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Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030

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    November 2020

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Overview

Over time, several gene therapies have been developed for the treatment of both simple (single defect) and complex (multiple defects) genetic disorders. In fact, there are 10 approved gene therapies (recent examples include Zolgensma®, Zynteglo™ and Collategene®), till date, and more than a thousand product candidates being evaluated in clinical trials, worldwide. Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods (such as genome sequencing), and genome editing and manipulation technologies (such as molecular switches), have enabled the development of novel therapy development tools / platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

Scope of the Report

The “Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030” report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene 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 market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).
  • A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain.
  • An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.
  • 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, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies. 
  • Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company.
  • An elaborate discussion on the various commercialization strategies that can be adopted by drug developers across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing. 
  • A review of the 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 various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2016. The analysis assesses several relevant parameters associated with the patents, including type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation.
  • A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company’ product and target therapeutic area.
  • An analysis of the investments made at various stages of development in companies that are focused in this area, between 2015-2020, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings.
  • A detailed geographical clinical trial analysis of completed, ongoing and planned studies of numerous gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.
  • An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.
  • 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. 
  • An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.
  • A case study on the prevalent and emerging trends related to vector manufacturing, along 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.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with 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 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vectors used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] 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:

  • Adam Rogers (CEO, Hemera Biosciences)
  • Al Hawkins (CEO, Milo Biotechnology)
  • Buel Dan Rodgers (Founder & CEO, AAVogen)
  • Christopher Reinhard (CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics))
  • Michael Triplett (CEO, Myonexus Therapeutics)
  • Robert Jan Lamers (CEO, Arthrogen)
  • Ryo Kubota (CEO, Chairman & President, Acucela)
  • Tom Wilton (CBO, LogicBio Therapeutics)
  • Jeffrey Hung (CCO, Vigene Biosciences)
  • Cedric Szpirer (Executive & Scientific Director, Delphi Genetics)
  • Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory)
  • Molly Cameron (Corporate Communications Manager, Orchard 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.

Key Questions Answered

  • Who are the leading industry players engaged in the development of gene therapies? 
  • How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products? 
  • Which types of vectors are most commonly used for effective delivery of gene therapies?
  • What are the key regulatory requirements for gene therapy approval, across various geographies?
  • Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?
  • What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?
  • What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?
  • Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development?
  • What are the key value drivers of the merger and acquisition activity in the gene therapy industry?
  • Who are the key stakeholders that have actively made investments in the gene therapy domain?
  • Which are the most active trial sites (in terms of number of clinical studies being conducted) related to this domain?
  • How is the current and future market opportunity likely to be distributed across key market segments?

Contents

Chapter Outline

Chapter 2 provides 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 further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it 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 and limitations 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, Australia, China, Hong Kong, Japan and South Korea. 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 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.

Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. 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 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the company’s patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any). 

Chapter 11 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 features 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 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic disorders, metabolic disorders, nervous system disorders, oncological disorders and ophthalmic diseases. 

Chapter 13 provides an overview of the various patents that have been filed / granted in relation to gene therapy and gene editing technologies. It also features a detailed analysis, highlighting the prevalent trends related to type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation. For the purpose of this analysis, we have taken into consideration patents that have been filed / granted since 2016. 

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company’ product and target therapeutic area.

Chapter 15 presents details on various funding instances, investments and grants reported 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 2015 and 2020, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents a detailed, geographical clinical trial analysis of completed, ongoing and planned studies focused on gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 highlights top big biopharma players engaged in the field of gene therapy, featuring a heat map analysis based on several parameters, including therapeutic area, type of vector used, therapeutic approach and type of gene therapy.

Chapter 19 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 20 presents an elaborate market 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 [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vector used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world).

Chapter 21 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. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors. 

Chapter 22 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain. 

Chapter 23 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 24 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), Christopher Reinhard (CEO & Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (CEO, Chairman & President, Acucela), Tom Wilton (CBO, LogicBio Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Molly Cameron (Corporate Communications Manager, Orchard Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 25 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 26 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. Key Questions Answered
1.4. 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 Therapies
3.3.2. Ex Vivo and In Vivo Therapies
3.4. Routes of Administration
3.5. Mechanism of Action of Gene Therapies
3.6. Overview of the Basic Concepts of Gene Editing
3.6.1. Evolution of Genome Editing 
3.6.2. Applications of Genome Editing
3.6.3. Genome Editing Techniques

 

3.7. Advantages and Disadvantages of Gene Therapies
3.8. Ethical and Social Concerns Related to Gene Therapies
3.9. Constraints and Challenges Related to Gene Therapies
3.9.1. Therapy Development Concerns
3.9.2. Manufacturing Concerns
3.9.3. Commercial Viability Concerns

4. GENE DELIVERY VECTORS
4.1. Chapter Overview
4.2. Viral and Non-Viral Methods of Gene Transfer
4.3. Viral Vectors for Genetically Modified Therapies
4.4. Types of Viral Vectors
 
4.4.1. Adeno-associated Viral Vectors
4.4.1.1. Overview
4.4.1.2. Design and Manufacturing
4.4.1.3. Advantages
4.4.1.4. Limitations
 
4.4.2. Adenoviral Vectors
4.4.2.1. Overview
4.4.2.2. Design and Manufacturing
4.4.2.3. Advantages
4.4.2.4. Limitations
 
4.4.3. Lentiviral Vectors
4.4.3.1. Overview
4.4.3.2. Design and Manufacturing
4.4.3.3. Advantages
4.4.3.4. Limitations
 
4.4.4. Retroviral Vectors
4.4.4.1. Overview
4.4.4.2. Design and Manufacturing
4.4.4.3. Advantages
4.4.4.4. Limitations
 
4.4.5. Other Viral Vectors
4.4.5.1. Alphavirus
4.4.5.2. Foamy Virus
4.4.5.3. Herpes Simplex Virus
4.4.5.4. Sendai Virus
4.4.5.5. Simian Virus
4.4.5.6. Vaccinia Virus
 
4.5. Types of Non-Viral Vectors
4.5.1. Plasmid DNA
4.5.2. Liposomes, Lipoplexes and Polyplexes
4.5.3. Oligonucleotides
4.5.4. Other Non-Viral Vectors
 
4.5.5. Gene Delivery Using Non-Viral Vectors
4.5.5.1. Biolistic Methods
4.5.5.2. Electroporation
4.5.5.3. Receptor Mediated Gene Delivery
4.5.5.4. 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
5.4.5. Hong Kong Scenario
 
5.5. Reimbursement Scenario
5.5.1. Challenges Related to Reimbursement
5.6. Commonly Offered Payment Models for Gene Therapies
 
6. MARKET OVERVIEW
6.1. Chapter Overview
6.1.1. Analysis by Phase of Development of Gene Therapy
6.2. Gene Therapy Market: Clinical and Commercial 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 Therapeutic Approach
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 Phase 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 Therapeutic Approach
6.3.6. Analysis by Type of Gene Therapy
  
6.4. Gene Therapy Candidates: Special Designations 
6.4.1. Analysis by Special Designation(s) Awarded
6.5. Grid Representation: Analysis by Phase of Development, Therapeutic Area and Therapeutic Approach
 
7. COMPETITIVE LANDSCAPE
7.1. Chapter Overview
7.2. Gene Therapy Market: List of Developers
7.2.1. Analysis by Year of Establishment
7.2.2. Analysis by Company Size
7.2.3. Analysis by Geography
7.3. Key Players: Analysis by Number of Product Candidates
 
8. MARKETED GENE THERAPIES
8.1. Chapter Overview
8.2. Gendicine® (Shenzhen Sibiono GeneTech)
8.2.1. Company Overview
8.2.2. Development Timeline
8.2.3. Mechanism of Action and Vectors Used
8.2.4. Target Indication(s)
8.2.5. Current Status of Development
8.2.6. Manufacturing, Dosage and Sales
 
8.3. Oncorine® (Shanghai Sunway Biotech)
8.3.1. Company Overview
8.3.2. Development Timeline
8.3.3. Mechanism of Action and Vectors Used
8.3.4. Target Indication(s)
8.3.5. Current Status of Development
8.3.6. Manufacturing, Dosage and Sales
 
8.4. Rexin-G® (Epeius Biotechnologies)
8.4.1. Company Overview
8.4.2. Development Timeline
8.4.3. Mechanism of Action and Vector Used
8.4.4. Target Indication(s)
8.4.5. Current Status of Development
8.4.6. Manufacturing, Dosage and Sales
 
8.5. Neovasculgen® (Human Stem Cells Institute)
8.5.1. Company Overview
8.5.2. Development Timeline 
8.5.3. Mechanism of Action and Vectors Used
8.5.4. Target Indication(s)
8.5.5. Current Status of Development
8.5.6. Manufacturing, Dosage and Sales
 
8.6. Imlygic® (Amgen)
8.6.1. Company Overview
8.6.2. Development Timeline 
8.6.3. Mechanism of Action and Vectors Used
8.6.4. Target Indication(s)
8.6.5. Current Status of Development
8.6.6. Manufacturing, Dosage and Sales
 
8.7. Strimvelis® (Orchard Therapeutics)
8.7.1. Company Overview
8.7.2. Development Timeline 
8.7.3. Mechanism of Action and Vectors Used
8.7.4. Target Indication(s)
8.7.5. Current Status of Development
8.7.6. Manufacturing, Dosage and Sales
 
8.8. Luxturna™ (Spark Therapeutics)
8.8.1. Company Overview
8.8.2. Development Timeline 
8.8.3. Mechanism of Action and Vector Used
8.8.4. Target Indication(s)
8.8.5. Current Status of Development
8.8.6. Manufacturing, Dosage and Sales
 
8.9. Zolgensma™ (Novartis Gene Therapies)
8.9.1. Company Overview
8.9.2. Development Timeline 
8.9.3. Mechanism of Action and Vector Used
8.9.4. Target Indication(s)
8.9.5. Current Status of Development
8.9.6. Manufacturing, Dosage and Sales
 
8.10. Collategene® (AnGes)
8.10.1. Company Overview
8.10.2. Development Timeline 
8.10.3. Mechanism of Action and Vector Used
8.10.4. Target Indication(s)
8.10.5. Current Status of Development
8.10.6. Manufacturing, Dosage and Sales
 
8.11. Zyntelgo™ (bluebird bio)
8.11.1. Company Overview
8.11.2. Development Timeline 
8.11.3. Mechanism of Action and Vector Used
8.11.4. Target Indication(s)
8.11.5. Current Status of Development
8.11.6. Manufacturing, Dosage and Sales
 
9. KEY COMMERCIALIZATION STRATEGIES
9.1. Chapter Overview
9.2. Successful Drug Launch Strategy: ROOTS Framework
9.3. Successful Drug Launch Strategy: Product Differentiation
9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product
9.5. List of Currently Approved Gene Therapies
9.6. Key Commercialization Strategies Adopted by Companies Focused on Gene Therapy
9.6.1. Strategies Adopted Before Therapy Approval 
9.6.2. Strategies Adopted During / Post Therapy Approval 
9.7. Concluding Remarks
 
10. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES
10.1. Chapter Overview
10.2. GS010
10.3. PTC-AADC
10.4. BMN 270
10.5. rAd-IFN/Syn3
10.6. TAVO™
10.7. OTL-200
10.8. EB-101
10.9. ProstAtak®
10.10. BIIB111
10.11. LentiGlobin BB305®
10.12. Lenti-D™
10.13. FCX-007
10.14. CG0070
10.15. Vigil®
10.16. VM202
10.17. VGX-3100
10.18. Invossa™
10.19. KB103
10.20. PF-06939926
10.21. PF-06838435
10.22. PF-07055480
10.23. SPK-8011
10.24. AMT-061
10.25. VB-111
10.26. OTL-101
10.27. BIIB112
10.28. FLT180a
10.29. LYS-SAF302
10.30. NFS-01
10.31. OTL-103
 
11. EMERGING TECHNOLOGIES
11.1. Chapter Overview
11.2. Gene Editing Technologies
11.2.1. Overview
11.2.2. Applications
 
11.3. Emerging Gene Editing Platforms
11.3.1. CRISPR / Cas9 System
11.3.2. TALENs
11.3.3. megaTAL
11.3.4. Zinc Finger Nuclease
 
11.4. Gene Expression Regulation Technologies
11.5. Technology Platforms for Developing / Delivering Gene Therapies
 
12. KEY THERAPEUTICS AREAS
12.1. Chapter Overview
12.2. Analysis by Therapeutic Area and Special Designation(s) Awarded
 
12.3. Oncological Disorders
12.3.1. Analysis by Target Indication
12.3.2. Analysis by Type of Vector Used
 
12.4. Nervous System Disorders
12.4.1. Analysis by Target Indication
12.4.2. Analysis by Type of Vector Used
 
12.5. Metabolic Disorders
12.5.1. Analysis by Target Indication
12.5.2. Analysis by Type of Vector Used
 
12.6. Ophthalmic Diseases
12.6.1. Analysis by Target Indication
12.6.2. Analysis by Type of Vector Used
 
12.7. Genetic Disorders
12.7.1. Analysis by Target Indication
12.7.2. Analysis by Type of Vector Used
 
13. PATENT ANALYSIS
13.1. Chapter Overview
13.2. Gene Therapy Market: List of Patents 
13.2.1. Scope and Methodology
13.2.1.1. Analysis by Publication Year
13.2.1.2. Analysis by Publication Year and Type of Patent
13.2.1.3. Analysis by Geography 
13.2.1.4. Analysis by CPC Symbols 
13.2.1.5. Analysis by Emerging Focus Areas
13.2.1.6. Leading Players: Analysis by Number of Patents
13.2.1.7. Patent Benchmark Analysis
13.2.1.8. Patent Valuation Analysis
 
13.3. Gene Editing Market: List of Patents 
13.3.1. Scope and Methodology
13.3.1.1. Analysis by Publication Year
13.3.1.2. Analysis by Publication Year and Type of Patent
13.3.1.3. Analysis by Geography 
13.3.1.4. Analysis by CPC Symbols 
13.3.1.5. Analysis by Emerging Focus Areas
13.3.1.6. Leading Players: Analysis by Number of Patents
13.3.1.7. Patent Benchmark Analysis
13.3.1.8. Patent Valuation Analysis
13.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization
 
14. MERGERS AND ACQUISITIONS
14.1. Chapter Overview
14.2. Merger and Acquisition Models
14.3. Gene Therapy Market: Mergers and Acquisitions
14.3.1. Analysis by Year of Agreement 
14.3.2. Analysis by Type of Agreement
14.3.3. Analysis by Geography 
14.3.3.1. Continent-wise Distribution
14.3.3.2. Intercontinental and Intracontinental Deals
14.3.3.3. Country-wise Distribution
 
14.3.4. Analysis by Key Value Drivers
14.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
14.3.5. Analysis by Phase of Development of the Acquired Company’s Product
14.3.6. Analysis by Therapeutic Area
 
15. FUNDING AND INVESTMENT ANALYSIS
15.1. Chapter Overview
15.2. Types of Funding
15.3. Gene Therapy Market: Funding and Investment Analysis
15.3.1. Analysis by Number of Funding Instances
15.3.2. Analysis by Amount Invested
15.3.3. Analysis by Type of Funding
15.3.4. Analysis by Type of Funding and Year
15.3.5. Analysis of Amount Invested by Type of Gene Therapy 
15.3.6. Analysis of Amount Invested by Geography 
15.3.7. Most Active Players: Analysis by Number of Funding Instances and Amount Invested
15.3.8. Key Investors: Analysis by Number of Funding Instances
15.3.9. Analysis by Phase of Development
15.3.10. Analysis by Therapeutic Area
15.4. Concluding Remarks
 
16. CLINICAL TRIAL ANALYSIS
16.1. Chapter Overview
16.2. Scope and Methodology
16.3. Gene Therapy Market: Clinical Trial Analysis
16.3.1. Analysis by Trial Registration Year
16.3.2. Analysis by Trial Status
16.3.3. Analysis by Trial Phase
16.3.4. Analysis by Therapeutic Area
16.3.5. Analysis by Geography
16.3.6. Geographical Analysis by Trial Registration Year
16.3.7. Geographical Analysis by Trial Status
16.3.8. Geographical Analysis by Trial Status and Trial Phase
16.3.9. Geographical Analysis by Therapeutic Area
16.4. Analysis by Type of Sponsor
16.5. Analysis by Prominent Treatment Sites
 
16.6. Gene therapy Market: Analysis of Enrolled Patient Population
16.6.1. Analysis by Trial Registration Year
16.6.2. Analysis by Trial Status
16.6.3. Analysis by Trial Phase
16.6.4. Analysis by Therapeutic Area
16.6.5. Analysis by Location of Trial Site
16.6.6. Analysis by Trial Status and Location of Trial Site
16.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
16.6.8. Analysis by Therapeutic Area and Location of Trial Site
 
16.7. Concluding Remarks
16.7.1. Emerging Molecules to Watch
16.7.2. Most Important Trials to Watch
 
17. COST PRICE ANALYSIS
17.1. Chapter Overview
17.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
17.3. Gene Therapy Market: Pricing Models
17.3.1. Based on Associated Product / Component Costs
17.3.2. Based on Competition
17.3.3. Based on Patient Segment
17.3.4. Based on Opinions of Industry Experts
 
18. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES
18.1. Chapter Overview
18.2. Gene Therapy Market: List of Most Prominent Big Pharmaceutical Players 
18.2.1. Analysis by Therapeutic Area
18.2.2. Analysis by Type of Vector Used
18.2.3. Analysis by Therapeutic Approach
18.2.4. Analysis by Type of Gene Therapy
18.3. Other Big Pharma Players
 
19. DEMAND ANALYSIS
19.1. Chapter Overview
19.2. Assumptions and Methodology
19.3. Clinical Demand for Gene Therapies
19.4. Commercial Demand for Gene Therapies
 
20. MARKET FORECAST AND OPPORTUNITY ANALYSIS
20.1. Chapter Overview
20.2. Scope and Limitations
20.3. Key Assumptions and Forecast Methodology
20.4. Global Gene Therapy Market, 2020-2030
20.4.1. Gene Therapy Market: Analysis by Type of Gene Modification
20.4.2. Gene Therapy Market: Analysis by Type of Therapy
20.4.3. Gene Therapy Market: Analysis by Type of Vector Used
20.4.4. Gene Therapy Market: Analysis by Therapeutic Area
20.4.5. Gene Therapy Market: Analysis by Route of Administration
20.4.6. Gene Therapy Market: Analysis by Geography
 
20.5. Gene Therapy Market: Value Creation Analysis
20.6. Gene Therapy Market: Product-wise Sales Forecasts
20.6.1. Gendicine®
20.6.1.1. Target Patient Population
20.6.1.2. Sales Forecast
20.6.1.3. Net Present Value
20.6.1.4. Value Creation Analysis
 
20.6.2. Oncorine®
20.6.2.1. Target Patient Population
20.6.2.2. Sales Forecast
20.6.2.3. Net Present Value
20.6.2.4. Value Creation Analysis
 
20.6.3. Rexin-G®
20.6.3.1. Target Patient Population
20.6.3.2. Sales Forecast
20.6.3.3. Net Present Value
20.6.3.4. Value Creation Analysis
 
20.6.4. Neovasculgen®
20.6.4.1. Target Patient Population
20.6.4.2. Sales Forecast
20.6.4.3. Net Present Value
20.6.4.4. Value Creation Analysis
 
20.6.5. Strimvelis®
20.6.5.1. Target Patient Population
20.6.5.2. Sales Forecast
20.6.5.3. Net Present Value
20.6.5.4. Value Creation Analysis
 
20.6.6. Imlygic® 
20.6.6.1. Target Patient Population
20.6.6.2. Sales Forecast
20.6.6.3. Net Present Value
20.6.6.4. Value Creation Analysis
 
20.6.7. Luxturna™
20.6.7.1. Target Patient Population
20.6.7.2. Sales Forecast
20.6.7.3. Net Present Value
20.6.7.4. Value Creation Analysis
 
20.6.8. Zolgensma™
20.6.8.1. Target Patient Population
20.6.8.2. Sales Forecast
20.6.8.3. Net Present Value
20.6.8.4. Value Creation Analysis
 
20.6.9. Collategene®
20.6.9.1. Target Patient Population
20.6.9.2. Sales Forecast
20.6.9.3. Net Present Value
20.6.9.4. Value Creation Analysis
 
20.6.10. Zyntelgo™
20.6.10.1. Target Patient Population
20.6.10.2. Sales Forecast
20.6.10.3. Net Present Value
20.6.10.4. Value Creation Analysis
 
20.6.11. GS010
20.6.11.1. Target Patient Population
20.6.11.2. Sales Forecast
20.6.11.3. Net Present Value
20.6.11.4. Value Creation Analysis
 
20.6.12. PTC-AADC
20.6.12.1. Target Patient Population
20.6.12.2. Sales Forecast
20.6.12.3. Net Present Value
20.6.12.4. Value Creation Analysis
 
20.6.13. BMN 270
20.6.13.1. Target Patient Population
20.6.13.2. Sales Forecast
20.6.13.3. Net Present Value
20.6.13.4. Value Creation Analysis
 
20.6.14. rAd-IFN/Syn3
20.6.14.1. Target Patient Population
20.6.14.2. Sales Forecast
20.6.14.3. Net Present Value
20.6.14.4. Value Creation Analysis
 
20.6.15. TAVO™
20.6.15.1. Target Patient Population
20.6.15.2. Sales Forecast
20.6.15.3. Net Present Value
20.6.15.4. Value Creation Analysis
 
20.6.16. OTL-200
20.6.16.1. Target Patient Population
20.6.16.2. Sales Forecast
20.6.16.3. Net Present Value
20.6.16.4. Value Creation Analysis

20.6.17. EB-101
20.6.17.1. Target Patient Population
20.6.17.2. Sales Forecast
20.6.17.3. Net Present Value
20.6.17.4. Value Creation Analysis
 
20.6.18. ProstAtak®
20.6.18.1. Target Patient Population
20.6.18.2. Sales Forecast
20.6.18.3. Net Present Value
20.6.18.4. Value Creation Analysis
 
20.6.19. BIIB111
20.6.19.1. Target Patient Population
20.6.19.2. Sales Forecast
20.6.19.3. Net Present Value
20.6.19.4. Value Creation Analysis

20.6.20. LentiGlobin BB305®
20.6.20.1. Target Patient Population
20.6.20.2. Sales Forecast
20.6.20.3. Net Present Value
20.6.20.4. Value Creation Analysis

20.6.21. Lenti-D™
20.6.21.1. Target Patient Population
20.6.21.2. Sales Forecast
20.6.21.3. Net Present Value
20.6.21.4. Value Creation Analysis

20.6.22. FCX-007
20.6.22.1. Target Patient Population
20.6.22.2. Sales Forecast
20.6.22.3. Net Present Value
20.6.22.4. Value Creation Analysis

20.6.23. CG0070
20.6.23.1. Target Patient Population
20.6.23.2. Sales Forecast
20.6.23.3. Net Present Value
20.6.23.4. Value Creation Analysis

20.6.24. Vigil®
20.6.24.1. Target Patient Population
20.6.24.2. Sales Forecast
20.6.24.3. Net Present Value
20.6.24.4. Value Creation Analysis

20.6.25. VM202
20.6.25.1. Target Patient Population
20.6.25.2. Sales Forecast
20.6.25.3. Net Present Value
20.6.25.4. Value Creation Analysis

20.6.26. VGX-3100
20.6.26.1. Target Patient Population
20.6.26.2. Sales Forecast
20.6. 26.3. Net Present Value
20.6.26.4. Value Creation Analysis

20.6.27. Invossa™
20.6.27.1. Target Patient Population
20.6.27.2. Sales Forecast
20.6.27.3. Net Present Value
20.6.27.4. Value Creation Analysis

20.6.28. KB103
20.6.28.1. Target Patient Population
20.6.28.2. Sales Forecast
20.6.28.3. Net Present Value
20.6.28.4. Value Creation Analysis

20.6.29. PF-06939926
20.6.29.1. Target Patient Population
20.6.29.2. Sales Forecast
20.6.29.3. Net Present Value
20.6.29.4. Value Creation Analysis

20.6.30. PF-06838435
20.6.30.1. Target Patient Population
20.6.30.2. Sales Forecast
20.6.30.3. Net Present Value
20.6.30.4. Value Creation Analysis

20.6.31. PF-07055480
20.6.31.1. Target Patient Population
20.6.31.2. Sales Forecast
20.6.31.3. Net Present Value
20.6.31.4. Value Creation Analysis

20.6.32. SPK-8011
20.6.32.1. Target Patient Population
20.6.32.2. Sales Forecast
20.6.32.3. Net Present Value
20.6.32.4. Value Creation Analysis

20.6.33. AMT-061
20.6.33.1. Target Patient Population
20.6.33.2. Sales Forecast
20.6.33.3. Net Present Value
20.6.33.4. Value Creation Analysis

20.6.34. VB-111
20.6.34.1. Target Patient Population
20.6.34.2. Sales Forecast
20.6.34.3. Net Present Value
20.6.34.4. Value Creation Analysis

20.6.35. OTL-101
20.6.35.1. Target Patient Population
20.6.35.2. Sales Forecast
20.6.35.3. Net Present Value
20.6.35.4. Value Creation Analysis

20.6.36. BIIB112
20.6.36.1. Target Patient Population
20.6.36.2. Sales Forecast
20.6.36.3. Net Present Value
20.6.36.4. Value Creation Analysis

20.6.37. FLT180a
20.6.37.1. Target Patient Population
20.6.37.2. Sales Forecast
20.6.37.3. Net Present Value
20.6.37.4. Value Creation Analysis

20.6.38. LYS-SAF302
20.6.38.1. Target Patient Population
20.6.38.2. Sales Forecast
20.6.38.3. Net Present Value
20.6.38.4. Value Creation Analysis

20.6.39. NFS-01
20.6.39.1. Target Patient Population
20.6.39.2. Sales Forecast
20.6.39.3. Net Present Value
20.6.39.4. Value Creation Analysis

20.6.40. OTL-103
20.6.40.1. Target Patient Population
20.6.40.2. Sales Forecast
20.6.40.3. Net Present Value
20.6.40.4. Value Creation Analysis

21. VECTOR MANUFACTURING
21.1. Chapter Overview
21.2. Overview of Viral Vector Manufacturing
21.3. Viral Vector Manufacturing Processes
21.3.1. Mode of Vector Production
21.3.2. Adherent and Suspension Cultures
21.3.3. Unit and Parallel Processes
21.3.4. Cell Culture Systems for Production of Viral Vectors
21.3.5. Culture Media Specifications 

21.4. Bioprocessing of Viral Vectors
21.4.1. Adeno-associated Viral Vector Production
21.4.2. Adenoviral Vector Production
21.4.3. Lentiviral Vector Production
21.4.4. ? -Retroviral Vector Production

21.5. Challenges Associated with Vector Manufacturing
21.6. Companies Offering Contract Services for Viral and Plasmid Vectors

22. CASE STUDY: GENE THERAPY SUPPLY CHAIN
22.1. Chapter Overview
22.2. Overview of the Gene Therapy Supply Chain
22.3. Implementation of Supply Chain Models
22.4. Logistics Associated with Gene Therapies
22.4.1. Autologous and Allogeneic Therapies
22.5. Regulatory Supply Chain Across the Globe
22.6. Challenges Associated with Gene Therapy Supply Chain

22.7. Optimizing Cell and Advanced Therapies Supply Chain Management
22.7.1. Enterprise Manufacturing Systems
22.7.2. Laboratory Information Management Systems
22.7.3. Inventory Management Systems
22.7.4. Quality Management Systems
22.7.5. Logistics Management Systems
22.7.6. Patient Management Systems
22.7.7. Electronic Clinical Outcome Assessments Systems
22.7.8. Supply Chain Orchestration Platforms
22.8. Recent Developments and Upcoming Trends

23. CONCLUSION

24. INTERVIEW TRANSCRIPTS
24.1. Chapter Overview
24.2. Adam Rogers, CEO, Hemera Biosciences 
24.3. Al Hawkins, CEO, Milo Biotechnology 
24.4. Buel Dan Rodgers, Founder and CEO, AAVogen 
24.5. Christopher Reinhard, CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)
24.6. Michael Tripletti, CEO, Myonexus Therapeutics
24.7. Robert Jan Lamers, CEO, Arthrogen 
24.8. Ryo Kubota, CEO, Chairman and President, Acucela
24.9. Tom Wilton, CBO, LogicBio Therapeutics
24.10.  Jeffrey Hung, CCO, Vigene Biosciences 
24.11. Cedric Szpirer, Executive & Scientific Director, Delphi Genetics 
24.12. Marco Schmeer, Project Manager and Tatjana Buchholz, Marketing Manager, PlasmidFactory 
24.13. Molly Cameron, Corporate Communications Manager, Orchard Therapeutics

25. APPENDIX 1: TABULATED DATA

26. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

List of Figures

Figure 3.1 Evolution of Gene Therapies
Figure 3.2 Ex Vivo Gene Therapies
Figure 3.3 In Vivo Gene Therapies
Figure 3.4 Mechanism of Action of Gene Therapies
Figure 3.5 Genome Editing: Historical Milestones
Figure 3.6 Genome Editing Techniques
Figure 3.7 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 the 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: Distribution by Phase of Development
Figure 6.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
Figure 6.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
Figure 6.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Figure 6.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
Figure 6.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene
Figure 6.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Modification
Figure 6.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Modification
Figure 6.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Therapy
Figure 6.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of
Figure 6.11 Early Stage Pipeline of Gene Therapies: Distribution by Phase of Development
Figure 6.12 Early Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
Figure 6.13 Early Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Figure 6.14 Early Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
Figure 6.15 Early Stage Pipeline of Gene Therapies: Distribution by Type of Gene
Figure 6.16 Early Stage Pipeline of Gene Therapies: Distribution by Therapeutic Approach
Figure 6.17 Early Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used and Modification
Figure 6.18 Early Stage Pipeline of Gene Therapies: Distribution by Type of Gene Therapy
Figure 6.19 Early Stage Pipeline of Gene Therapies: Distribution by Route of Administration
Figure 6.20 Gene Therapies: Distribution by Special Designation(s) Awarded
Figure 6.21 Grid Representation: Distribution by Phase of Development, Therapeutic Area and Therapeutic Approach
Figure 7.1 Gene Therapy Developers: Distribution by Year of Establishment
Figure 7.2 Gene Therapy Developers: Distribution by Company Size
Figure 7.3 Gene Therapy Developers: Distribution by Location of Headquarters
Figure 7.4 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
Figure 7.5 Gene Therapy Developers: Most Prominent Hubs
Figure 7.6 Gene Therapy Developers: Distribution of Players based in North America
Figure 7.7 Gene Therapy Developers: Distribution of Players based in Europe
Figure 7.8 Gene Therapy Developers: Distribution of Players based in Asia-Pacific
Figure 7.9 Gene Therapy Developers: Key Players
Figure 7.10 Gene Therapy Developers: Distribution by Key Players and Phase of Development
Figure 8.1 Gendicine®: Development Timeline
Figure 8.2 Oncorine®: Development Timeline
Figure 8.3 Oncorine®: Mechanism of Action
Figure 8.4 Rexin-G®: Development Timeline
Figure 8.5 Neovasculgen®: Development Timeline
Figure 8.6 Imlygic®: Development Timeline
Figure 8.7 Imlygic®: Mechanism of Action
Figure 8.8 Strimvelis®: Development Timeline
Figure 8.9 Luxturna™: Development Timeline
Figure 8.10 Zolgensma™: Development Timeline
Figure 8.11 Collategene®: Development Timeline
Figure 8.12 Zyntelgo™: Development Timeline
Figure 9.1 Successful Drug Launch Strategy: ROOTS Framework
Figure 9.2 Successful Drug Launch Strategy: Product Differentiation
Figure 9.3 Commonly Adopted Commercialization Strategies based on Phase of Development of the Product
Figure 9.4 Harvey Ball Analysis: Commercialization Strategies Adopted by Companies Focused on Gene Therapies
Figure 9.5 Approved Gene Therapies: Historical Timeline of Geographical Expansion
Figure 9.6 Approved Gene Therapies: Snapshot of Promotional Activities on Product Websites
Figure 9.7 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
Figure 11.1 Gene Editing: Key Application Areas
Figure 11.2 Gene Editing: Emerging Technology Platforms
Figure 12.1 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
Figure 12.2 Gene Therapies for Oncological Disorders: Distribution by Target Indication and Phase of Development
Figure 12.3 Gene Therapies for Oncological Disorders: Distribution by Type of Vector Used
Figure 12.4 Gene Therapies for Nervous System Disorders: Distribution by Target Indication and Phase of Development
Figure 12.5 Gene Therapies for Nervous System Disorders: Distribution by Type of Vector Used
Figure 12.6 Gene Therapies for Metabolic Disorders: Distribution by Target Indication and Phase of Development
Figure 12.7 Gene Therapies for Metabolic Disorders: Distribution by Type of Vector Used
Figure 12.8 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Figure 12.9 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
Figure 12.10 Gene Therapies for Genetic Disorders: Distribution by Target Indication and Phase of Development
Figure 12.11 Gene Therapies for Genetic Disorders: Distribution by Type of Vector Used
Figure 13.1 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Figure 13.2 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2020
Figure 13.3 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 13.4 Gene Therapy Patent Portfolio: Distribution by Geography
Figure 13.5 Gene Therapy Patent Portfolio: Distribution by Region (North America)
Figure 13.6 Gene Therapy Patent Portfolio: Distribution by Region (Europe)
Figure 13.7 Gene Therapy Patent Portfolio: Distribution by Region (Asia-Pacific)
Figure 13.8 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
Figure 13.9 Gene Therapy Patent Portfolio: Emerging Focus Areas
Figure 13.10 Gene Therapy Patent Portfolio: Leading Industry Players
Figure 13.11 Gene Therapy Patent Portfolio (Sangamo Therapeutics and Genzyme): Benchmarking by Patent Characteristics
Figure 13.12 Gene Therapy Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 13.13 Gene Therapy Patent Portfolio: Distribution by Patent Age (2016-2020)
Figure 13.14 Gene Therapy Patent Portfolio: Valuation Analysis
Figure 13.15 Gene Editing Patent Portfolio: Distribution by Type of Patent
Figure 13.16 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2020
Figure 13.17 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 13.18 Gene Editing Patent Portfolio: Distribution by Geography
Figure 13.19 Gene Editing Patent Portfolio: Distribution by Region (North America)
Figure 13.20 Gene Editing Patent Portfolio: Distribution by Region (Europe)
Figure 13.21 Gene Editing Patent Portfolio: Distribution by Region (Asia-Pacific)
Figure 13.22 Gene Editing Patent Portfolio: Distribution by CPC Symbols
Figure 13.23 Gene Editing Patent Portfolio: Emerging Focus Areas
Figure 13.24 Gene Editing Patent Portfolio: Leading Industry Players
Figure 13.25 Gene Editing Patent Portfolio (Sangamo Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
Figure 13.26 Gene Editing Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 13.27 Gene Editing Patent Portfolio: Distribution by Patent Age (2016-2020)
Figure 13.28 Gene Editing Patent Portfolio: Valuation Analysis
Figure 13.29 Gene Therapy and Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 14.1 Mergers and Acquisitions: Cumulative Year-Wise Trend (2015-2020)
Figure 14.2 Mergers and Acquisitions: Distribution by Type of Agreement
Figure 14.3 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Figure 14.4 Mergers and Acquisitions: Continent-wise Distribution
Figure 14.5 Mergers and Acquisitions: Distribution by Intercontinental and Intracontinental Acquisitions
Figure 14.6 Mergers and Acquisitions: Country-wise Distribution
Figure 14.7 Acquisitions: Distribution by Key Value Drivers
Figure 14.8 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
Figure 14.9 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Figure 14.10 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company’s Product and
Figure 14.11 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product and Deal Amount
Figure 14.12 Mergers and Acquisitions: Distribution by Therapeutic Area
Figure 15.1 Funding and Investment Analysis: Cumulative Year-wise Distribution by Number of Instances, 2015-2020
Figure 15.2 Funding and Investment Analysis: Cumulative Amount Invested, 2015-2020 (USD Million)
Figure 15.3 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2015-2020
Figure 15.4 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2015-2020 (USD Million)
Figure 15.5 Funding and Investment Analysis: Distribution by Year and Type of Funding, 2015-2020 (USD Million)
Figure 15.6 Funding and Investment Analysis: Distribution by Year, Type of Funding and Amount Invested, 2015-2020 (USD Million)
Figure 15.7 Funding and Investment Analysis: Summary of Investments, 2015-2020 (USD Million)
Figure 15.8 Funding and Investments: Distribution by Focus Area
Figure 15.9 Funding and Investment Analysis: Distribution by Geography
Figure 15.10 Funding and Investment Analysis: Regional Distribution of Funding Instances
Figure 15.11 Funding and Investment Analysis: Most Active Players, based on the Number of Funding Instances, 2015-2020 (USD Million)
Figure 15.12 Funding and Investment Analysis: Most Active Players, based on the Amount Invested, 2015-2020 (USD Million)
Figure 15.13 Funding and Investment Analysis: Distribution by Type of Investors
Figure 15.14 Funding and Investment Analysis: Distribution by Leading Investors
Figure 15.15 Funding and Investment Analysis: Distribution of Investments by Phase of Development of the Company’s Product
Figure 15.16 Funding and Investment Analysis: Distribution by Therapeutic Area
Figure 15.17 Funding and Investment Summary, 2015-2020 (USD Million)
Figure 16.1 Clinical Trial Analysis: Distribution by Trial Registration Year, Pre 2010-2020
Figure 16.2 Clinical Trial Analysis: Distribution by Trial Status
Figure 16.3 Clinical Trial Analysis: Distribution by Trial Phase
Figure 16.4 Clinical Trial Analysis: Distribution by Therapeutic Area
Figure 16.5 Clinical Trial Analysis: Distribution by Geography
Figure 16.6 Clinical Trial Analysis: Geographical Distribution by Trial Registration Year
Figure 16.7 Clinical Trial Analysis: Geographical Distribution by Trial Status
Figure 16.8 Clinical Trial Analysis: Geographical Distribution by Trial Status and Trial Phase
Figure 16.9 Clinical Trial Analysis: Geographical Distribution by Therapeutic Area
Figure 16.10 Clinical Trial Analysis: Distribution by Type of Sponsor
Figure 16.11 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Figure 16.12 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year
Figure 16.13 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Figure 16.14 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Figure 16.15 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Figure 16.16 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
Figure 16.17 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
Figure 16.18 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status, Phase and Location of Trial Site
Figure 16.19 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area and Location of Trial Site
Figure 17.1 Gene Therapy: Pricing Model Based on Patient Segment
Figure 18.1 Big Pharma Players: Heat Map Analysis of Top Pharmaceutical Companies
Figure 18.2 Big Pharma Players: Analysis by Therapeutic Area
Figure 18.3 Big Pharma Players: Analysis by Type of Vector Used
Figure 18.4 Big Pharma Players: Analysis by Therapeutic Approach
Figure 18.5 Big Pharma Players: Analysis by Type of Gene Therapy
Figure 18.6 Big Pharma Players: Heat Map Analysis of Other Players
Figure 19.1 Demand Analysis: Clinical Demand for Gene Therapies
Figure 19.2 Demand Analysis: Commercial Demand for Gene Therapies
Figure 20.1 Global Gene Therapy Market, 2020-2030: Base Case Scenario (USD Billion)
Figure 20.2 Gene Therapy Market: Distribution by Therapeutic Approach, 2020, 2025 and 2030
Figure 20.3 Gene Therapy Market: Distribution by Type of Gene Therapy, 2020, 2025 and 2030
Figure 20.4 Gene Therapy Market: Distribution by Type of Vector Used, 2020, 2025 and 2030
Figure 20.5 Gene Therapy Market: Distribution by Therapeutic Area, 2020, 2025 and 2030
Figure 20.6 Gene Therapy Market: Distribution by Route of Administration, 2020, 2025 and 2030
Figure 20.7 Gene Therapy Market: Distribution by Geography, 2020, 2025 and 2030
Figure 20.8 Gene Therapy Market: Country-wise Distribution of Asia-Pacific Region, 2020, 2025 and 2030
Figure 20. 9 Gendicine® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.10 Oncorine® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.11 Rexin-G® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.12 Neovasculgen® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.13 Strimvelis® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.14 Imlygic® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.15 Luxturna™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.16 Zolgensma™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.17 Collategene® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.18 Zyntelgo™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.19 GS010 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.20 PTC-AADC Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.21 BMN 270 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.22 rAd-IFN/Syn3 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.23 TAVO™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.24 OTL-200 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.25 EB-101 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.26 ProstAtak® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.27 BIIB111 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.28 LentiGlobin BB305® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.29 Lenti-D™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.30 FCX-007 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.31 CG0070 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.32 Vigil® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.33 VM202 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.34 VGX-3100 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.35 Invossa™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.36 KB103 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.37 PF-06939926 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.38 PF-06838435 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.39 PF-07055480 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.40 SPK-8011 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.41 AMT-061 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.42 VB-111 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.43 OTL-101 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.44 BIIB112 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.45 FLT180a Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.46 LYS-SAF302 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.47 NFS-01 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 20.48 OTL-103 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 21.1 Viral Vectors: Manufacturing Steps
Figure 22.1 Steps Involved in the Development of Gene Therapies
Figure 22.2 Overall Development Process of Cell-based Gene Therapies
Figure 22.3 Key Responsibilities of Stakeholders Involved in Advanced Therapies Supply Chain
Figure 22.4 Key Steps Involved in the Implementation of a Supply Chain Strategy
Figure 22.5 Keys Steps Involved in the Logistics Process of Autologous Therapies
Figure 22.6 Comparison of Logistics Processes for Autologous and Allogeneic Therapies
Figure 22.7 Global Regulatory Authorities for Gene Therapy Supply Chain
Figure 22.8 Overview of Supply Chain Orchestration Platform
Figure 22.9 Functions of Key Technological Solutions for Cell and Advanced Therapies Supply Chain Management
Figure 23.1 Concluding Remarks: Summary of the Current Market Landscape
Figure 23.2 Concluding Remarks: Summary of the Current Developer Landscape
Figure 23.3 Concluding Remarks: Recent Mergers and Acquisitions
Figure 23.4 Concluding Remarks: Recent Funding and Investments
Figure 23.5 Concluding Remarks: Market Sizing and Opportunity Analysis

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 and Commercial Pipeline
Table 6.2 Gene Therapy Market: Early Stage Pipeline
Table 6.3 Gene Therapy Market: Special Designation(s) Awarded
Table 7.1 Gene Therapy Market: List of Developers
Table 8.1 Gene Therapies: Marketed and Approved Products
Table 8.2 Shenzhen Sibiono GeneTech: Company Overview
Table 8.3 Gendicine®: Patent Portfolio
Table 8.4 Gendicine®: Status of Development
Table 8.5 Shanghai Sunway Biotech: Company Overview
Table 8.6 Oncorine®: Status of Development
Table 8.7 Epeius Biotechnologies: Company Overview
Table 8.8 Rexin-G®: Status of Development
Table 8.9 Human Stem Cell Institute: Company Overview
Table 8.10 Neovasculgen®: Status of Development
Table 8.11 Amgen: Company Overview
Table 8.12 Imlygic®: Status of Development
Table 8.13 Imlygic®: Recommended Dose and Schedule
Table 8.14 Imlygic®: Determination of Injection Volume based upon Lesion Size
Table 8.15 Orchard Therapeutics: Company Overview
Table 8.16 Strimvelis®: Status of Development
Table 8.17 Spark Therapeutics: Company Overview
Table 8.18 Luxturna™: Status of Development
Table 8.19 Novartis Gene Therapies: Company Overview
Table 8.20 Zolgensma™: Status of Development
Table 8.21 AnGes: Company Overview
Table 8.22 Collategene®: Status of Development
Table 8.23 bluebird bio: Company Overview
Table 8.24 Zyntelgo™: Status of Development
Table 9.1 Luxturna™: Presentations in Conferences Before Product Approval
Table 9.2 Zolgensma™: Presentations in Conferences Before Product Approval
Table 9.3 Zyntelgo™: Presentations in Conferences Before Product Approval
Table 9.4 Luxturna™: Presentations in Conferences After Product Approval
Table 9.5 Strimvelis™: Presentations in Conferences After Product Approval
Table 9.6 Zyntelgo™: Presentations in Conferences After Product Approval
Table 9.7 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
Table 10.1 Gene Therapies: Late Stage (Phase II/III and Above) Drug Candidates
Table 10.2 GS010: Drug Profile
Table 10.3 PTC-AADC: Drug Profile
Table 10.4 BMN 270: Drug Profile
Table 10.5 rAd-IFN/Syn3: Drug Profile
Table 10.6 TAVO™: Drug Profile
Table 10.7 OTL-200: Drug Profile
Table 10.8 EB-101: Drug Profile
Table 10.9 ProstAtak®: Drug Profile
Table 10.10 BIIB111: Drug Profile
Table 10.11 LentiGlobin BB305®: Drug Profile
Table 10.12 Lenti-D™: Drug Profile
Table 10.13 FCX-007: Drug Profile
Table 10.14 CG0070: Drug Profile
Table 10.15 Vigil®: Drug Profile
Table 10.16 VM202: Drug Profile
Table 10.17 VGX-3100: Drug Profile
Table 10.18 Invossa™: Drug Profile
Table 10.19 KB103: Drug Profile
Table 10.20 PF-06939926: Drug Profile
Table 10.21 PF-06838435: Drug Profile
Table 10.22 PF-07055480: Drug Profile
Table 10.23 SPK-8011: Drug Profile
Table 10.24 AMT-061: Drug Profile
Table 10.25 VB-111: Drug Profile
Table 10.26 OTL-101: Drug Profile
Table 10.27 BIIB112: Drug Profile
Table 10.28 FLT180a: Drug Profile
Table 10.29 LYS-SAF302: Drug Profile
Table 10.30 NFS-01: Drug Profile
Table 10.31 OTL-103: Drug Profile
Table 11.1 Gene Editing Platforms
Table 11.2 CRISPR Therapeutics: Funding Instances
Table 11.3 CRISPR Therapeutics: Collaborations
Table 11.4 Editas Medicine: Funding Instances
Table 11.5 Editas Medicine: Collaborations
Table 11.6 Intellia Therapeutics: Funding Instances
Table 11.7 Intellia Therapeutics: Collaborations
Table 11.8 Cellectis: Funding Instances
Table 11.9 Cellectis: Collaborations
Table 11.10 bluebird bio: Funding Instances
Table 11.11 bluebird bio: Collaborations
Table 11.12 Sangamo Therapeutics: Funding Instances
Table 11.13 Sangamo Therapeutics: Collaborations
Table 11.14 Gene Switch Technology Platforms
Table 11.15 Intrexon: Funding Instances
Table 11.16 Intrexon: Collaborations
Table 11.17 MeiraGTx: Funding Instances
Table 11.18 MeiraGTx: Collaborations
Table 11.19 Gene Therapy: Technology Platforms
Table 12.1 Gene Therapies for Oncological Disorders
Table 12.2 Gene Therapies for Nervous System Disorders
Table 12.3 Gene Therapies for Metabolic Disorders
Table 12.4 Gene Therapies for Ophthalmic Diseases
Table 12.5 Gene Therapies for Genetic Disorders
Table 13.1 Gene Therapy Patent Portfolio: List of Top CPC Symbols
Table 13.2 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
Table 13.3 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 13.4 Gene Therapy Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
Table 13.5 Gene Editing Patent Portfolio: List of Top CPC Symbols
Table 13.6 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
Table 13.7 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 13.8 Gene Editing Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
Table 14.1 Gene Therapy: List of Mergers and Acquisitions, 2015-2020
Table 14.2 Acquisitions: Key Value Drivers
Table 14.3 Mergers and Acquisitions: Information on Phase of Development and Therapeutic Area of the Acquired Company’s Gene Therapy Product
Table 15.1 Gene Therapy Market: Funding and Investments, 2020-2030
Table 15.2 Funding and Investment Analysis: Summary of Investments
Table 15.3 Funding and Investment Analysis: Summary of Venture Capital Funding
Table 16.1 Clinical Trial Analysis: Emerging Molecules to Watch
Table 16.2 Clinical Trial Analysis: Most Important Trials to Watch
Table 17.1 Pricing Model: Price of Marketed Gene / Cell Therapies
Table 17.2 Pricing Model: Price of Marketed Targeted Drugs
Table 17.3 Pricing Model: Opinions of Experts / Other Analysts
Table 20.1 Gene Therapies: Expected Launch Years of Advanced Stage Drug Candidates
Table 20.2 Gendicine®: Target Patient Population
Table 20.3 Gendicine®: Net Present Value (USD Million)
Table 20.4 Gendicine®: Value Creation Analysis (USD Million)
Table 20.5 Oncorine®: Target Patient Population
Table 20.6 Oncorine®: Net Present Value (USD Million)
Table 20.7 Oncorine®: Value Creation Analysis (USD Million)
Table 20.8 Rexin-G®: Target Patient Population
Table 20.9 Rexin-G®: Net Present Value (USD Million)
Table 20.10 Rexin-G®: Value Creation Analysis (USD Million)
Table 20.11 Neovasculgen®: Target Patient Population
Table 20.12 Neovasculgen®: Net Present Value (USD Million)
Table 20.13 Neovasculgen®: Value Creation Analysis (USD Million)
Table 20.14 Strimvelis®: Target Patient Population
Table 20.15 Strimvelis®: Net Present Value (USD Million)
Table 20.16 Strimvelis®: Value Creation Analysis (USD Million)
Table 20.17 Imlygic®: Target Patient Population
Table 20.18 Imlygic®: Net Present Value (USD Million)
Table 20.19 Imlygic®: Value Creation Analysis (USD Million)
Table 20.20 Luxturna™: Target Patient Population
Table 20.21 Luxturna™: Net Present Value (USD Million)
Table 20.22 Luxturna™: Value Creation Analysis (USD Million)
Table 20.23 Zolgensma™: Target Patient Population
Table 20.24 Zolgensma™: Net Present Value (USD Million)
Table 20.25 Zolgensma™: Value Creation Analysis (USD Million)
Table 20.26 Collategene®: Target Patient Population
Table 20.27 Collategene®: Net Present Value (USD Million)
Table 20.28 Collategene®: Value Creation Analysis (USD Million)
Table 20.29 Zynteglo™: Target Patient Population
Table 20.30 Zynteglo™: Net Present Value (USD Million)
Table 20.31 Zynteglo™: Value Creation Analysis (USD Million)
Table 20.32 GS010: Target Patient Population
Table 20.33 GS010: Net Present Value (USD Million)
Table 20.34 GS010: Value Creation Analysis (USD Million)
Table 20.35 PTC-AADC: Target Patient Population
Table 20.36 PTC-AADC: Net Present Value (USD Million)
Table 20.37 PTC-AADC: Value Creation Analysis (USD Million)
Table 20.38 BMN 270: Target Patient Population
Table 20.39 BMN 270: Net Present Value (USD Million)
Table 20.40 BMN 270: Value Creation Analysis (USD Million)
Table 20.41 rAd-IFN/Syn3: Target Patient Population
Table 20.42 rAd-IFN/Syn3: Net Present Value (USD Million)
Table 20.43 rAd-IFN/Syn3: Value Creation Analysis (USD Million)
Table 20.44 TAVO™: Target Patient Population
Table 20.45 TAVO™: Net Present Value (USD Million)
Table 20.46 TAVO™: Value Creation Analysis (USD Million)
Table 20.47 OTL-200: Target Patient Population
Table 20.48 OTL-200: Net Present Value (USD Million)
Table 20.49 OTL-200: Value Creation Analysis (USD Million)
Table 20.50 EB-101: Target Patient Population
Table 20.51 EB-101: Net Present Value (USD Million)
Table 20.52 EB-101: Value Creation Analysis (USD Million)
Table 20.53 ProstAtak®: Target Patient Population
Table 20.54 ProstAtak®: Net Present Value (USD Million)
Table 20.55 ProstAtak®: Value Creation Analysis (USD Million)
Table 20.56 BIIB111: Target Patient Population
Table 20.57 BIIB111: Net Present Value (USD Million)
Table 20.58 BIIB111: Value Creation Analysis (USD Million)
Table 20.59 LentiGlobin BB305®: Target Patient Population
Table 20.60 LentiGlobin BB305®: Net Present Value (USD Million)
Table 20.61 LentiGlobin BB305®: Value Creation Analysis (USD Million)
Table 20.62 Lenti-D™: Target Patient Population
Table 20.63 Lenti-D™: Net Present Value (USD Million)
Table 20.64 Lenti-D™: Value Creation Analysis (USD Million)
Table 20.65 FCX-007: Target Patient Population
Table 20.66 FCX-007: Net Present Value (USD Million)
Table 20.67 FCX-007: Value Creation Analysis (USD Million)
Table 20.68 CG0070: Target Patient Population
Table 20.69 CG0070: Net Present Value (USD Million)
Table 20.70 CG0070: Value Creation Analysis (USD Million)
Table 20.71 Vigil®: Target Patient Population
Table 20.72 Vigil®: Net Present Value (USD Million)
Table 20.73 Vigil®: Value Creation Analysis (USD Million)
Table 20.74 VM202: Target Patient Population
Table 20.75 VM202: Net Present Value (USD Million)
Table 20.76 VM202: Value Creation Analysis (USD Million)
Table 20.77 VGX-3100: Target Patient Population
Table 20.78 VGX-3100: Net Present Value (USD Million)
Table 20.79 VGX-3100: Value Creation Analysis (USD Million)
Table 20.80 Invossa™: Target Patient Population
Table 20.81 Invossa™: Net Present Value (USD Million)
Table 20.82 Invossa™: Value Creation Analysis (USD Million)
Table 20.83 KB103: Target Patient Population
Table 20.84 KB103: Net Present Value (USD Million)
Table 20.85 KB103: Value Creation Analysis (USD Million)
Table 20.86 PF-06939926: Target Patient Population
Table 20.87 PF-06939926: Net Present Value (USD Million)
Table 20.88 PF-06939926: Value Creation Analysis (USD Million)
Table 20.89 PF-06838435: Target Patient Population
Table 20.90 PF-06838435: Net Present Value (USD Million)
Table 20.91 PF-06838435: Value Creation Analysis (USD Million)
Table 20.92 PF-07055480: Target Patient Population
Table 20.93 PF-07055480: Net Present Value (USD Million)
Table 20.94 PF-07055480: Value Creation Analysis (USD Million)
Table 20.95 SPK-8011: Target Patient Population
Table 20.96 SPK-8011: Net Present Value (USD Million)
Table 20.97 SPK-8011: Value Creation Analysis (USD Million)
Table 20.98 AMT-061: Target Patient Population
Table 20.99 AMT-061: Net Present Value (USD Million)
Table 20.100 AMT-061: Value Creation Analysis (USD Million)
Table 20.101 VB-111: Target Patient Population
Table 20.102 VB-111: Net Present Value (USD Million)
Table 20.103 VB-111: Value Creation Analysis (USD Million)
Table 20.104 OTL-101: Target Patient Population
Table 20.105 OTL-101: Net Present Value (USD Million)
Table 20.106 OTL-101: Value Creation Analysis (USD Million)
Table 20.107 BIIB112: Target Patient Population
Table 20.108 BIIB112: Net Present Value (USD Million)
Table 20.109 BIIB112: Value Creation Analysis (USD Million)
Table 20.110 FLT180a: Target Patient Population
Table 20.111 FLT180a: Net Present Value (USD Million)
Table 20.112 FLT180a: Value Creation Analysis (USD Million)
Table 20.113 LYS-SAF302: Target Patient Population
Table 20.114 LYS-SAF302: Net Present Value (USD Million)
Table 20.115 LYS-SAF302: Value Creation Analysis (USD Million)
Table 20.116 NFS-01: Target Patient Population
Table 20.117 NFS-01: Net Present Value (USD Million)
Table 20.118 NFS-01: Value Creation Analysis (USD Million)
Table 20.119 OTL-103: Target Patient Population
Table 20.120 OTL-103: Net Present Value (USD Million)
Table 20.121 OTL-103: Value Creation Analysis (USD Million)
Table 21.1 Small Scale Cell Culture Systems
Table 21.2 Contract Manufacturing Service Providers for Viral Vectors and Plasmid DNA
Table 25.1 Gene Therapies: Distribution by Phase of Development
Table 25.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
Table 25.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
Table 25.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Table 25.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
Table 25.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene
Table 25.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Modification
Table 25.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Modification
Table 25.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Therapy
Table 25.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of Administration
Table 25.11 Early Stage Pipeline of Gene Therapies: Distribution by Phase of Development
Table 25.12 Early Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
Table 25.13 Early Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
Table 25.14 Early Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
Table 25.15 Early Stage Pipeline of Gene Therapies: Distribution by Type of Gene
Table 25.16 Early Stage Pipeline of Gene Therapies: Distribution by Therapeutic Approach
Table 25.17 Early Stage Pipeline of Gene Therapies: Distribution by Vector Used and Modification
Table 25.18 Early Stage Pipeline of Gene Therapies: Distribution by Type of Gene Therapy
Table 25.19 Early Stage Pipeline of Gene Therapies: Distribution by Route of Administration
Table 25.20 Gene Therapies: Distribution by Special Designation Awarded
Table 25.21 Gene Therapy Developers: Distribution by Year of Establishment
Table 25.22 Gene Therapy Developers: Distribution by Company Size
Table 25.23 Gene Therapy Developers: Distribution by Location of Headquarters
Table 25.24 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
Table 25.25 Gene Therapy Developers: Key Players
Table 25.26 Gene Therapy Developers: Distribution by Key Players and Phase of Development
Table 25.27 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
Table 25.28 Gene Therapies for Oncological Disorders: Distribution by Target Indication and Phase of Development
Table 25.29 Gene Therapies for Oncological Disorders: Distribution by Type of Vector Used
Table 25.30 Gene Therapies for Nervous System Disorders: Distribution by Target Indication and Phase of Development
Table 25.31 Gene Therapies for Nervous System Disorders: Distribution by Type of Vector Used
Table 25.32 Gene Therapies for Metabolic Disorders: Distribution by Target Indication and Phase of Development
Table 25.33 Gene Therapies for Metabolic Disorders: Distribution by Type of Vector Used
Table 25.34 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Table 25.35 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
Table 25.36 Gene Therapies for Genetic Disorders: Distribution by Target Indication and Phase of Development
Table 25.37 Gene Therapies for Genetic Disorders: Distribution by Type of Vector Used
Table 25.38 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Table 25.39 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2020
Table 25.40 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 25.41 Gene Therapy Patent Portfolio: Distribution by Geography
Table 25.42 Gene Therapy Patent Portfolio: Distribution by Region (North America)
Table 25.43 Gene Therapy Patent Portfolio: Distribution by Region (Europe)
Table 25.44 Gene Therapy Patent Portfolio: Distribution by Region (Asia-Pacific)
Table 25.45 Gene Therapy Patent Portfolio: Leading Industry Players
Table 25.46 Gene Therapy Patent Portfolio: Distribution by Patent Age (2016-2020)
Table 25.47 Gene Editing Patent Portfolio: Distribution by Type of Patent
Table 25.48 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2020
Table 25.49 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 25.50 Gene Editing Patent Portfolio: Distribution by Geography
Table 25.51 Gene Editing Patent Portfolio: Distribution by Region (North America)
Table 25.52 Gene Editing Patent Portfolio: Distribution by Region (Europe)
Table 25.53 Gene Editing Patent Portfolio: Distribution by Region (Asia-Pacific)
Table 25.54 Gene Editing Patent Portfolio: Leading Industry Players
Table 25.55 Gene Editing Patent Portfolio: Distribution by Patent Age (2016-2020)
Table 25.56 Gene Therapy and Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
Table 25.57 Mergers and Acquisitions: Cumulative Year-Wise Trend (2015-2020)
Table 25.58 Mergers and Acquisitions: Distribution by Type of Agreement
Table 25.59 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Table 25.60 Mergers and Acquisitions: Continent-wise Distribution
Table 25.61 Mergers and Acquisitions: Country-wise Distribution
Table 25.62 Acquisitions: Distribution by Key Value Drivers
Table 25.63 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
Table 25.64 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Table 25.65 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company’s Product
Table 25.66 Mergers and Acquisitions: Distribution by Therapeutic Area
Table 25.67 Funding and Investment Analysis: Cumulative Year-wise Distribution by Number of Instances, 2015-2020
Table 25.68 Funding and Investment Analysis: Cumulative Amount Invested, 2015-2020 (USD Million)
Table 25.69 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2015-2020
Table 25.70 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2015-2020 (USD Million)
Table 25.71 Funding and Investment Analysis: Distribution of Year and Type of Funding, 2015-2020 (USD Million)
Table 25.72 Funding and Investments: Distribution by Focus Area
Table 25.73 Funding and Investment Analysis: Distribution by Geography
Table 25.74 Funding and Investment Analysis: Regional Distribution of Funding Instances
Table 25.75 Funding and Investment Analysis: Most Active Players, based on the Number of Funding Instances, 2015-2020 (USD Million)
Table 25.76 Funding and Investment Analysis: Most Active Players, based on the Amount Invested, 2015-2020 (USD Million)
Table 25.77 Funding and Investment Analysis: Distribution by Type of Investors
Table 25.78 Funding and Investment Analysis: Distribution by Leading Investors
Table 25.79 Funding and Investment Analysis: Distribution of Investments by Phase of Development of the Company’s Product
Table 25.80 Funding and Investment Analysis: Distribution by Therapeutic Area
Table 25.81 Clinical Trial Analysis: Distribution by Trial Registration Year, Pre 2010-2020
Table 25.82 Clinical Trial Analysis: Distribution by Trial Status
Table 25.83 Clinical Trial Analysis: Distribution by Trial Phase
Table 25.84 Clinical Trial Analysis: Distribution by Therapeutic Area
Table 25.85 Clinical Trial Analysis: Geographical Distribution by Trial Registration Year
Table 25.86 Clinical Trial Analysis: Distribution by Type of Sponsor
Table 25.87 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Table 25.88 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year
Table 25.89 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Table 25.90 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Table 25.91 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Table 25.92 Demand Analysis: Clinical Demand for Gene Therapies
Table 25.93 Demand Analysis: Commercial Demand for Gene Therapies
Table 25.94 Global Gene Therapy Market, 2020-2030: Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.95 Gene Therapy Market: Distribution by Therapeutic Approach, 2020, 2025 and 2030
Table 25.96 Gene Therapy Market: Distribution by Type of Gene Therapy, 2020, 2025 and 2030
Table 25.97 Gene Therapy Market: Distribution by Type of Vector Used, 2020, 2025 and 2030
Table 25.98 Gene Therapy Market: Distribution by Therapeutic Area, 2020, 2025 and 2030
Table 25.99 Gene Therapy Market: Distribution by Route of Administration, 2020, 2025 and 2030
Table 25.100 Gene Therapy Market: Distribution by Geography, 2020, 2025 and 2030
Table 25.101 Gene Therapy Market: Country-wise Distribution of Asia-Pacific Region, 2020, 2025 and 2030
Table 25.102 Gendicine® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.103 Oncorine® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.104 Rexin-G® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.105 Neovasculgen® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.106 Strimvelis® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.107 Imlygic® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.108 Luxturna™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.109 Zolgensma™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.110 Collategene® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.111 Zyntelgo™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.112 GS010 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.113 PTC-AADC Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.114 BMN 270 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.115 rAd-IFN/Syn3 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.116 TAVO™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.117 OTL-200 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.118 EB-101 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.119 ProstAtak® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.120 BIIB111 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.121 LentiGlobin BB305® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.122 Lenti-D™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.123 FCX-007 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.124 CG0070 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.125 Vigil® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.126 VM202 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.127 VGX-3100 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.128 Invossa™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.129 KB103 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.130 PF-06939926 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.131 PF-06838435 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.132 PF-07055480 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.133 SPK-8011 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.134 AMT-061 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.135 VB-111 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.136 OTL-101 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.137 BIIB112 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.138 FLT180a Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.139 LYS-SAF302 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.140 NFS-01 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
Table 25.141 OTL-103 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)

List of Companies

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

  1. 4BIO Capital
  2. 4D Molecular Therapeutics
  3. 5AM Ventures
  4. AavantiBio
  5. aaVective
  6. AAVogen
  7. AbbVie
  8. Abby Grace Foundation
  9. Abeona Therapeutics
  10. Abintus Bio
  11. Adage Capital Management
  12. Adrenas Therapeutics
  13. Advantagene (also known as Candel Therapeutics)
  14. Advaxis
  15. Adverum Biotechnologies
  16. Aevitas Therapeutics
  17. Affinia Therapeutics
  18. Agent Capital
  19. Agilis Biotherapeutics
  20. AGTC
  21. Aisling Capital
  22. AJU IB Investment
  23. Akouos
  24. Alexandria Venture Investments
  25. Allergan
  26. Ally Bridge Group
  27. Alpha Holdings
  28. AlphaVax
  29. Altheia Science
  30. Altitude Life Science Ventures
  31. Amarna Therapeutics
  32. Ambulero
  33. American Gene Technologies™
  34. Amgen
  35. Amicus Therapeutics
  36. Amplo
  37. Anaeropharma Science
  38. AnGes
  39. Angionetics
  40. Annapurna Therapeutics (acquired by Avalanche Biotechnologies)
  41. ApolloBio
  42. Apple Tree Partners
  43. ARCH Venture Partners
  44. Arix Bioscience
  45. Arkin Bio Ventures
  46. ArrowMark Partners
  47. Arthrogen
  48. ARTIS Ventures
  49. Aruvant Sciences
  50. ASC Therapeutics
  51. Asia Alpha
  52. Asklepios BioPharmaceutical (AskBio)
  53. Aspa Therapeutics
  54. Astellas Pharma
  55. AstraZeneca
  56. Atlas Venture
  57. Atsena Therapeutics
  58. Audentes Therapeutics
  59. Audition Therapeutics
  60. Auriga Partners
  61. Avalanche Biotechnologies
  62. AVROBIO
  63. Axovant Gene Therapies
  64. Baillie Gifford
  65. Bain Capital Life Sciences
  66. Baldota Group
  67. Bamboo Therapeutics
  68. Bayer
  69. Beam Therapeutics
  70. Berkeley Catalyst Fund
  71. Biogen
  72. BioInvent International
  73. BioMarin Pharmaceutical
  74. Biomatics Capital 
  75. Bionic Sight
  76. BioVec Pharma
  77. Bioverativ (a Sanofi company)
  78. Blue Bear Ventures
  79. bluebird bio
  80. BlueWater Angels
  81. Boehringer Ingelheim
  82. Boxer Capital
  83. Bpifrance 
  84. Brace Pharma Capital
  85. Brain Neurotherapy Bio
  86. BrainVectis
  87. Breakout Ventures
  88. BridgeBio
  89. Bristol-Myers Squibb
  90. Brookside Capital
  91. BVF Partners
  92. California Institute for Regenerative Medicine
  93. Calimmune
  94. Cambridge Enterprise
  95. Cambridge Gene Therapy
  96. Cambridge Innovation Capital
  97. Cancer Research Institute
  98. CapDecisif Management
  99. Cardiogen Sciences
  100. Carmine Therapeutics
  101. Casdin Capital
  102. Case Western Reserve University 
  103. Castle Creek Biosciences
  104. Catalyst Biosciences
  105. CavoGene LifeSciences
  106. Celenex (Nationwide Children’s Hospital Spin Out)
  107. CellGenTech
  108. Celsion
  109. CG Oncology
  110. Chiba University
  111. Children’s Medical Research Foundation
  112. Children’s Oncology Group
  113. Coalition for Epidemic Preparedness Innovations (CEPI)
  114. CODA Biotherapeutics
  115. Codexis
  116. Columbia University
  117. Columbus Venture Partners
  118. CombiGene
  119. Consort Medical
  120. Copernicus Therapeutics
  121. Cormorant Asset Management
  122. Cowen Group
  123. Cowen Private Investments
  124. CRISPR Therapeutics
  125. CSL Behring
  126. CureDuchenne Ventures
  127. Cynvec
  128. Cyprium Therapeutics
  129. Cystic Fibrosis Foundation Therapeutics
  130. DARPA
  131. Deerfield Management
  132. DEFTA Partners
  133. Denovo Biopharma
  134. Dexcel Pharma
  135. Dimension Therapeutics
  136. DiNAQOR
  137. Discovery Genomics
  138. DNAlite Therapeutics
  139. DNAtrix
  140. Driehaus Capital Management
  141. Duke University
  142. Dyno Therapeutics
  143. EB Medical Research Foundation
  144. EcoR1 Capital
  145. Editas Medicine
  146. Edmond de Rothschild Investment Partners
  147. Eight Roads Ventures
  148. Emendo Biotherapeutics
  149. Emory University
  150. Emrys Bio
  151. Encoded Therapeutics
  152. Endsulin
  153. Enochian BioSciences
  154. Entos Pharmaceuticals
  155. Epeius Biotechnologies
  156. EPS Holdings
  157. Errant Gene Therapeutics
  158. Esco Ventures
  159. ESTEVE
  160. European Innovation Council Accelerator
  161. European Investment Bank
  162. Eventide Asset Management
  163. Evotec
  164. Excision BioTherapeutics
  165. Exegenesis Bio
  166. Exonics Therapeutics
  167. Expression Therapeutics
  168. ExSight Capital
  169. Eyevensys
  170. FerGene (Ferring Pharmaceuticals subsidiary)
  171. FF Science
  172. Fibrocell Science
  173. Fidelity Management & Research Company
  174. Flagship Pioneering
  175. Flash Therapeutics
  176. Flerie Invest AB
  177. Flexion Therapeutics
  178. Fondation Sanfilippo Suisse
  179. Foresite Capital
  180. Forge Biologics
  181. Forte Biosciences
  182. Fosun Capital
  183. FoxKiser
  184. F-Prime Capital
  185. Franklin Templeton Investments
  186. Frazier Healthcare Partners
  187. Freeline Therapeutics
  188. Friedreich’s Ataxia Research Alliance
  189. Fund+
  190. Fundación Sanfilippo B
  191. Galia Gestion
  192. Gemini Therapeutics
  193. Genable Technologies
  194. Gene Therapy Research Institution
  195. GeneCure Biotechnologies
  196. Genelux
  197. GeneMedicine
  198. GeneOne Life Science
  199. GeneQuine Biotherapeutics
  200. Generation Bio
  201. Genespire
  202. Genethon
  203. GeneTx
  204. Genexine
  205. Genprex
  206. GenSight Biologics
  207. GenVec 
  208. Ghost Tree Capital Group
  209. GO CAPITAL
  210. GOFAR
  211. GOG Foundation
  212. Google Ventures
  213. Gradalis
  214. Green Cross Holdings
  215. GSK
  216. Gyroscope Therapeutics
  217. Handl Therapeutics
  218. Harvard University
  219. Hatteras Venture Partners
  220. Hayfin Capital Management
  221. HBM Healthcare Investments
  222. HealthCap
  223. Helixmith
  224. Hemera Biosciences
  225. Herantis Pharma
  226. Hiroshima Venture Capital
  227. Histogenics
  228. Holostem Terapie Avanzate
  229. Homology Medicines
  230. HORAMA
  231. Horizon Technology Finance
  232. Hovione Scientia
  233. Huapont Life Sciences
  234. Human Stem Cells Institute PJSC (HSCI)
  235. Idinvest Partners
  236. Ikarovec
  237. Illumina Ventures
  238. Ilya Pharma
  239. Immusoft
  240. Imperial Innovations
  241. Innovate UK
  242. Innovation Network Corporation of Japan
  243. Inotek Pharmaceuticals
  244. Inovio Pharmaceuticals
  245. Inserm Transfert
  246. Institute of Myology
  247. Intellia Therapeutics
  248. Intrexon
  249. Invus
  250. Israel Innovation Authority
  251. IVERIC bio
  252. Janssen Pharmaceuticals
  253. Janus Capital Management
  254. Jennison Associates
  255. Johnson & Johnson Development
  256. Juda Capital
  257. K2 Venture Capital
  258. Kiwoom Securities
  259. Kleiner Perkins
  260. Kodikaz Therapeutic Solutions
  261. Kolon TissueGene
  262. Korea Investment Partners
  263. Kriya Therapeutics
  264. Krystal Biotech
  265. Kubota Vision
  266. Kurma Partners
  267. Kyorin Pharmaceutical
  268. Lacerta Therapeutics
  269. Leerink Partners
  270. Legend Capital
  271. Leiden University Medical Center 
  272. Lentistem® Biotech
  273. LEXEO Therapeutics
  274. LifeArc
  275. Lilly Asia Ventures
  276. Lime Asset Management
  277. Limelight Bio
  278. Lipigon Pharmaceuticals
  279. LogicBio Therapeutics
  280. Lokon Pharma
  281. Longitude Capital
  282. Ludwig Cancer Research
  283. Lundbeckfonden Ventures
  284. Lysogene
  285. Mangrove Partners
  286. Maryland Technology Development
  287. Matrix Capital Management
  288. Maverick Ventures
  289. Medison Ventures
  290. MeiraGTx
  291. Memorial Sloan Kettering Cancer Center
  292. Mercia Technologies
  293. Merck
  294. MERITZ Securities
  295. Mesa Verde Venture Partners
  296. Michigan Economic Development
  297. MidCap Financial
  298. Milo Biotechnology
  299. Mirae Asset Financial Group
  300. Mita Securities
  301. Mitsubishi Tanabe Pharma
  302. Mitsubishi UFJ Asset Management
  303. Momotaro-Gene
  304. MultiVir
  305. Muscular Dystrophy Association
  306. Mustang Bio
  307. Myonexus Therapeutics
  308. Myosana Therapeutics
  309. NanoCor Therapeutics
  310. Nanogenic Solutions
  311. Narya Capital
  312. National Cancer Institute
  313. National Institute of Allergy and Infectious Diseases
  314. National Institute of Health
  315. National MPS Society
  316. Nationwide Children’s Hospital
  317. Navega Therapeutics
  318. NeoMed
  319. Nerveda
  320. Netherlands Enterprise Agency
  321. NeuExcell Therapeutics
  322. Neuracle Genetics
  323. Neurimmune
  324. Neurocrine Biosciences
  325. Neurogene
  326. Neurophth Therapeutics
  327. New Energy and Industrial Technology Development Organization
  328. New Enterprise Associates
  329. New Leaf Venture Partners
  330. Nightstar Therapeutics
  331. Ninevah Therapeutics
  332. Noga Therapeutics
  333. Northpond Ventures
  334. Novartis
  335. Novo Holdings
  336. Novo Nordisk
  337. Oberland Capital
  338. Ocugen
  339. Odylia Therapeutics
  340. Okayama University
  341. Omega Funds
  342. Omnes Capital
  343. Oncolys BioPharma
  344. Onconetics Pharmaceuticals
  345. Oncos Therapeutics
  346. OncoSec Medical
  347. OncoSenX
  348. OrbiMed
  349. Orbit Biomedical
  350. ORCA Therapeutics
  351. Orchard Therapeutics
  352. ORI Capital
  353. Osage University Partners
  354. Otonomy
  355. Oxford BioMedica
  356. Oxford Finance
  357. Oxular
  358. Panmure Gordon
  359. Paragon Biosciences
  360. Parker Institute for Cancer Immunotherapy
  361. Partners Innovation Fund
  362. Passage Bio
  363. Pattern Bioscience
  364. Pavilion Capital
  365. PBM Capital
  366. Pentwater Capital Management
  367. Perceptive Advisors
  368. PeriphaGen
  369. Perseverance Capital Management
  370. Pfizer
  371. Pharmakon Advisors
  372. Phoenix Nest
  373. PhorMed
  374. Pivotal bioVenture Partners
  375. PlasmaTech Biopharmaceuticals
  376. Pontifax Venture Capital
  377. POSCO Capital
  378. Poseida Therapeutics
  379. Precigen
  380. Precision BioSciences
  381. Prevail Therapeutics
  382. Progenics Pharmaceuticals
  383. PsiOxus Therapeutics
  384. PTC Therapeutics
  385. RA Capital Management
  386. RBV Capital
  387. Red Sanfilippo Foundation
  388. Redbiotec
  389. Redmile Group
  390. Redpin Therapeutics
  391. Regeneron Pharmaceuticals
  392. REGENXBIO
  393. Renova Therapeutics
  394. Rentschler Biopharma
  395. Rev1 Ventures
  396. Reyon Pharmaceutical
  397. Ridgeback Capital Investments
  398. Ring Therapeutics
  399. Roche
  400. Rock Springs Capital
  401. Rocket Pharmaceuticals
  402. Roswell Park Comprehensive Cancer Center
  403. RoverMed BioSciences
  404. RTW Investments
  405. Ryboquin
  406. Samsara BioCapital
  407. Sanfilippo Children’s Foundation
  408. Sangamo Therapeutics
  409. Sanofi
  410. Sanofi Genzyme 
  411. Santen Pharmaceutical
  412. Sarepta Therapeutics
  413. Scancell
  414. SDL Ventures
  415. Seattle Children’s Research Institute
  416. Selecta Biosciences
  417. Sequoia Capital China
  418. Seraph Research Institute
  419. Seventure Partners
  420. Sham Innovation Sante
  421. Shanghai Sunway Biotech
  422. Shenzhen Qianhai Taxus
  423. Shinhan Capital
  424. Shinsei Corporate Investment
  425. Shire
  426. Sibiono GeneTech
  427. SillaJen
  428. Sofinnova Ventures
  429. Solid Biosciences
  430. Sorrento Therapeutics
  431. SOSV
  432. SOTIO
  433. Spark Therapeutics
  434. Sphera Global Healthcare Fund
  435. Spirovant Sciences
  436. SR-Tiget
  437. St. Jude Children’s Research Hospital
  438. Stanford University
  439. StrideBio
  440. Surveyor Capital
  441. SwanBio Therapeutics
  442. Syncona
  443. Synpromics
  444. T. Rowe Price
  445. Takeda Pharmaceutical
  446. Tamid Bio
  447. Target ALS Foundation
  448. Targovax
  449. Taysha Gene Therapies
  450. Temasek 
  451. Tenaya Therapeutics
  452. The Army Small Business Innovation Research
  453. The Column Group
  454. TheraBiologics
  455. Theravectys
  456. TNK Therapeutics
  457. Tocagen
  458. Tolerion
  459. TPG Capital
  460. Transgene
  461. Transhuman Capital
  462. Trucode Gene Repair
  463. UCL Technology Fund
  464. UK Cystic Fibrosis Gene Therapy Consortium
  465. Ultragenyx Pharmaceutical
  466. uniQure Biopharma
  467. Universitat Autònoma de Barcelona (UAB)
  468. University College London
  469. University Hospitals Cleveland Medical Center
  470. University of California San Diego School of Medicine
  471. University of Florida
  472. University of Massachusetts
  473. University of North Carolina at Chapel Hill
  474. University of Pennsylvania
  475. University of Pittsburgh
  476. University of Washington
  477. Urovant Sciences
  478. Valor Equity Partners
  479. V-Bio Ventures
  480. VBL Therapeutics
  481. VCN Biosciences
  482. Vectalys
  483. venBio
  484. Venrock
  485. Versant Ventures
  486. Vertex Pharmaceuticals
  487. Verve Therapeutics
  488. Vida Ventures
  489. ViGeneron (spin-off of the Ludwig-Maximilians-University)
  490. Viking Global Investors
  491. Virttu Biologics
  492. Vivet Therapeutics
  493. Vivo Capital
  494. Voyager Therapeutics
  495. VREX Therapeutics
  496. Washington Research Foundation
  497. Washington State University's Office of Commercialization
  498. Waverly Capital
  499. Weill Cornell Medicine
  500. Wellcome Trust
  501. Whitesun Healthcare Ventures
  502. WI Harper Group
  503. Wize Pharma
  504. Woodford Investment Management
  505. WuXi AppTec
  506. Wyvern Pharmaceuticals
  507. XyloCor Therapeutics
  508. Ysios Capital
  509. Ziopharm Oncology

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