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Gene Therapy Market (2nd Edition), 2018-2030

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  1. Around 300 product candidates are currently under various stages of development for a diverse range of indications. Eight gene therapies are commercially available; of these, Imlygic® (Amgen), Strimvelis® (GSK), Invossa™ (TissueGene / Kolon Life Science / Mitsubishi Tanabe Pharma) and Luxturna™ (Spark Therapeutics) were approved after 2015. Nearly 48% of the pipeline molecules are under clinical development; of these, 21 molecules are being investigated in phase II/III and above, 8 molecules in phase III (planned), 21 molecules in phase II, 48 molecules in phase I/II and 49 molecules in phase I clinical trials. However, majority (49%) of the product candidates in the pipeline are still in the preclinical and discovery stages.
  2. A significant proportion (59%) of product candidates in the pipeline follow the gene augmentation modification strategy, by delivering functional copies of missing genes into diseased hosts. Other types of modifications include immunotherapy (20%; delivering genes coding for antigens in order to generate an immune response) and oncolytic therapy (10%; using replication competent viruses to infect and kill cancer cells). Late stage (phase II/III and above) oncolytic therapies that are under development include ProsAtak® (prostate cancer), Pexa-Vec (HCC) and Toca-511 (GBM). In addition, a significant share of the pipeline comprises of in vivo gene therapies against various indications.
  3. The market landscape is characterized by the presence of large-sized (17), mid-sized (34) and small-sized companies (63). Some of the prominent large-sized companies engaged in this domain include (in alphabetical order) Amgen, Biogen, BioMarin Pharmaceuticals, BMS, GSK, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi. Similarly, mid-sized companies that are actively contributing to the development of gene therapies include (based on year of approval) Spark Therapeutics, Kolon Life Science and Human Stem Cell Institute. In addition, small companies, such as (in alphabetical order) Agilis Biotherapeutics, Angionetics (subsidiary of Taxus Cardium Therapeutics), AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics and XyloCor, are also actively involved in this domain.
  4. Close to 25% of the products in the development pipeline are designed to treat various oncological indications, including (ordered by decreasing number of gene therapies in the pipeline) prostate cancer, bladder cancer, head and neck cancer, breast cancer, lung cancer, ovarian cancer and pancreatic cancer. Nearly 15% of therapy candidates are being developed for the treatment of metabolic disorders (ordered by decreasing number of gene therapies under development), including mucopolysaccharidosis (MPS) IIIA, MPS IIIB, ornithine transcarbamylase (OTC) deficiency and diabetes. We also observed that close to 14% of the pipeline molecules are being developed for treating ocular disorders, such as (ordered by decreasing number of gene therapies under development) retinitis pigmentosa, wet age associated macular degeneration, achromatopsia and Leber's hereditary optic neuropathy.
  5. AAV (38%) is presently the preferred type of vector used by drug developers to design gene therapies. This can be attributed to the advantages offered by these vectors, such as high viral yields, mild immunogenic response, ability to infect a wide spectrum of cells and a better safety profile. Other prominent delivery vehicles include adenovirus (16.5%), lentivirus (14.5%) and non-viral vectors (12%), such as plasmid DNA.
  6. Over 34,000 patents, related to gene therapies, have been filed in last five years alone. Research efforts in this domain are led by both the industry and academia. Leading academic players that are actively engaged in developing gene therapies include (in decreasing order of number of patents) University of California, University of Texas, Stanford University, Harvard University, Massachusetts Institute of Technology and Johns Hopkins University.
  7. Around 240 funding instances, amounting to USD 9.5 billion worth of capital, have taken place in order to support R&D in gene therapies in the last five years. California Institute for Regenerative Medicine (CIRM), OrbiMed Advisors, Deerfield Management, Versant Ventures, Novartis Venture Fund, National Institute of Health (NIH) and 5AM Ventures have emerged as some of the prominent investors (in terms of number of funding instances).
  8. With many candidates in late stages of development, the market is likely to witness several approvals and grow steadily over the coming decade. Despite the fact that the first gene therapies were approved in Asian countries, North America and the EU together are likely to capture the over 75% of the market share by 2030. Within Asia Pacific region, South Korea is expected to dominate the market by 2030.

Report Description

The market has the potential to be 10 times higher than what most people are predicting; however, there is also a possibility that the market stalls, one of the reasons being the occurrence of serious adverse reactions. Nevertheless, the potential of gene therapies cannot be easily ignored especially for the treatment of rare/orphan diseases.

-Founder and Chief Executive Officer, a US based therapy developer

The concept of gene transfer into mammalian cells can be traced back to the 1920s. However, the first gene therapy, Gendicine® , was only approved in 2003 in China; since then, the domain has evolved significantly. The year 2017 was particularly eventful; despite the withdrawal of Glybera from the European market in early 2017, the latter half of the year witnessed the approval of two gene therapies, namely Invossa™ and Luxturna™. In fact, Luxturna™ became the first gene therapy to gain approval in the US. Further, promising clinical results were reported for several gene therapies that are currently in late phases of development. The growing popularity and potential of gene therapies can be correlated with an exponential increase in the number of patents that have been filed; the cumulative number has increased from 7,300 patents in 2013 to 42,300 patents till the third quarter of 2017. Further, over the past five years, capital worth more than USD 9.8 billion has been invested by venture capital (VC) firms and government bodies to fund research activities in this domain.

The overall market is expected to witness significant growth in opportunities for a variety of stakeholders in the coming decade. It is important to highlight that several technology providers, aiming to develop and / or support the development of gene therapies, with improved efficacy and safety, have designed and already introduced advanced platforms for the engineering of vectors. Innovation in this domain has also led to the discovery of novel molecular targets and strengthened the research pipelines of companies focused in this space. The capability to target diverse therapeutic areas is considered to be amongst the most prominent growth drivers of this market.

 

Scope of Report

The ‘Gene Therapy Market (2nd Edition), 2018-2030’ report provides an extensive study on the current market landscape of gene therapies, with a prime focus on gene augmentation based therapies and oncolytic viral therapies, featuring an elaborate discussion on the future potential of this evolving market. Amongst other elements, the report features:

  • A detailed assessment of the current market landscape of gene therapies, providing information on various drug / therapy developers, phase of development (clinical, preclinical or discovery stage) of product candidates, key therapeutic areas and indication(s), information on gene type, vector type, type of therapy (ex vivo, in vivo) and type of modification (gene augmentation, oncolytic viral therapy and others).
  • A discussion on the various types of viral and non-viral vectors, highlighting information on design, manufacturing requirements, advantages, limitations and applications of currently available gene delivery vectors.
  • A world map representation, depicting the most active geographies in terms of the presence of companies developing gene therapies, and a bull’s eye analysis highlighting the distribution of clinical pipeline candidates in terms of phase of development, type of vector and type of therapy (ex vivo / in vivo).
  • Comprehensive profiles of marketed drugs, highlighting their history of development, current developmental status, mechanism of action, affiliated technology, patent portfolio, dosage and manufacturing, and information on the developer.
  • Comprehensive profiles of clinical stage (phase II/III and above) drug candidates, highlighting their current status of development, mechanism of action, affiliated technology, patent portfolio, clinical trial information and recent developments.
  • A section on emerging technologies and platforms that are aiding the development of gene therapies, featuring detailed profiles of technologies that are presently being used in the development of four or more products / product candidates.
  • An overview of the most commonly targeted therapeutic indications and details on the gene therapies that are being developed against them.
  • An analysis of the investments made at various stages of development in companies that are focused in this area, including seed financing, venture capital financing, debt financing, grants, capital raised from IPOs and subsequent offerings.
  • A detailed analysis of the recently filed patents (since 2013); the study highlights the emerging trends in innovation and identifies the key players involved. In addition, it presents a high-level view on the valuation of these patents as well.
  • A case study on the prevalent and emerging trends in vector manufacturing, with information on companies offering contract services for manufacturing vectors. The study includes a detailed discussion on the manufacturing processes of various types of vectors as well.
  • An analysis on the various factors that may form the basis for the pricing of gene therapies, featuring different models / approaches that may be adopted in order to decide the price of a product that is likely to be marketed in the coming years.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of the market. Based on parameters, such as target consumer segments, likely adoption rates and expected pricing, we have provided an informed estimate of the likely evolution of the market in the short to mid-term and mid to long term, for the period 2018-2030. To account for the uncertainties associated with the development of gene therapies and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the market’s evolution.

The opinions and insights presented in the report were also influenced by discussions held with senior stakeholders in the industry. The study includes detailed transcripts of discussions held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Christopher Reinhard (CEO and Chairman, Cardium Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Michael Triplett (CEO, Myonexus Therapeutics). All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

Contents

Chapter 2  provides an executive summary of the insights captured during our research. It offers a high-level view on the likely evolution of the gene therapiesin the short to mid-term and mid to long term.

Chapter 3  provides a general overview of gene therapies covering their historical background, popular types (somatic versus germline;  in vivo  versus  ex vivo ), application areas and route of administration. It also includes information on the steps required to transfer gene(s) into the body, along with a discussion on the advantages and disadvantages of the process. Further, the chapter features a discussion on the ethical and social concerns related to gene therapies, and highlights certain future constraints and challenges in terms of manufacturing, reimbursements and commercial viability. It also presents details on the regulatory guidelines for these therapies in the US, Europe and China.

Chapter 4  provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of currently available vectors.

Chapter 5  includes information on over 300 gene therapies that are currently approved or are in different stages of development. It features a comprehensive analysis of pipeline molecules, highlighting the drug developers, target therapeutic areas and indications, phases of development, vectors, target gene segment, type of therapy (gene augmentation / oncolytic viral therapy / others) and type of somatic cell therapy ( in vivo  /  ex vivo ). In addition, it features a schematic world map representation, highlighting the key regional hubs where gene therapies are being developed for the treatment of various disorders. Further, we have provided a logo landscape of product developers in North America, Europe and the Asia Pacific region on the basis of company size.

Chapter 6  provides detailed profiles of marketed gene therapies. Each profile includes an overview of the developer and information on various other parameters, such as history of development, indication, mechanism of action, patent portfolio, current developmental status, target, clinical trial results and manufacturing information.

Chapter 7  contains detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each profile provides information on the mechanism of action, current status of development, route of administration, affiliated technology platform ( if applicable ), dosage form, clinical studies and key clinical trial results.

Chapter 8  provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. It features brief profiles of some of the key technologies as well. Each profile contains details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships established related to the same. In addition, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 9  highlights the potential target indications ( segregated by therapeutic areas ) that are currently the prime focus of companies developing gene therapies. These include cardiovascular disorders, hematological disorders, metabolic disorders, muscular disorders, neurological disorders, ocular disorders and oncology.

Chapter 10  presents details on various investments and grants received by companies that are engaged in this domain. It includes an analysis of the funding instances that have taken place in the period between 2013-2017, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 11  highlights our view point on the various factors that may be taken into consideration while pricing gene therapies. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt to decide the price at which their gene therapy product is likely to be marketed.

Chapter 12  provides a detailed analysis of the patents that have been filed in this domain. The study highlights emerging trends in recently filed patents, and identifies the key players that are driving the innovation in this space. Moreover, it presents a high-level view on the valuation of these patents as well.

Chapter 13  presents a comprehensive forecast analysis, highlighting the future potential of the market till the year 2030. It includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of key therapeutic areas (cardiovascular disorders, hematological disorders, muscular disorders, ocular disorders, oncology and others), type of vector (AAV, adenovirus, lentivirus, plasmid DNA, retrovirus and others), type of somatic cell therapy ( ex vivo  and  in vivo ) and geography (the US, EU, RoW (Australia, China, Israel Japan and South Korea)).

Chapter 14  provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. It also features the challenges that exist in this domain, and highlights some of the recent collaborations and developments related to manufacturing processes related to gene therapies. In addition, the chapterprovides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 15  is a summary of the overall report. In this chapter, we have provided a list of key takeaways from the report, and expressed our independent opinion related to the research and analysis described in the previous chapters.

Chapter 16  is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Christopher Reinhard (CEO and Chairman, Cardium Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Michael Triplett (CEO, Myonexus Therapeutics).

Chapter 17  is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 18  is an appendix, which provides the list of companies and organizations mentioned 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. Evolution of Gene Therapy
3.3. Classification of Gene Therapy
3.3.1. Somatic versus Germline Gene Therapy
3.3.2. Ex Vivo versus In Vivo Gene Therapy
3.4. Routes of Administration
3.5. Mechanism of Action of Gene Therapy
3.6. Advantages and Disadvantages
3.7. Ethical and Social Concerns
3.7.1. Concerns related to Somatic Gene Therapy
3.7.2. Concerns related to Germline Gene Therapy
3.8. Gene Therapy: Affiliated Methods and Technologies
3.9. Future Constraints and Challenges
3.9.1. Issues related to Manufacturing
3.9.2. Issues related to Reimbursement
3.9.3. Issues related to Commercial Viability
3.10. Regulatory Guidelines
3.10.1. European Scenario
3.10.2. The US Scenario
3.10.3. Chinese Scenario
 
4. VIRAL AND NON-VIRAL VECTORS
4.1. Chapter Overview
4.2. Viral Vectors
4.3. Types of Viral Vectors
4.3.1. Adeno-associated Viral Vectors
4.3.1.1. Overview
4.3.1.2. Design and Manufacturing
4.3.1.3. Advantages
4.3.1.4. Limitations
4.3.2. Adenoviral Vectors
4.3.2.1. Overview
4.3.2.2. Design and Manufacturing
4.3.2.3. Advantages
4.3.2.4. Limitations
4.3.3. Lentiviral Vectors
4.3.3.1. Overview
4.3.3.2. Design and Manufacturing
4.3.3.3. Advantages
4.3.3.4. Limitations
4.3.4. Retroviral Vectors
4.3.4.1. Overview
4.3.4.2. Design and Manufacturing
4.3.4.3. Advantages
4.3.4.4. Limitations
4.3.5. Other Viral Vectors
4.3.5.1.1. Alphavirus
4.3.5.1.2. Herpes Simplex Virus
4.3.5.1.3. Simian Virus
4.3.5.1.4. Vaccinia Virus
4.4. Non-Viral Vectors
4.4.1. Types of Non-Viral Vectors
4.4.1.1. Plasmid DNA
4.4.1.2. Liposomes, Lipoplexes and Polyplexes
4.4.1.3. Oligonucleotides
4.4.2. Methods of Transfection
4.4.2.1. Biolistic Methods: Gene Gun
4.4.2.2. Electroporation
4.4.2.3. Receptor Mediated Gene Delivery Methods
4.4.2.4. Gene Activated Matrix (GAM)
4.5. Applications of Viral and Non-Viral Vectors
4.5.1. In Gene Therapy
4.5.2. In Vaccinology
 
5. COMPETITIVE LANDSCAPE
5.1. Chapter Overview
5.2. Gene Therapy Market: Clinical Pipeline
5.2.1. Analysis by Phase of Development
5.2.2. Analysis by Therapeutic Area
5.2.3. Analysis by Indication
5.2.4. Analysis by Vector Type
5.2.5. Analysis by Gene Type
5.2.6. Analysis by Type of Modification
5.2.7. Analysis by Type of Gene Therapy (In Vivo / Ex Vivo)
5.3. Gene Therapy Market: Preclinical / Discovery Pipeline
5.3.1. Analysis by Therapeutic Area
5.3.2. Analysis by Target Indication
5.3.3. Analysis by Vector Type
5.3.4. Analysis by Gene Type
5.3.5. Analysis by Type of Modification
5.3.6. Analysis by Type of Gene Therapy (In Vivo / Ex Vivo)
5.4. Gene Therapy: Key Players
5.5. Gene Therapy: Prominent Hubs
5.6. Gene Therapy: Regional Landscape
 
6. MARKETED GENE THERAPIES
6.1. Chapter Overview
6.2. Gendicine® (Shenzhen SiBiono GeneTech)
6.2.1. Overview of Company and Development Pipeline
6.2.2. History of Development and Approval
6.2.3. Mechanism of Action and Vectors
6.2.4. Target Indication(s)
6.2.5. Current Status of Development
6.2.6. Manufacturing, Dosage and Sales
6.3. Oncorine™ (Shanghai Sunway Biotech)
6.3.1. Overview of Company and Development Pipeline
6.3.2. History of Development and Approval
6.3.3. Mechanism of Action and Vectors
6.3.4. Target Indication(s)
6.3.5. Current Status of Development
6.3.6. Dosage and Sales
6.4. Rexin-G (Epeius Biotechnologies)
6.4.1. Overview of Company and Development Pipeline
6.4.2. History of Development and Approval
6.4.3. Mechanism of Action and Vectors
6.4.4. Target Indication(s)
6.4.5. Current Status of Development
6.4.6. Manufacturing, Dosage and Sales
6.5. Neovasculgen® (Human Stem Cell Institute)
6.5.1. Overview of Company and Development Pipeline
6.5.2. History of Development and Approval
6.5.3. Mechanism of Action and Vectors
6.5.4. Target Indication(s)
6.5.5. Current Status of Development
6.5.6. Manufacturing, Dosage and Sales
 
6.6. Strimvelis® (GSK)
6.6.1. Overview of Company and Development Pipeline
6.6.2. History of Development and Approval
6.6.3. Mechanism of Action and Vectors
6.6.4. Target Indication(s)
6.6.5. Current Status of Development
6.6.6. Manufacturing, Dosage and Sales
 
6.7. Imlygic®  (Amgen)
6.7.1. Overview of Company and Development Pipeline
6.7.2. History of Development and Approval
6.7.3. Mechanism of Action and Vectors
6.7.4. Target Indication(s)
6.7.5. Current Status of Development
6.7.6. Manufacturing, Dosage and Sales
6.8. Invossa™ (TissueGene)
6.8.1. Overview of Company and Development Pipeline
6.8.2. History of Development and Approval
6.8.3. Mechanism of Action and Vectors
6.8.4. Target Indication(s)
6.8.5. Current Status of Development
6.8.6. Manufacturing, Dosage and Sales
6.9. Luxturna™ (Spark Therapeutics)
6.9.1. Overview of Company and Development Pipeline
6.9.2. History of Development and Approval
6.9.3. Mechanism of Action and Vectors
6.9.4. Target Indication(s)
6.9.5. Development Status
6.9.6. Manufacturing, Dosage and Sales
 
7. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES
7.1. Chapter Overview
7.2. ASP0113: Overview of Therapy, Key Developments and Clinical Results
7.3. Axalimogene Filolisbac: Overview of Therapy, Key Developments and Clinical Results
7.4. AVXS-101: Overview of Therapy, Key Developments and Clinical Results
7.5. Beperminogene Perplasmid (Collategene): Overview of Therapy, Key Developments and Clinical Results
7.6. Donaperminogene Seltoplasmid (VM202): Overview of Therapy, Key Developments and Clinical Results
7.7. E10A: Overview of Therapy, Key Developments and Clinical Results
7.8. GS-010: Overview of Therapy, Key Developments and Clinical Results
7.9. GSK2696274: Overview of Therapy, Key Developments and Clinical Results
7.10. GSK2696275: Overview of Therapy, Key Developments and Clinical Results
7.11. ImmunoPulse ®: Overview of Therapy, Key Developments and Clinical Results
7.12. Instiladrin ®: Overview of Therapy, Key Developments and Clinical Results
7.13. Lenti-D™: Overview of Therapy, Key Developments and Clinical Results
7.14. LentiGlobin® BB305: Overview of Therapy, Key Developments and Clinical Results
7.15. Ofranergene Obadenovec (VB-111): Overview of Therapy, Key Developments and Clinical Results
7.16. OTL-101: Overview of Therapy, Key Developments and Clinical Results
7.17. Pexastimogene Devacirepvec (Pexa-Vec): Overview of Therapy, Key Developments and Clinical Results
7.18. ProstAtak ®: Overview of Therapy, Key Developments and Clinical Results
7.19. Valoctocogene roxaparvovec (BMN 270): Overview of Therapy, Key Developments and Clinical Results
7.20. Vigil ®: Overview of Therapy, Key Developments and Clinical Results
7.21. VGX-3100: Overview of Therapy, Key Developments and Clinical Results
7.22. Vocimagene Amiretrorepvec (Toca-511): Overview of Therapy, Key Developments and Clinical Results
 
8. EMERGING TECHNOLOGIES
8.1. Chapter Overview
8.2. Genome Editing Technologies
8.2.1. Overview
8.2.2. Applications
8.3. Emerging Technology Platforms
8.3.1. CRISPR / Cas9 System
8.3.2. megaTAL
8.3.3. TALENs
8.3.4. Zinc Finger Nuclease
8.4. Gene Expression Regulation Technologies
8.4.1. RheoSwitch™ Therapeutic System
8.4.2. Transformative Gene Regulation Technology
8.5. Technology Platforms for Developing / Delivering Gene Therapy
8.5.1. Gene Mediated Cytotoxic Immunotherapy Technology
8.5.2. Innovative Delivery Method
8.5.3. Innovative Modular Technology
8.5.4. In Situ Delivery and Production System (i-DPS)
8.5.5. LentiVector® Platform
8.5.6. NAV® Technology Platform
 
9. THERAPEUTICS AREAS
9.1. Chapter Overview
9.2. Cardiovascular Disorders
9.3. Hematological Disorders
9.4. Inflammatory & Infectious Disorders
9.5. Metabolic Disorders
9.6. Muscular Disorders
9.7. Neurological Disorders
9.8. Ocular Disorders
9.9. Oncology
 
10. FUNDING AND INVESTMENT ANALYSIS
10.1. Chapter Overview
10.2. Types of Funding
10.3. Gene Therapy Market: Funding and Investment Analysis
10.3.1. Analysis by Number of Funding Instances
10.3.2. Analysis by Amount Invested
10.3.3. Analysis by Types of Funding
10.3.4. Analysis by Key Players
10.3.5. Analysis by Key Investors
 
11. COST PRICE ANALYSIS
11.1. Chapter Overview
11.2. Gene Therapy Market: Factors Contributing to Price Tags
11.3. Gene Therapy Market: Pricing Models
11.3.1. On the Basis of Associated Costs
11.3.2. On the Basis of Competition
11.3.3. On the Basis of Patient Segment
11.3.4. On the Basis of Experts’ Opinion
 
12. PATENT ANALYSIS
12.1. Chapter Overview
12.2. Scope and Methodology
12.3. Gene Therapy Patents: Distribution by Filing Year
12.4. Gene Therapy Patents: Distribution by Geographical Location of Patent Offices
12.5. Gene Therapy Patents: Distribution by CPC Classification
12.6. Gene Therapy Patents: Emerging Areas
12.7. Gene Therapy Patents: Leading Players
12.8. Gene Therapy Patents: Competitive Benchmarking
12.8.1. Analysis by Patent Characteristics
12.8.2. Analysis by Geographical Distribution
12.9. Gene Therapy Patents: Valuation Analysis
12.10. Gene Therapy Patents: Leading Citations
 
13. MARKET SIZING AND OPPORTUNITY ANALYSIS
13.1. Chapter Overview
13.2. Forecast Methodology
13.3. Overall Gene Therapy Market, 2018-2030
13.3.1. Gene Therapy Market: Distribution by Therapeutic Area
13.3.2. Gene Therapy Market: Distribution by Vector Type
13.3.3. Gene Therapy Market: Distribution by Therapy Type
13.3.4. Gene Therapy Market: Distribution by Geography
 
13.4. Gene Therapy Market: Product-wise Sales Forecasts
13.4.1. Gendicine® 
13.4.1.1. Target Patient Population
13.4.1.2. Sales Forecast
 
13.4.2. Oncorine
13.4.2.1. Target Patient Population
13.4.2.2. Sales Forecast
 
13.4.3. Rexin-G® 
13.4.3.1. Target Patient Population
13.4.3.2. Sales Forecast
 
13.4.4. Neovasculgen®
13.4.4.1. Target Patient Population
13.4.4.2. Sales Forecast
 
13.4.5. Strimvelis®
13.4.5.1. Target Patient Population
13.4.5.2. Sales Forecast
 
13.4.6. Imlygic®
13.4.6.1. Target Patient Population
13.4.6.2. Sales Forecast
 
13.4.7. Invossa™
13.4.7.1. Target Patient Population
13.4.7.2. Sales Forecast
 
13.4.8. Luxturna™
13.4.8.1. Target Patient Population
13.4.8.2. Sales Forecast
 
13.4.9. Axalimogene Filolisbac
13.4.9.1. Target Patient Population
13.4.9.2. Sales Forecast
 
13.4.10. AVXS-101
13.4.10.1. Target Patient Population
13.4.10.2. Sales Forecast
 
13.4.11. Collategene® (Beperminogene Perplasmid)
13.4.11.1. Target Patient Population
13.4.11.2. Sales Forecast
 
13.4.12. Donaperminogene Seltoplasmid (VM202)
13.4.12.1. Target Patient Population
13.4.12.2. Sales Forecast
 
13.4.13. E10A
13.4.13.1. Target Patient Population
13.4.13.2. Sales Forecast
 
13.4.14. GS-010
13.4.14.1. Target Patient Population
13.4.14.2. Sales Forecast
 
13.4.15. GSK2696274
13.4.15.1. Target Patient Population
13.4.15.2. Sales Forecast
 
13.4.16. GSK2696275
13.4.16.1. Target Patient Population
13.4.16.2. Sales Forecast
 
13.4.17. ImmunoPulse®
13.4.17.1. Target Patient Population
13.4.17.2. Sales Forecast
 
13.4.18. Instiladrin®
13.4.18.1. Target Patient Population
13.4.18 .2. Sales Forecast
 
13.4.19. Lenti-D™
13.4.19.1. Target Patient Population
13.4.19.2. Sales Forecast
 
13.4.20. LentiGlobin® BB305
13.4.20.1. Target Patient Population
13.4.20.2. Sales Forecast
 
13.4.21. Ofranergene Obadenovec (VB-111)
13.4.21.1. Target Patient Population
13.4.21.2. Sales Forecast
 
13.4.22. OTL-101
13.4.22.1. Target Patient Population
13.4.22.2. Sales Forecast
 
13.4.23. Pexastimogene Devacirepvec (Pexa-Vec)
13.4.23.1. Target Patient Population
13.4.23.2. Sales Forecast
 
13.4.24. ProstAtak®
13.4.24.1. Target Patient Population
13.4.24.2. Sales Forecast
 
13.4.25. Valoctocogene roxaparvovec (BMN 270)
13.4.25.1. Target Patient Population
13.4.25.2. Sales Forecast
 
13.4.26. Vigil®
13.4.26.1. Target Patient Population
13.4.26.2. Sales Forecast
 
13.4.27. VGX-3100
13.4.27.1. Target Patient Population
13.4.27.2. Sales Forecast
 
13.4.28. Vocimagene Amiretrorepvec (Toca-511)
13.4.28.1. Target Patient Population
13.4.28.2. Sales Forecast
 
14. VIRAL VECTOR MANUFACTURING
14.1. Chapter Overview
14.2. Manufacturing Viral Vectors
14.3. Viral Vector Manufacturing Processes
14.3.1. Mode of Vector Production
14.3.2. Adherent Cells Versus Suspension Cells
14.3.3. Unit Process Versus Multiple Parallel Processes
14.3.4. Cell Culture Systems for Production of Different Viral Vectors
14.4. Serum-Containing Versus Serum-Free Media
14.5. Bioprocessing of Viral Vectors
14.5.1. AAV Vector Production
14.5.2. Adenoviral Vector Production
14.5.3. Lentiviral Vector Production
14.5.4. γ-Retroviral Vector Production
14.6. Challenges Related to Vector Manufacturing
14.7. Companies Offering Contract Services for Viral and Plasmid Vectors
14.8. Partnerships
14.9. Recent Developments
 
15. CONCLUSION
15.1. Gene Therapy Market is Characterized by a Robust Pipeline of Candidates Being Developed Using Various Types of Vectors
15.2. Although Product Candidates are Being Developed to Treat a Number of Clinical Conditions, the Primary Focus is on Oncology
15.3. Currently, the Market is Led by Big Pharma Players; However, Many Start-ups, Supported by Both Private and Public Investors, are Spearheading Innovation in this Domain
15.4. CMOs are Expected to Continue to Play a Key Role in Driving Manufacturing Efforts in the Industry
15.5. Given the Advancements in Technology Platforms and the Approval of Late Stage Product Candidates, the Market is poised to Grow Significantly in the Foreseen Future
 
16. INTERVIEW TRANSCRIPTS
16.1. Chapter Overview
16.2. Adam Rogers, CEO, Hemera Biosciences
16.3. Al Hawkins, CEO, Milo Biotechnology
16.4. Cedric Szpirer, Executive & Scientific Director, Delphi Genetics
16.5. Christopher Reinhard, CEO and Chairman, Cardium Therapeutics
16.6. Jeffrey Hung, CCO, Vigene Biosciences
16.7. Marco Schmeer, Project Manager and Tatjana Buchholz, Marketing Manager, PlasmidFactory
16.8. Michael Triplett, CEO, Myonexus Therapeutics
 
17. APPENDIX 1: TABULATED DATA
 
18. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

List of Figures

Figure 3.1  History of Evolution of Gene Therapies
Figure 3.2  Ex Vivo Gene Therapy
Figure 3.3  In Vivo Gene Therapy
Figure 3.4  Mechanism of Action of Gene Therapies
Figure 3.5  Viral Vectors as Gene Transfer Tools
Figure 3.6  Advantages and Disadvantages of Gene Therapies 
Figure 3.7  Gene Therapy: Affiliated Methods and Technologies 
Figure 3.8  Construction of DNA Expression Cassette and the Gene Delivery System
Figure 3.9  China: Manufacturing Requirements for Gene Therapy Products
Figure 3.10 China: Key Elements of Quality Control
Figure 4.1  Gene Transfer: Viral and Non-Viral Methods
Figure 5.1  Gene Therapies, Clinical Pipeline: Distribution by Phase of Development
Figure 5.2  Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Areas
Figure 5.3  Gene Therapies, Clinical Pipeline: Distribution by Indications 
Figure 5.4  Gene Therapies, Clinical Pipeline: Distribution by Vector Type
Figure 5.5  Gene Therapies, Clinical Pipeline: Distribution by Gene Type
Figure 5.6  Gene Therapies, Clinical Pipeline: Distribution by Type of Modification
Figure 5.7  Gene Therapies, Clinical Pipeline: Distribution by Type of Gene Therapy
Figure 5.8 Gene Therapies, Preclinical Pipeline: Distribution by Therapeutic Areas
Figure 5.9  Gene Therapies, Preclinical Pipeline: Distribution by Indications
Figure 5.10  Gene Therapies, Preclinical Pipeline: Distribution by Vector Type
Figure 5.11  Gene Therapies, Preclinical Pipeline: Distribution by Gene Type
Figure 5.12  Gene Therapies, Preclinical Pipeline: Distribution by Type of Modification
Figure 5.13  Gene Therapies, Preclinical Pipeline: Distribution by Type of Gene Therapy
Figure 5.14  Gene Therapies: Key Players
Figure 5.15  Gene Therapies: Prominent Hubs
Figure 5.16  Gene Therapy Developers: North America
Figure 5.17  Gene Therapy Developers: Europe 
Figure 5.18  Gene Therapy Developers: Asia Pacific
Figure 6.1  Gendicine®: Development Timeline
Figure 6.2  Oncorine: Development Timeline
Figure 6.3  Oncorine: Mechanism of Action
Figure 6.4  Rexin-G: Development Timeline
Figure 6.5  Neovasculgen®: Development Timeline
Figure 6.6  Invossa™: Development Timeline
Figure 6.7  Invossa™: Mechanism of Action 
Figure 6.8  Strimvelis®: Development Timeline
Figure 6.9  Imlygic®: Development Timeline
Figure 6.10  Imlygic®: Mechanism of Action
Figure 6.11  Luxturna™: Development Timeline
Figure 8.1  Genome Editing Technologies: Applications
Figure 8.2  Genome Editing Technologies: Emerging Technology Platforms 
Figure 9.1  Gene Therapies for Cardiovascular Disorders: Distribution by Indication and Phase of Development
Figure 9.2  Gene Therapies for Cardiovascular Disorders : Distribution by Vector Type
Figure 9.3  Gene Therapies for Hematological Disorders: Distribution by Indication and Phase of Development
Figure 9.4  Gene Therapies for Hematological Disorders: Distribution by Vector Type 
Figure 9.5  Gene Therapies for I&I Disorders: Distribution by Indication and Phase of Development
Figure 9.6  Gene Therapies for I&I Disorders: Distribution by Vector Type 
Figure 9.7  Gene Therapies for Metabolic Disorders: Distribution by Indication and Phase of Development
Figure 9.8  Gene Therapies for Metabolic Disorders: Distribution by Vector Type 
Figure 9.9  Gene Therapies for Muscular Disorders: Distribution by Indication and Phase of Development
Figure 9.10  Gene Therapies for Muscular Disorders: Distribution by Vector Type 
Figure 9.11  Gene Therapies for Neurological Disorders: Distribution by Indication and Phase of Development
Figure 9.12  Gene Therapies for Neurological Disorders: Distribution by Vector Type 
Figure 9.13  Gene Therapies for Ocular Disorders: Distribution by Indication and Phase of Development
Figure 9.14  Gene Therapies for Ocular Disorders: Distribution by Vector Type 
Figure 9.15  Gene Therapies for Oncology: Distribution by Indication and Phase of Development
Figure 9.16  Gene Therapies for Oncology: Distribution by Vector Type 
Figure 10.1 Gene Therapy Market: Cumulative Number of Funding Instances, Pre-2013-2018
Figure 10.2  Gene Therapy Market: Cumulative Amount Invested, Pre-2013-2018 (USD Billion)
Figure 10.3  Gene Therapy Market: Distribution by Type of Funding, Pre-2013-2018
Figure 10.4  Gene Therapy Market: Amount Invested under each Type of Funding, Pre-2013 – 2018 (USD Million)
Figure 10.5  Gene Therapy Market: Funding and Investment Summary
Figure 10.6  Gene Therapy Market: Key Players
Figure 10.7  Gene Therapy Market: Key Investors
Figure 11.1  Gene Therapy: Pricing Model Based on Patient Segment
Figure 12.1  Gene Therapy Patents: Distribution by Types of Patents
Figure 12.2  Gene Therapy Patents: Distribution (Cumulative) by Publication Year
Figure 12.3  Gene Therapy Patents: Distribution by Patent Offices
Figure 12.4  Gene Therapy Patents: Cumulative Distribution by CPC Classification Symbol
Figure 12.5  Gene Therapy Patents: Emerging Areas
Figure 12.6  Gene Therapy Patents: Leading Industry and Non-Industry Players 
Figure 12.7  Gene Therapy Patents: Distribution by Industry Players 
Figure 12.8  Gene Therapy Patents: Distribution by Non-Industry Players 
Figure 12.9  Gene Therapy Patents: Geographical Distribution of Leading Players
Figure 12.10  Gene Therapy Patents (Industry Players): Benchmarking by Patent Characteristics
Figure 12.11  Gene Therapy Patents (Non-Industry Players): Benchmarking by Patent Characteristics 
Figure 12.12  Gene Therapy Patents (Industry Players): Benchmarking by Geography
Figure 12.13  Gene Therapy Patents (Industry Players): Benchmarking by International Patents
Figure 12.14  Gene Therapy Patents (Non-Industry Players): Benchmarking by Geography 
Figure 12.15  Gene Therapy (Non-Industry) Patents: Benchmarking by International Patents
Figure 12.16  Gene Therapy Patents: Distribution by Age of Patents (January 2013-October 2017)
Figure 12.17  Gene Therapy Patents: Valuation Analysis
Figure 13.1  Overall Gene Therapy Market, 2018-2030: Base Scenario (USD Million)
Figure 13.2  Gene Therapy Market: Distribution by Therapeutic Area, 2018, 2024 and 2030 (USD Million)
Figure 13.3  Gene Therapy Market: Distribution by Vector Type, 2018, 2024 and 2030 (USD Million)
Figure 13.4  Gene Therapy Market: Distribution by Therapy Type, 2018, 2024 and 2030 (USD Million)
Figure 13.5  Gene Therapy Market: Distribution by Geography, 2018, 2024 and 2030 (USD Million)
Figure 13.6  Gendicine® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.7  Oncorine Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.8  Rexin-G Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.9  Neovasculgen® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.10  Strimvelis® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.11  Imlygic® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.12  Invossa™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.13  Luxturna™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.14  ASP0113 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.15  Axalimogene Filolisbac Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.16  AVXS-101 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.17  Beperminogene Perplasmid (Collategene®) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.18  Donaperminogene Seltoplasmid (VM202) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.19  E10A Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.20  GS-010 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.21  GSK2696274 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.22  GSK2696275 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.23  ImmunoPulse® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.24  Instiladrin® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.25  Lenti-D™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.26  LentiGlobin® BB305 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.27  Ofranergene Obadenovec (VB-111) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.28  OTL-101 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.29  Pexastimogene Devacirepvec (Pexa-Vec) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.30  ProstAtak® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.31  Valoctocogene Roxaparvovec (BMN 270) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.32  Vigil® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.33  VGX-3100 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 13.34  Vocimagene Amiretrorepvec (Toca-511) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 14.1  Viral Vectors: Manufacturing Steps 
Figure 15.1  Gene Therapy: Distribution of Clinical Stage Product Candidates by Vector Type and Therapy Type 
Figure 15.2  Gene Therapy: Key Therapeutic Areas
Figure 15.3  Gene Therapy Market: Conservative, Base and Optimistic Forecast Scenarios, 2018, 2024 and 2030 (USD Billion)

List of Tables

Table 3.1 Differences between Ex Vivo and In Vivo Techniques
Table 3.2 Administration Routes for Targeted Gene Delivery to Specific Organs / Tissues
Table 3.3 Routes of Administration used for Targeted Gene Delivery to Tissues / Organs
Table 3.4  Routes of Administration of Gene Therapies: Advantages and Disadvantages 
Table 3.5  Prices of Marketed Gene Therapies 
Table 3.6  Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Statuses in Europe
Table 4.1  Viral Vectors: Key Features
Table 5.1  Gene Therapies: Clinical Pipeline
Table 5.2  Gene Therapies: Preclinical Pipeline
Table 6.1  Gene Therapies: Marketed and Approved Products
Table 6.2  Company Overview: Shenzhen SiBiono GeneTech
Table 6.3  Gendicine®: Status of Development 
Table 6.4  Gendicine®: Patent Portfolio 
Table 6.5  Company Overview: Shanghai Sunway Biotech
Table 6.6  Oncorine: Status of Development
Table 6.7  Company Overview: Epeius Biotechnologies
Table 6.8  Rexin-G: Status of Development
Table 6.9  Company Overview: Human Stem Cell Institute
Table 6.10  Neovasculgen®: Status of Development
Table 6.11  Company Overview: GSK
Table 6.12  Strimvelis®: Status of Development
Table 6.13  Company Overview: Amgen
Table 6.14  Imlygic®: Status of Development
Table 6.15  Imlygic®: Recommended Dose and Schedule 
Table 6.16  Imylgic®: Determination of Injection Volume based upon Lesion Size
Table 6.17  Company Overview: TissueGene
Table 6.18  Invossa™: Status of Development
Table 6.19  Company Overview: Spark Therapeutics
Table 6.20  Luxturna™: Status of Development
Table 7.1  Gene Therapies: Phase III Drugs
Table 7.2  Drug Profile: ASP0113
Table 7.3  Drug Profile: Axalimogene Filolisbac
Table 7.4  Drug Profile: AVXS-101
Table 7.5  Drug Profile: Beperminogene Perplasmid (Collategene®)
Table 7.6  Drug Profile: Donaperminogene Seltoplasmid (VM202)
Table 7.7  Drug Profile: E10A
Table 7.8  Drug Profile: GS-010
Table 7.9  Drug Profile: GSK2696274
Table 7.10  Drug Profile: GSK2696275
Table 7.11  Drug Profile: ImmunoPulse®
Table 7.12  Drug Profile: Instiladrin®
Table 7.13  Drug Profile: Lenti-D™
Table 7.14  Drug Profile: LentiGlobin® BB305
Table 7.15  Drug Profile: Ofranergene Obadenovec (VB-111)
Table 7.16  Drug Profile: OTL-101
Table 7.17  Drug Profile: Pexastimogene Devacirepvec (Pexa-Vec)
Table 7.18  Drug Profile: ProstAtak®
Table 7.19  Drug Profile: Valoctocogene roxaparvovec (BMN 270)
Table 7.20  Drug Profile: Vigil®
Table 7.21  Drug Profile: VGX-3100
Table 7.22  Drug Profile: Vocimagene Amiretrorepvec (Toca-511)
Table 8.1  Gene Editing Technology Platforms
Table 8.2  Editas Medicine: CRISPR / Cas9 Technology, Research Publications 
Table 8.3  Editas Medicine: Funding Instances
Table 8.4  Editas Medicine: Collaborations
Table 8.5  Intellia Therapeutics: CRISPR / Cas9 Technology, Research Publications
Table 8.6  Intellia Therapeutics: Funding Instances
Table 8.7  Intellia Therapeutics: Collaborations
Table 8.8  CRISPR Therapeutics: CRIPSR / Cas9 Technology, Research Publications
Table 8.9  CRISPR Therapeutics: Funding Instances
Table 8.10  CRISPR Therapeutics: Collaborations
Table 8.11  Cellectis: Funding Instances
Table 8.12  Cellectis: Collaborations
Table 8.13  bluebird bio: megaTAL Technology, Research Publications
Table 8.14  bluebird bio: Funding Instances
Table 8.15  bluebird bio: Collaborations
Table 8.16  Sangamo BioSciences: Funding Instances
Table 8.17  Sangamo BioSciences: Collaborations
Table 8.18  Gene Switch Technology Platforms
Table 8.19  Intrexon: Funding Instances
Table 8.20  Intrexon: Collaborations
Table 8.21  MeiraGTx: Funding Instances
Table 8.22  MeiraGTx: Collaborations
Table 8.23  Gene Therapies: Technology Platforms
Table 9.1  Gene Therapies for Cardiovascular Disorders
Table 9.2  Gene Therapies for Hematological Disorders
Table 9.3  Gene Therapies for I&I Disorders
Table 9.4  Gene Therapies for Metabolic Disorders
Table 9.5  Gene Therapies for Muscular Disorders
Table 9.6  Gene Therapies for Neurological Disorders
Table 9.7  Gene Therapies for Ocular Disorders
Table 9.8  Gene Therapies for Oncology
Table 10.1  Gene Therapy Market: Funding and Investment Analysis
Table 10.2  Gene Therapy Market: Summary of Investments
Table 11.1  Pricing Model: Price of Marketed Gene / Cell Therapies
Table 11.2  Pricing Model: Price of Marketed Targeted Drugs
Table 11.3  Pricing Model: Opinions of Experts / Other Analysts
Table 12.1  Gene Therapy Patents: CPC Symbol Definitions
Table 12.2  Gene Therapy Patents: Most Popular CPC Symbols
Table 12.3  Gene Therapy Patents: List of Top CPC Classifications
Table 12.4  Gene Therapy Patents: List of Top Leading Citations
Table 13.1  Gene Therapies: Expected Launch Years of Advanced Stage Drug Candidates
Table 13.2  Gendicine®: Target Patient Population 
Table 13.3  Oncorine: Target Patient Population
Table 13.4  Rexin-G: Target Patient Population 
Table 13.5  Neovasculgen®: Target Patient Population
Table 13.6  Strimvelis®: Target Patient Population 
Table 13.7  Imlygic®: Target Patient Population 
Table 13.8  Invossa™: Target Patient Population
Table 13.9  Luxturna™: Target Patient Population
Table 13.10  ASP0113: Target Patient Population
Table 13.11  Axalimogene Filolisbac: Target Patient Population
Table 13.12  AVXS-101: Target Patient Population
Table 13.13  Beperminogene Perplasmid (Collategene®): Target Patient Population
Table 13.14  Donaperminogene Seltoplasmid (VM202): Target Patient Population
Table 13.15  E10A: Target Patient Population
Table 13.16  GS-010: Target Patient Population
Table 13.17  GSK2696274: Target Patient Population
Table 13.18  GSK2696275: Target Patient Population
Table 13.19  ImmunoPulse®: Target Patient Population
Table 13.20  Instiladrin®: Target Patient Population
Table 13.21  Lenti-D™: Target Patient Population
Table 13.22  LentiGlobin® BB305: Target Patient Population
Table 13.23  Ofranergene Obadenovec (VB-111): Target Patient Population
Table 13.24  OTL-101: Target Patient Population
Table 13.25  Pexastimogene Devacirepvec (Pexa-Vec): Target Patient Population
Table 13.26  ProstAtak®: Target Patient Population
Table 13.27  Valoctocogene Roxaparvovec (BMN 270): Target Patient Population
Table 13.28  Vigil®: Target Patient Population
Table 13.29  VGX-3100: Target Patient Population
Table 13.30  Vocimagene Amiretrorepvec (Toca-511): Target Patient Population
Table 14.1  Small Scale Cell Culture Systems
Table 14.2  Contract Viral and Plasmid Vector Manufacturing Service Providers  
Table 14.3  Gene Therapy Manufacturing Partnerships 
Table 17.1  Gene Therapies, Clinical Pipeline: Distribution by Phase of Development
Table 17.2  Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Areas
Table 17.3  Gene Therapies, Clinical Pipeline: Distribution by Indications 
Table 17.4  Gene Therapies, Clinical Pipeline: Distribution by Vector Type
Table 17.5  Gene Therapies, Clinical Pipeline: Distribution by Gene Type
Table 17.6  Gene Therapies, Clinical Pipeline: Distribution by Type of Modification
Table 17.7  Gene Therapies, Clinical Pipeline: Distribution by Type of Gene Therapy
Table 17.8  Gene Therapies, Preclinical Pipeline: Distribution by Therapeutic Areas
Table 17.9  Gene Therapies, Preclinical Pipeline: Distribution by Indications
Table 17.10  Gene Therapies, Preclinical Pipeline: Distribution by Vector Type
Table 17.11  Gene Therapies, Preclinical Pipeline: Distribution by Gene Type
Table 17.12  Gene Therapies, Preclinical Pipeline: Distribution by Type of Modification
Table 17.13  Gene Therapies, Preclinical Pipeline: Distribution by Type of Gene Therapy
Table 17.14  Gene Therapies for Cardiovascular Disorders: Distribution by Indication and Phase of Development
Table 17.15  Gene Therapies for Cardiovascular Diseases: Distribution by Vector Type
Table 17.16  Gene Therapies for Hematological Disorders: Distribution by Indication and Phase of Development
Table 17.17  Gene Therapies for Hematological Disorders: Distribution by Vector Type
Table 17.18  Gene Therapies for I&I Disorders: Distribution by Indication and Phase of Development
Table 17.19  Gene Therapies for I&I Disorders: Distribution by Vector Type
Table 17.20  Gene Therapies for Metabolic Disorders: Distribution by Indication and Phase of Development
Table 17.21  Gene Therapies for Metabolic Disorders: Distribution by Vector Type
Table 17.22  Gene Therapies for Muscular Disorders: Distribution by Indication and Phase of Development
Table 17.23  Gene Therapies for Muscular Disorders: Distribution by Vector Type
Table 17.24  Gene Therapies for Neurological Disorders: Distribution by Indication and Phase of Development
Table 17.25  Gene Therapies for Neurological Disorders: Distribution by Vector Type
Table 17.26  Gene Therapies for Ocular Disorders: Distribution by Indication and Phase of Development
Table 17.27  Gene Therapies for Ocular Disorders: Distribution by Vector Type
Table 17.28  Gene Therapies for Oncology: Distribution by Indication and Phase of Development
Table 17.29  Gene Therapies for Oncology: Distribution by Vector Type
Table 17.31  Gene Therapy Market: Cumulative Number of Funding Instances, Pre-2013-2018
Table 17.32  Gene Therapy Market: Cumulative Amount Invested, Pre-2013-2018 (USD Billion)
Table 17.33  Gene Therapy Market: Distribution by Type of Funding, Pre-2013-2018
Table 17.34  Gene Therapy Market: Amount Invested under each Type of Funding, Pre-2013 – 2018 (USD Million)
Table 17.35  Gene Therapy Market: Funding and Investment Summary
Table 17.36  Gene Therapy Market: Key Players
Table 17.37  Gene Therapy Market: Key Investors
Table 17.38  Gene Therapy Patents: Distribution by Type of Patent
Table 17.39  Gene Therapy Patents: Cumulative Number by Publication Year
Table 17.40  Gene Therapy Patents: Distribution by Patent Offices
Table 17.41  Gene Therapy Patents: Distribution by Industry Player Data
Table 17.42  Gene Therapy Patents: Distribution by Non-Industry Player Data 
Table 17.43  Gene Therapy Patents (Industry Players): Benchmarking by Geography
Table 17.44  Gene Therapy Patents (Industry Players): Benchmarking by International Patents
Table 17.45  Gene Therapy Patents (Non-Industry Players): Benchmarking by Geography 
Table 17.46  Gene Therapy (Non-Industry) Patents: Benchmarking by International Patents
Table 17.47  Gene Therapy Patents: Distribution by Age of Patents (January 2013-October 2017)
Table 17.48  Overall Gene Therapy Market, 2018-2030: Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.49  Gene Therapy Market: Distribution by Therapeutic Area, 2018, 2024 and 2030 (USD Million)
Table 17.50  Gene Therapy Market: Distribution by Vector Type, 2018, 2024 and 2030 (USD Million)
Table 17.51  Gene Therapy Market: Distribution by Therapy Type, 2018, 2024 and 2030 (USD Million)
Table 17.52  Gene Therapy Market: Distribution by Geography, 2018, 2024 and 2030 (USD Million)
Table 17.53  Gendicine® Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.54  Oncorine Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.55  Rexin-G Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.56  Neovasculgen® Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.57  Strimvelis® Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.58  Imlygic® Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.59  Invossa™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.60  Luxturna™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.61  ASP0113 Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.62  Axalimogene Filolisbac Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.63  AVXS-101 Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.64  Beperminogene Perplasmid (Collategene) Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.65  Donaperminogene Seltoplasmid (VM202) Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.66  E10A Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.67  GS-010 Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.68  GSK2696274 Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.69  GSK2696275 Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.70  ImmunoPulse® Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.71  Instiladrin® Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.72  Lenti-D™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.73  LentiGlobin®BB305 Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.74  Ofranergene Obadenovec (VB-111) Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.75  OTL-101 Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.76  Pexastimogene Devacirepvec (Pexa-Vec) Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.77  ProstAtak® Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.78  Valoctocogene Roxaparvovec (BMN 270) Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.79  Vigil® Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.81  VGX-3100 Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.82  Vocimagene Amiretrorepvec (Toca-511) Sales Forecast (Till 2030): Conservative, Base and Optimistic Forecast Scenarios (USD Million)
Table 17.83  Gene Therapy Market: Conservative, Base and Optimistic Forecast Scenarios, 2018, 2024 and 2030 (USD Billion)

Listed Companies

The following companies and organizations have been mentioned in the report.

  1. 4BIO Capital
  2. 5AM Ventures
  3. Abbott
  4. Abby Grace Foundation
  5. Abeona Therapeutics
  6. Abingworth
  7. ABL Europe 
  8. Ablynx
  9. Abraxis BioScience
  10. Acceleron Pharma
  11. Acucela (Subsidiary of Kubota Pharmaceutical)
  12. Adage Capital Management
  13. Aduro Biotech
  14. Advanced BioScience Laboratories (ABL)
  15. Advanced Biotherapeutics Consulting 
  16. Advantagene
  17. Advaxis Immunotherapies
  18. Adverum Biotechnologies
  19. Agent Capital
  20. Agilis   Biotherapeutics
  21. Ajinomoto Althea
  22. AJU IB Investment  
  23. Aldevron
  24. Alexandria Venture Investments
  25. Allele Biotechnology and Pharmaceuticals
  26. Ally Bridge Group (ABG)
  27. Alpha-1 Foundation
  28. AlphaVax
  29. Amgen
  30. AMSBIO
  31. Anaeropharma Science
  32. Anemocyte (previously known as Areta International)
  33. AnGes
  34. Angionetics  
  35. Apic Bio
  36. Applied Genetic Technologies Corporation (AGTC)
  37. Applied Viromics
  38. ARCH Venture Partners
  39. Arthrogen
  40. AstraZeneca
  41. Athena Vision
  42. Atlas Venture
  43. aTyr Pharma
  44. Audentes Therapeutics
  45. AveXis
  46. AVROBIO
  47. Baillie Gifford
  48. Bamboo Therapeutics (acquired   by Pfizer)
  49. Batavia Biosciences
  50. Bayer
  51. Beckman Research Institute 
  52. Beijing Capital Grand (previously known as Beijing Capital Juda)
  53. Benitec Biopharma  
  54. BioCanCell Therapeutic
  55. Biogen
  56. BioMarin  Pharmaceutical 
  57. Biomay
  58. Biomiga 
  59. BioMotiv
  60. BioNTech Innovative Manufacturing Services (previously known as EUFETS)
  61. BioReliance / SAFC
  62. Bioverativ
  63. Biovian
  64. BioViva
  65. Blue Sky BioServices
  66. bluebird bio
  67. BlueWater Angels
  68. Boehringer Ingelheim BioXcellence™ 
  69. Boehringer Ingelheim Venture Fund
  70. Bpifrance 
  71. Brammer Bio
  72. Bristol-Myers Squibb  
  73. Broad Institute
  74. Brookside Capital Partners Fund
  75. California Institute for Regenerative Medicine (CIRM)
  76. Cambridge Enterprise
  77. Cancer Prevention and Research Institute of Texas (CPRIT) 
  78. CapDecisif Management
  79. Caribou Biosciences
  80. Casdin Capital
  81. Celgene
  82. Cell and Gene Therapy Catapult
  83. Cellectis
  84. CellGenTech
  85. Celsion
  86. Center for the Development of Industrial Technology (CDTI)
  87. Centre for Process Innovation
  88. Centre national de la recherche scientifique (CNRS)
  89. CEVEC Pharmaceuticals
  90. CHDI Foundation
  91. Chiesi Farmaceutici
  92. Children’s Medical Research Foundation
  93. Children's Hospital of Philadelphia
  94. Children's Medical Research Foundation
  95. Clarus Ventures  
  96. Clough Capital Partners
  97. Cobra Biologics and Pharmaceutical Services
  98. Cold Genesys
  99. Columbus Venture Partners
  100. Commercial & External Partnership, Industrial Affairs (CEPiA)
  101. Copernicus Therapeutics   
  102. Cormorant Asset Management
  103. Cowen 
  104. Creative Biogene
  105. CRISPR Therapeutics
  106. Cure Sanfilippo Foundation
  107. CureVac
  108. Cystic Fibrosis Foundation Therapeutics (CFFT)
  109. Deerfield Management Company 
  110. Dimension Therapeutics (acquired by Ultragenyx Pharmaceutical)
  111. DNAtrix Therapeutics
  112. Easton Capital
  113. EcoR1 Capital 
  114. Editas Medicine
  115. Elk OrthoBiologics  
  116. Emergent BioSolutions
  117. Eminent Venture Capital 
  118. enGenes 
  119. Ensysce Biosciences
  120. Epeius Biotechnologies   
  121. Errant Gene Therapeutics
  122. Esteve
  123. Etubics 
  124. Eurofins Genomics
  125. Eurogentec
  126. European Investment Bank (EIB)
  127. Expression Therapeutics 
  128. ExSight Capital
  129. Eyevensys
  130. Federal Holding and Investment Company (SFPI-FPIM) 
  131. Fibrocell Science
  132. Fireman Capital Partners
  133. FKD Therapies 
  134. Flagship Pioneering (previously known as Flagship Ventures)
  135. Fondación Genoma España
  136. Fondation Sanfilippo Suisse
  137. Fondazione Telethon and Ospedale San Raffaele
  138. Forbion Capital Partners
  139. Foresite Capital Management
  140. FoxKiser 
  141. F-Prime Capital Partners (previously known as Fidelity Biosciences) 
  142. Franklin Advisers
  143. Franklin Templeton Investments
  144. Fred Hutchinson Cancer Research Center
  145. Freeline Therapeutics
  146. Friedreich’s Ataxia Research Alliance (FARA)
  147. FUJIFILM Diosynth Biotechnologies
  148. Fund+ 
  149. Fundación Sanfilippo B
  150. Fundación Stop Sanfilippo
  151. Further Lane Securities
  152. Gene Medicine Japan (GMJ) / Kobe Biomedical Accelerator
  153. GeneCure Biotechnologies
  154. GeneDetect
  155. GeneImmune Biotechnology
  156. Genelux 
  157. Genentech
  158. GeneOne Life Science
  159. Genethon
  160. Genexine 
  161. GenIbet Biopharmaceuticals
  162. Genprex
  163. GenScript Biotech
  164. GenSight Biologics
  165. GenVec
  166. GlaxoSmithKline  
  167. Global BioTherapeutics   
  168. GlobeImmune
  169. GO Capital Amorçage
  170. Google Ventures (GV)
  171. Gradalis®  
  172. Green Cross Holdings
  173. Guangzhou Double Bio-products
  174. Harvard University
  175. HealthCap
  176. Hemera Biosciences
  177. Herantis Pharma
  178. Hercules Capital
  179. Hoffmann-La Roche
  180. HORAMA   
  181. Horizon Discovery 
  182. Horizon Pharma  
  183. Huapont Life Sciences
  184. Human Stem Cells Institute
  185. I2BF Global Ventures
  186. Ichor Therapeutics
  187. Ichor  Systems 
  188. ID Pharma  
  189. Idinvest Partners
  190. Immune Technology
  191. Imperial Innovations
  192. Inception Capital Management
  193. Index Ventures
  194. Innovation Network Corporation of Japan (INCJ)
  195. Innovative Genomics Institute (IGI)
  196. Inovio Pharmaceuticals
  197. Inserm  
  198. Intellia Therapeutics
  199. Intrexon 
  200. Invesco Perpetual 
  201. InvivoGen
  202. Ion Channel Innovations
  203. Iowa State University
  204. Israel Innovation Authority
  205. Janssen
  206. Janus Capital Management
  207. Jennison Associates 
  208. Johns Hopkins University
  209. Johnson & Johnson Development Corporation 
  210. JumpStart Ventures
  211. Juventas Therapeutics
  212. Kaneka Eurogentec 
  213. Khosla Ventures
  214. Kiwoom Securities
  215. Knight Therapeutics
  216. KOLON LIFE SCIENCE
  217. Krystal Group  
  218. Kubota Pharmaceutical   
  219. Kurma Partners
  220. Leerink Transformation Partners
  221. Leland Stanford Junior University
  222. Lentigen Technology (wholly owned subsidiary of Miltenyi Biotec)
  223. Leukon Investments
  224. Lime Asset Management
  225. Lonza  
  226. Louisiana State University (LSU)
  227. Luminous BioSciences
  228. Lundbeckfonden Ventures
  229. Lysogene
  230. Marsala Biotech
  231. Maryland Technology Development Corporation (TEDCO) 
  232. Massachusetts General Hospital
  233. Massachusetts Institute of Technology
  234. MassBiologics
  235. Medarex
  236. Medigen Biotechnology 
  237. MedImmune
  238. Medtronic
  239. MeiraGTx
  240. Mercia Technologies
  241. Merck
  242. Mercury Fund
  243. Meridian Life Science 
  244. MERITZ Securities
  245. MidCap Financial
  246. Milo Biotechnology
  247. Miltenyi Biotec
  248. Mita Securities
  249. Mitsubishi Tanabe Pharma
  250. Mitsubishi UFJ Capital
  251. Moderna Therapeutics
  252. Molecular Diagnostic Services(MDS)
  253. MolMed 
  254. MOLOGEN
  255. Momotaro-Gene 
  256. Morningside Group
  257. Movember Foundation
  258. Myonexus Therapeutics
  259. NanoCor Therapeutics
  260. NanoDimension Venture Firm
  261. NantCell
  262. Nantes Gene Therapy Institute 
  263. National Cancer Institute (NCI)
  264. National Eye Institute (NEI)
  265. National Institute of Allergy and Infectious Diseases (NIAID)
  266. National Institute on Drug Abuse (NIDA)
  267. National Institutes of Health (NIH)
  268. National MPS Society
  269. National Securities Corporation (NSC)
  270. Nature Technology 
  271. Neuralgene
  272. Neurotech Pharmaceuticals
  273. New Energy and Industrial Technology Development Organization (NEDO)
  274. New Enterprise Associates (NEA)
  275. New Leaf Venture Partners
  276. New River Management
  277. NewVa Capital Partners
  278. Nightstar Therapeutics
  279. Nohla therapeutics
  280. Novartis
  281. Novartis Venture Fund
  282. Novasep
  283. Novo Seeds
  284. Oberland Capital
  285. Okairos
  286. Omega Fund Management
  287. Omnes Capital
  288. Omnia Biologics
  289. Oncolys BioPharma
  290. OncoSec Medical 
  291. OrbiMed Advisors
  292. ORCA Therapeutics
  293. Orchard Therapeutics
  294. ORI Capital
  295. Osage University Partners
  296. Oxford BioMedica
  297. Oxford Finance
  298. Oxford Genetics 
  299. PacificGMP (a part of Abzena Group)
  300. Pangu BioPharma
  301. Panmure Gordon 
  302. Paragon Bioservices
  303. Partners Innovation Fund
  304. Pavilion Capital Partners
  305. PeriphaGen
  306. Pfizer
  307. PhaseRx
  308. PlasmidFactory
  309. PNP Therapeutics®
  310. Polaris Partners
  311. Pontifax
  312. Posco Capital Partners 
  313. Poseida Therapeutics
  314. Precision BioSciences 
  315. Profectus BioSciences  
  316. Promethera Biosciences
  317. PsiOxus Therapeutics
  318. Puresyn
  319. Quethera
  320. RA Capital Management®
  321. RBV Capital
  322. Red Sanfilippo Foundation
  323. Redmile Group
  324. Regeneron Pharmaceuticals   
  325. REGENXBIO
  326. Relieve Genetics
  327. Renova Therapeutics 
  328. RetroSense Therapeutics (acquired by Allergan)
  329. Rev1 Ventures (previously known as TechColumbus)
  330. REYON Pharmaceutical   
  331. Richter-Helm BioLogics
  332. Ridgeback Capital Management
  333. Roche
  334. Roche Venture Fund
  335. Rock Springs Capital Management
  336. Rocket Pharma
  337. RTW Investments
  338. Rusnano
  339. SAB Technology
  340. Sabby Capital
  341. San Raffaele Telethon Institute for Gene Therapy (SR-TIGET)  
  342. SanBio
  343. Sanfilippo Children’s Foundation
  344. Sanfilippo Children's Research Foundation (SCRF)
  345. Sanfilippo Research Foundation (SRF)
  346. Sangamo Therapeutics
  347. Sanofi
  348. Sanofi Genzyme
  349. Sanofi-Genzyme BioVentures
  350. Santen Pharmaceutical 
  351. Sarepta Therapeutics
  352. Scancell
  353. Sectoral Asset Management
  354. Selecta Biosciences
  355. Seventure Partners' Health for Life Capital fund
  356. Sham Innovation Santé
  357. Shanghai Sunway Biotech
  358. Shenzhen Qianhai Taxus Industry Capital Management
  359. Shenzhen Sibiono GeneTech
  360. Shinhan Capital
  361. Shinsei Corporate Investment
  362. Shire
  363. Sigma-Aldrich (now a part of Merck)
  364. SignaGen Laboratories
  365. Sino Biological 
  366. SIRION Biotech
  367. Skolkovo Foundation
  368. SMS Investments
  369. Sofinnova Ventures
  370. Song Hong Fang
  371. Spark Therapeutics
  372. Sphera Global Health Care
  373. SR One (investment arm of GlaxoSmithKline)
  374. Stratophase 
  375. StrideBio
  376. SV Life Sciences Investors
  377. Syncona Partners (a subsidiary of the Wellcome Trust)
  378. Synpromics
  379. Synthace
  380. System Biosciences
  381. T. Rowe Price 
  382. Takeda Pharmaceutical
  383. Target ALS Foundation
  384. Targeted Technology Fund
  385. Targovax
  386. Team Sanfilippo 
  387. Tekes
  388. Temasek Holdings
  389. Tethys Ventures
  390. The Finnish Funding Agency for Technology and Innovation
  391. The French Muscular Dystrophy Association (AFM-Téléthon)
  392. The Jagen Group
  393. The Scripps Research Institute
  394. TheraBiologics
  395. TheraVectys 
  396. Thermo Fisher Scientific
  397. Third Rock Ventures 
  398. Third Security
  399. TissueGene
  400. Tocagen
  401. Transgene
  402. Tufts Financial Group (TFG)
  403. Turenne Santé
  404. TVM Capital 
  405. Two Blades Foundation
  406. UB Securities 
  407. UCL Technology Fund
  408. UK Innovation & Science Seed Fund (UKI2S, previously known as The Rainbow Seed Fund)
  409. uniQure 
  410. University of California
  411. University of Florida
  412. University of Minnesota 
  413. University of Pennsylvania
  414. University of Texas
  415. University of Washington
  416. Valneva
  417. Vascular Biologics (also known as VBL Therapeutics)  
  418. VCN Biosciences
  419. Vectalys
  420. Vector Biolabs
  421. Vecura (Karolinska University Hospital)
  422. Venrock 
  423. Versant Ventures 
  424. Vertex Pharmaceuticals
  425. VESSL    (previously known as MultiGene Vascular Systems  )
  426. Vibalogics
  427. Vical
  428. Vigene Biosciences
  429. Viking Global Investors
  430. Virginia Biosciences Health Research Corporation (VBHRC)
  431. ViroMed 
  432. Virovek
  433. Virttu Biologics (acquired by Sorrento Therapeutics)
  434. VIVEbiotech
  435. Vivet Therapeutics
  436. Vivo Capital
  437. Voyager Therapeutics
  438. Waisman Biomanufacturing
  439. Wellcome Trust
  440. Wellington Management Company
  441. Wellstat Ophthalmics 
  442. Whitesun Healthcare Ventures 
  443. WI Harper Group
  444. Woodford Investment Management
  445. WuXi AppTec
  446. XyloCor   Therapeutics
  447. Ysios Capital
  448. Yuhan 
  449. ZIOPHARM Oncology
     

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