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遺伝子治療薬市場:第2版

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

発行 ROOTS ANALYSIS 商品コード 324981
出版日 ページ情報 英文 450 Pages
納期: 即日から翌営業日
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本日の銀行送金レート: 1USD=110.58円で換算しております。
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遺伝子治療薬市場:第2版 Gene Therapy Market (2nd Edition), 2018-2030
出版日: 2018年02月20日 ページ情報: 英文 450 Pages
概要

当レポートでは、遺伝子治療薬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
  • 遺伝子治療薬の歴史的な発展経緯
  • 遺伝子治療薬の分類
  • 投与経路
  • 遺伝子治療薬の作用機序
  • 遺伝子治療薬のメリット・デメリット
  • 遺遺伝子治療薬の倫理的・社会的懸念事項
  • 将来の抑制因子・課題
  • 規制ガイドライン

第4章 ウィルス・非ウィルスベクター

  • 本章の概要
  • ウィルスベクター
  • ウィルスベクターの種類
  • 非ウィルスベクター
  • ウィルスベクター・非ウィルスベクターのアプリケーション

第5章 競合情勢

  • 本章の概要
  • 遺伝子治療薬市場:臨床パイプライン
  • 遺伝子治療薬市場:前臨床/創薬パイプライン
  • 遺伝子治療薬市場:主要企業
  • 遺伝子治療薬市場:有望な拠点
  • 遺伝子治療薬市場:地域情勢

第6章 市販の遺伝子治療薬

  • 本章の概要
  • Gendicine (Shenzhen SiBiono GeneTech)
  • Oncorine (Shanghai Sunway Biotech)
  • Rexin-G (Epeius Biotechnologies)
  • Neovasculgen (Human Stem Cell Institute)
  • Strimvelis (GSK)
  • Imlygic (Amgen)
  • Invossa (TissueGene)
  • Luxturna (Spark Therapeutics)

第7章 後期段階 (フェーズII/III・それ以降) の遺伝子治療薬

  • 本章の概要
  • ASP0113
  • Axalimogene Filolisbac
  • AVXS-101
  • Beperminogene Perplasmid (Collategene)
  • Donaperminogene Seltoplasmid (VM202)
  • E10A
  • GS-010
  • GSK2696274
  • GSK269627
  • ImmunoPulse
  • Instiladrin
  • Lenti-D
  • LentiGlobin
  • Ofranergene Obadenovec (VB-111)
  • OTL-101
  • Pexastimogene Devacirepvec (Pexa-Vec)
  • ProstAtak
  • Valoctocogene roxaparvovec (BMN 270)
  • Vigil
  • VGX-3100
  • Vocimagene Amiretrorepvec (Toca-511)

第8章 新興技術

  • 本章の概要
  • ゲノム編集技術
  • 新興技術プラットフォーム
  • 遺伝子発現調整技術
  • 開発向け技術プラットフォーム/遺伝子治療薬の提供

第9章 治療領域

  • 本章の概要
  • 心血管疾患
  • 血液疾患
  • 炎症・感染症
  • 代謝疾患
  • 筋疾患
  • 神経疾患
  • 眼疾患

第10章 資金・投資分析

  • 本章の概要
  • 資金の種類
  • 遺伝子治療薬市場:資金・投資分析

第11章 コスト分析

  • 本章の概要
  • 価格の一因となる要因
  • 価格決定モデル

第12章 特許分析

  • 本章の概要
  • 調査範囲・調査手法
  • 遺伝子治療薬特許:出願年度別
  • 遺伝子治療薬特許:特許事務所の地域別
  • 遺伝子治療薬特許:CPC分類別
  • 遺伝子治療薬特許:新興地域
  • 遺伝子治療薬特許:主要地域
  • 遺伝子治療薬特許:競合ベンチマーキング
  • 遺伝子治療薬特許:評価分析
  • 遺伝子治療薬特許:主な引用

第13章 市場規模・機会分析

  • 本章の概要
  • 予測手法
  • 全体的な遺伝子治療薬市場
  • 遺伝子治療薬市場:製品別の売上予測

第14章 ウィルスベクターの製造

  • 本章の概要
  • ウィルスベクターの製造
  • ウィルスベクターの製造工程
  • 血清含有 vs. 無血清培地 (SFM)
  • ウィルスベクターの
  • ベクター製造関連の課題
  • ウィルスベクター・プラスミドベクター向け受託サービスを提供する企業
  • 提携
  • 近年の発展

第15章 結論

第16章 インタビュー記録

第17章 付録1:データ表

第18章 付録2:企業リスト

目次
Product Code: RA100100

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.

Synopsis:

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.

Example Highlights:

  • 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.

Research Methodology:

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 12 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 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. ASP0113
      • 13.4.9.1. Target Patient Population
      • 13.4.9.2. Sales Forecast
    • 13.4.10. Axalimogene Filolisbac
      • 13.4.10.1. Target Patient Population
      • 13.4.10.2. Sales Forecast
    • 13.4.11. AVXS-101
      • 13.4.11.1. Target Patient Population
      • 13.4.11.2. Sales Forecast
    • 13.4.12. Beperminogene Perplasmid (Collategene)
      • 13.4.12.1. Target Patient Population
      • 13.4.12.2. Sales Forecast
    • 13.4.13. Donaperminogene Seltoplasmid (VM202)
      • 13.4.13.1. Target Patient Population
      • 13.4.13.2. Sales Forecast
    • 13.4.14. E10A
      • 13.4.14.1. Target Patient Population
      • 13.4.14.2. Sales Forecast
    • 13.4.15. GS-010
      • 13.4.15.1. Target Patient Population
      • 13.4.15.2. Sales Forecast
    • 13.4.16. GSK2696274
      • 13.4.16.1. Target Patient Population
      • 13.4.16.2. Sales Forecast
    • 13.4.17. GSK2696275
      • 13.4.17.1. Target Patient Population
      • 13.4.17.2. Sales Forecast
    • 13.4.18. ImmunoPulse®
      • 13.4.18.1. Target Patient Population
      • 13.4.18.2. Sales Forecast
    • 13.4.19. Instiladrin®
      • 13.4.19.1. Target Patient Population
      • 13.4.19.2. Sales Forecast
    • 13.4.20. Lenti-D™
      • 13.4.20.1. Target Patient Population
      • 13.4.20.2. Sales Forecast
    • 13.4.21. LentiGlobin® BB305
      • 13.4.21.1. Target Patient Population
      • 13.4.21.2. Sales Forecast
    • 13.4.22. Ofranergene Obadenovec (VB-111)
      • 13.4.22.1. Target Patient Population
      • 13.4.22.2. Sales Forecast
    • 13.4.23. OTL-101
      • 13.4.23.1. Target Patient Population
      • 13.4.23.2. Sales Forecast
    • 13.4.24. Pexastimogene Devacirepvec (Pexa-Vec)
      • 13.4.24.1. Target Patient Population
      • 13.4.24.2. Sales Forecast
    • 13.4.25. ProstAtak®
      • 13.4.25.1. Target Patient Population
      • 13.4.25.2. Sales Forecast
    • 13.4.26. Valoctocogene roxaparvovec (BMN 270)
      • 13.4.26.1. Target Patient Population
      • 13.4.26.2. Sales Forecast
    • 13.4.27. Vigil®
      • 13.4.27.1. Target Patient Population
      • 13.4.27.2. Sales Forecast
    • 13.4.28. VGX-3100
      • 13.4.28.1. Target Patient Population
      • 13.4.28.2. Sales Forecast
    • 13.4.29. Vocimagene Amiretrorepvec (Toca-511)
      • 13.4.29.1. Target Patient Population
      • 13.4.29.2. 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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