionoGeneTech)
  • Oncorine (Shanghai Sunway Biotech)
  • Rexin-G (Epeius Biotechnologies)
  • Neovasculgen (Human Stem Cell Institute)
  • Glybera (uniQure)

第7章 遺伝子治療:パイプライン製品

  • 本章の概要
  • Generx (Taxus Cardium)
  • TK (MolMedS.p.A)
  • Collategene (AnGes MG)
  • TissueGene-C (TissueGene Inc./Kolon Life Sciences)
  • SPK-RPE65 (Spark Therapeutics)
  • Prostvac (Bavarian Nordic)
  • T-VEC (Amgen)
  • ProstAtak (Advantagene)
  • TroVax (Oxford BioMedica)
  • Algenpantucel-L (Newlink Genetics Corporation)
  • ASP0113 (Vical/Astellas Pharma)
  • E10A (Marsala Biotech)
  • その他の後期段階の遺伝子治療
  • 全体的な遺伝子治療市場

第8章 有望な治療領域

  • 本章の概要
  • がん
  • 神経疾患
  • 眼疾患
  • 筋疾患
  • 血液疾患 (貧血・血友病)
  • 免疫不全症

第9章 遺伝子治療:追加の検討事項

  • 本章の概要
  • 遺伝子治療におけるベンチャー資金投資
  • 遺伝子治療に関する会議・エキシビション
  • 遺伝子治療における受託生産

第10章 結論

  • 単一遺伝子疾患から癌への移行
  • 遺伝子発現を最適化するための遺伝子治療の管理
  • mRNAを媒介した遺伝子治療:トランスフェクション効率を改善するための有望なアプローチ
  • 生殖系列遺伝子治療:まだ明らかにされていない可能性
  • 強力なパイプラインは数十億ドル市場になる可能性

第11章 インタビュー記録

第12章 付録1:データ表

第13章 付録2:企業リスト

図表

目次
Product Code: RA10033

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.

Synopsis:

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.

Example Highlights

  • 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.

Research Methodology

Most of the data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include

  • Annual reports
  • Investor presentations
  • SEC filings
  • Industry databases
  • News releases from company websites
  • Government policy documents
  • Industry analysts' views

While the focus has been on forecasting the market over the coming ten years, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

Chapter Outlines

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
  • Figure 4.1: 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)
  • Figure 7.1: 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
  • Figure 7.26: 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

List of 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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