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

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  • Published
    August 2019

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    550

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Overview

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

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.

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.

Contents

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 Content

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

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.

  1. 4D Molecular Therapeutics
  2. 5AM Ventures
  3. A1ATD Investors
  4. AAVogen
  5. AbbVie
  6. Abby Grace Foundation
  7. Abeona Therapeutics 
  8. Actus Therapeutics
  9. Acucela
  10. Adage Capital Management
  11. Aduro Biotech
  12. Advantagene
  13. Advaxis
  14. Adverum Biotechnologies
  15. Agent Capital
  16. Agilis Biotherapeutics
  17. Aisling Capital
  18. AJU IB Investment
  19. Akouos
  20. Alexandria Venture Investments
  21. Allergan
  22. Ally Bridge Group
  23. Alpha-1 Foundation
  24. AlphaVax
  25. ALS Investment Fund
  26. Altitude Life Science Ventures
  27. Ambys Medicines
  28. American Gene Technologies
  29. Amgen
  30. Amicus Therapeutics
  31. Anaeropharma Science
  32. Anchiano Therapeutics
  33. AnGes
  34. Angionetics
  35. Apic Bio
  36. Applied Genetic Technologies (AGTC)
  37. ARCH Venture Partners
  38. Arix Bioscience
  39. ArrowMark Partners
  40. Arthrogen
  41. Aruvant Sciences
  42. AskBio
  43. Astellas Pharma
  44. Atlas Venture
  45. Auburn University
  46. Audentes Therapeutics
  47. Avalanche Biotechnologies
  48. AveXis
  49. AVROBIO
  50. Axovant Gene Therapies
  51. Baillie Gifford
  52. Bain Capital Life Sciences
  53. Bamboo Therapeutics
  54. Baxter
  55. Bayer
  56. Beam Therapeutics
  57. Berkeley Catalyst Fund
  58. Biogen
  59. BioInvent
  60. BioMarin
  61. Biomatics Capital
  62. Bionic Sight
  63. Biotechnology Value Fund
  64. Bioverativ
  65. Blue Bear Ventures
  66. bluebird bio
  67. BlueWater Angels
  68. Boehringer Ingelheim
  69. Boston Children's Hospital
  70. Boxer Capital
  71. Bpifrance
  72. Brace Pharma Capital
  73. Breakout Ventures
  74. BrightGene Bio-Medical Technology
  75. Bristol-Myers Squibb
  76. Brookside Capital
  77. Butterfly sciences
  78. BVF Partners
  79. California Institute for Regenerative Medicine (CIRM)
  80. Calimmune
  81. Cambridge Enterprise
  82. Cambridge Innovation Capital
  83. Cancer Prevention and Research Institute of Texas (CPRIT)
  84. CapDecisif Management
  85. Cardiogen Sciences
  86. CareNet Group
  87. Casdin Capital
  88. Castle Creek Pharma
  89. Celenex (Nationwide Children’s Hospital Spin Out)
  90. Cellectis
  91. CellGenTech
  92. Celsion
  93. Center for Applied Medical Research (CIMA)
  94. Chatham Therapeutics
  95. Chiba University
  96. Children’s Medical Research Foundation
  97. Clarus Ventures
  98. Clough Capital Partners
  99. Cold Genesys
  100. Columbus Venture Partners
  101. CombiGene
  102. Copernicus Therapeutics
  103. Cormorant Asset Management
  104. Cowen Private Investments
  105. CRISPR Therapeutics
  106. CSL Behring
  107. Cure Sanfilippo Foundation
  108. CureDuchenne Ventures
  109. Cynvec
  110. Cystic Fibrosis Foundation Therapeutics
  111. Deerfield Management
  112. Defense Advanced Research Projects Agency (DARPA)
  113. DEFTA Partners
  114. Dimension Therapeutics
  115. Discovery Genomics
  116. DNAlite Therapeutics
  117. DNAtrix
  118. Driehaus Capital Management 
  119. Duke University Medical Center
  120. EB Medical Research Foundation (EBMRF)
  121. EB Research Partnership 
  122. EcoR1 Capital
  123. EdiGene
  124. Editas Medicine
  125. Edmond de Rothschild Investment Partners
  126. Eight Roads Ventures
  127. Endsulin
  128. enGene
  129. Enochian BioSciences
  130. Ensysce Biosciences
  131. Epeius Biotechnologies
  132. EPS Holdings
  133. Errant Gene Therapeutics
  134. Esteve
  135. Etubics
  136. European Investment Bank (EIB)
  137. Eventide Asset Management
  138. Exonics Therapeutics
  139. Expression Therapeutics
  140. ExSight Capital
  141. Eyevensys
  142. FF Science
  143. Fibrocell
  144. Fidelity Management & Research Company
  145. FIT Biotech
  146. FKD Therapies
  147. Flexion Therapeutics
  148. Fondation Sanfilippo Suisse
  149. Foresite Capital
  150. Foundation Sanfilippo B
  151. FoxKiser
  152. F-Prime Capital
  153. Franklin Templeton Investments
  154. Frazier Healthcare Partners
  155. Freeline Therapeutics
  156. Friedreich’s Ataxia Research Alliance
  157. Fund+
  158. Gemini Therapeutics
  159. Genable Technologies
  160. GeneCure Biotechnologies
  161. Genelux
  162. Genentech
  163. GeneOne Life Science
  164. GeneQuine Biotherapeutics
  165. Generation Bio
  166. Genethon
  167. Genexine 
  168. Genprex
  169. GenSight Biologics
  170. GenVec
  171. Ghost Tree Capital Group
  172. Global BioTherapeutics (GBT)
  173. Go Capital
  174. GOG foundation
  175. Gradalis
  176. Green Cross Holdings
  177. Green Pine Capital Partners
  178. GSK
  179. Guangzhou Double Bioproducts
  180. GV (formerly Google Ventures)
  181. Gyroscope Therapeutics
  182. Harvard College
  183. Hatteras Venture Partners
  184. HBM Healthcare Investments
  185. HealthCap
  186. Hemera Biosciences
  187. Herantis Pharma
  188. Hercules Capital
  189. Hiroshima Venture Capital
  190. Histogenics
  191. Holostem Terapie Avanzate
  192. Homology Medicines
  193. HORAMA
  194. Horizon 2020
  195. Horizon Discovery 
  196. Horizon Technology Finance
  197. Huagai Capital
  198. Huapont Life Sciences
  199. Human Stem Cells Institute (HSCI)
  200. Icahn School of Medicine at Mount Sinai
  201. Ichor
  202. ID Pharma
  203. IDG Capital
  204. Idinvest Partners
  205. Immusoft
  206. Imperial Innovations
  207. Inception Capital Management
  208. Innovation Network Corporation of Japan
  209. Inotek Pharmaceuticals
  210. Inovio Pharmaceuticals
  211. Inserm Transfert
  212. Intellia Therapeutics
  213. Intrexon
  214. Israel Innovation Authority
  215. IVERIC bio
  216. Janssen Pharmaceuticals
  217. Janus Capital Management
  218. Janus Henderson Investors
  219. Jennerex
  220. Jennison Associates
  221. Juda Capital
  222. JumpStart
  223. Juventas Therapeutics
  224. Kiwoom Securities
  225. Kolon TissueGene
  226. Krystal Biotech
  227. Kurma Partners
  228. Lacerta Therapeutics
  229. Leerink Partners
  230. Lentigen Technology, a Miltenyi Biotec Company
  231. Life Sciences Partners (LSP)
  232. Lilly Asia Ventures
  233. LIME Asset Management
  234. LogicBio Therapeutics
  235. Lokon Pharma
  236. Longitude Capital
  237. Lonza
  238. Ludwig Institute for Cancer Research
  239. Lundbeckfonden Ventures
  240. Lysogene
  241. Marsala Biotech
  242. Maryland Technology Development Corporation (TEDCO)
  243. Maverick Ventures
  244. MaxCyte
  245. Mayo Clinic
  246. MedImmune
  247. Medison Ventures
  248. MeiraGTx
  249. Memorial Sloan Kettering Cancer Center
  250. Mercia Technologies
  251. Merck
  252. Mercury Fund
  253. MERITZ Securities
  254. Mesa Verde Venture Partners
  255. Michigan Economic Development 
  256. MidCap Financial
  257. Midven
  258. Milo Biotechnology
  259. Mirae Asset
  260. Mita Securities
  261. Mizuho Capital Partners
  262. MOLOGEN
  263. Momotaro-Gene
  264. Morningside Ventures
  265. MultiVir
  266. Mustang Bio
  267. Myonexus Therapeutics
  268. NanoCor Therapeutics
  269. Nanogenic Solutions
  270. National Institute of Allergy and Infectious Diseases
  271. National Institutes of Health (NIH)
  272. National MPS Society
  273. National Securities
  274. Nationwide Children's Hospital
  275. Nature Technology
  276. Nerveda
  277. Neuracle Genetics
  278. Neuralgene
  279. Neurocrine Biosciences
  280. Neurogene
  281. Neurotech Pharmaceuticals
  282. New Energy and Industrial Technology Development Organization
  283. New Enterprise Associates (NEA)
  284. New Leaf Venture Partners
  285. Nightstar Therapeutics
  286. Nissei Capital
  287. Nohla Therapeutics
  288. Nottingham Technology Grant Fund
  289. Novartis
  290. Oberland Capital
  291. Ocugen
  292. Office of Commercialization, Washington State University
  293. Omega Funds
  294. Omnes Capital
  295. Oncolys BioPharma
  296. Oncos Therapeutics
  297. OncoSec Medical
  298. OrbiMed
  299. Orbit Biomedical
  300. ORCA Therapeutics
  301. Orchard Therapeutics
  302. ORI Healthcare Fund
  303. Osage University Partners
  304. Oxford BioMedica
  305. Oxford Finance
  306. Panmure Gordon
  307. Pappas Capital
  308. Parker Institute for Cancer Immunotherapy
  309. Partner Fund Management
  310. Partners Innovation Fund
  311. Passage Bio
  312. Pattern BioSciences
  313. Pavilion Capital
  314. Pentwater Capital Management
  315. Perceptive Advisors
  316. PeriphaGen
  317. Perseverance Capital Management
  318. Pfizer
  319. PlasmaTech Biopharmaceuticals
  320. PNP Therapeutics
  321. Pontifax Venture Capital
  322. Posco Capital Partners
  323. Poseida Therapeutics
  324. Precision BioSciences
  325. Precision Genome Engineering
  326. Prevail Therapeutics
  327. Promethera Biosciences
  328. Prostate Cancer Foundation
  329. PsiOxus Therapeutics
  330. PTC Therapeutics
  331. Quethera 
  332. QVT Financial
  333. RA Capital Management
  334. RBV Capital
  335. Red Sanfilippo Foundation
  336. Redmile Group
  337. Regeneron Pharmaceuticals
  338. REGENXBIO
  339. Renova Therapeutics
  340. Renovacor
  341. Rentschler Biopharma
  342. RetroSense Therapeutics
  343. Rev1 Ventures
  344. Reyon Pharmaceutical
  345. Ridgeback Capital
  346. Roche
  347. Rock Springs Capital
  348. Rocket Pharmaceuticals
  349. RTW Investments
  350. Ryboquin
  351. Sabby Capital
  352. Samsara BioCapital
  353. Sanfilippo Children’s Foundation
  354. Sangamo Therapeutics
  355. Sanofi
  356. Sarepta Therapeutics
  357. Santhera Pharmaceuticals
  358. SBI Japan-Israel Innovation Fund
  359. Scancell
  360. SDL Ventures
  361. Sectoral Asset Management
  362. Selecta Biosciences
  363. Seventure Partners
  364. Sham Innovation Santé
  365. Shanghai Sunway Biotech
  366. Shavit Capital
  367. Shenzhen Qianhai Taxus
  368. Shinhan Capital
  369. Shinsei Corporate Investment
  370. Shire
  371. Sibiono GeneTech
  372. SillaJen
  373. SIRION Biotech
  374. SMBC Venture Capital
  375. Sofinnova Ventures
  376. Solid Biosciences
  377. SOSV
  378. Spark Therapeutics
  379. Sphera Global Health Care Fund
  380. SR-TIGET
  381. St. Jude Children’s Research Hospital
  382. Stop Sanfilippo Foundation
  383. StrideBio
  384. Surveyor Capital (a Citadel company)
  385. SV Health Investors
  386. SV Life Sciences
  387. SVE Capital
  388. T. Rowe Price Associates
  389. Takeda
  390. Target ALS Foundation
  391. Targeted Technology Funds
  392. Targovax
  393. Tasly Biopharmaceuticals
  394. Taxus Cardium Pharmaceuticals
  395. Tech Coast Angels
  396. Technium Partners
  397. Temasek
  398. Tenaya Therapeutics
  399. The Board of Trustees of the Leland Stanford Junior University
  400. The Column Group (TCG)
  401. The Movember Foundation
  402. TheraBiologics
  403. THERAVECTYS
  404. Third Rock Ventures
  405. TNK Therapeutics, subsidiary of Sorrento Therapeutics
  406. Tocagen
  407. TPG Capital
  408. Transgene
  409. Transposagen Biopharmaceuticals
  410. U.S. Army’s Small Business Innovation Research
  411. UB Securities
  412. UC Davis Medical Center
  413. UK Cystic Fibrosis Gene Therapy Consortium
  414. Ultragenyx Pharmaceutical
  415. uniQure Biopharma
  416. Universitat Autònoma de Barcelona (UAB)
  417. University College London
  418. University of California
  419. University of Massachusetts Medical School
  420. University of Oxford
  421. University of Pennsylvania
  422. Urovant Sciences
  423. UT Southwestern Medical Center
  424. V-Bio Ventures
  425. VBL Therapeutics
  426. VCN Biosciences
  427. Vector Neurosciences
  428. Venrock
  429. Versant Ventures
  430. Vertex Pharmaceuticals
  431. Verve Therapeutics
  432. Vessl™
  433. Vical
  434. Vida Ventures
  435. Viking Global Investors
  436. Virginia Catalyst
  437. ViroMed
  438. Virttu Biologics
  439. Vivet Therapeutics
  440. Vivo Capital
  441. Voyager Therapeutics
  442. Washington Research Foundation
  443. Weill Cornell Medical College
  444. Wellcome Trust
  445. Wellington Capital Management
  446. Wellstat Ophthalmics
  447. Whitesun Healthcare Ventures
  448. WI Harper Group
  449. Woodford Investment Management
  450. Wyvern Pharmaceuticals
  451. XyloCor Therapeutics
  452. Ysios Capital
  453. Yuhan Corporation
  454. Ziopharm Oncology

Segmentation

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

  • Key therapeutic areas
    • Cardiovascular disorders 
    • Muscle-related disorders
    • Nervous system disorders
    • Ophthalmic disorders
    • Oncological disorders
    • Others
  • Type of vector
    • Adeno associated virus
    • Adenovirus
    • Lentivirus
    • Plasmid DNA
    • Retrovirus
    • Others
  • Type of therapy
    • Ex vivo
    • In vivo
  • Type of gene modification
    • Gene augmentation
    • Immunotherapy
    • Oncolytic viral therapy
    • Others
  • Route of administration
    • Intravenous
    • Intravitreal
    • Intravesical
    • Intracerebellar
    • Subretinal
    • Others
  • Key geographical regions 
    • US
    • EU5
    • Rest of the World

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