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市场调查报告书

基因治疗药市场:第2版

Gene Therapy Market (3rd Edition), 2019-2030

出版商 ROOTS ANALYSIS 商品编码 324981
出版日期 内容资讯 英文 550 Pages
商品交期: 最快1-2个工作天内
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基因治疗药市场:第2版 Gene Therapy Market (3rd Edition), 2019-2030
出版日期: 2019年08月31日内容资讯: 英文 550 Pages
简介

本报告以基因治疗药市场为研究主题,提供主要企业,市售的基因治疗产品,临床/前临床研究中的产品,基因治疗相关的伦理性问题,未来发展预定及相关利益者的机会等分析,市场短期、中期的未来展望,为您概述为以下内容。

第1章 序论

第2章 摘要整理

第3章 简介

  • 情况、背景
  • 基因治疗药历史性的发展过程
  • 基因治疗药的分类
  • 给药途径
  • 基因治疗药的作用机制
  • 基因治疗药的优点及缺点
  • 遗基因治疗药的伦理的、社会顾虑事项
  • 未来的抑制因素、课题
  • 法规指南

第4章 病毒、非病毒媒介

  • 本章概要
  • 病毒媒介
  • 病毒媒介的种类
  • 非病毒媒介
  • 病毒媒介、非病毒媒介的应用

第5章 竞争情形

  • 本章概要
  • 基因治疗药市场:临床实验平台
  • 基因治疗药市场:前临床/药物研发开发平台
  • 基因治疗药市场:主要企业
  • 基因治疗药市场:有前途的据点
  • 基因治疗药市场:地区形势

第6章 市售的基因治疗药

  • 本章概要
  • 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 后期阶段 (阶段II/III、之后)的基因治疗药

  • 本章概要
  • 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 新兴技术

  • 本章概要
  • 基因编辑技术
  • 新兴技术平台
  • 基因表现调整技术
  • 开发用技术平台/基因治疗药的提供

第9章 治疗领域

  • 本章概要
  • 心血管疾病
  • 血液疾病
  • 发炎、感染疾病
  • 代谢疾病
  • 筋疾病
  • 神经疾病
  • 眼科疾病
  • 癌症

第10章 资金、投资分析

  • 本章概要
  • 资金的种类
  • 基因治疗药市场:资金、投资分析

第11章 成本分析

  • 本章概要
  • 成为价格的原因之一的要素
  • 价格决策模式

第12章 专利分析

  • 本章概要
  • 调查范围、调查手法
  • 基因治疗药专利:各申请年度
  • 基因治疗药专利:专利事务所的各地区
  • 基因治疗药专利:各CPC分类
  • 基因治疗药专利:新兴地区
  • 基因治疗药专利:主要地区
  • 基因治疗药专利:竞争基准
  • 基因治疗药专利:评估分析
  • 基因治疗药专利:主要的引用

第13章 市场规模、机会分析

  • 本章概要
  • 预测手法
  • 整体基因治疗药市场
  • 基因治疗药市场:各产品的销售额预测

第14章 病毒媒介的制造

  • 本章概要
  • 病毒媒介的制造
  • 病毒媒介的制造工程
  • 血清含有 vs. 无血清培养基 (SFM)
  • 病毒媒介
  • 媒介制造相关的课题
  • 提供病毒媒介、质体媒介的受托服务的企业
  • 联盟
  • 近几年的发展

第15章 结论

第16章 采访记录

第17章 附录1:资料表

第18章 附录2:企业清单

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目录

Since the approval of the first therapy, Genedicine® in 2003, the gene therapy domain has evolved significantly. Specifically, in 2019, three gene therapies, namely Zolgensma® (US), Zynteglo™ (Europe) and Beperminogene Perplasmid (Japan), have received approval / conditional approval, leading to a marked upward surge in the interest in this field. In fact, the growing popularity can be correlated to the substantial increase (more than 90%) in the number of patents that have been filed / granted in the last three years. Moreover, in the same time period, more than USD 12.5 billion in capital has been invested by various private and public investors to fund research activities. Presently, there are more than 10 approved gene therapies in the market, while many others are being investigated across various phases of clinical research.

Over time, the efforts of industry stakeholders and clinical researchers have led to the discovery of novel molecular targets, thereby, strengthening the research pipelines of companies involved in the development of gene therapies. Further, several technology developers have designed innovative ways to improve the efficacy and safety of gene therapies and introduced advanced therapy development and vector engineering platforms. It is also worth mentioning that, in the last 4-5 years, there has been a marked rise in the M&A activity in this domain, including the involvement of several big pharma players as well. The capability of such therapies to target diverse disease indications is considered to be amongst the most prominent growth drivers of this market. Backed by promising clinical results and several therapy candidates in late phases of development, we believe that the overall market is expected to witness tremendous growth in the coming decade.

"Although the price of such therapies is high, it is estimated that the number of patients who are likely to opt for such a treatment option is also on the rise. Moreover, as these are one-time treatments offering a cure for blindness, I believe that the high price is both reasonable and justified"

-Ryo Kubota, Chairman, President and Chief Executive Officer, Acucela, A Kubota Pharmaceutical Company

Scope of the Report

The "Gene Therapy Market (3rd Edition), 2019-2030" report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies and genome editing therapies. The study also features an elaborate discussion on the future potential of this evolving market. Amongst other elements, the report features:

  • A detailed review of the overall landscape of gene therapies and genome editing therapies, including information on various drug / therapy developer companies, phase of development (marketed, clinical, and preclinical / discovery stage) of pipeline candidates, key therapeutic areas (cardiovascular disorders, muscular disorders, neurological disorders, ocular disorders, oncology and others) and target disease indication(s), information on gene type, type of vector used, type of therapy (ex vivo and in vivo), mechanism of action, type of gene modification (gene augmentation, oncolytic viral therapy and others) and special drug designation (if any).
  • A discussion on the various types of viral and non-viral vectors, along with 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 engaged in developing gene therapies, and a bull's eye analysis, highlighting the distribution of clinical-stage pipeline candidates by phase of development, type of vector and type of therapy (ex vivo and in vivo).
  • A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.
  • Detailed profiles of marketed and phase II/III and gene therapies, including a brief history of development, information on current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, along with information on the developer company.
  • An elaborate discussion on the various commercialization strategies that can be adopted by drug developers for use across different stages of therapy development, namely prior to drug launch, at / during drug launch and post-marketing.
  • A review of various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates.
  • An in-depth analysis of the various patents that have been filed / granted related to gene therapies and genome editing therapies, since 2016. The analysis also highlights the key parameters associated with the patents, including information on patent type (granted patents, patent applications and others), publication year, regional applicability, CPC classification, emerging focus areas, leading industry / non-industry players (in terms of the number of patents filed / granted), and patent valuation.
  • A analysis of the various mergers and acquisitions that have taken place in this domain, highlighting the trend in the number of companies acquired between 2014-2019. The analysis also provides information on the key value drivers and deal multiples related to the mergers and acquisitions that we came across.
  • An analysis of the investments made at various stages of development in companies that are focused in this area, between 2014-2019, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other offerings.
  • An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio.
  • A case study on the prevalent and emerging trends related to vector manufacturing, with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.
  • A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.
  • An analysis of the various factors that are likely to influence the pricing of gene-based therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.

One of the key objectives of the report was to estimate the existing market size and the future opportunity for gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2019-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] key therapeutic areas (cardiovascular disorders, muscular disorders, neurological disorders, ocular disorders, oncology and others), [B] various types of vectors used for therapy development (adeno associated virus, adenovirus, lentivirus, plasmid DNA, retrovirus and others), [C] type of therapy (ex vivo and in vivo), [D] type of gene modification (gene augmentation, oncolytic viral therapy and others) and [E] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry's growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals (in alphabetical order):

  • Adam Rogers (CEO, Hemera Biosciences)
  • Al Hawkins (CEO, Milo Biotechnology)
  • Buel Dan Rodgers (Founder & CEO, AAVogen)
  • 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)
  • Michael Triplett (CEO, Myonexus Therapeutics)
  • Robert Jan Lamers (CEO, Arthrogen)
  • Ryo Kubota (Chairman, President and Chief Executive Officer, Acucela)
  • Tom Wilton (Chief Business Officer, LogicBio 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.

Chapter Outline

Chapter 2 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas and the route of administration of such therapeutic interventions. In addition, it includes information on the various steps involved in the transfer of therapeutic gene(s) into the body, along with a discussion on the advantages and disadvantages of this treatment paradigm. Further, the chapter features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

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 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, China, Japan, South Korea and Australia. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 450 gene therapies and genome editing therapies that are currently approved or are in different stages of development. It features a comprehensive analysis of pipeline molecules, highlighting the drug developers, key therapeutic areas and target disease indication(s), phases of development, type of vector used, target gene type, type of therapy (gene augmentation / oncolytic viral therapy / others), type of somatic cell therapy (in vivo / ex vivo), mechanism of action and drug designation (if any). 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 presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.

Chapter 7 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 information related to manufacturing.

Chapter 8 features an elaborate discussion on the various strategies that can be adopted by the therapy developers across key commercialization stages, namely prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis on the key strategies adopted by drug developers for the commercialization of their gene therapies that were approved post 2014.

Chapter 9 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 10 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. In addition, it features brief profiles of some of the key technologies. Each profile contains details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 11 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 12 provides insights from a detailed patent analysis, presenting an overview on the filed / granted patents related to gene therapies and genome editing therapies since 2016. For this analysis, we looked at the patents that have been published by various players till May 2019. It also highlights the important information and trends associated with these patents, including patent type (granted patents, patent applications and others), patent publication year, regional distribution, CPC classification, emerging focus areas and the leading industry / academic players (in terms of the number of patents filed / granted). The chapter also includes a patent benchmarking analysis and a detailed valuation analysis.

Chapter 13 features a detailed analysis of the various mergers and acquisitions that have taken place in this domain, highlighting the trend in the number of companies acquired between 2014-2019, based on parameters such as key value drivers, year of acquisition, type of acquisition, geographical location of the acquirer and the acquired company, and financial details of the deal (if available). In addition, the chapter presents a schematic world map representation of the geographical distribution of this activity, highlighting intracontinental and intercontinental deals.

Chapter 14 presents details on various funding instances, investments and grants that have been made within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2014 and 2019, highlighting the growing interest of the venture capital community and other strategic investors in this domain.

Chapter 15 highlights our views 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 of its proprietary products.

Chapter 16 highlights top ten big biopharma players in the field of gene therapy. It features tabulated profiles of the companies and each profile includes a brief overview of the company, its financial information (if available), information on its product portfolio and recent developments.

Chapter 17 presents a comprehensive forecast analysis, highlighting the future potential of the market till the year 2030. It also 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, muscular disorders, neurological disorders, ocular disorders, oncology and others), type of vector (adeno associated virus, adenovirus, lentivirus, plasmid DNA, retrovirus and others), type of somatic cell therapy (ex vivo and in vivo), type of gene modification (gene augmentation, oncolytic viral therapy and others) and geography (the US, EU5, RoW (Australia, China, Israel Japan and South Korea)).

Chapter 18 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain, and highlights some of the recent collaborations and developments related to manufacturing processes of gene therapies. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 19 provides a general overview on the supply chain of gene therapies. It features the process and steps followed in the supply chain to deliver gene therapies to target patients for the treatment of various rare disorders.

Chapter 20 summarizes the entire report. It presents a list of key takeaways and offers our independent opinion on the current market scenario. Further, it captures the evolutionary trends that are likely to determine the future of this segment of the gene therapies industry.

Chapter 21 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), Buel Dan Rodgers (Founder & CEO, AAVogen), 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), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (Chairman, President and Chief Executive Officer, Acucela) and Tom Wilton (Chief Business Officer, LogicBio Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 22 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 23 is an appendix, which contains a list of companies and organizations mentioned in this 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 Therapies
  • 3.3. Classification of Gene Therapies
    • 3.3.1. Somatic and Germline Gene Therapy
    • 3.3.2. Ex Vivo and In Vivo Gene Therapy
  • 3.4. Routes of Administration
  • 3.5. Mechanism of Action of Gene Therapies
  • 3.6. Concept of Gene Editing
  • 3.7. Advantages and Disadvantages of Gene Therapies
  • 3.8. Ethical and Social Concerns Related to Gene Therapies
  • 3.9. Future Constraints and Challenges Related to Gene Therapies
    • 3.9.1. Concerns Related to Manufacturing
    • 3.9.2. Concerns Related to Commercial Viability

4 GENE DELIVERY VECTORS

  • 4.1. Chapter Overview
  • 4.2. Viral Vectors
    • 4.2.1 Types of Viral Vectors
      • 4.2.1.1. Adeno-associated Viral Vectors
        • 4.2.1.1.1. Overview
        • 4.2.1.1.2. Design
        • 4.2.1.1.3. Advantages
        • 4.2.1.1.4. Limitations
      • 4.2.1.2. Adenoviral Vectors
        • 4.2.1.2.1. Overview
        • 4.2.1.2.2. Design
        • 4.2.1.2.3. Advantages
        • 4.2.1.2.4. Limitations
      • 4.2.1.3. Lentiviral Vectors
        • 4.2.1.3.1. Overview
        • 4.2.1.3.2. Design
        • 4.2.1.3.3. Advantages
        • 4.2.1.3.4. Limitations
      • 4.2.1.4. Retroviral Vectors
        • 4.2.1.4.1. Overview
        • 4.2.1.4.2. Design
        • 4.2.1.4.3. Advantages
        • 4.2.1.4.4. Limitations
      • 4.2.1.5. Other Viral Vectors
        • 4.2.1.5.1. Alphavirus
        • 4.2.1.5.2. Herpes Simplex Virus
        • 4.2.1.5.3. Simian Virus
        • 4.2.1.5.4. Vaccinia Virus
  • 4.3. Non-Viral Vectors
    • 4.3.1. Types of Non-Viral Vectors
      • 4.3.1.1. Plasmid DNA
      • 4.3.1.2. Liposomes, Lipoplexes and Polyplexes
      • 4.3.1.3. Oligonucleotides
  • 4.4. Methods of Transfection
    • 4.4.1. Biolistic Method
    • 4.4.2. Electroporation
    • 4.4.3. Receptor Mediated Gene Delivery
    • 4.4.4. Gene Activated Matrix (GAM)

5 REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

  • 5.1. Chapter Overview
  • 5.2. Regulatory Guidelines in North America
    • 5.2.1. The US Scenario
    • 5.2.2. The Canadian Scenario
  • 5.3. Regulatory Guidelines in Europe
  • 5.4. Regulatory Guidelines in Asia Pacific
    • 5.4.1. Chinese Scenario
    • 5.4.2. Japanese Scenario
    • 5.4.3. South Korean Scenario
    • 5.4.4. Australian Scenario
  • 5.5. Reimbursement Scenario
    • 5.5.1. Challenges Related to Reimbursement
  • 5.6. Payment Models for Gene Therapies

6 COMPETITIVE LANDSCAPE

  • 6.1. Chapter Overview
  • 6.2. Gene Therapy Market: Clinical Pipeline
    • 6.2.1. Analysis by Phase of Development
    • 6.2.2. Analysis by Therapeutic Area
    • 6.2.3. Analysis by Type of Vector Used
    • 6.2.4. Analysis by Type of Gene
    • 6.2.5. Analysis by Type of Modification
    • 6.2.6. Analysis by Type of Gene Therapy
    • 6.2.7. Analysis by Route of Administration
  • 6.3. Gene Therapy Market: Early Stage Pipeline
    • 6.3.1. Analysis by Stage of Development
    • 6.3.2. Analysis by Therapeutic Area
    • 6.3.3. Analysis by Type of Vector Used
    • 6.3.4. Analysis by Type of Gene
    • 6.3.5. Analysis by Type of Modification
    • 6.3.6. Analysis by Type of Gene Therapy
  • 6.4. Key Players
  • 6.5. Developer Landscape
  • 6.6. Regional Landscape

7 MARKETED GENE THERAPIES

  • 7.1. Chapter Overview
  • 7.2. Gendicine® (Shenzhen Sibiono GeneTech)
    • 7.2.1. Company Overview
    • 7.2.2. Development Timeline
    • 7.2.3. Mechanism of Action and Vectors Used
    • 7.2.4. Target Indication(s)
    • 7.2.5. Current Status of Development
    • 7.2.6. Manufacturing, Dosage and Sales
  • 7.3. Oncorine® (Shanghai Sunway Biotech)
    • 7.3.1. Company Overview
    • 7.3.2. Development Timeline
    • 7.3.3. Mechanism of Action and Vectors Used
    • 7.3.4. Target Indication(s)
    • 7.3.5. Current Status of Development
    • 7.3.6. Dosage and Sales
  • 7.4. Rexin-G® (Epeius Biotechnologies)
    • 7.4.1. Company Overview
    • 7.4.2. Development Timeline
    • 7.4.3. Mechanism of Action and Vector Used
    • 7.4.4. Target Indication(s)
    • 7.4.5. Current Status of Development
    • 7.4.6. Manufacturing, Dosage and Sales
  • 7.5. Neovasculgen® (Human Stem Cells Institute)
    • 7.5.1. Company Overview
    • 7.5.2. Development Timeline
    • 7.5.3. Mechanism of Action and Vectors Used
    • 7.5.4. Target Indication(s)
    • 7.5.5. Current Status of Development
    • 7.5.6. Manufacturing, Dosage and Sales
  • 7.6. Imlygic® (Amgen)
    • 7.6.1. Company Overview
    • 7.6.2. Development Timeline
    • 7.6.3. Mechanism of Action and Vectors Used
    • 7.6.4. Target Indication(s)
    • 7.6.5. Current Status of Development
    • 7.6.6. Manufacturing, Dosage and Sales
  • 7.7. Strimvelis® (Orchard Therapeutics)
    • 7.7.1. Company Overview
    • 7.7.2. Development Timeline
    • 7.7.3. Mechanism of Action and Vectors Used
    • 7.7.4. Target Indication(s)
    • 7.7.5. Current Status of Development
    • 7.7.6. Manufacturing, Dosage and Sales
  • 7.8. Invossa™ (TissueGene)
    • 7.8.1. Company Overview
    • 7.8.2. Development Timeline
    • 7.8.3. Mechanism of Action and Vectors Used
    • 7.8.4. Target Indication(s)
    • 7.8.5. Current Status of Development
    • 7.8.6. Manufacturing, Dosage and Sales
  • 7.9. Luxturna™ (Spark Therapeutics)
    • 7.9.1. Company Overview
    • 7.9.2. Development Timeline
    • 7.9.3. Mechanism of Action and Vector Used
    • 7.9.4. Target Indication(s)
    • 7.9.5. Current Status of Development
    • 7.9.6. Manufacturing, Dosage and Sales
  • 7.10. Zolgensma™ (AveXis / Novartis)
    • 7.10.1. Company Overview
    • 7.10.2. Development Timeline
    • 7.10.3. Mechanism of Action and Vector Used
    • 7.10.4. Target Indication(s)
    • 7.10.5. Current Status of Development
    • 7.10.6. Manufacturing, Dosage and Sales
  • 7.11. Collategene® / Beperminogene Perplasmid (AnGes)
    • 7.11.1. Company Overview
    • 7.11.2. Development Timeline
    • 7.11.3. Mechanism of Action and Vector Used
    • 7.11.4. Target Indication(s)
    • 7.11.5. Current Status of Development
    • 7.11.6. Manufacturing, Dosage and Sales
  • 7.12. Zyntelgo™ (bluebird bio)
    • 7.12.1. Company Overview
    • 7.12.2. Development Timeline
    • 7.12.3. Mechanism of Action and Vector Used
    • 7.12.4. Target Indication(s)
    • 7.12.5. Current Status of Development
    • 7.12.6. Manufacturing, Dosage and Sales

8 KEY COMMERCIALIZATION STRATEGIES

  • 8.1. Chapter Overview
  • 8.2. Successful Drug Launch Strategy: ROOTS Framework
  • 8.3. Successful Drug Launch Strategy: Product Differentiation
  • 8.4. Commonly Adopted Commercialization Strategies based on Development Stage of the Product
  • 8.5. Approved Gene Therapies
  • 8.6. Key Commercialization Strategies Adopted by Companies Focused on Gene Therapy
    • 8.6.1. Strategies Adopted Before Therapy Approval
    • 8.6.2. Strategies Adopted During / Post Therapy Approval
  • 8.7. Concluding Remarks

9 LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES

  • 9.1. Chapter Overview
  • 9.2. Axalimogene Filolisbac: Overview of Therapy, Current Development Status and Clinical Results
  • 9.3. AMT-061: Overview of Therapy, Current Development Status and Clinical Results
  • 9.4. BIIB111: Overview of Therapy, Current Development Status and Clinical Results
  • 9.5. BIIB112: Overview of Therapy, Current Development Status and Clinical Results
  • 9.6. Donaperminogene Seltoplasmid (VM202): Overview of Therapy, Current Development Status and Clinical Results
  • 9.7. E10A: Overview of Therapy, Current Development Status and Clinical Results
  • 9.8. Fidanacogene Elaparvovec (PF-06838435): Overview of Therapy, Current Development Status and Clinical Results
  • 9.9. FLT180a: Overview of Therapy, Current Development Status and Clinical Results
  • 9.10. GS010: Overview of Therapy, Current Development Status and Clinical Results
  • 9.11. Instiladrin®: Overview of Therapy, Current Development Status and Clinical Results
  • 9.12. Lenti-D™: Overview of Therapy, Current Development Status and Clinical Results
  • 9.13. LYS-SAF302: Overview of Therapy, Current Development Status and Clinical Results
  • 9.14. Ofranergene Obadenovec (VB-111): Overview of Therapy, Current Development Status and Clinical Results
  • 9.15. OTL-101: Overview of Therapy, Current Development Status and Clinical Results
  • 9.16. OTL-103: Overview of Therapy, Current Development Status and Clinical Results
  • 9.17. OTL-200: Overview of Therapy, Current Development Status and Clinical Results
  • 9.18. Pexastimogene Devacirepvec (PEXA-VEC): Overview of Therapy, Current Development Status and Clinical Results
  • 9.19. ProstAtak®: Overview of Therapy, Current Development Status and Clinical Results
  • 9.20. SPK-8011: Overview of Therapy, Current Development Status and Clinical Results
  • 9.21. Unnamed Therapy: Overview of Therapy, Current Development Status and Clinical Results
  • 9.22. Valoctocogene Roxaparvovec (BMN 270): Overview of Therapy, Current Development Status and Clinical Results
  • 9.23. Vigil®: Overview of Therapy, Current Development Status and Clinical Results
  • 9.24. VGX-3100: Overview of Therapy, Current Development Status and Clinical Results
  • 9.25. Vocimagene Amiretrorepvec (Toca-511): Overview of Therapy, Current Development Status and Clinical Results

10 EMERGING TECHNOLOGIES

  • 10.1. Chapter Overview
  • 10.2. Gene Editing Technologies
    • 10.2.1. Overview
    • 10.2.2. Applications
  • 10.3. Emerging Gene Editing Platforms
    • 10.3.1. CRISPR / Cas9 System
    • 10.3.2. TALENs
    • 10.3.3. megaTAL
    • 10.3.4. Zinc Finger Nuclease
  • 10.4. Gene Expression Regulation Technologies
  • 10.5. Technology Platforms for Developing / Delivering Gene Therapies

11 PROMISING THERAPEUTICS AREAS

  • 11.1. Chapter Overview
  • 11.2 Analysis by Special Designations Awarded
  • 11.3. Cardiovascular Disorders
    • 11.3.1. Analysis by Target Indication
    • 11.3.2. Analysis by Type of Vector Used
  • 11.4. Hematological Disorders
    • 11.4.1. Analysis by Target Indication
    • 11.4.2. Analysis by Type of Vector Used
  • 11.5. Inflammatory & Infectious (I&I) Diseases
    • 11.5.1. Analysis by Target Indication
    • 11.5.2. Analysis by Type of Vector Used
  • 11.6. Metabolic Disorders
    • 11.6.1. Analysis by Target Indication
    • 11.6.2. Analysis by Type of Vector Used
  • 11.7. Muscular Disorders
    • 11.7.1. Analysis by Target Indication
    • 11.7.2. Analysis by Type of Vector Used
  • 11.8. Neurological Disorders
    • 11.8.1. Analysis by Target Indication
    • 11.8.2. Analysis by Type of Vector Used
  • 11.9. Ophthalmic Disorders
    • 11.9.1. Analysis by Target Indication
    • 11.9.2. Analysis by Type of Vector Used
  • 11.10. Oncology
    • 11.10.1. Analysis by Target Indication
    • 11.10.2. Analysis by Type of Vector Used

12 PATENT ANALYSIS

  • 12.1. Chapter Overview
  • 12.2. Gene Therapy-related Patents
    • 12.2.1. Scope and Methodology
      • 12.2.1.1. Analysis by Publication Year
      • 12.2.1.2. Analysis by Geographical Location
      • 12.2.1.3. Analysis by CPC Classification
      • 12.2.1.4. Emerging Focus Areas
      • 12.2.1.5. Leading Players: Analysis by Number of Patents
      • 12.2.1.6. Patent Benchmark Analysis
      • 12.2.1.7. Patent Valuation Analysis
  • 12.2. Gene Editing-related Patents
    • 12.2.1. Scope and Methodology
      • 12.2.1.1. Analysis by Publication Year
      • 12.2.1.2. Analysis by Geographical Location
      • 12.2.1.3. Analysis by CPC Classification
      • 12.2.1.4. Emerging Focus Areas
      • 12.2.1.5. Leading Players: Analysis by Number of Patents
      • 12.2.1.6. Patent Benchmark Analysis
      • 12.2.1.7. Patent Valuation Analysis
  • 12.3. Overall Intellectual Property Portfolio: Analysis by Type of Organization

13 MERGERS AND ACQUISITIONS

  • 13.1. Chapter Overview
  • 13.2. Merger and Acquisition Models
  • 13.3. Gene Therapy: Mergers and Acquisitions
    • 13.3.1. Analysis by Year of Mergers and Acquisitions
    • 13.3.2. Analysis by Type of Mergers and Acquisitions
    • 13.3.3. Regional Analysis
      • 13.3.3.1. Continent-wise Distribution
      • 13.3.3.2. Intercontinental and Intracontinental Deals
      • 13.3.3.3. Country-wise Distribution
    • 13.3.4. Analysis by Key Value Drivers
      • 13.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
    • 13.3.5. Analysis by Phase of Development of the Acquired Company's Product
    • 13.3.6. Analysis by Therapeutic Area

14 FUNDING AND INVESTMENT ANALYSIS

  • 14.1. Chapter Overview
  • 14.2. Types of Funding
  • 14.3. Funding and Investment Analysis
    • 14.3.1. Analysis by Number of Funding Instances
    • 14.3.2. Analysis by Amount Invested
    • 14.3.3. Analysis by Type of Funding
    • 14.3.4. Analysis by Amount Invested across Different Types of Therapies
    • 14.3.5. Regional Analysis by Amount Invested
    • 14.3.6. Most Active Players
    • 14.3.7. Key Investors
    • 14.3.8. Analysis by Stage of Development
  • 14.4. Concluding Remarks

15 COST PRICE ANALYSIS

  • 13.1. Chapter Overview
  • 13.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
  • 13.3. Gene Therapy Market: Pricing Models
    • 13.3.1. On the Basis of Associated Product / Component Costs
    • 13.3.2. On the Basis of Competition
    • 13.3.3. On the Basis of Patient Segment
    • 13.3.4. On the Basis of Opinions of Industry Experts

16 BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES

  • 16.1. Chapter Overview
  • 16.2. Top Pharmaceutical Companies
    • 16.2.1. Analysis by Therapeutic Area
    • 16.2.2. Analysis by Type of Vector Used
    • 16.2.3. Analysis by Type of Modification
    • 16.2.4. Analysis by Type of Gene Therapy
  • 16.3. Other Big Pharma Players

17 MARKET FORECAST AND OPPORTUNITY ANALYSIS

  • 17.1. Chapter Overview
  • 17.2. Scope and Limitations
  • 17.3. Key Assumptions and Forecast Methodology
  • 17.4. Overall Gene Therapy Market, 2019-2030
    • 17.4.1. Gene Therapy Market: Analysis by Type of Gene Modification
    • 17.4.2. Gene Therapy Market: Analysis by Type of Therapy
    • 17.4.3. Gene Therapy Market: Analysis by Type of Vector Used
    • 17.4.4. Gene Therapy Market: Analysis by Therapeutic Area
    • 17.4.5. Gene Therapy Market: Analysis by Route of Administration
    • 17.4.6. Gene Therapy Market: Analysis by Geography
  • 17.5. Gene Therapy Market: Value Creation Analysis
  • 17.6. Gene Therapy Market: Product-wise Sales Forecasts
    • 17.6.1. Gendicine®
      • 17.6.1.1. Target Patient Population
      • 17.6.1.2. Sales Forecast
      • 17.6.1.3. Net Present Value
      • 17.6.1.4. Value Creation Analysis
    • 17.6.2. Oncorine®
      • 17.6.2.1. Target Patient Population
      • 17.6.2.2. Sales Forecast
      • 17.6.2.3. Net Present Value
      • 17.6.2.4. Value Creation Analysis
    • 17.6.3. Rexin-G®
      • 17.6.3.1. Target Patient Population
      • 17.6.3.2. Sales Forecast
      • 17.6.3.3. Net Present Value
      • 17.6.3.4. Value Creation Analysis
    • 17.6.4. Neovasculgen®
      • 17.6.4.1. Target Patient Population
      • 17.6.4.2. Sales Forecast
      • 17.6.4.3. Net Present Value
      • 17.6.4.4. Value Creation Analysis
    • 17.6.5. Strimvelis®
      • 17.6.5.1. Target Patient Population
      • 17.6.5.2. Sales Forecast
      • 17.6.5.3. Net Present Value
      • 17.6.5.4. Value Creation Analysis
    • 17.6.6. Imlygic®
      • 17.6.6.1. Target Patient Population
      • 17.6.6.2. Sales Forecast
      • 17.6.6.3. Net Present Value
      • 17.6.6.4. Value Creation Analysis
    • 17.6.7. Invossa™
      • 17.6.7.1. Target Patient Population
      • 17.6.7.2. Sales Forecast
      • 17.6.7.3. Net Present Value
      • 17.6.7.4. Value Creation Analysis
    • 17.6.8. Luxturna™
      • 17.6.8.1. Target Patient Population
      • 17.6.8.2. Sales Forecast
      • 17.6.8.3. Net Present Value
      • 17.6.8.4. Value Creation Analysis
    • 17.6.9. Zolgensma™
      • 17.6.9.1. Target Patient Population
      • 17.6.9.2. Sales Forecast
      • 17.6.9.3. Net Present Value
      • 17.6.9.4. Value Creation Analysis
    • 17.6.10. Collategene® / Beperminogene Perplasmid
      • 17.6.10.1. Target Patient Population
      • 14.6.10.2. Sales Forecast
      • 17.6.10.3. Net Present Value
      • 17.6.10.4. Value Creation Analysis
    • 17.6.11. Zyntelgo™
      • 17.6.11.1. Target Patient Population
      • 17.6.11.2. Sales Forecast
      • 17.6.11.3. Net Present Value
      • 17.6.11.4. Value Creation Analysis
    • 17.6.12. Axalimogene Filolisbac
      • 17.6.12.1. Target Patient Population
      • 17.6.12.2. Sales Forecast
      • 17.6.12.3. Net Present Value
      • 17.6.12.4. Value Creation Analysis
    • 17.6.13. AMT-061
      • 17.6.13.1. Target Patient Population
      • 17.6.13.2. Sales Forecast
      • 17.6.13.3. Net Present Value
      • 17.6.13.4. Value Creation Analysis
    • 17.6.14. BIIB111
      • 17.6.14.1. Target Patient Population
      • 17.6.14.2. Sales Forecast
      • 17.6.14.3. Net Present Value
      • 17.6.14.4. Value Creation Analysis
    • 17.6.15. BIIB112
      • 17.6.15.1. Target Patient Population
      • 17.6.15.2. Sales Forecast
      • 17.6.15.3. Net Present Value
      • 17.6.15.4. Value Creation Analysis
    • 17.6.16. Donaperminogene Seltoplasmid (VM202)
      • 17.6.16.1. Target Patient Population
      • 17.6.16.2. Sales Forecast
      • 17.6.16.3. Net Present Value
      • 17.6.16.4. Value Creation Analysis
    • 17.6.17. E10A
      • 17.6.17.1. Target Patient Population
      • 17.6.17.2. Sales Forecast
      • 17.6.17.3. Net Present Value
      • 17.6.17.4. Value Creation Analysis
    • 17.6.18. Fidanacogene Elaparvovec (PF-06838435)
      • 17.6.18.1. Target Patient Population
      • 17.6.18.2. Sales Forecast
      • 17.6.18.3. Net Present Value
      • 17.6.18.4. Value Creation Analysis
    • 17.6.19. FLT180a
      • 17.6.19.1. Target Patient Population
      • 17.6.19.2. Sales Forecast
      • 17.6.19.3. Net Present Value
      • 17.6.19.4. Value Creation Analysis
    • 17.6.20. GS010
      • 17.6.20.1. Target Patient Population
      • 17.6.20.2. Sales Forecast
      • 17.6.20.3. Net Present Value
      • 17.6.20.4. Value Creation Analysis
    • 17.6.21. Instiladrin®
      • 17.6.21.1. Target Patient Population
      • 17.6.21.2. Sales Forecast
      • 17.6.21.3. Net Present Value
      • 17.6.21.4. Value Creation Analysis
    • 17.6.22. Lenti-D™
      • 17.6.22.1. Target Patient Population
      • 17.6.22.2. Sales Forecast
      • 17.6.22.3. Net Present Value
      • 17.6.22.4. Value Creation Analysis
    • 17.6.23. LYS-SAF302
      • 17.6.23.1. Target Patient Population
      • 17.6.23.2. Sales Forecast
      • 17.6.23.3. Net Present Value
      • 17.6.23.4. Value Creation Analysis
    • 17.6.24. Ofranergene Obadenovec (VB-111)
      • 17.6.24.1. Target Patient Population
      • 17.6.24.2. Sales Forecast
      • 17.6.24.3. Net Present Value
      • 17.6.24.4. Value Creation Analysis
    • 17.6.25. OTL-101
      • 17.6.25.1. Target Patient Population
      • 17.6.25.2. Sales Forecast
      • 17.6.25.3. Net Present Value
      • 17.6.25.4. Value Creation Analysis
    • 17.6.26. OTL-103
      • 17.6.26.1. Target Patient Population
      • 17.6.26.2. Sales Forecast
      • 17.6. 26.3. Net Present Value
      • 17.6.26.4. Value Creation Analysis
    • 17.6.27. OTL-200
      • 17.6.27.1. Target Patient Population
      • 17.6.27.2. Sales Forecast
      • 17.6.27.3. Net Present Value
      • 17.6.27.4. Value Creation Analysis
    • 17.6.28. Pexastimogene Devacirepvec (PEXA-VEC)
      • 17.6.28.1. Target Patient Population
      • 17.6.28.2. Sales Forecast
      • 17.6.28.3. Net Present Value
      • 17.6.28.4. Value Creation Analysis
    • 17.6.29. ProstAtak®
      • 17.6.29.1. Target Patient Population
      • 17.6.29.2. Sales Forecast
      • 17.6.29.3. Net Present Value
      • 17.6.29.4. Value Creation Analysis
    • 17.6.30. SPK-8011
      • 17.6.30.1. Target Patient Population
      • 17.6.30.2. Sales Forecast
      • 17.6.30.3. Net Present Value
      • 17.6.30.4. Value Creation Analysis
    • 17.6.31. Unnamed Therapy
      • 17.6.31.1. Target Patient Population
      • 17.6.31.2. Sales Forecast
      • 17.6.31.3. Net Present Value
      • 17.6.31.4. Value Creation Analysis
    • 17.6.32. Valoctocogene Roxaparvovec (BMN 270)
      • 17.6.32.1. Target Patient Population
      • 17.6.32.2. Sales Forecast
      • 17.6.32.3. Net Present Value
      • 17.6.32.4. Value Creation Analysis
    • 17.6.33. Vigil®
      • 17.6.33.1. Target Patient Population
      • 17.6.33.2. Sales Forecast
      • 17.6.33.3. Net Present Value
      • 17.6.33.4. Value Creation Analysis
    • 17.6.34. VGX-3100
      • 17.6.34.1. Target Patient Population
      • 17.6.34.2. Sales Forecast
      • 17.6.34.3. Net Present Value
      • 17.6.34.4. Value Creation Analysis
    • 17.6.35. Vocimagene Amiretrorepvec (Toca-511)
      • 17.6.35.1. Target Patient Population
      • 17.6.35.2. Sales Forecast
      • 17.6.35.3. Net Present Value
      • 17.6.35.4. Value Creation Analysis

18 VECTOR MANUFACTURING

  • 18.1. Chapter Overview
  • 18.2. Overview of Viral Vector Manufacturing
  • 18.3. Viral Vector Manufacturing Processes
    • 18.3.1. Mode of Vector Production
    • 18.3.2. Adherent and Suspension Cultures
    • 18.3.3. Unit Processes and Multiple Parallel Processes
    • 18.3.4. Cell Culture Systems for Production of Viral Vectors
    • 18.3.5. Culture Media Specifications
  • 18.4. Bioprocessing of Viral Vectors
    • 18.4.1. AAV Vector Production
    • 18.4.2. Adenoviral Vector Production
    • 18.4.3. Lentiviral Vector Production
    • 18.4.4. γ -Retroviral Vector Production
  • 18.5. Challenges Associated with Vector Manufacturing
  • 18.6. Companies Offering Contract Services for Viral and Plasmid Vectors

19 CASE STUDY: GENE THERAPY SUPPLY CHAIN

  • 19.1. Chapter Overview
  • 19.2. Overview of the Gene Therapy Supply Chain
  • 19.3. Implementation of Supply Chain Models
  • 19.4. Logistics in Gene Therapy
    • 19.4.1. Logistics Processes for Autologous and Allogeneic Therapies
  • 19.5. Regulatory Supply Chain across the Globe
  • 19.6. Challenges Associated with Gene Therapy Supply Chain
  • 19.7. Optimizing Cell and Advanced Therapies Supply Chain Management
  • 19.8. Recent Developments and Upcoming Trends

20 CONCLUSION

  • 20.1. Chapter Overview
  • 20.2. Key Takeaways

21 INTERVIEW TRANSCRIPTS

  • 21.1. Chapter Overview
  • 21.2. Adam Rogers, Chief Executive Officer, Hemera Biosciences
  • 21.3. Al Hawkins, Chief Executive Officer, Milo Biotechnology
  • 21.4. Buel Dan Rodgers, Founder & Chief Executive Officer, AAVogen
  • 21.5. Cedric Szpirer, Executive & Scientific Director, Delphi Genetics
  • 21.6. Christopher Reinhard, Chief Executive Officer and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)
  • 21.7. Jeffrey HunG, Chief Commercial Officer, Vigene Biosciences
  • 21.8. Marco Schmeer, Project Manager and Tatjana Buchholz, Marketing Manager, PlasmidFactory
  • 21.9. Michael Tripletti, Chief Executive Officer, Myonexus Therapeutics
  • 21.10. Robert Jan Lamers, Chief Executive Officer, Arthrogen
  • 21.11. Ryo Kubota, Chairman, President and Chief Executive Officer, Acucela
  • 21.12. Tom Wilton, Chief Business Officer, LogicBio Therapeutics

22 APPENDIX 1: TABULATED DATA

23 APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

  • Read More

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 Advantages and Disadvantages of Gene Therapies
  • Figure 4.1 Gene Transfer: Viral and Non-Viral Methods
  • Figure 5.1 Centralized Regulatory Pathway for Market Approval in the US
  • Figure 5.2 Centralized Regulatory Pathway for Market Approval in Europe
  • Figure 5.3 Manufacturing Requirements for Gene Therapy Products in China
  • Figure 5.4 Key Elements of Quality Control in China
  • Figure 5.5 Centralized Regulatory Pathway for Market Approval in Japan
  • Figure 6.1 Gene Therapies, Clinical Pipeline: Distribution by Phase of Development
  • Figure 6.2 Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Area
  • Figure 6.3 Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Area and Phase of Development
  • Figure 6.4 Gene Therapies, Clinical Pipeline: Distribution by Type of Vector Used
  • Figure 6.5 Gene Therapies, Clinical Pipeline: Distribution by Type of Gene
  • Figure 6.6 Gene Therapies, Clinical Pipeline: Distribution by Type of Modification
  • Figure 6.7 Gene Therapies, Clinical Pipeline: Distribution by Type of Modification and Type of Vector Used
  • Figure 6.8 Gene Therapies, Clinical Pipeline: Distribution by Type of Gene Therapy
  • Figure 6.9 Gene Therapies, Clinical Pipeline: Distribution by Route of Administration
  • Figure 6.10 Gene Therapies, Early Stage Pipeline: Distribution by Stage of Development
  • Figure 6.11 Gene Therapies, Early Stage Pipeline: Distribution by Therapeutic Area
  • Figure 6.12 Gene Therapies, Early Stage Pipeline: Distribution by Type of Vector Used
  • Figure 6.13 Gene Therapies, Early Stage Pipeline: Distribution by Type of Gene
  • Figure 6.14 Gene Therapies, Early Stage Pipeline: Distribution by Type of Modification
  • Figure 6.15 Gene Therapies, Early Stage Pipeline: Distribution by Type of Modification and Type of Vector Used
  • Figure 6.16 Gene Therapies, Early Stage Pipeline: Distribution by Type of Gene Therapy
  • Figure 6.17 Gene Therapies: Key Players
  • Figure 6.18 Gene Therapy Developers: Distribution by Year of Establishment
  • Figure 6.19 Gene Therapy Developers: Distribution by Size of Employee Base
  • Figure 6.20 Gene Therapies Developers: Distribution by Location of Headquarter
  • Figure 6.21 Gene Therapy Developers: North America
  • Figure 6.22 Gene Therapy Developers: Europe
  • Figure 6.23 Gene Therapy Developers: Asia Pacific
  • Figure 7.1 Gendicine®: Development Timeline
  • Figure 7.2 Oncorine®: Development Timeline
  • Figure 7.3 Oncorine®: Mechanism of Action
  • Figure 7.4 Rexin-G®: Development Timeline
  • Figure 7.5 Neovasculgen®: Development Timeline
  • Figure 7.6 Imlygic®: Development Timeline
  • Figure 7.7 Imlygic®: Mechanism of Action
  • Figure 7.8 Strimvelis®: Development Timeline
  • Figure 7.9 Invossa™: Development Timeline
  • Figure 7.10 Invossa™: Mechanism of Action
  • Figure 7.11 Luxturna™: Development Timeline
  • Figure 7.12 Zolgensma™: Development Timeline
  • Figure 7.13 Collategene®: Development Timeline
  • Figure 7.14 Zyntelgo™: Development Timeline
  • Figure 8.1 Successful Drug Launch Strategy: ROOTS Framework
  • Figure 8.2 Successful Drug Launch Strategy: Product Differentiation
  • Figure 8.3 Commonly Adopted Commercialization Strategies based on Development Stage of the Product
  • Figure 8.4 Harvey Ball Analysis: Commercialization Strategies Adopted by Companies Focused on Gene Therapies
  • Figure 8.5 Approved Gene Therapies: Historical Timeline of Geographical Expansion
  • Figure 8.6 Approved Gene Therapies: Snapshot of Promotional Activities on Product Websites
  • Figure 10.1 Gene Editing: Key Application Areas
  • Figure 10.2 Gene Editing: Emerging Technology Platforms
  • Figure 11.1 Gene Therapies: Distribution by Therapeutic Area and Special Designations Awarded
  • Figure 11.2 Gene Therapies for Cardiovascular Disorders: Distribution by Target Indication and Phase of Development
  • Figure 11.3 Gene Therapies for Cardiovascular Disorders: Distribution by Type of Vector Used
  • Figure 11.4 Gene Therapies for Hematological Disorders: Distribution by Target Indication and Phase of Development
  • Figure 11.5 Gene Therapies for Hematological Disorders: Distribution by Type of Vector Used
  • Figure 11.6 Gene Therapies for I&I Disorders: Distribution by Target Indication and Phase of Development
  • Figure 11.7 Gene Therapies for I&I Disorders: Distribution by Type of Vector Used
  • Figure 11.8 Gene Therapies for Metabolic Disorders: Distribution by Target Indication and Phase of Development
  • Figure 11.9 Gene Therapies for Metabolic Disorders: Distribution by Type of Vector Used
  • Figure 11.10 Gene Therapies for Muscular Disorders: Distribution by Target Indication and Phase of Development
  • Figure 11.11 Gene Therapies for Muscular Disorders: Distribution by Type of Vector Used
  • Figure 11.12 Gene Therapies for Neurological Disorders: Distribution by Target Indication and Phase of Development
  • Figure 11.13 Gene Therapies for Neurological Disorders: Distribution by Type of Vector Used
  • Figure 11.14 Gene Therapies for Ophthalmic Disorders: Distribution by Target Indication and Phase of Development
  • Figure 11.15 Gene Therapies for Ophthalmic Disorders: Distribution by Type of Vector Used
  • Figure 11.16 Gene Therapies for Oncology: Distribution by Target Indication and Phase of Development
  • Figure 11.17 Gene Therapies for Oncology: Distribution by Type of Vector Used
  • Figure 12.1 Gene Therapy Patent Portfolio: Distribution by Type of Patent
  • Figure 12.2 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2019
  • Figure 12.3 Gene Therapy Patent Portfolio: Distribution by Geographical Location
  • Figure 12.4 Gene Therapy Patent Portfolio: Distribution by Geographical Location, North America
  • Figure 12.5 Gene Therapy Patent Portfolio: Distribution by Geographical Location, Europe
  • Figure 12.6 Gene Therapy Patent Portfolio: Distribution by Geographical Location, Asia-Pacific
  • Figure 12.7 Gene Therapy Patent Portfolio: Distribution by CPC Classification Symbols
  • Figure 12.8 Gene Therapy Patent Portfolio: Emerging Focus Areas
  • Figure 12.9 Gene Therapy Patent Portfolio: Leading Industry Players
  • Figure 12.10 Gene Therapy Patent Portfolio: Leading Non-Industry Players
  • Figure 12.11 Gene Therapy Patent Portfolio (Genentech and GSK): Benchmarking by Patent Characteristics
  • Figure 12.12 Gene Therapy Patent Portfolio, Leading Industry Players: Benchmarking by Patent Characteristics
  • Figure 12.13 Gene Therapy Patent Portfolio: Distribution of Patents by Age (2016-2019)
  • Figure 12.14 Gene Therapy Patent Portfolio: Valuation Analysis
  • Figure 12.15 Gene Editing Patent Portfolio: Distribution by Type of Patent
  • Figure 12.16 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2019
  • Figure 12.17 Gene Editing Patent Portfolio: Distribution by Geographical Location
  • Figure 12.18 Gene Editing Patent Portfolio: Distribution by Geographical Location, North America
  • Figure 12.19 Gene Editing Patent Portfolio: Distribution by Geographical Location, Europe
  • Figure 12.20 Gene Editing Patent Portfolio: Distribution by Geographical Location, Asia-Pacific
  • Figure 12.21 Gene Editing Patent Portfolio: Distribution by CPC Classification Symbols
  • Figure 12.22 Gene Editing Patent Portfolio: Emerging Focus Areas
  • Figure 12.23 Gene Editing Patent Portfolio: Leading Industry Players
  • Figure 12.24 Gene Editing Patent Portfolio: Leading Non-Industry Players
  • Figure 12.25 Gene Editing Patent Portfolio (Sangamo Therapeutics and Cellectis): Benchmarking by Patent Characteristics
  • Figure 12.26 Gene Editing Patent Portfolio (Leading Industry Players): Benchmarking by Patent Characteristics
  • Figure 12.27 Gene Editing Patent Portfolio: Distribution of Patents by Age (2016- 2019)
  • Figure 12.28 Gene Editing Patent Portfolio: Valuation Analysis
  • Figure 12.29 Gene Therapy and Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
  • Figure 13.1 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2014- 2019
  • Figure 13.2 Mergers and Acquisitions: Distribution by Type of Mergers and Acquisitions, 2014 - 2019
  • Figure 13.3 Mergers and Acquisitions: Distribution by Year and Type of Merger and Acquisition, 2014 - 2019
  • Figure 13.4 Mergers and Acquisitions: Continent-wise Distribution
  • Figure 13.5 Mergers and Acquisitions: Intercontinental and Intracontinental Acquisitions
  • Figure 13.6 Mergers and Acquisitions: Country-wise Distribution
  • Figure 13.7 Acquisitions: Distribution by Key Value Drivers
  • Figure 13.8 Acquisitions: Distribution by Key Value Drivers and Year of Acquisition, 2014-2019
  • Figure 13.9 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product
  • Figure 13.10 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product and Year of Acquisition
  • Figure 13.11 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product and Deal Amount
  • Figure 13.12 Mergers and Acquisitions: Distribution by Therapeutic Area
  • Figure 14.1 Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2019
  • Figure 14.2 Funding and Investment Analysis: Cumulative Number of Instances by Year, 2014-2019
  • Figure 14.3 Funding and Investment Analysis: Cumulative Amount Invested, 2014-2019 (USD Million)
  • Figure 14.4 Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2019
  • Figure 14.5 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2019
  • Figure 14.6 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2019 (USD Million)
  • Figure 14.7 Funding and Investment Analysis: Summary of Investments, 2014-2019 (USD Million)
  • Figure 14.8 Funding and Investments: Distribution by Amount Invested across Different Types of Gene Modification
  • Figure 14.9 Funding and Investment Analysis: Distribution by Geography
  • Figure 14.10 Funding and Investment Analysis: Regional Distribution of Funding Instances
  • Figure 14.11 Funding and Investment Analysis: Most Active Players, on the basis of Number of Instances, 2014-2019 (USD Million)
  • Figure 14.12 Funding and Investment Analysis: Most Active Players, on the basis of Amount Invested, 2014-2019 (USD Million)
  • Figure 14.13 Funding and Investment Analysis: Distribution by Type of Investors
  • Figure 14.14 Funding and Investment Analysis: Distribution by Big Pharma Players (Investors)
  • Figure 14.15 Funding and Investment Analysis: Distribution by Government Institutes (Investors)
  • Figure 14.16 Funding and Investment Analysis: Leading Investors
  • Figure 14.17 Funding and Investment Analysis: Distribution of Investments by Stage of Development of the Company's Product
  • Figure 14.18 Funding and Investment Analysis: Distribution by Highest Phase of Development of the Company's Product
  • Figure 14.19 Funding and Investment Summary, 2014-2019 (USD Million)
  • Figure 15.1 Gene Therapy: Pricing Model Based on Patient Segment
  • Figure 16.1 Big Pharma Players: Heat Map Analysis of Top Pharmaceutical Companies
  • Figure 16.2 Big Pharma Players: Analysis by Therapeutic Area
  • Figure 16.3 Big Pharma Players: Analysis by Type of Vector Used
  • Figure 16.4 Big Pharma Players: Analysis by Type of Modification
  • Figure 16.5 Big Pharma Players: Analysis by Type of Gene Therapy
  • Figure 16.6 Big Pharma Players: Heat Map Analysis of Other Players
  • Figure 17.1 Overall Gene Therapy Market, 2018-2030: Base Scenario (USD Million)
  • Figure 17.2 Gene Therapy Market: Distribution by Type of Gene Modification, 2019, 2025 and 2030
  • Figure 17.3 Gene Therapy Market: Distribution by Type of Therapy, 2019, 2025 and 2030
  • Figure 17.4 Gene Therapy Market: Distribution by Type of Vector Used, 2019, 2025 and 2030
  • Figure 17.5 Gene Therapy Market: Distribution by Therapeutic Area, 2019, 2025 and 2030
  • Figure 17.6 Gene Therapy Market: Distribution by Route of Administration, 2019, 2025 and 2030
  • Figure 17.7 Gene Therapy Market: Distribution by Geography, 2019, 2025 and 2030
  • Figure 17.8 Gene Therapy Market: Country-wise Distribution of Rest of the World Region, 2019, 2025 and 2030
  • Figure 17.9 Gene Therapy Market: Distribution by Key Players, 2019, 2025 and 2030
  • Figure 17.10 Gendicine® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.11 Oncorine® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.12 Rexin-G® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.13 Neovasculgen® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.14 Strimvelis® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.15 Imlygic® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.16 Invossa™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.17 Luxturna™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.18 Zolgensma™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.19 Collategene® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.20 Zyntelgo™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.21 Axalimogene Filolisbac Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.22 AMT-061 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.23 BIIB111 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.24 BIIB112 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.25 Donaperminogene Seltoplasmid (VM202) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.26 E10A Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.27 Fidanacogene Elaparvovec (PF-06838435) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.28 FLT180a Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.29 GS010 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.30 Instiladrin® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.31 Lenti-D™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.32 LYS-SAF302 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.33 Ofranergene Obadenovec (VB-111) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.34 OTL-101Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.35 OTL-103 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.36 OTL-200 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.37 Pexastimogene Devacirepvec (PEXA-VEC) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.38 ProstAtak® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.39 SPK-8011 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.40 Unnamed Therapy Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.41 Valoctocogene Roxaparvovec (BMN 270) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.42 Vigil® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.43 VGX-3100 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.44 Vocimagene Amiretrorepvec (Toca-511) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 18.1 Viral Vectors: Manufacturing Steps
  • Figure 19.1 Steps Involved in the Development of Gene Therapies
  • Figure 19.2 Workflow of Cell-based Gene Therapies
  • Figure 19.3 Key Responsibilities of Stakeholders Involved in Advanced Therapies Supply Chain
  • Figure 19.4 Key Steps Involved in the Implementation of a Supply Chain Strategy
  • Figure 19.5 Keys Steps in the Logistics of Autologous Therapies
  • Figure 19.6 Comparison of Logistics Processes for Autologous and Allogeneic Therapies
  • Figure 19.7 Supply Chain Regulatory Authorities Across the Globe
  • Figure 19.8 Overview of Supply Chain Orchestration Platform
  • Figure 19.9 Functions of Key Technological Solutions for Cell and Advanced Therapies Supply Chain Management

List of Tables

  • Table 3.1 Comparison of Ex Vivo and In Vivo Techniques
  • Table 3.2 Vectors used for Targeted Gene Delivery to Tissues / Organs
  • Table 3.3 Routes of Administration of Gene Therapies: Advantages and Disadvantages
  • Table 4.1 Viral Vectors: Key Features
  • Table 5.1 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
  • Table 5.2 Payment Options Available for Gene Therapies
  • Table 6.1 Gene Therapy Market: Clinical Pipeline
  • Table 6.2 Gene Therapy Market: Early Stage Pipeline
  • Table 6.3 Gene Therapy Market: Developer Overview
  • Table 7.1 Gene Therapies: Marketed and Approved Products
  • Table 7.2 Company Overview: Shenzhen Sibiono GeneTech
  • Table 7.3 Gendicine®: Patent Portfolio
  • Table 7.4 Gendicine®: Status of Development
  • Table 7.5 Company Overview: Shanghai Sunway Biotech
  • Table 7.6 Oncorine®: Status of Development
  • Table 7.7 Company Overview: Epeius Biotechnologies
  • Table 7.8 Rexin-G®: Status of Development
  • Table 7.9 Company Overview: Human Stem Cell Institute
  • Table 7.10 Neovasculgen®: Status of Development
  • Table 7.11 Company Overview: Amgen
  • Table 7.12 Imlygic®: Status of Development
  • Table 7.13 Imlygic®: Recommended Dose and Schedule
  • Table 7.14 Imlygic®: Determination of Injection Volume based upon Lesion Size
  • Table 7.15 Company Overview: Orchard Therapeutics
  • Table 7.16 Strimvelis®: Status of Development
  • Table 7.17 Company Overview: TissueGene
  • Table 7.18 Invossa™: Status of Development
  • Table 7.19 Company Overview: Spark Therapeutics
  • Table 7.20 Luxturna™: Status of Development
  • Table 7.21 Company Overview: AveXis (A Novartis Company)
  • Table 7.22 Zolgensma™: Status of Development
  • Table 7.23 Company Overview: AnGes
  • Table 7.24 Collategene®: Status of Development
  • Table 7.25 Company Overview: bluebird bio
  • Table 7.26 Zyntelgo™: Status of Development
  • Table 8.1 Luxturna™: List of Conferences Attended Before Approval
  • Table 8.2 Zolgensma™: List of Conferences Attended Before Approval
  • Table 8.3 Luxturna™: List of Conferences Attended Post Approval
  • Table 8.4 Strimvelis™: List of Conferences Attended Post Approval
  • Table 8.5 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
  • Table 8.6 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
  • Table 9.1 Gene Therapies: Phase III Drugs
  • Table 9.2 Drug Profile: Axalimogene filolisbac
  • Table 9.3 Drug Profile: AMT-061
  • Table 9.4 Drug Profile: BIIB111
  • Table 9.5 Drug Profile: BIIB112
  • Table 9.6 Drug Profile: Donaperminogene Seltoplasmid (VM202)
  • Table 9.7 Drug Profile: E10A
  • Table 9.8 Drug Profile: Fidanacogene Elaparvovec (PF-06838435)
  • Table 9.9 Drug Profile: FLT180a
  • Table 9.10 Drug Profile: GS010
  • Table 9.11 Drug Profile: Instiladrin®
  • Table 9.12 Drug Profile: Lenti-D™
  • Table 9.13 Drug Profile: LYS-SAF302
  • Table 9.14 Drug Profile: Ofranergene Obadenovec (VB-111)
  • Table 9.15 Drug Profile: OTL-101
  • Table 9.16 Drug Profile: OTL-103
  • Table 9.17 Drug Profile: OTL-200
  • Table 9.18 Drug Profile: Pexastimogene Devacirepvec (PEXA-VEC)
  • Table 9.19 Drug Profile: ProstAtak®
  • Table 9.20 Drug Profile: SPK-8011
  • Table 9.21 Drug Profile: Unnamed Therapy
  • Table 9.22 Drug Profile: Valoctocogene Roxaparvovec (BMN 270)
  • Table 9.23 Drug Profile: Vigil®
  • Table 9.24 Drug Profile: VGX-3100
  • Table 9.25 Drug Profile: Vocimagene Amiretrorepvec (Toca-511)
  • Table 10.1 Gene Editing Technology Platforms
  • Table 10.2 CRISPR Therapeutics: Funding Instances
  • Table 10.3 CRISPR Therapeutics: Collaborations
  • Table 10.4 Editas Medicine: Funding Instances
  • Table 10.5 Editas Medicine: Collaborations
  • Table 10.6 Intellia Therapeutics: Funding Instances
  • Table 10.7 Intellia Therapeutics: Collaborations
  • Table 10.8 Cellectis: Funding Instances
  • Table 10.9 Cellectis: Collaborations
  • Table 10.10 bluebird bio: Funding Instances
  • Table 10.11 bluebird bio: Collaborations
  • Table 10.12 Sangamo Therapeutics: Funding Instances
  • Table 10.13 Sangamo Therapeutics: Collaborations
  • Table 10.14 Gene Switch Technology Platforms
  • Table 10.15 Intrexon: Funding Instances
  • Table 10.16 Intrexon: Collaborations
  • Table 10.17 MeiraGTx: Funding Instances
  • Table 10.18 MeiraGTx: Collaborations
  • Table 10.19 Gene Therapy: Technology Platforms
  • Table 11.1 Gene Therapies for Cardiovascular Disorders
  • Table 11.2 Gene Therapies for Hematological Disorders
  • Table 11.3 Gene Therapies for I&I Disorders
  • Table 11.4 Gene Therapies for Metabolic Disorders
  • Table 11.5 Gene Therapies for Muscular Disorders
  • Table 11.6 Gene Therapies for Neurological Disorders
  • Table 11.7 Gene Therapies for Ophthalmic Disorders
  • Table 11.8 Gene Therapies for Oncology
  • Table 12.1 Gene Therapy Patent Portfolio: CPC Classification Symbol Definitions
  • Table 12.2 Gene Therapy Patent Portfolio: Most Popular CPC Classification Symbols
  • Table 12.3 Gene Therapy Patent Portfolio: List of Top CPC Classifications
  • Table 12.4 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
  • Table 12.5 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
  • Table 12.6 Gene Therapy Patent Portfolio: List of Leading Patents (by Highest Relative Valuation)
  • Table 12.7 Gene Editing Patent Portfolio: CPC Classification Symbol Definitions
  • Table 12.8 Gene Editing Patent Portfolio: Most Popular CPC Classification Symbols
  • Table 12.9 Gene Editing Patent Portfolio: List of Top CPC Classifications
  • Table 12.10 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
  • Table 12.11 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
  • Table 12.12 Gene Editing Patent Portfolio: List of Leading Patents (by Highest Relative Valuation)
  • Table 13.1 Gene Therapy: List of Mergers and Acquisitions, 2014-2019
  • Table 13.2 Acquisitions: Key Value Drivers
  • Table 13.3 Mergers and Acquisitions: Therapeutic Areas and Phase of Development of the Acquired Company's Product
  • Table 14.1 Gene Therapy Market: Funding and Investments, 2014-2019
  • Table 14.2 Funding and Investment Analysis: Summary of Investments
  • Table 14.3 Funding and Investment Analysis: Summary of Venture Capital Funding
  • Table 15.1 Pricing Model: Price of Marketed Gene / Cell Therapies
  • Table 15.2 Pricing Model: Price of Marketed Targeted Drugs
  • Table 15.3 Pricing Model: Opinions of Experts / Other Analysts
  • Table 17.1 Gene Therapies: Expected Launch Years of Advanced Stage Drug Candidates
  • Table 17.2 Gendicine®: Target Patient Population
  • Table 17.3 Gendicine®: Net Present Value (USD Million)
  • Table 17.4 Gendicine®: Value Creation Analysis (USD Million)
  • Table 17.5 Oncorine®: Target Patient Population
  • Table 17.6 Oncorine®: Net Present Value (USD Million)
  • Table 17.7 Oncorine®: Value Creation Analysis (USD Million)
  • Table 17.8 Rexin-G®: Target Patient Population
  • Table 17.9 Rexin-G®: Net Present Value (USD Million)
  • Table 17.10 Rexin-G®: Value Creation Analysis (USD Million)
  • Table 17.11 Neovasculgen®: Target Patient Population
  • Table 17.12 Neovasculgen®: Net Present Value (USD Million)
  • Table 17.13 Neovasculgen®: Value Creation Analysis (USD Million)
  • Table 17.14 Strimvelis®: Target Patient Population
  • Table 17.15 Strimvelis®: Net Present Value (USD Million)
  • Table 17.16 Strimvelis®: Value Creation Analysis (USD Million)
  • Table 17.17 Imlygic®: Target Patient Population
  • Table 17.18 Imlygic®: Net Present Value (USD Million)
  • Table 17.19 Imlygic®: Value Creation Analysis (USD Million)
  • Table 17.20 Invossa™: Target Patient Population
  • Table 17.21 Invossa™: Net Present Value (USD Million)
  • Table 17.22 Invossa™: Value Creation Analysis (USD Million)
  • Table 17.23 Luxturna™: Target Patient Population
  • Table 17.24 Luxturna™: Net Present Value (USD Million)
  • Table 17.25 Luxturna™: Value Creation Analysis (USD Million)
  • Table 17.26 Axalimogene Filolisbac: Target Patient Population
  • Table 17.27 Axalimogene Filolisbac: Net Present Value (USD Million)
  • Table 17.28 Axalimogene Filolisbac: Value Creation Analysis (USD Million)
  • Table 17.29 AMT-061: Target Patient Population
  • Table 17.30 AMT-061: Net Present Value (USD Million)
  • Table 17.31 AMT-061: Value Creation Analysis (USD Million)
  • Table 17.32 BIIB111: Target Patient Population
  • Table 17.33 BIIB111: Net Present Value (USD Million)
  • Table 17.34 BIIB111: Value Creation Analysis (USD Million)
  • Table 17.35 BIIB112: Target Patient Population
  • Table 17.36 BIIB112: Net Present Value (USD Million)
  • Table 17.37 BIIB112: Value Creation Analysis (USD Million)
  • Table 17.38 Donaperminogene Seltoplasmid (VM202): Target Patient Population
  • Table 17.39 Donaperminogene Seltoplasmid (VM202): Net Present Value (USD Million)
  • Table 17.40 Donaperminogene Seltoplasmid (VM202): Value Creation Analysis (USD Million)
  • Table 17.41 E10A: Target Patient Population
  • Table 17.42 E10A: Net Present Value (USD Million)
  • Table 17.43 E10A: Value Creation Analysis (USD Million)
  • Table 17.44 Fidanacogene Elaparvovec (PF-06838435): Target Patient Population
  • Table 17.45 Fidanacogene Elaparvovec (PF-06838435): Net Present Value (USD Million)
  • Table 17.46 Fidanacogene Elaparvovec (PF-06838435): Value Creation Analysis (USD Million)
  • Table 17.47 FLT180a: Target Patient Population
  • Table 17.48 FLT180a: Net Present Value (USD Million)
  • Table 17.49 FLT180a: Value Creation Analysis (USD Million)
  • Table 17.50 GS010: Target Patient Population
  • Table 17.51 GS010: Net Present Value (USD Million)
  • Table 17.52 GS010: Value Creation Analysis (USD Million)
  • Table 17.53 Instiladrin®: Target Patient Population
  • Table 17.54 Instiladrin®: Net Present Value (USD Million)
  • Table 17.55 Instiladrin®: Value Creation Analysis (USD Million)
  • Table 17.56 Lenti-D™: Target Patient Population
  • Table 17.57 Lenti-D™: Net Present Value (USD Million)
  • Table 17.58 Lenti-D™: Value Creation Analysis (USD Million)
  • Table 17.59 LYS-SAF302: Target Patient Population
  • Table 17.60 LYS-SAF302: Net Present Value (USD Million)
  • Table 17.61 LYS-SAF302: Value Creation Analysis (USD Million)
  • Table 17.62 Ofranergene Obadenovec (VB-111): Target Patient Population
  • Table 17.63 Ofranergene Obadenovec (VB-111): Net Present Value (USD Million)
  • Table 17.64 Ofranergene Obadenovec (VB-111): Value Creation Analysis (USD Million)
  • Table 17.65 OTL-101: Target Patient Population
  • Table 17.66 OTL-101: Net Present Value (USD Million)
  • Table 17.67 OTL-101: Value Creation Analysis (USD Million)
  • Table 17.68 OTL-103: Target Patient Population
  • Table 17.69 OTL-103: Net Present Value (USD Million)
  • Table 17.70 OTL-103: Value Creation Analysis (USD Million)
  • Table 17.71 OTL-200: Target Patient Population
  • Table 17.72 OTL-200: Net Present Value (USD Million)
  • Table 17.73 OTL-200: Value Creation Analysis (USD Million)
  • Table 17.74 Pexastimogene Devacirepvec (PEXA-VEC): Target Patient Population
  • Table 17.75 Pexastimogene Devacirepvec (PEXA-VEC): Net Present Value (USD Million)
  • Table 17.76 Pexastimogene Devacirepvec (PEXA-VEC): Value Creation Analysis (USD Million)
  • Table 17.77 ProstAtak®: Target Patient Population
  • Table 17.78 ProstAtak®: Net Present Value (USD Million)
  • Table 17.79 ProstAtak®: Value Creation Analysis (USD Million)
  • Table 17.80 SPK-8011: Target Patient Population
  • Table 17.81 SPK-8011: Net Present Value (USD Million)
  • Table 17.82 SPK-8011: Value Creation Analysis (USD Million)
  • Table 17.83 Unnamed Therapy: Target Patient Population
  • Table 17.84 Unnamed Therapy: Net Present Value (USD Million)
  • Table 17.85 Unnamed Therapy: Value Creation Analysis (USD Million)
  • Table 17.86 Valoctocogene Roxaparvovec (BMN 270): Target Patient Population
  • Table 17.87 Valoctocogene Roxaparvovec (BMN 270): Net Present Value (USD Million)
  • Table 17.88 Valoctocogene Roxaparvovec (BMN 270): Value Creation Analysis (USD Million)
  • Table 17.89 Vigil®: Target Patient Population
  • Table 17.90 Vigil®: Net Present Value (USD Million)
  • Table 17.91 Vigil®: Value Creation Analysis (USD Million)
  • Table 17.92 VGX-3100: Target Patient Population
  • Table 17.93 VGX-3100: Net Present Value (USD Million)
  • Table 17.94 VGX-3100: Value Creation Analysis (USD Million)
  • Table 17.95 Vocimagene Amiretrorepvec (Toca-511): Target Patient Population
  • Table 17.96 Vocimagene Amiretrorepvec (Toca-511): Net Present Value (USD Million)
  • Table 17.97 Vocimagene Amiretrorepvec (Toca-511): Value Creation Analysis (USD Million)
  • Table 18.1 Small Scale Cell Culture Systems
  • Table 18.2 Contract Manufacturing Service Providers for Viral Vectors and Plasmid DNA
  • Table 20.1 Gene Therapy Market: Key Takeaways
  • Table 22.1 Gene Therapies, Clinical Pipeline: Distribution by Phase of Development
  • Table 22.2 Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Area
  • Table 22.3 Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Area and Phase of Development
  • Table 22.4 Gene Therapies, Clinical Pipeline: Distribution by Type of Vector Used
  • Table 22.5 Gene Therapies, Clinical Pipeline: Distribution by Type of Gene
  • Table 22.6 Gene Therapies, Clinical Pipeline: Distribution by Type of Modification
  • Table 22.7 Gene Therapies, Clinical Pipeline: Distribution by Type of Modification and Type of Vector Used
  • Table 22.8 Gene Therapies, Clinical Pipeline: Distribution by Type of Gene Therapy
  • Table 22.9 Gene Therapies, Clinical Pipeline: Distribution by Route of Administration
  • Table 22.10 Gene Therapies, Early Stage Pipeline: Distribution by Stage of Development
  • Table 22.11 Gene Therapies, Early Stage Pipeline: Distribution by Therapeutic Areas
  • Table 22.12 Gene Therapies, Early Stage Pipeline: Distribution by Type of Vector Used
  • Table 22.13 Gene Therapies, Early Stage Pipeline: Distribution by Type of Gene
  • Table 22.14 Gene Therapies, Early Stage Pipeline: Distribution by Type of Modification
  • Table 22.15 Gene Therapies, Early Stage Pipeline: Distribution by Type of Modification and Type of Vector Used
  • Table 22.16 Gene Therapies, Early Stage Pipeline: Distribution by Type of Gene Therapy
  • Table 22.17 Gene Therapies, Early Stage Pipeline: Distribution by Route of Administration
  • Table 22.18 Gene Therapies: Key Players
  • Table 22.19 Gene Therapy Developers: Distribution by Year of Establishment
  • Table 22.20 Gene Therapy Developers : Distribution by Size of Employee Base
  • Table 22.21 Gene Therapy Developers : Distribution by Location of Headquarter
  • Table 22.22 Gene Therapies: Distribution by Therapeutic Area and Special Designations Awarded
  • Table 22.23 Gene Therapies for Cardiovascular Disorders: Distribution by Target Indication and Phase of Development
  • Table 22.24 Gene Therapies for Cardiovascular Disorders: Distribution by Type of Vector Used
  • Table 22.25 Gene Therapies for Hematological Disorders: Distribution by Target Indication and Phase of Development
  • Table 22.26 Gene Therapies for Hematological Disorders: Distribution by Type of Vector Used
  • Table 22.27 Gene Therapies for I&I Disorders: Distribution by Target Indication and Phase of Development
  • Table 22.28 Gene Therapies for I&I Disorders: Distribution by Type of Vector Used
  • Table 22.29 Gene Therapies for Metabolic Disorders: Distribution by Target Indication and Phase of Development
  • Table 22.30 Gene Therapies for Metabolic Disorders: Distribution by Type of Vector Used
  • Table 22.31 Gene Therapies for Muscular Disorders: Distribution by Target Indication and Phase of Development
  • Table 22.32 Gene Therapies for Muscular Disorders: Distribution by Type of Vector Used
  • Table 22.33 Gene Therapies for Neurological Disorders: Distribution by Target Indication and Phase of Development
  • Table 22.34 Gene Therapies for Neurological Disorders: Distribution by Type of Vector Used
  • Table 22.35 Gene Therapies for Ophthalmic Disorders: Distribution by Target Indication and Phase of Development
  • Table 22.36 Gene Therapies for Ophthalmic Disorders: Distribution by Type of Vector Used
  • Table 22.37 Gene Therapies for Oncology: Distribution by Target Indication and Phase of Development
  • Table 22.38 Gene Therapies for Oncology: Distribution by Type of Vector Used
  • Table 22.39 Gene Therapy Patent Portfolio: Distribution by Type of Patent
  • Table 22.40 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2019
  • Table 22.41 Gene Therapy Patent Portfolio: Distribution by Geographical Location
  • Table 22.42 Gene Therapy Patent Portfolio: Distribution by Geographical Location, North America
  • Table 22.43 Gene Therapy Patent Portfolio: Distribution by Geographical Location, Europe
  • Table 22.44 Gene Therapy Patent Portfolio: Distribution by Geographical Location, Asia Pacific
  • Table 22.45 Gene Therapy Patent Portfolio: Distribution by CPC Classification Symbols
  • Table 22.46 Gene Therapy Patent Portfolio: Emerging Focus Areas
  • Table 22.47 Gene Therapy Patent Portfolio: Leading Industry Players
  • Table 22.48 Gene Therapy Patent Portfolio: Leading Non-Industry Players
  • Table 22.49 Gene Therapy Patent Portfolio (Genentech and GSK): Benchmarking by Patent Characteristics
  • Table 22.50 Gene Therapy Patent Portfolio, Leading Industry Players: Benchmarking by Patent Characteristics
  • Table 22.51 Gene Therapy Patent Portfolio: Distribution of Patents by Age (2016-2019)
  • Table 22.52 Gene Therapy Patent Portfolio: Valuation Analysis
  • Table 22.53 Gene Editing Patent Portfolio: Distribution by Type of Patent
  • Table 22.54 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2019
  • Table 22.55 Gene Editing Patent Portfolio: Distribution by Geographical Location
  • Table 22.56 Gene Editing Patent Portfolio: Distribution by Geographical Location, North America
  • Table 22.57 Gene Editing Patent Portfolio: Distribution by Geographical Location, Europe
  • Table 22.58 Gene Editing Patent Portfolio: Distribution by Geographical Location, Asia-Pacific
  • Table 22.59 Gene Editing Patent Portfolio: Distribution by CPC Classification Symbols
  • Table 22.60 Gene Editing Patent Portfolio: Emerging Focus Areas
  • Table 22.61 Gene Editing Patent Portfolio: Leading Industry Players
  • Table 22.62 Gene Editing Patent Portfolio: Leading Non-Industry Players
  • Table 22.63 Gene Editing Patent Portfolio (Sangamo Therapeutics and Cellectis): Benchmarking by Patent Characteristics
  • Table 22.64 Gene Editing Patent Portfolio, Leading Industry Players: Benchmarking by Patent Characteristics
  • Table 22.65 Gene Editing Patent Portfolio: Distribution of Patents by Age (2016- 2019)
  • Table 22.66 Gene Editing Patent Portfolio: Valuation Analysis
  • Table 22.67 Gene Therapy and Gene Editing Patent Portfolio: Distribution by Type of Organization
  • Table 22.68 Mergers and Acquisitions: Cumulative Year-Wise Trend (2014- 2019)
  • Table 22.69 Mergers and Acquisitions: Distribution by Type of Mergers and Acquisitions (2014 - 2019)
  • Table 22.70 Mergers and Acquisitions: Distribution by Year and Type of Mergers and Acquisitions (2014 - 2019)
  • Table 22.71 Mergers and Acquisitions: Continent-wise Distribution
  • Table 22.72 Mergers and Acquisitions: Intercontinental and Intracontinental Acquisitions
  • Table 22.73 Mergers and Acquisitions: Country-wise Distribution
  • Table 22.74 Acquisitions: Distribution by Key Value Drivers
  • Table 22.75 Acquisitions: Distribution by Key Value Drivers and Year of Acquisition (2014-2019)
  • Table 22.76 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product
  • Table 22.77 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product and Year of Acquisition
  • Table 22.78 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product and Year of Acquisition
  • Table 22.79 Mergers and Acquisitions: Distribution by Therapeutic Area
  • Table 22.80 Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2019
  • Table 22.81 Funding and Investment Analysis: Cumulative Number of Instances by Year, 2014-2019
  • Table 22.82 Funding and Investment Analysis: Cumulative Amount Invested, 2014-2019 (USD Million)
  • Table 22.83 Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2019
  • Table 22.84 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2019
  • Table 22.85 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2019 (USD Million)
  • Table 22.86 Funding and Investment Analysis: Summary of Investments, 2014-2019 (USD Million)
  • Table 22.87 Funding and Investments: Distribution by Amount Invested across Different Types of Therapies
  • Table 22.88 Funding and Investment Analysis: Distribution by Geography
  • Table 22.89 Funding and Investment Analysis: Regional Distribution of Funding Instances
  • Table 22.90 Funding and Investment Analysis: Most Active Players, on the basis of Number of Instances, 2014-2019 (USD Million)
  • Table 22.91 Funding and Investment Analysis: Most Active Players, on the basis of Amount Invested, 2014-2019 (USD Million)
  • Table 22.92 Funding and Investment Analysis: Distribution by Type of Investors
  • Table 22.93 Funding and Investment Analysis: Distribution by Big Pharma Players (Investors)
  • Table 22.94 Funding and Investment Analysis: Distribution by Government Institutes (Investors)
  • Table 22.95 Funding and Investment Analysis: Leading Investors
  • Table 22.96 Funding and Investment Analysis: Distribution of Investments by Stages of Development
  • Table 22.97 Funding and Investment Analysis: Distribution by Highest Phase of Development of the Company's Product for Funding
  • Table 22.98 Funding and Investment Summary, 2014-2019 (USD Million)
  • Table 22.99 Overall Gene Therapy Market, 2018-2030: Base Scenario (USD Million)
  • Table 22.100 Gene Therapy Market: Distribution by Type of Gene Modification, 2019, 2025 and 2030
  • Table 22.101 Gene Therapy Market: Distribution by Type of Therapy, 2019, 2025 and 2030
  • Table 22.102 Gene Therapy Market: Distribution by Type of Vector Used, 2019, 2025 and 2030
  • Table 22.103 Gene Therapy Market: Distribution by Therapeutic Area, 2019, 2025 and 2030
  • Table 22.104 Gene Therapy Market: Distribution by Route of Administration, 2019, 2025 and 2030
  • Table 22.105 Gene Therapy Market: Distribution by Geography, 2019, 2025 and 2030
  • Table 22.106 Gene Therapy Market: Distribution by Geography (Rest of the World), 2019, 2025 and 2030
  • Table 22.107 Gene Therapy Market: Key Players, 2019, 2025 and 2030
  • Table 22.108 Gendicine® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.109 Oncorine® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.110 Rexin-G® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.111 Neovasculgen® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.112 Strimvelis® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.113 Imlygic® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.114 Invossa™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.115 Luxturna™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.116 Zolgensma™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.117 Collategene® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.118 Zyntelgo™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.119 Axalimogene Filolisbac Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.120 AMT-061 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.121 BIIB111 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.122 BIIB112 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.123 Donaperminogene Seltoplasmid (VM202) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.124 E10A Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.125 Fidanacogene Elaparvovec (PF-06838435) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.126 FLT180a Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.127 GS010 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.128 Instiladrin® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.129 Lenti-D™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.130 LYS-SAF302 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.131 Ofranergene Obadenovec (VB-111) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.132 OTL-101Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.133 OTL-103 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.134 OTL-200 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.135 Pexastimogene Devacirepvec (PEXA-VEC) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.136 ProstAtak® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.137 SPK-8011 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.138 Unnamed Therapy Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.139 Valoctocogene Roxaparvovec (BMN 270) Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.140 Vigil® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.141 VGX-3100 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 22.142 Vocimagene Amiretrorepvec (Toca-511) Sales Forecast (Till 2030): Base Scenario (USD Million)

List of Companies

The following companies / institutes / government bodies and organizations have been mentioned in this report.

  • 4D Molecular Therapeutics
  • 5AM Ventures
  • A1ATD Investors
  • AAVogen
  • AbbVie
  • Abby Grace Foundation
  • Abeona Therapeutics
  • Actus Therapeutics
  • Acucela
  • Adage Capital Management
  • Aduro Biotech
  • Advantagene
  • Advaxis
  • Adverum Biotechnologies
  • Agent Capital
  • Agilis Biotherapeutics
  • Aisling Capital
  • AJU IB Investment
  • Akouos
  • Alexandria Venture Investments
  • Allergan
  • Ally Bridge Group
  • Alpha-1 Foundation
  • AlphaVax
  • ALS Investment Fund
  • Altitude Life Science Ventures
  • Ambys Medicines
  • American Gene Technologies
  • Amgen
  • Amicus Therapeutics
  • Anaeropharma Science
  • Anchiano Therapeutics
  • AnGes
  • Angionetics
  • Apic Bio
  • Applied Genetic Technologies (AGTC)
  • ARCH Venture Partners
  • Arix Bioscience
  • ArrowMark Partners
  • Arthrogen
  • Aruvant Sciences
  • AskBio
  • Astellas Pharma
  • Atlas Venture
  • Auburn University
  • Audentes Therapeutics
  • Avalanche Biotechnologies
  • AveXis
  • AVROBIO
  • Axovant Gene Therapies
  • Baillie Gifford
  • Bain Capital Life Sciences
  • Bamboo Therapeutics
  • Baxter
  • Bayer
  • Beam Therapeutics
  • Berkeley Catalyst Fund
  • Biogen
  • BioInvent
  • BioMarin
  • Biomatics Capital
  • Bionic Sight
  • Biotechnology Value Fund
  • Bioverativ
  • Blue Bear Ventures
  • bluebird bio
  • BlueWater Angels
  • Boehringer Ingelheim
  • Boston Children's Hospital
  • Boxer Capital
  • Bpifrance
  • Brace Pharma Capital
  • Breakout Ventures
  • BrightGene Bio-Medical Technology
  • Bristol-Myers Squibb
  • Brookside Capital
  • Butterfly sciences
  • BVF Partners
  • California Institute for Regenerative Medicine (CIRM)
  • Calimmune
  • Cambridge Enterprise
  • Cambridge Innovation Capital
  • Cancer Prevention and Research Institute of Texas (CPRIT)
  • CapDecisif Management
  • Cardiogen Sciences
  • CareNet Group
  • Casdin Capital
  • Castle Creek Pharma
  • Celenex (Nationwide Children's Hospital Spin Out)
  • Cellectis
  • CellGenTech
  • Celsion
  • Center for Applied Medical Research (CIMA)
  • Chatham Therapeutics
  • Chiba University
  • Children's Medical Research Foundation
  • Clarus Ventures
  • Clough Capital Partners
  • Cold Genesys
  • Columbus Venture Partners
  • CombiGene
  • Copernicus Therapeutics
  • Cormorant Asset Management
  • Cowen Private Investments
  • CRISPR Therapeutics
  • CSL Behring
  • Cure Sanfilippo Foundation
  • CureDuchenne Ventures
  • Cynvec
  • Cystic Fibrosis Foundation Therapeutics
  • Deerfield Management
  • Defense Advanced Research Projects Agency (DARPA)
  • DEFTA Partners
  • Dimension Therapeutics
  • Discovery Genomics
  • DNAlite Therapeutics
  • DNAtrix
  • Driehaus Capital Management
  • Duke University Medical Center
  • EB Medical Research Foundation (EBMRF)
  • EB Research Partnership
  • EcoR1 Capital
  • EdiGene
  • Editas Medicine
  • Edmond de Rothschild Investment Partners
  • Eight Roads Ventures
  • Endsulin
  • enGene
  • Enochian BioSciences
  • Ensysce Biosciences
  • Epeius Biotechnologies
  • EPS Holdings
  • Errant Gene Therapeutics
  • Esteve
  • Etubics
  • European Investment Bank (EIB)
  • Eventide Asset Management
  • Exonics Therapeutics
  • Expression Therapeutics
  • ExSight Capital
  • Eyevensys
  • FF Science
  • Fibrocell
  • Fidelity Management & Research Company
  • FIT Biotech
  • FKD Therapies
  • Flexion Therapeutics
  • Fondation Sanfilippo Suisse
  • Foresite Capital
  • Foundation Sanfilippo B
  • FoxKiser
  • F-Prime Capital
  • Franklin Templeton Investments
  • Frazier Healthcare Partners
  • Freeline Therapeutics
  • Friedreich's Ataxia Research Alliance
  • Fund+
  • Gemini Therapeutics
  • Genable Technologies
  • GeneCure Biotechnologies
  • Genelux
  • Genentech
  • GeneOne Life Science
  • GeneQuine Biotherapeutics
  • Generation Bio
  • Genethon
  • Genexine
  • Genprex
  • GenSight Biologics
  • GenVec
  • Ghost Tree Capital Group
  • Global BioTherapeutics (GBT)
  • Go Capital
  • GOG foundation
  • Gradalis
  • Green Cross Holdings
  • Green Pine Capital Partners
  • GSK
  • Guangzhou Double Bioproducts
  • GV (formerly Google Ventures)
  • Gyroscope Therapeutics
  • Harvard College
  • Hatteras Venture Partners
  • HBM Healthcare Investments
  • HealthCap
  • Hemera Biosciences
  • Herantis Pharma
  • Hercules Capital
  • Hiroshima Venture Capital
  • Histogenics
  • Holostem Terapie Avanzate
  • Homology Medicines
  • HORAMA
  • Horizon 2020
  • Horizon Discovery
  • Horizon Technology Finance
  • Huagai Capital
  • Huapont Life Sciences
  • Human Stem Cells Institute (HSCI)
  • Icahn School of Medicine at Mount Sinai
  • Ichor
  • ID Pharma
  • IDG Capital
  • Idinvest Partners
  • Immusoft
  • Imperial Innovations
  • Inception Capital Management
  • Innovation Network Corporation of Japan
  • Inotek Pharmaceuticals
  • Inovio Pharmaceuticals
  • Inserm Transfert
  • Intellia Therapeutics
  • Intrexon
  • Israel Innovation Authority
  • IVERIC bio
  • Janssen Pharmaceuticals
  • Janus Capital Management
  • Janus Henderson Investors
  • Jennerex
  • Jennison Associates
  • Juda Capital
  • JumpStart
  • Juventas Therapeutics
  • Kiwoom Securities
  • Kolon TissueGene
  • Krystal Biotech
  • Kurma Partners
  • Lacerta Therapeutics
  • Leerink Partners
  • Lentigen Technology, a Miltenyi Biotec Company
  • Life Sciences Partners (LSP)
  • Lilly Asia Ventures
  • LIME Asset Management
  • LogicBio Therapeutics
  • Lokon Pharma
  • Longitude Capital
  • Lonza
  • Ludwig Institute for Cancer Research
  • Lundbeckfonden Ventures
  • Lysogene
  • Marsala Biotech
  • Maryland Technology Development Corporation (TEDCO)
  • Maverick Ventures
  • MaxCyte
  • Mayo Clinic
  • MedImmune
  • Medison Ventures
  • MeiraGTx
  • Memorial Sloan Kettering Cancer Center
  • Mercia Technologies
  • Merck
  • Mercury Fund
  • MERITZ Securities
  • Mesa Verde Venture Partners
  • Michigan Economic Development
  • MidCap Financial
  • Midven
  • Milo Biotechnology
  • Mirae Asset
  • Mita Securities
  • Mizuho Capital Partners
  • MOLOGEN
  • Momotaro-Gene
  • Morningside Ventures
  • MultiVir
  • Mustang Bio
  • Myonexus Therapeutics
  • NanoCor Therapeutics
  • Nanogenic Solutions
  • National Institute of Allergy and Infectious Diseases
  • National Institutes of Health (NIH)
  • National MPS Society
  • National Securities
  • Nationwide Children's Hospital
  • Nature Technology
  • Nerveda
  • Neuracle Genetics
  • Neuralgene
  • Neurocrine Biosciences
  • Neurogene
  • Neurotech Pharmaceuticals
  • New Energy and Industrial Technology Development Organization
  • New Enterprise Associates (NEA)
  • New Leaf Venture Partners
  • Nightstar Therapeutics
  • Nissei Capital
  • Nohla Therapeutics
  • Nottingham Technology Grant Fund
  • Novartis
  • Oberland Capital
  • Ocugen
  • Office of Commercialization, Washington State University
  • Omega Funds
  • Omnes Capital
  • Oncolys BioPharma
  • Oncos Therapeutics
  • OncoSec Medical
  • OrbiMed
  • Orbit Biomedical
  • ORCA Therapeutics
  • Orchard Therapeutics
  • ORI Healthcare Fund
  • Osage University Partners
  • Oxford BioMedica
  • Oxford Finance
  • Panmure Gordon
  • Pappas Capital
  • Parker Institute for Cancer Immunotherapy
  • Partner Fund Management
  • Partners Innovation Fund
  • Passage Bio
  • Pattern BioSciences
  • Pavilion Capital
  • Pentwater Capital Management
  • Perceptive Advisors
  • PeriphaGen
  • Perseverance Capital Management
  • Pfizer
  • PlasmaTech Biopharmaceuticals
  • PNP Therapeutics
  • Pontifax Venture Capital
  • Posco Capital Partners
  • Poseida Therapeutics
  • Precision BioSciences
  • Precision Genome Engineering
  • Prevail Therapeutics
  • Promethera Biosciences
  • Prostate Cancer Foundation
  • PsiOxus Therapeutics
  • PTC Therapeutics
  • Quethera
  • QVT Financial
  • RA Capital Management
  • RBV Capital
  • Red Sanfilippo Foundation
  • Redmile Group
  • Regeneron Pharmaceuticals
  • REGENXBIO
  • Renova Therapeutics
  • Renovacor
  • Rentschler Biopharma
  • RetroSense Therapeutics
  • Rev1 Ventures
  • Reyon Pharmaceutical
  • Ridgeback Capital
  • Roche
  • Rock Springs Capital
  • Rocket Pharmaceuticals
  • RTW Investments
  • Ryboquin
  • Sabby Capital
  • Samsara BioCapital
  • Sanfilippo Children's Foundation
  • Sangamo Therapeutics
  • Sanofi
  • Sarepta Therapeutics
  • Santhera Pharmaceuticals
  • SBI Japan-Israel Innovation Fund
  • Scancell
  • SDL Ventures
  • Sectoral Asset Management
  • Selecta Biosciences
  • Seventure Partners
  • Sham Innovation Santé
  • Shanghai Sunway Biotech
  • Shavit Capital
  • Shenzhen Qianhai Taxus
  • Shinhan Capital
  • Shinsei Corporate Investment
  • Shire
  • Sibiono GeneTech
  • SillaJen
  • SIRION Biotech
  • SMBC Venture Capital
  • Sofinnova Ventures
  • Solid Biosciences
  • SOSV
  • Spark Therapeutics
  • Sphera Global Health Care Fund
  • SR-TIGET
  • St. Jude Children's Research Hospital
  • Stop Sanfilippo Foundation
  • StrideBio
  • Surveyor Capital (a Citadel company)
  • SV Health Investors
  • SV Life Sciences
  • SVE Capital
  • T. Rowe Price Associates
  • Takeda
  • Target ALS Foundation
  • Targeted Technology Funds
  • Targovax
  • Tasly Biopharmaceuticals
  • Taxus Cardium Pharmaceuticals
  • Tech Coast Angels
  • Technium Partners
  • Temasek
  • Tenaya Therapeutics
  • The Board of Trustees of the Leland Stanford Junior University
  • The Column Group (TCG)
  • The Movember Foundation
  • TheraBiologics
  • THERAVECTYS
  • Third Rock Ventures
  • TNK Therapeutics, subsidiary of Sorrento Therapeutics
  • Tocagen
  • TPG Capital
  • Transgene
  • Transposagen Biopharmaceuticals
  • U.S. Army's Small Business Innovation Research
  • UB Securities
  • UC Davis Medical Center
  • UK Cystic Fibrosis Gene Therapy Consortium
  • Ultragenyx Pharmaceutical
  • uniQure Biopharma
  • Universitat Autònoma de Barcelona (UAB)
  • University College London
  • University of California
  • University of Massachusetts Medical School
  • University of Oxford
  • University of Pennsylvania
  • Urovant Sciences
  • UT Southwestern Medical Center
  • V-Bio Ventures
  • VBL Therapeutics
  • VCN Biosciences
  • Vector Neurosciences
  • Venrock
  • Versant Ventures
  • Vertex Pharmaceuticals
  • Verve Therapeutics
  • Vessl™
  • Vical
  • Vida Ventures
  • Viking Global Investors
  • Virginia Catalyst
  • ViroMed
  • Virttu Biologics
  • Vivet Therapeutics
  • Vivo Capital
  • Voyager Therapeutics
  • Washington Research Foundation
  • Weill Cornell Medical College
  • Wellcome Trust
  • Wellington Capital Management
  • Wellstat Ophthalmics
  • Whitesun Healthcare Ventures
  • WI Harper Group
  • Woodford Investment Management
  • Wyvern Pharmaceuticals
  • XyloCor Therapeutics
  • Ysios Capital
  • Yuhan Corporation
  • Ziopharm Oncology
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