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Although the price of such therapies is high, it is estimated that the number of patients who are likely to opt for such a treatment option is also on the rise. Moreover, as these are one-time treatments offering a cure for blindness, I believe that the high price is both reasonable and justified
-Ryo Kubota, Chairman, President and Chief Executive Officer, Acucela, A Kubota Pharmaceutical Company
Since the approval of the first therapy, Genedicine® in 2003, the gene therapy domain has evolved significantly. Specifically, in 2019, three gene therapies, namely Zolgensma® (US), Zynteglo™ (Europe) and Beperminogene Perplasmid (Japan), have received approval / conditional approval, leading to a marked upward surge in the interest in this field. In fact, the growing popularity can be correlated to the substantial increase (more than 90%) in the number of patents that have been filed / granted in the last three years. Moreover, in the same time period, more than USD 12.5 billion in capital has been invested by various private and public investors to fund research activities. Presently, there are more than 10 approved gene therapies in the market, while many others are being investigated across various phases of clinical research.
Over time, the efforts of industry stakeholders and clinical researchers have led to the discovery of novel molecular targets, thereby, strengthening the research pipelines of companies involved in the development of gene therapies. Further, several technology developers have designed innovative ways to improve the efficacy and safety of gene therapies and introduced advanced therapy development and vector engineering platforms. It is also worth mentioning that, in the last 4-5 years, there has been a marked rise in the M&A activity in this domain, including the involvement of several big pharma players as well. The capability of such therapies to target diverse disease indications is considered to be amongst the most prominent growth drivers of this market. Backed by promising clinical results and several therapy candidates in late phases of development, we believe that the overall market is expected to witness tremendous growth in the coming decade.
The “Gene Therapy Market (3rd Edition), 2019-2030” report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies and genome editing therapies. The study also features an elaborate discussion on the future potential of this evolving market. Amongst other elements, the report features:
One of the key objectives of the report was to estimate the existing market size and the future opportunity for gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2019-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] key therapeutic areas (cardiovascular disorders, muscular disorders, neurological disorders, ocular disorders, oncology and others), [B] various types of vectors used for therapy development (adeno associated virus, adenovirus, lentivirus, plasmid DNA, retrovirus and others), [C] type of therapy (ex vivo and in vivo), [D] type of gene modification (gene augmentation, oncolytic viral therapy and others) and [E] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.
The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals (in alphabetical order):
All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.
Chapter 2 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.
Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas and the route of administration of such therapeutic interventions. In addition, it includes information on the various steps involved in the transfer of therapeutic gene(s) into the body, along with a discussion on the advantages and disadvantages of this treatment paradigm. Further, the chapter features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.
Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages, limitations and applications of currently available vectors.
Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, China, Japan, South Korea and Australia. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.
Chapter 6 includes information on over 450 gene therapies and genome editing therapies that are currently approved or are in different stages of development. It features a comprehensive analysis of pipeline molecules, highlighting the drug developers, key therapeutic areas and target disease indication(s), phases of development, type of vector used, target gene type, type of therapy (gene augmentation / oncolytic viral therapy / others), type of somatic cell therapy (in vivo / ex vivo), mechanism of action and drug designation (if any). In addition, it features a schematic world map representation, highlighting the key regional hubs where gene therapies are being developed for the treatment of various disorders. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.
Chapter 7 provides detailed profiles of marketed gene therapies. Each profile includes an overview of the developer and information on various other parameters, such as history of development, indication, mechanism of action, patent portfolio, current developmental status, target, clinical trial results and information related to manufacturing.
Chapter 8 features an elaborate discussion on the various strategies that can be adopted by the therapy developers across key commercialization stages, namely prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis on the key strategies adopted by drug developers for the commercialization of their gene therapies that were approved post 2014.
Chapter 9 contains detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each profile provides information on the mechanism of action, current status of development, route of administration, affiliated technology platform (if applicable), dosage form, clinical studies and key clinical trial results.
Chapter 10 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile contains details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.
Chapter 11 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include cardiovascular disorders, hematological disorders, metabolic disorders, muscular disorders, neurological disorders, ocular disorders and oncology.
Chapter 12 provides insights from a detailed patent analysis, presenting an overview on the filed / granted patents related to gene therapies and genome editing therapies since 2016. For this analysis, we looked at the patents that have been published by various players till May 2019. It also highlights the important information and trends associated with these patents, including patent type (granted patents, patent applications and others), patent publication year, regional distribution, CPC classification, emerging focus areas and the leading industry / academic players (in terms of the number of patents filed / granted). The chapter also includes a patent benchmarking analysis and a detailed valuation analysis.
Chapter 13 features a detailed analysis of the various mergers and acquisitions that have taken place in this domain, highlighting the trend in the number of companies acquired between 2014-2019, based on parameters such as key value drivers, year of acquisition, type of acquisition, geographical location of the acquirer and the acquired company, and financial details of the deal (if available). In addition, the chapter presents a schematic world map representation of the geographical distribution of this activity, highlighting intracontinental and intercontinental deals.
Chapter 14 presents details on various funding instances, investments and grants that have been made within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2014 and 2019, highlighting the growing interest of the venture capital community and other strategic investors in this domain.
Chapter 15 highlights our views on the various factors that may be taken into consideration while pricing gene therapies. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt to decide the price of its proprietary products.
Chapter 16 highlights top ten big biopharma players in the field of gene therapy. It features tabulated profiles of the companies and each profile includes a brief overview of the company, its financial information (if available), information on its product portfolio and recent developments.
Chapter 17 presents a comprehensive forecast analysis, highlighting the future potential of the market till the year 2030. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of key therapeutic areas (cardiovascular disorders, muscular disorders, neurological disorders, ocular disorders, oncology and others), type of vector (adeno associated virus, adenovirus, lentivirus, plasmid DNA, retrovirus and others), type of somatic cell therapy (ex vivo and in vivo), type of gene modification (gene augmentation, oncolytic viral therapy and others) and geography (the US, EU5, RoW (Australia, China, Israel Japan and South Korea)).
Chapter 18 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain, and highlights some of the recent collaborations and developments related to manufacturing processes of gene therapies. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.
Chapter 19 provides a general overview on the supply chain of gene therapies. It features the process and steps followed in the supply chain to deliver gene therapies to target patients for the treatment of various rare disorders.
Chapter 20 summarizes the entire report. It presents a list of key takeaways and offers our independent opinion on the current market scenario. Further, it captures the evolutionary trends that are likely to determine the future of this segment of the gene therapies industry.
Chapter 21 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Christopher Reinhard (CEO and Chairman, Cardium Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (Chairman, President and Chief Executive Officer, Acucela) and Tom Wilton (Chief Business Officer, LogicBio Therapeutics). In addition, a brief profile of each company has been provided.
Chapter 22 is an appendix, which provides tabulated data and numbers for all the figures included in the report.
Chapter 23 is an appendix, which contains a list of companies and organizations mentioned in this report.
1 PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Chapter Outlines
2 EXECUTIVE SUMMARY
3 INTRODUCTION
3.1. Context and Background
3.2. Evolution of Gene Therapies
3.3. Classification of Gene Therapies
3.3.1. Somatic and Germline Gene Therapy
3.3.2. Ex Vivo and In Vivo Gene Therapy
3.4. Routes of Administration
3.5. Mechanism of Action of Gene Therapies
3.6. Concept of Gene Editing
3.7. Advantages and Disadvantages of Gene Therapies
3.8. Ethical and Social Concerns Related to Gene Therapies
3.9. Future Constraints and Challenges Related to Gene Therapies
3.9.1. Concerns Related to Manufacturing
3.9.2. Concerns Related to Commercial Viability
4 GENE DELIVERY VECTORS
4.1. Chapter Overview
4.2. Viral Vectors
4.2.1 Types of Viral Vectors
4.2.1.1. Adeno-associated Viral Vectors
4.2.1.1.1. Overview
4.2.1.1.2. Design
4.2.1.1.3. Advantages
4.2.1.1.4. Limitations
4.2.1.2. Adenoviral Vectors
4.2.1.2.1. Overview
4.2.1.2.2. Design
4.2.1.2.3. Advantages
4.2.1.2.4. Limitations
4.2.1.3. Lentiviral Vectors
4.2.1.3.1. Overview
4.2.1.3.2. Design
4.2.1.3.3. Advantages
4.2.1.3.4. Limitations
4.2.1.4. Retroviral Vectors
4.2.1.4.1. Overview
4.2.1.4.2. Design
4.2.1.4.3. Advantages
4.2.1.4.4. Limitations
4.2.1.5. Other Viral Vectors
4.2.1.5.1. Alphavirus
4.2.1.5.2. Herpes Simplex Virus
4.2.1.5.3. Simian Virus
4.2.1.5.4. Vaccinia Virus
4.3. Non-Viral Vectors
4.3.1. Types of Non-Viral Vectors
4.3.1.1. Plasmid DNA
4.3.1.2. Liposomes, Lipoplexes and Polyplexes
4.3.1.3. Oligonucleotides
4.4. Methods of Transfection
4.4.1. Biolistic Method
4.4.2. Electroporation
4.4.3. Receptor Mediated Gene Delivery
4.4.4. Gene Activated Matrix (GAM)
5 REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO
5.1. Chapter Overview
5.2. Regulatory Guidelines in North America
5.2.1. The US Scenario
5.2.2. The Canadian Scenario
5.3. Regulatory Guidelines in Europe
5.4. Regulatory Guidelines in Asia Pacific
5.4.1. Chinese Scenario
5.4.2. Japanese Scenario
5.4.3. South Korean Scenario
5.4.4. Australian Scenario
5.5. Reimbursement Scenario
5.5.1. Challenges Related to Reimbursement
5.6. Payment Models for Gene Therapies
6 COMPETITIVE LANDSCAPE
6.1. Chapter Overview
6.2. Gene Therapy Market: Clinical Pipeline
6.2.1. Analysis by Phase of Development
6.2.2. Analysis by Therapeutic Area
6.2.3. Analysis by Type of Vector Used
6.2.4. Analysis by Type of Gene
6.2.5. Analysis by Type of Modification
6.2.6. Analysis by Type of Gene Therapy
6.2.7. Analysis by Route of Administration
6.3. Gene Therapy Market: Early Stage Pipeline
6.3.1. Analysis by Stage of Development
6.3.2. Analysis by Therapeutic Area
6.3.3. Analysis by Type of Vector Used
6.3.4. Analysis by Type of Gene
6.3.5. Analysis by Type of Modification
6.3.6. Analysis by Type of Gene Therapy
6.4. Key Players
6.5. Developer Landscape
6.6. Regional Landscape
7 MARKETED GENE THERAPIES
7.1. Chapter Overview
7.2. Gendicine® (Shenzhen Sibiono GeneTech)
7.2.1. Company Overview
7.2.2. Development Timeline
7.2.3. Mechanism of Action and Vectors Used
7.2.4. Target Indication(s)
7.2.5. Current Status of Development
7.2.6. Manufacturing, Dosage and Sales
7.3. Oncorine® (Shanghai Sunway Biotech)
7.3.1. Company Overview
7.3.2. Development Timeline
7.3.3. Mechanism of Action and Vectors Used
7.3.4. Target Indication(s)
7.3.5. Current Status of Development
7.3.6. Dosage and Sales
7.4. Rexin-G® (Epeius Biotechnologies)
7.4.1. Company Overview
7.4.2. Development Timeline
7.4.3. Mechanism of Action and Vector Used
7.4.4. Target Indication(s)
7.4.5. Current Status of Development
7.4.6. Manufacturing, Dosage and Sales
7.5. Neovasculgen® (Human Stem Cells Institute)
7.5.1. Company Overview
7.5.2. Development Timeline
7.5.3. Mechanism of Action and Vectors Used
7.5.4. Target Indication(s)
7.5.5. Current Status of Development
7.5.6. Manufacturing, Dosage and Sales
7.6. Imlygic® (Amgen)
7.6.1. Company Overview
7.6.2. Development Timeline
7.6.3. Mechanism of Action and Vectors Used
7.6.4. Target Indication(s)
7.6.5. Current Status of Development
7.6.6. Manufacturing, Dosage and Sales
7.7. Strimvelis® (Orchard Therapeutics)
7.7.1. Company Overview
7.7.2. Development Timeline
7.7.3. Mechanism of Action and Vectors Used
7.7.4. Target Indication(s)
7.7.5. Current Status of Development
7.7.6. Manufacturing, Dosage and Sales
7.8. Invossa™ (TissueGene)
7.8.1. Company Overview
7.8.2. Development Timeline
7.8.3. Mechanism of Action and Vectors Used
7.8.4. Target Indication(s)
7.8.5. Current Status of Development
7.8.6. Manufacturing, Dosage and Sales
7.9. Luxturna™ (Spark Therapeutics)
7.9.1. Company Overview
7.9.2. Development Timeline
7.9.3. Mechanism of Action and Vector Used
7.9.4. Target Indication(s)
7.9.5. Current Status of Development
7.9.6. Manufacturing, Dosage and Sales
7.10. Zolgensma™ (AveXis / Novartis)
7.10.1. Company Overview
7.10.2. Development Timeline
7.10.3. Mechanism of Action and Vector Used
7.10.4. Target Indication(s)
7.10.5. Current Status of Development
7.10.6. Manufacturing, Dosage and Sales
7.11. Collategene® / Beperminogene Perplasmid (AnGes)
7.11.1. Company Overview
7.11.2. Development Timeline
7.11.3. Mechanism of Action and Vector Used
7.11.4. Target Indication(s)
7.11.5. Current Status of Development
7.11.6. Manufacturing, Dosage and Sales
7.12. Zyntelgo™ (bluebird bio)
7.12.1. Company Overview
7.12.2. Development Timeline
7.12.3. Mechanism of Action and Vector Used
7.12.4. Target Indication(s)
7.12.5. Current Status of Development
7.12.6. Manufacturing, Dosage and Sales
8 KEY COMMERCIALIZATION STRATEGIES
8.1. Chapter Overview
8.2. Successful Drug Launch Strategy: ROOTS Framework
8.3. Successful Drug Launch Strategy: Product Differentiation
8.4. Commonly Adopted Commercialization Strategies based on Development Stage of the Product
8.5. Approved Gene Therapies
8.6. Key Commercialization Strategies Adopted by Companies Focused on Gene Therapy
8.6.1. Strategies Adopted Before Therapy Approval
8.6.2. Strategies Adopted During / Post Therapy Approval
8.7. Concluding Remarks
9 LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES
9.1. Chapter Overview
9.2. Axalimogene Filolisbac: Overview of Therapy, Current Development Status and Clinical Results
9.3. AMT-061: Overview of Therapy, Current Development Status and Clinical Results
9.4. BIIB111: Overview of Therapy, Current Development Status and Clinical Results
9.5. BIIB112: Overview of Therapy, Current Development Status and Clinical Results
9.6. Donaperminogene Seltoplasmid (VM202): Overview of Therapy, Current Development Status and Clinical Results
9.7. E10A: Overview of Therapy, Current Development Status and Clinical Results
9.8. Fidanacogene Elaparvovec (PF-06838435): Overview of Therapy, Current Development Status and Clinical Results
9.9. FLT180a: Overview of Therapy, Current Development Status and Clinical Results
9.10. GS010: Overview of Therapy, Current Development Status and Clinical Results
9.11. Instiladrin®: Overview of Therapy, Current Development Status and Clinical Results
9.12. Lenti-D™: Overview of Therapy, Current Development Status and Clinical Results
9.13. LYS-SAF302: Overview of Therapy, Current Development Status and Clinical Results
9.14. Ofranergene Obadenovec (VB-111): Overview of Therapy, Current Development Status and Clinical Results
9.15. OTL-101: Overview of Therapy, Current Development Status and Clinical Results
9.16. OTL-103: Overview of Therapy, Current Development Status and Clinical Results
9.17. OTL-200: Overview of Therapy, Current Development Status and Clinical Results
9.18. Pexastimogene Devacirepvec (PEXA-VEC): Overview of Therapy, Current Development Status and Clinical Results
9.19. ProstAtak®: Overview of Therapy, Current Development Status and Clinical Results
9.20. SPK-8011: Overview of Therapy, Current Development Status and Clinical Results
9.21. Unnamed Therapy: Overview of Therapy, Current Development Status and Clinical Results
9.22. Valoctocogene Roxaparvovec (BMN 270): Overview of Therapy, Current Development Status and Clinical Results
9.23. Vigil®: Overview of Therapy, Current Development Status and Clinical Results
9.24. VGX-3100: Overview of Therapy, Current Development Status and Clinical Results
9.25. Vocimagene Amiretrorepvec (Toca-511): Overview of Therapy, Current Development Status and Clinical Results
10 EMERGING TECHNOLOGIES
10.1. Chapter Overview
10.2. Gene Editing Technologies
10.2.1. Overview
10.2.2. Applications
10.3. Emerging Gene Editing Platforms
10.3.1. CRISPR / Cas9 System
10.3.2. TALENs
10.3.3. megaTAL
10.3.4. Zinc Finger Nuclease
10.4. Gene Expression Regulation Technologies
10.5. Technology Platforms for Developing / Delivering Gene Therapies
11 PROMISING THERAPEUTICS AREAS
11.1. Chapter Overview
11.2 Analysis by Special Designations Awarded
11.3. Cardiovascular Disorders
11.3.1. Analysis by Target Indication
11.3.2. Analysis by Type of Vector Used
11.4. Hematological Disorders
11.4.1. Analysis by Target Indication
11.4.2. Analysis by Type of Vector Used
11.5. Inflammatory & Infectious (I&I) Diseases
11.5.1. Analysis by Target Indication
11.5.2. Analysis by Type of Vector Used
11.6. Metabolic Disorders
11.6.1. Analysis by Target Indication
11.6.2. Analysis by Type of Vector Used
11.7. Muscular Disorders
11.7.1. Analysis by Target Indication
11.7.2. Analysis by Type of Vector Used
11.8. Neurological Disorders
11.8.1. Analysis by Target Indication
11.8.2. Analysis by Type of Vector Used
11.9. Ophthalmic Disorders
11.9.1. Analysis by Target Indication
11.9.2. Analysis by Type of Vector Used
11.10. Oncology
11.10.1. Analysis by Target Indication
11.10.2. Analysis by Type of Vector Used
12 PATENT ANALYSIS
12.1. Chapter Overview
12.2. Gene Therapy-related Patents
12.2.1. Scope and Methodology
12.2.1.1. Analysis by Publication Year
12.2.1.2. Analysis by Geographical Location
12.2.1.3. Analysis by CPC Classification
12.2.1.4. Emerging Focus Areas
12.2.1.5. Leading Players: Analysis by Number of Patents
12.2.1.6. Patent Benchmark Analysis
12.2.1.7. Patent Valuation Analysis
12.2. Gene Editing-related Patents
12.2.1. Scope and Methodology
12.2.1.1. Analysis by Publication Year
12.2.1.2. Analysis by Geographical Location
12.2.1.3. Analysis by CPC Classification
12.2.1.4. Emerging Focus Areas
12.2.1.5. Leading Players: Analysis by Number of Patents
12.2.1.6. Patent Benchmark Analysis
12.2.1.7. Patent Valuation Analysis
12.3. Overall Intellectual Property Portfolio: Analysis by Type of Organization
13 MERGERS AND ACQUISITIONS
13.1. Chapter Overview
13.2. Merger and Acquisition Models
13.3. Gene Therapy: Mergers and Acquisitions
13.3.1. Analysis by Year of Mergers and Acquisitions
13.3.2. Analysis by Type of Mergers and Acquisitions
13.3.3. Regional Analysis
13.3.3.1. Continent-wise Distribution
13.3.3.2. Intercontinental and Intracontinental Deals
13.3.3.3. Country-wise Distribution
13.3.4. Analysis by Key Value Drivers
13.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
13.3.5. Analysis by Phase of Development of the Acquired Company’s Product
13.3.6. Analysis by Therapeutic Area
14 FUNDING AND INVESTMENT ANALYSIS
14.1. Chapter Overview
14.2. Types of Funding
14.3. Funding and Investment Analysis
14.3.1. Analysis by Number of Funding Instances
14.3.2. Analysis by Amount Invested
14.3.3. Analysis by Type of Funding
14.3.4. Analysis by Amount Invested across Different Types of Therapies
14.3.5. Regional Analysis by Amount Invested
14.3.6. Most Active Players
14.3.7. Key Investors
14.3.8. Analysis by Stage of Development
14.4. Concluding Remarks
15 COST PRICE ANALYSIS
13.1. Chapter Overview
13.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
13.3. Gene Therapy Market: Pricing Models
13.3.1. On the Basis of Associated Product / Component Costs
13.3.2. On the Basis of Competition
13.3.3. On the Basis of Patient Segment
13.3.4. On the Basis of Opinions of Industry Experts
16 BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES
16.1. Chapter Overview
16.2. Top Pharmaceutical Companies
16.2.1. Analysis by Therapeutic Area
16.2.2. Analysis by Type of Vector Used
16.2.3. Analysis by Type of Modification
16.2.4. Analysis by Type of Gene Therapy
16.3. Other Big Pharma Players
17 MARKET FORECAST AND OPPORTUNITY ANALYSIS
17.1. Chapter Overview
17.2. Scope and Limitations
17.3. Key Assumptions and Forecast Methodology
17.4. Overall Gene Therapy Market, 2019-2030
17.4.1. Gene Therapy Market: Analysis by Type of Gene Modification
17.4.2. Gene Therapy Market: Analysis by Type of Therapy
17.4.3. Gene Therapy Market: Analysis by Type of Vector Used
17.4.4. Gene Therapy Market: Analysis by Therapeutic Area
17.4.5. Gene Therapy Market: Analysis by Route of Administration
17.4.6. Gene Therapy Market: Analysis by Geography
17.5. Gene Therapy Market: Value Creation Analysis
17.6. Gene Therapy Market: Product-wise Sales Forecasts
17.6.1. Gendicine®
17.6.1.1. Target Patient Population
17.6.1.2. Sales Forecast
17.6.1.3. Net Present Value
17.6.1.4. Value Creation Analysis
17.6.2. Oncorine®
17.6.2.1. Target Patient Population
17.6.2.2. Sales Forecast
17.6.2.3. Net Present Value
17.6.2.4. Value Creation Analysis
17.6.3. Rexin-G®
17.6.3.1. Target Patient Population
17.6.3.2. Sales Forecast
17.6.3.3. Net Present Value
17.6.3.4. Value Creation Analysis
17.6.4. Neovasculgen®
17.6.4.1. Target Patient Population
17.6.4.2. Sales Forecast
17.6.4.3. Net Present Value
17.6.4.4. Value Creation Analysis
17.6.5. Strimvelis®
17.6.5.1. Target Patient Population
17.6.5.2. Sales Forecast
17.6.5.3. Net Present Value
17.6.5.4. Value Creation Analysis
17.6.6. Imlygic®
17.6.6.1. Target Patient Population
17.6.6.2. Sales Forecast
17.6.6.3. Net Present Value
17.6.6.4. Value Creation Analysis
17.6.7. Invossa™
17.6.7.1. Target Patient Population
17.6.7.2. Sales Forecast
17.6.7.3. Net Present Value
17.6.7.4. Value Creation Analysis
17.6.8. Luxturna™
17.6.8.1. Target Patient Population
17.6.8.2. Sales Forecast
17.6.8.3. Net Present Value
17.6.8.4. Value Creation Analysis
17.6.9. Zolgensma™
17.6.9.1. Target Patient Population
17.6.9.2. Sales Forecast
17.6.9.3. Net Present Value
17.6.9.4. Value Creation Analysis
17.6.10. Collategene® / Beperminogene Perplasmid
17.6.10.1. Target Patient Population
14.6.10.2. Sales Forecast
17.6.10.3. Net Present Value
17.6.10.4. Value Creation Analysis
17.6.11. Zyntelgo™
17.6.11.1. Target Patient Population
17.6.11.2. Sales Forecast
17.6.11.3. Net Present Value
17.6.11.4. Value Creation Analysis
17.6.12. Axalimogene Filolisbac
17.6.12.1. Target Patient Population
17.6.12.2. Sales Forecast
17.6.12.3. Net Present Value
17.6.12.4. Value Creation Analysis
17.6.13. AMT-061
17.6.13.1. Target Patient Population
17.6.13.2. Sales Forecast
17.6.13.3. Net Present Value
17.6.13.4. Value Creation Analysis
17.6.14. BIIB111
17.6.14.1. Target Patient Population
17.6.14.2. Sales Forecast
17.6.14.3. Net Present Value
17.6.14.4. Value Creation Analysis
17.6.15. BIIB112
17.6.15.1. Target Patient Population
17.6.15.2. Sales Forecast
17.6.15.3. Net Present Value
17.6.15.4. Value Creation Analysis
17.6.16. Donaperminogene Seltoplasmid (VM202)
17.6.16.1. Target Patient Population
17.6.16.2. Sales Forecast
17.6.16.3. Net Present Value
17.6.16.4. Value Creation Analysis
17.6.17. E10A
17.6.17.1. Target Patient Population
17.6.17.2. Sales Forecast
17.6.17.3. Net Present Value
17.6.17.4. Value Creation Analysis
17.6.18. Fidanacogene Elaparvovec (PF-06838435)
17.6.18.1. Target Patient Population
17.6.18.2. Sales Forecast
17.6.18.3. Net Present Value
17.6.18.4. Value Creation Analysis
17.6.19. FLT180a
17.6.19.1. Target Patient Population
17.6.19.2. Sales Forecast
17.6.19.3. Net Present Value
17.6.19.4. Value Creation Analysis
17.6.20. GS010
17.6.20.1. Target Patient Population
17.6.20.2. Sales Forecast
17.6.20.3. Net Present Value
17.6.20.4. Value Creation Analysis
17.6.21. Instiladrin®
17.6.21.1. Target Patient Population
17.6.21.2. Sales Forecast
17.6.21.3. Net Present Value
17.6.21.4. Value Creation Analysis
17.6.22. Lenti-D™
17.6.22.1. Target Patient Population
17.6.22.2. Sales Forecast
17.6.22.3. Net Present Value
17.6.22.4. Value Creation Analysis
17.6.23. LYS-SAF302
17.6.23.1. Target Patient Population
17.6.23.2. Sales Forecast
17.6.23.3. Net Present Value
17.6.23.4. Value Creation Analysis
17.6.24. Ofranergene Obadenovec (VB-111)
17.6.24.1. Target Patient Population
17.6.24.2. Sales Forecast
17.6.24.3. Net Present Value
17.6.24.4. Value Creation Analysis
17.6.25. OTL-101
17.6.25.1. Target Patient Population
17.6.25.2. Sales Forecast
17.6.25.3. Net Present Value
17.6.25.4. Value Creation Analysis
17.6.26. OTL-103
17.6.26.1. Target Patient Population
17.6.26.2. Sales Forecast
17.6. 26.3. Net Present Value
17.6.26.4. Value Creation Analysis
17.6.27. OTL-200
17.6.27.1. Target Patient Population
17.6.27.2. Sales Forecast
17.6.27.3. Net Present Value
17.6.27.4. Value Creation Analysis
17.6.28. Pexastimogene Devacirepvec (PEXA-VEC)
17.6.28.1. Target Patient Population
17.6.28.2. Sales Forecast
17.6.28.3. Net Present Value
17.6.28.4. Value Creation Analysis
17.6.29. ProstAtak®
17.6.29.1. Target Patient Population
17.6.29.2. Sales Forecast
17.6.29.3. Net Present Value
17.6.29.4. Value Creation Analysis
17.6.30. SPK-8011
17.6.30.1. Target Patient Population
17.6.30.2. Sales Forecast
17.6.30.3. Net Present Value
17.6.30.4. Value Creation Analysis
17.6.31. Unnamed Therapy
17.6.31.1. Target Patient Population
17.6.31.2. Sales Forecast
17.6.31.3. Net Present Value
17.6.31.4. Value Creation Analysis
17.6.32. Valoctocogene Roxaparvovec (BMN 270)
17.6.32.1. Target Patient Population
17.6.32.2. Sales Forecast
17.6.32.3. Net Present Value
17.6.32.4. Value Creation Analysis
17.6.33. Vigil®
17.6.33.1. Target Patient Population
17.6.33.2. Sales Forecast
17.6.33.3. Net Present Value
17.6.33.4. Value Creation Analysis
17.6.34. VGX-3100
17.6.34.1. Target Patient Population
17.6.34.2. Sales Forecast
17.6.34.3. Net Present Value
17.6.34.4. Value Creation Analysis
17.6.35. Vocimagene Amiretrorepvec (Toca-511)
17.6.35.1. Target Patient Population
17.6.35.2. Sales Forecast
17.6.35.3. Net Present Value
17.6.35.4. Value Creation Analysis
18 VECTOR MANUFACTURING
18.1. Chapter Overview
18.2. Overview of Viral Vector Manufacturing
18.3. Viral Vector Manufacturing Processes
18.3.1. Mode of Vector Production
18.3.2. Adherent and Suspension Cultures
18.3.3. Unit Processes and Multiple Parallel Processes
18.3.4. Cell Culture Systems for Production of Viral Vectors
18.3.5. Culture Media Specifications
18.4. Bioprocessing of Viral Vectors
18.4.1. AAV Vector Production
18.4.2. Adenoviral Vector Production
18.4.3. Lentiviral Vector Production
18.4.4. γ -Retroviral Vector Production
18.5. Challenges Associated with Vector Manufacturing
18.6. Companies Offering Contract Services for Viral and Plasmid Vectors
19 CASE STUDY: GENE THERAPY SUPPLY CHAIN
19.1. Chapter Overview
19.2. Overview of the Gene Therapy Supply Chain
19.3. Implementation of Supply Chain Models
19.4. Logistics in Gene Therapy
19.4.1. Logistics Processes for Autologous and Allogeneic Therapies
19.5. Regulatory Supply Chain across the Globe
19.6. Challenges Associated with Gene Therapy Supply Chain
19.7. Optimizing Cell and Advanced Therapies Supply Chain Management
19.8. Recent Developments and Upcoming Trends
20 CONCLUSION
20.1. Chapter Overview
20.2. Key Takeaways
21 INTERVIEW TRANSCRIPTS
21.1. Chapter Overview
21.2. Adam Rogers, Chief Executive Officer, Hemera Biosciences
21.3. Al Hawkins, Chief Executive Officer, Milo Biotechnology
21.4. Buel Dan Rodgers, Founder & Chief Executive Officer, AAVogen
21.5. Cedric Szpirer, Executive & Scientific Director, Delphi Genetics
21.6. Christopher Reinhard, Chief Executive Officer and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)
21.7. Jeffrey HunG, Chief Commercial Officer, Vigene Biosciences
21.8. Marco Schmeer, Project Manager and Tatjana Buchholz, Marketing Manager, PlasmidFactory
21.9. Michael Tripletti, Chief Executive Officer, Myonexus Therapeutics
21.10. Robert Jan Lamers, Chief Executive Officer, Arthrogen
21.11. Ryo Kubota, Chairman, President and Chief Executive Officer, Acucela
21.12. Tom Wilton, Chief Business Officer, LogicBio Therapeutics
22 APPENDIX 1: TABULATED DATA
23 APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
Figure 3.1 History of Evolution of Gene Therapies
Figure 3.2 Ex Vivo Gene Therapy
Figure 3.3 In Vivo Gene Therapy
Figure 3.4 Mechanism of Action of Gene Therapies
Figure 3.5 Advantages and Disadvantages of Gene Therapies
Figure 4.1 Gene Transfer: Viral and Non-Viral Methods
Figure 5.1 Centralized Regulatory Pathway for Market Approval in the US
Figure 5.2 Centralized Regulatory Pathway for Market Approval in Europe
Figure 5.3 Manufacturing Requirements for Gene Therapy Products in China
Figure 5.4 Key Elements of Quality Control in China
Figure 5.5 Centralized Regulatory Pathway for Market Approval in Japan
Figure 6.1 Gene Therapies, Clinical Pipeline: Distribution by Phase of Development
Figure 6.2 Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Area
Figure 6.3 Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Area and Phase of Development
Figure 6.4 Gene Therapies, Clinical Pipeline: Distribution by Type of Vector Used
Figure 6.5 Gene Therapies, Clinical Pipeline: Distribution by Type of Gene
Figure 6.6 Gene Therapies, Clinical Pipeline: Distribution by Type of Modification
Figure 6.7 Gene Therapies, Clinical Pipeline: Distribution by Type of Modification and Type of Vector Used
Figure 6.8 Gene Therapies, Clinical Pipeline: Distribution by Type of Gene Therapy
Figure 6.9 Gene Therapies, Clinical Pipeline: Distribution by Route of Administration
Figure 6.10 Gene Therapies, Early Stage Pipeline: Distribution by Stage of Development
Figure 6.11 Gene Therapies, Early Stage Pipeline: Distribution by Therapeutic Area
Figure 6.12 Gene Therapies, Early Stage Pipeline: Distribution by Type of Vector Used
Figure 6.13 Gene Therapies, Early Stage Pipeline: Distribution by Type of Gene
Figure 6.14 Gene Therapies, Early Stage Pipeline: Distribution by Type of Modification
Figure 6.15 Gene Therapies, Early Stage Pipeline: Distribution by Type of Modification and Type of Vector Used
Figure 6.16 Gene Therapies, Early Stage Pipeline: Distribution by Type of Gene Therapy
Figure 6.17 Gene Therapies: Key Players
Figure 6.18 Gene Therapy Developers: Distribution by Year of Establishment
Figure 6.19 Gene Therapy Developers: Distribution by Size of Employee Base
Figure 6.20 Gene Therapies Developers: Distribution by Location of Headquarter
Figure 6.21 Gene Therapy Developers: North America
Figure 6.22 Gene Therapy Developers: Europe
Figure 6.23 Gene Therapy Developers: Asia Pacific
Figure 7.1 Gendicine®: Development Timeline
Figure 7.2 Oncorine®: Development Timeline
Figure 7.3 Oncorine®: Mechanism of Action
Figure 7.4 Rexin-G®: Development Timeline
Figure 7.5 Neovasculgen®: Development Timeline
Figure 7.6 Imlygic®: Development Timeline
Figure 7.7 Imlygic®: Mechanism of Action
Figure 7.8 Strimvelis®: Development Timeline
Figure 7.9 Invossa™: Development Timeline
Figure 7.10 Invossa™: Mechanism of Action
Figure 7.11 Luxturna™: Development Timeline
Figure 7.12 Zolgensma™: Development Timeline
Figure 7.13 Collategene®: Development Timeline
Figure 7.14 Zyntelgo™: Development Timeline
Figure 8.1 Successful Drug Launch Strategy: ROOTS Framework
Figure 8.2 Successful Drug Launch Strategy: Product Differentiation
Figure 8.3 Commonly Adopted Commercialization Strategies based on Development Stage of the Product
Figure 8.4 Harvey Ball Analysis: Commercialization Strategies Adopted by Companies Focused on Gene Therapies
Figure 8.5 Approved Gene Therapies: Historical Timeline of Geographical Expansion
Figure 8.6 Approved Gene Therapies: Snapshot of Promotional Activities on Product Websites
Figure 10.1 Gene Editing: Key Application Areas
Figure 10.2 Gene Editing: Emerging Technology Platforms
Figure 11.1 Gene Therapies: Distribution by Therapeutic Area and Special Designations Awarded
Figure 11.2 Gene Therapies for Cardiovascular Disorders: Distribution by Target Indication and Phase of Development
Figure 11.3 Gene Therapies for Cardiovascular Disorders: Distribution by Type of Vector Used
Figure 11.4 Gene Therapies for Hematological Disorders: Distribution by Target Indication and Phase of Development
Figure 11.5 Gene Therapies for Hematological Disorders: Distribution by Type of Vector Used
Figure 11.6 Gene Therapies for I&I Disorders: Distribution by Target Indication and Phase of Development
Figure 11.7 Gene Therapies for I&I Disorders: Distribution by Type of Vector Used
Figure 11.8 Gene Therapies for Metabolic Disorders: Distribution by Target Indication and Phase of Development
Figure 11.9 Gene Therapies for Metabolic Disorders: Distribution by Type of Vector Used
Figure 11.10 Gene Therapies for Muscular Disorders: Distribution by Target Indication and Phase of Development
Figure 11.11 Gene Therapies for Muscular Disorders: Distribution by Type of Vector Used
Figure 11.12 Gene Therapies for Neurological Disorders: Distribution by Target Indication and Phase of Development
Figure 11.13 Gene Therapies for Neurological Disorders: Distribution by Type of Vector Used
Figure 11.14 Gene Therapies for Ophthalmic Disorders: Distribution by Target Indication and Phase of Development
Figure 11.15 Gene Therapies for Ophthalmic Disorders: Distribution by Type of Vector Used
Figure 11.16 Gene Therapies for Oncology: Distribution by Target Indication and Phase of Development
Figure 11.17 Gene Therapies for Oncology: Distribution by Type of Vector Used
Figure 12.1 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Figure 12.2 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2019
Figure 12.3 Gene Therapy Patent Portfolio: Distribution by Geographical Location
Figure 12.4 Gene Therapy Patent Portfolio: Distribution by Geographical Location, North America
Figure 12.5 Gene Therapy Patent Portfolio: Distribution by Geographical Location, Europe
Figure 12.6 Gene Therapy Patent Portfolio: Distribution by Geographical Location, Asia-Pacific
Figure 12.7 Gene Therapy Patent Portfolio: Distribution by CPC Classification Symbols
Figure 12.8 Gene Therapy Patent Portfolio: Emerging Focus Areas
Figure 12.9 Gene Therapy Patent Portfolio: Leading Industry Players
Figure 12.10 Gene Therapy Patent Portfolio: Leading Non-Industry Players
Figure 12.11 Gene Therapy Patent Portfolio (Genentech and GSK): Benchmarking by Patent Characteristics
Figure 12.12 Gene Therapy Patent Portfolio, Leading Industry Players: Benchmarking by Patent Characteristics
Figure 12.13 Gene Therapy Patent Portfolio: Distribution of Patents by Age (2016-2019)
Figure 12.14 Gene Therapy Patent Portfolio: Valuation Analysis
Figure 12.15 Gene Editing Patent Portfolio: Distribution by Type of Patent
Figure 12.16 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2019
Figure 12.17 Gene Editing Patent Portfolio: Distribution by Geographical Location
Figure 12.18 Gene Editing Patent Portfolio: Distribution by Geographical Location, North America
Figure 12.19 Gene Editing Patent Portfolio: Distribution by Geographical Location, Europe
Figure 12.20 Gene Editing Patent Portfolio: Distribution by Geographical Location, Asia-Pacific
Figure 12.21 Gene Editing Patent Portfolio: Distribution by CPC Classification Symbols
Figure 12.22 Gene Editing Patent Portfolio: Emerging Focus Areas
Figure 12.23 Gene Editing Patent Portfolio: Leading Industry Players
Figure 12.24 Gene Editing Patent Portfolio: Leading Non-Industry Players
Figure 12.25 Gene Editing Patent Portfolio (Sangamo Therapeutics and Cellectis): Benchmarking by Patent Characteristics
Figure 12.26 Gene Editing Patent Portfolio (Leading Industry Players): Benchmarking by Patent Characteristics
Figure 12.27 Gene Editing Patent Portfolio: Distribution of Patents by Age (2016- 2019)
Figure 12.28 Gene Editing Patent Portfolio: Valuation Analysis
Figure 12.29 Gene Therapy and Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 13.1 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2014- 2019
Figure 13.2 Mergers and Acquisitions: Distribution by Type of Mergers and Acquisitions, 2014 - 2019
Figure 13.3 Mergers and Acquisitions: Distribution by Year and Type of Merger and Acquisition, 2014 - 2019
Figure 13.4 Mergers and Acquisitions: Continent-wise Distribution
Figure 13.5 Mergers and Acquisitions: Intercontinental and Intracontinental Acquisitions
Figure 13.6 Mergers and Acquisitions: Country-wise Distribution
Figure 13.7 Acquisitions: Distribution by Key Value Drivers
Figure 13.8 Acquisitions: Distribution by Key Value Drivers and Year of Acquisition, 2014-2019
Figure 13.9 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Figure 13.10 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product and Year of Acquisition
Figure 13.11 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product and Deal Amount
Figure 13.12 Mergers and Acquisitions: Distribution by Therapeutic Area
Figure 14.1 Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2019
Figure 14.2 Funding and Investment Analysis: Cumulative Number of Instances by Year, 2014-2019
Figure 14.3 Funding and Investment Analysis: Cumulative Amount Invested, 2014-2019 (USD Million)
Figure 14.4 Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2019
Figure 14.5 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2019
Figure 14.6 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2019 (USD Million)
Figure 14.7 Funding and Investment Analysis: Summary of Investments, 2014-2019 (USD Million)
Figure 14.8 Funding and Investments: Distribution by Amount Invested across Different Types of Gene Modification
Figure 14.9 Funding and Investment Analysis: Distribution by Geography
Figure 14.10 Funding and Investment Analysis: Regional Distribution of Funding Instances
Figure 14.11 Funding and Investment Analysis: Most Active Players, on the basis of Number of Instances, 2014-2019 (USD Million)
Figure 14.12 Funding and Investment Analysis: Most Active Players, on the basis of Amount Invested, 2014-2019 (USD Million)
Figure 14.13 Funding and Investment Analysis: Distribution by Type of Investors
Figure 14.14 Funding and Investment Analysis: Distribution by Big Pharma Players (Investors)
Figure 14.15 Funding and Investment Analysis: Distribution by Government Institutes (Investors)
Figure 14.16 Funding and Investment Analysis: Leading Investors
Figure 14.17 Funding and Investment Analysis: Distribution of Investments by Stage of Development of the Company’s Product
Figure 14.18 Funding and Investment Analysis: Distribution by Highest Phase of Development of the Company’s Product
Figure 14.19 Funding and Investment Summary, 2014-2019 (USD Million)
Figure 15.1 Gene Therapy: Pricing Model Based on Patient Segment
Figure 16.1 Big Pharma Players: Heat Map Analysis of Top Pharmaceutical Companies
Figure 16.2 Big Pharma Players: Analysis by Therapeutic Area
Figure 16.3 Big Pharma Players: Analysis by Type of Vector Used
Figure 16.4 Big Pharma Players: Analysis by Type of Modification
Figure 16.5 Big Pharma Players: Analysis by Type of Gene Therapy
Figure 16.6 Big Pharma Players: Heat Map Analysis of Other Players
Figure 17.1 Overall Gene Therapy Market, 2018-2030: Base Scenario (USD Million)
Figure 17.2 Gene Therapy Market: Distribution by Type of Gene Modification, 2019, 2025 and 2030
Figure 17.3 Gene Therapy Market: Distribution by Type of Therapy, 2019, 2025 and 2030
Figure 17.4 Gene Therapy Market: Distribution by Type of Vector Used, 2019, 2025 and 2030
Figure 17.5 Gene Therapy Market: Distribution by Therapeutic Area, 2019, 2025 and 2030
Figure 17.6 Gene Therapy Market: Distribution by Route of Administration, 2019, 2025 and 2030
Figure 17.7 Gene Therapy Market: Distribution by Geography, 2019, 2025 and 2030
Figure 17.8 Gene Therapy Market: Country-wise Distribution of Rest of the World Region, 2019, 2025 and 2030
Figure 17.9 Gene Therapy Market: Distribution by Key Players, 2019, 2025 and 2030
Figure 17.10 Gendicine® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.11 Oncorine® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.12 Rexin-G® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.13 Neovasculgen® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.14 Strimvelis® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.15 Imlygic® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.16 Invossa™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.17 Luxturna™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.18 Zolgensma™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.19 Collategene® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.20 Zyntelgo™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.21 Axalimogene Filolisbac Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.22 AMT-061 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.23 BIIB111 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.24 BIIB112 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.25 Donaperminogene Seltoplasmid (VM202) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.26 E10A Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.27 Fidanacogene Elaparvovec (PF-06838435) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.28 FLT180a Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.29 GS010 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.30 Instiladrin® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.31 Lenti-D™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.32 LYS-SAF302 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.33 Ofranergene Obadenovec (VB-111) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.34 OTL-101Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.35 OTL-103 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.36 OTL-200 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.37 Pexastimogene Devacirepvec (PEXA-VEC) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.38 ProstAtak® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.39 SPK-8011 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.40 Unnamed Therapy Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.41 Valoctocogene Roxaparvovec (BMN 270) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.42 Vigil® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.43 VGX-3100 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.44 Vocimagene Amiretrorepvec (Toca-511) Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 18.1 Viral Vectors: Manufacturing Steps
Figure 19.1 Steps Involved in the Development of Gene Therapies
Figure 19.2 Workflow of Cell-based Gene Therapies
Figure 19.3 Key Responsibilities of Stakeholders Involved in Advanced Therapies Supply Chain
Figure 19.4 Key Steps Involved in the Implementation of a Supply Chain Strategy
Figure 19.5 Keys Steps in the Logistics of Autologous Therapies
Figure 19.6 Comparison of Logistics Processes for Autologous and Allogeneic Therapies
Figure 19.7 Supply Chain Regulatory Authorities Across the Globe
Figure 19.8 Overview of Supply Chain Orchestration Platform
Figure 19.9 Functions of Key Technological Solutions for Cell and Advanced Therapies Supply Chain Management
Table 3.1 Comparison of Ex Vivo and In Vivo Techniques
Table 3.2 Vectors used for Targeted Gene Delivery to Tissues / Organs
Table 3.3 Routes of Administration of Gene Therapies: Advantages and Disadvantages
Table 4.1 Viral Vectors: Key Features
Table 5.1 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
Table 5.2 Payment Options Available for Gene Therapies
Table 6.1 Gene Therapy Market: Clinical Pipeline
Table 6.2 Gene Therapy Market: Early Stage Pipeline
Table 6.3 Gene Therapy Market: Developer Overview
Table 7.1 Gene Therapies: Marketed and Approved Products
Table 7.2 Company Overview: Shenzhen Sibiono GeneTech
Table 7.3 Gendicine®: Patent Portfolio
Table 7.4 Gendicine®: Status of Development
Table 7.5 Company Overview: Shanghai Sunway Biotech
Table 7.6 Oncorine®: Status of Development
Table 7.7 Company Overview: Epeius Biotechnologies
Table 7.8 Rexin-G®: Status of Development
Table 7.9 Company Overview: Human Stem Cell Institute
Table 7.10 Neovasculgen®: Status of Development
Table 7.11 Company Overview: Amgen
Table 7.12 Imlygic®: Status of Development
Table 7.13 Imlygic®: Recommended Dose and Schedule
Table 7.14 Imlygic®: Determination of Injection Volume based upon Lesion Size
Table 7.15 Company Overview: Orchard Therapeutics
Table 7.16 Strimvelis®: Status of Development
Table 7.17 Company Overview: TissueGene
Table 7.18 Invossa™: Status of Development
Table 7.19 Company Overview: Spark Therapeutics
Table 7.20 Luxturna™: Status of Development
Table 7.21 Company Overview: AveXis (A Novartis Company)
Table 7.22 Zolgensma™: Status of Development
Table 7.23 Company Overview: AnGes
Table 7.24 Collategene®: Status of Development
Table 7.25 Company Overview: bluebird bio
Table 7.26 Zyntelgo™: Status of Development
Table 8.1 Luxturna™: List of Conferences Attended Before Approval
Table 8.2 Zolgensma™: List of Conferences Attended Before Approval
Table 8.3 Luxturna™: List of Conferences Attended Post Approval
Table 8.4 Strimvelis™: List of Conferences Attended Post Approval
Table 8.5 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
Table 8.6 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
Table 9.1 Gene Therapies: Phase III Drugs
Table 9.2 Drug Profile: Axalimogene filolisbac
Table 9.3 Drug Profile: AMT-061
Table 9.4 Drug Profile: BIIB111
Table 9.5 Drug Profile: BIIB112
Table 9.6 Drug Profile: Donaperminogene Seltoplasmid (VM202)
Table 9.7 Drug Profile: E10A
Table 9.8 Drug Profile: Fidanacogene Elaparvovec (PF-06838435)
Table 9.9 Drug Profile: FLT180a
Table 9.10 Drug Profile: GS010
Table 9.11 Drug Profile: Instiladrin®
Table 9.12 Drug Profile: Lenti-D™
Table 9.13 Drug Profile: LYS-SAF302
Table 9.14 Drug Profile: Ofranergene Obadenovec (VB-111)
Table 9.15 Drug Profile: OTL-101
Table 9.16 Drug Profile: OTL-103
Table 9.17 Drug Profile: OTL-200
Table 9.18 Drug Profile: Pexastimogene Devacirepvec (PEXA-VEC)
Table 9.19 Drug Profile: ProstAtak®
Table 9.20 Drug Profile: SPK-8011
Table 9.21 Drug Profile: Unnamed Therapy
Table 9.22 Drug Profile: Valoctocogene Roxaparvovec (BMN 270)
Table 9.23 Drug Profile: Vigil®
Table 9.24 Drug Profile: VGX-3100
Table 9.25 Drug Profile: Vocimagene Amiretrorepvec (Toca-511)
Table 10.1 Gene Editing Technology Platforms
Table 10.2 CRISPR Therapeutics: Funding Instances
Table 10.3 CRISPR Therapeutics: Collaborations
Table 10.4 Editas Medicine: Funding Instances
Table 10.5 Editas Medicine: Collaborations
Table 10.6 Intellia Therapeutics: Funding Instances
Table 10.7 Intellia Therapeutics: Collaborations
Table 10.8 Cellectis: Funding Instances
Table 10.9 Cellectis: Collaborations
Table 10.10 bluebird bio: Funding Instances
Table 10.11 bluebird bio: Collaborations
Table 10.12 Sangamo Therapeutics: Funding Instances
Table 10.13 Sangamo Therapeutics: Collaborations
Table 10.14 Gene Switch Technology Platforms
Table 10.15 Intrexon: Funding Instances
Table 10.16 Intrexon: Collaborations
Table 10.17 MeiraGTx: Funding Instances
Table 10.18 MeiraGTx: Collaborations
Table 10.19 Gene Therapy: Technology Platforms
Table 11.1 Gene Therapies for Cardiovascular Disorders
Table 11.2 Gene Therapies for Hematological Disorders
Table 11.3 Gene Therapies for I&I Disorders
Table 11.4 Gene Therapies for Metabolic Disorders
Table 11.5 Gene Therapies for Muscular Disorders
Table 11.6 Gene Therapies for Neurological Disorders
Table 11.7 Gene Therapies for Ophthalmic Disorders
Table 11.8 Gene Therapies for Oncology
Table 12.1 Gene Therapy Patent Portfolio: CPC Classification Symbol Definitions
Table 12.2 Gene Therapy Patent Portfolio: Most Popular CPC Classification Symbols
Table 12.3 Gene Therapy Patent Portfolio: List of Top CPC Classifications
Table 12.4 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
Table 12.5 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 12.6 Gene Therapy Patent Portfolio: List of Leading Patents (by Highest Relative Valuation)
Table 12.7 Gene Editing Patent Portfolio: CPC Classification Symbol Definitions
Table 12.8 Gene Editing Patent Portfolio: Most Popular CPC Classification Symbols
Table 12.9 Gene Editing Patent Portfolio: List of Top CPC Classifications
Table 12.10 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
Table 12.11 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 12.12 Gene Editing Patent Portfolio: List of Leading Patents (by Highest Relative Valuation)
Table 13.1 Gene Therapy: List of Mergers and Acquisitions, 2014-2019
Table 13.2 Acquisitions: Key Value Drivers
Table 13.3 Mergers and Acquisitions: Therapeutic Areas and Phase of Development of the Acquired Company’s Product
Table 14.1 Gene Therapy Market: Funding and Investments, 2014-2019
Table 14.2 Funding and Investment Analysis: Summary of Investments
Table 14.3 Funding and Investment Analysis: Summary of Venture Capital Funding
Table 15.1 Pricing Model: Price of Marketed Gene / Cell Therapies
Table 15.2 Pricing Model: Price of Marketed Targeted Drugs
Table 15.3 Pricing Model: Opinions of Experts / Other Analysts
Table 17.1 Gene Therapies: Expected Launch Years of Advanced Stage Drug Candidates
Table 17.2 Gendicine®: Target Patient Population
Table 17.3 Gendicine®: Net Present Value (USD Million)
Table 17.4 Gendicine®: Value Creation Analysis (USD Million)
Table 17.5 Oncorine®: Target Patient Population
Table 17.6 Oncorine®: Net Present Value (USD Million)
Table 17.7 Oncorine®: Value Creation Analysis (USD Million)
Table 17.8 Rexin-G®: Target Patient Population
Table 17.9 Rexin-G®: Net Present Value (USD Million)
Table 17.10 Rexin-G®: Value Creation Analysis (USD Million)
Table 17.11 Neovasculgen®: Target Patient Population
Table 17.12 Neovasculgen®: Net Present Value (USD Million)
Table 17.13 Neovasculgen®: Value Creation Analysis (USD Million)
Table 17.14 Strimvelis®: Target Patient Population
Table 17.15 Strimvelis®: Net Present Value (USD Million)
Table 17.16 Strimvelis®: Value Creation Analysis (USD Million)
Table 17.17 Imlygic®: Target Patient Population
Table 17.18 Imlygic®: Net Present Value (USD Million)
Table 17.19 Imlygic®: Value Creation Analysis (USD Million)
Table 17.20 Invossa™: Target Patient Population
Table 17.21 Invossa™: Net Present Value (USD Million)
Table 17.22 Invossa™: Value Creation Analysis (USD Million)
Table 17.23 Luxturna™: Target Patient Population
Table 17.24 Luxturna™: Net Present Value (USD Million)
Table 17.25 Luxturna™: Value Creation Analysis (USD Million)
Table 17.26 Axalimogene Filolisbac: Target Patient Population
Table 17.27 Axalimogene Filolisbac: Net Present Value (USD Million)
Table 17.28 Axalimogene Filolisbac: Value Creation Analysis (USD Million)
Table 17.29 AMT-061: Target Patient Population
Table 17.30 AMT-061: Net Present Value (USD Million)
Table 17.31 AMT-061: Value Creation Analysis (USD Million)
Table 17.32 BIIB111: Target Patient Population
Table 17.33 BIIB111: Net Present Value (USD Million)
Table 17.34 BIIB111: Value Creation Analysis (USD Million)
Table 17.35 BIIB112: Target Patient Population
Table 17.36 BIIB112: Net Present Value (USD Million)
Table 17.37 BIIB112: Value Creation Analysis (USD Million)
Table 17.38 Donaperminogene Seltoplasmid (VM202): Target Patient Population
Table 17.39 Donaperminogene Seltoplasmid (VM202): Net Present Value (USD Million)
Table 17.40 Donaperminogene Seltoplasmid (VM202): Value Creation Analysis (USD Million)
Table 17.41 E10A: Target Patient Population
Table 17.42 E10A: Net Present Value (USD Million)
Table 17.43 E10A: Value Creation Analysis (USD Million)
Table 17.44 Fidanacogene Elaparvovec (PF-06838435): Target Patient Population
Table 17.45 Fidanacogene Elaparvovec (PF-06838435): Net Present Value (USD Million)
Table 17.46 Fidanacogene Elaparvovec (PF-06838435): Value Creation Analysis (USD Million)
Table 17.47 FLT180a: Target Patient Population
Table 17.48 FLT180a: Net Present Value (USD Million)
Table 17.49 FLT180a: Value Creation Analysis (USD Million)
Table 17.50 GS010: Target Patient Population
Table 17.51 GS010: Net Present Value (USD Million)
Table 17.52 GS010: Value Creation Analysis (USD Million)
Table 17.53 Instiladrin®: Target Patient Population
Table 17.54 Instiladrin®: Net Present Value (USD Million)
Table 17.55 Instiladrin®: Value Creation Analysis (USD Million)
Table 17.56 Lenti-D™: Target Patient Population
Table 17.57 Lenti-D™: Net Present Value (USD Million)
Table 17.58 Lenti-D™: Value Creation Analysis (USD Million)
Table 17.59 LYS-SAF302: Target Patient Population
Table 17.60 LYS-SAF302: Net Present Value (USD Million)
Table 17.61 LYS-SAF302: Value Creation Analysis (USD Million)
Table 17.62 Ofranergene Obadenovec (VB-111): Target Patient Population
Table 17.63 Ofranergene Obadenovec (VB-111): Net Present Value (USD Million)
Table 17.64 Ofranergene Obadenovec (VB-111): Value Creation Analysis (USD Million)
Table 17.65 OTL-101: Target Patient Population
Table 17.66 OTL-101: Net Present Value (USD Million)
Table 17.67 OTL-101: Value Creation Analysis (USD Million)
Table 17.68 OTL-103: Target Patient Population
Table 17.69 OTL-103: Net Present Value (USD Million)
Table 17.70 OTL-103: Value Creation Analysis (USD Million)
Table 17.71 OTL-200: Target Patient Population
Table 17.72 OTL-200: Net Present Value (USD Million)
Table 17.73 OTL-200: Value Creation Analysis (USD Million)
Table 17.74 Pexastimogene Devacirepvec (PEXA-VEC): Target Patient Population
Table 17.75 Pexastimogene Devacirepvec (PEXA-VEC): Net Present Value (USD Million)
Table 17.76 Pexastimogene Devacirepvec (PEXA-VEC): Value Creation Analysis (USD Million)
Table 17.77 ProstAtak®: Target Patient Population
Table 17.78 ProstAtak®: Net Present Value (USD Million)
Table 17.79 ProstAtak®: Value Creation Analysis (USD Million)
Table 17.80 SPK-8011: Target Patient Population
Table 17.81 SPK-8011: Net Present Value (USD Million)
Table 17.82 SPK-8011: Value Creation Analysis (USD Million)
Table 17.83 Unnamed Therapy: Target Patient Population
Table 17.84 Unnamed Therapy: Net Present Value (USD Million)
Table 17.85 Unnamed Therapy: Value Creation Analysis (USD Million)
Table 17.86 Valoctocogene Roxaparvovec (BMN 270): Target Patient Population
Table 17.87 Valoctocogene Roxaparvovec (BMN 270): Net Present Value (USD Million)
Table 17.88 Valoctocogene Roxaparvovec (BMN 270): Value Creation Analysis (USD Million)
Table 17.89 Vigil®: Target Patient Population
Table 17.90 Vigil®: Net Present Value (USD Million)
Table 17.91 Vigil®: Value Creation Analysis (USD Million)
Table 17.92 VGX-3100: Target Patient Population
Table 17.93 VGX-3100: Net Present Value (USD Million)
Table 17.94 VGX-3100: Value Creation Analysis (USD Million)
Table 17.95 Vocimagene Amiretrorepvec (Toca-511): Target Patient Population
Table 17.96 Vocimagene Amiretrorepvec (Toca-511): Net Present Value (USD Million)
Table 17.97 Vocimagene Amiretrorepvec (Toca-511): Value Creation Analysis (USD Million)
Table 18.1 Small Scale Cell Culture Systems
Table 18.2 Contract Manufacturing Service Providers for Viral Vectors and Plasmid DNA
Table 20.1 Gene Therapy Market: Key Takeaways
Table 22.1 Gene Therapies, Clinical Pipeline: Distribution by Phase of Development
Table 22.2 Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Area
Table 22.3 Gene Therapies, Clinical Pipeline: Distribution by Therapeutic Area and Phase of Development
Table 22.4 Gene Therapies, Clinical Pipeline: Distribution by Type of Vector Used
Table 22.5 Gene Therapies, Clinical Pipeline: Distribution by Type of Gene
Table 22.6 Gene Therapies, Clinical Pipeline: Distribution by Type of Modification
Table 22.7 Gene Therapies, Clinical Pipeline: Distribution by Type of Modification and Type of Vector Used
Table 22.8 Gene Therapies, Clinical Pipeline: Distribution by Type of Gene Therapy
Table 22.9 Gene Therapies, Clinical Pipeline: Distribution by Route of Administration
Table 22.10 Gene Therapies, Early Stage Pipeline: Distribution by Stage of Development
Table 22.11 Gene Therapies, Early Stage Pipeline: Distribution by Therapeutic Areas
Table 22.12 Gene Therapies, Early Stage Pipeline: Distribution by Type of Vector Used
Table 22.13 Gene Therapies, Early Stage Pipeline: Distribution by Type of Gene
Table 22.14 Gene Therapies, Early Stage Pipeline: Distribution by Type of Modification
Table 22.15 Gene Therapies, Early Stage Pipeline: Distribution by Type of Modification and Type of Vector Used
Table 22.16 Gene Therapies, Early Stage Pipeline: Distribution by Type of Gene Therapy
Table 22.17 Gene Therapies, Early Stage Pipeline: Distribution by Route of Administration
Table 22.18 Gene Therapies: Key Players
Table 22.19 Gene Therapy Developers: Distribution by Year of Establishment
Table 22.20 Gene Therapy Developers : Distribution by Size of Employee Base
Table 22.21 Gene Therapy Developers : Distribution by Location of Headquarter
Table 22.22 Gene Therapies: Distribution by Therapeutic Area and Special Designations Awarded
Table 22.23 Gene Therapies for Cardiovascular Disorders: Distribution by Target Indication and Phase of Development
Table 22.24 Gene Therapies for Cardiovascular Disorders: Distribution by Type of Vector Used
Table 22.25 Gene Therapies for Hematological Disorders: Distribution by Target Indication and Phase of Development
Table 22.26 Gene Therapies for Hematological Disorders: Distribution by Type of Vector Used
Table 22.27 Gene Therapies for I&I Disorders: Distribution by Target Indication and Phase of Development
Table 22.28 Gene Therapies for I&I Disorders: Distribution by Type of Vector Used
Table 22.29 Gene Therapies for Metabolic Disorders: Distribution by Target Indication and Phase of Development
Table 22.30 Gene Therapies for Metabolic Disorders: Distribution by Type of Vector Used
Table 22.31 Gene Therapies for Muscular Disorders: Distribution by Target Indication and Phase of Development
Table 22.32 Gene Therapies for Muscular Disorders: Distribution by Type of Vector Used
Table 22.33 Gene Therapies for Neurological Disorders: Distribution by Target Indication and Phase of Development
Table 22.34 Gene Therapies for Neurological Disorders: Distribution by Type of Vector Used
Table 22.35 Gene Therapies for Ophthalmic Disorders: Distribution by Target Indication and Phase of Development
Table 22.36 Gene Therapies for Ophthalmic Disorders: Distribution by Type of Vector Used
Table 22.37 Gene Therapies for Oncology: Distribution by Target Indication and Phase of Development
Table 22.38 Gene Therapies for Oncology: Distribution by Type of Vector Used
Table 22.39 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Table 22.40 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2019
Table 22.41 Gene Therapy Patent Portfolio: Distribution by Geographical Location
Table 22.42 Gene Therapy Patent Portfolio: Distribution by Geographical Location, North America
Table 22.43 Gene Therapy Patent Portfolio: Distribution by Geographical Location, Europe
Table 22.44 Gene Therapy Patent Portfolio: Distribution by Geographical Location, Asia Pacific
Table 22.45 Gene Therapy Patent Portfolio: Distribution by CPC Classification Symbols
Table 22.46 Gene Therapy Patent Portfolio: Emerging Focus Areas
Table 22.47 Gene Therapy Patent Portfolio: Leading Industry Players
Table 22.48 Gene Therapy Patent Portfolio: Leading Non-Industry Players
Table 22.49 Gene Therapy Patent Portfolio (Genentech and GSK): Benchmarking by Patent Characteristics
Table 22.50 Gene Therapy Patent Portfolio, Leading Industry Players: Benchmarking by Patent Characteristics
Table 22.51 Gene Therapy Patent Portfolio: Distribution of Patents by Age (2016-2019)
Table 22.52 Gene Therapy Patent Portfolio: Valuation Analysis
Table 22.53 Gene Editing Patent Portfolio: Distribution by Type of Patent
Table 22.54 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2019
Table 22.55 Gene Editing Patent Portfolio: Distribution by Geographical Location
Table 22.56 Gene Editing Patent Portfolio: Distribution by Geographical Location, North America
Table 22.57 Gene Editing Patent Portfolio: Distribution by Geographical Location, Europe
Table 22.58 Gene Editing Patent Portfolio: Distribution by Geographical Location, Asia-Pacific
Table 22.59 Gene Editing Patent Portfolio: Distribution by CPC Classification Symbols
Table 22.60 Gene Editing Patent Portfolio: Emerging Focus Areas
Table 22.61 Gene Editing Patent Portfolio: Leading Industry Players
Table 22.62 Gene Editing Patent Portfolio: Leading Non-Industry Players
Table 22.63 Gene Editing Patent Portfolio (Sangamo Therapeutics and Cellectis): Benchmarking by Patent Characteristics
Table 22.64 Gene Editing Patent Portfolio, Leading Industry Players: Benchmarking by Patent Characteristics
Table 22.65 Gene Editing Patent Portfolio: Distribution of Patents by Age (2016- 2019)
Table 22.66 Gene Editing Patent Portfolio: Valuation Analysis
Table 22.67 Gene Therapy and Gene Editing Patent Portfolio: Distribution by Type of Organization
Table 22.68 Mergers and Acquisitions: Cumulative Year-Wise Trend (2014- 2019)
Table 22.69 Mergers and Acquisitions: Distribution by Type of Mergers and Acquisitions (2014 - 2019)
Table 22.70 Mergers and Acquisitions: Distribution by Year and Type of Mergers and Acquisitions (2014 - 2019)
Table 22.71 Mergers and Acquisitions: Continent-wise Distribution
Table 22.72 Mergers and Acquisitions: Intercontinental and Intracontinental Acquisitions
Table 22.73 Mergers and Acquisitions: Country-wise Distribution
Table 22.74 Acquisitions: Distribution by Key Value Drivers
Table 22.75 Acquisitions: Distribution by Key Value Drivers and Year of Acquisition (2014-2019)
Table 22.76 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Table 22.77 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product and Year of Acquisition
Table 22.78 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product and Year of Acquisition
Table 22.79 Mergers and Acquisitions: Distribution by Therapeutic Area
Table 22.80 Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2019
Table 22.81 Funding and Investment Analysis: Cumulative Number of Instances by Year, 2014-2019
Table 22.82 Funding and Investment Analysis: Cumulative Amount Invested, 2014-2019 (USD Million)
Table 22.83 Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2019
Table 22.84 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2019
Table 22.85 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2019 (USD Million)
Table 22.86 Funding and Investment Analysis: Summary of Investments, 2014-2019 (USD Million)
Table 22.87 Funding and Investments: Distribution by Amount Invested across Different Types of Therapies
Table 22.88 Funding and Investment Analysis: Distribution by Geography
Table 22.89 Funding and Investment Analysis: Regional Distribution of Funding Instances
Table 22.90 Funding and Investment Analysis: Most Active Players, on the basis of Number of Instances, 2014-2019 (USD Million)
Table 22.91 Funding and Investment Analysis: Most Active Players, on the basis of Amount Invested, 2014-2019 (USD Million)
Table 22.92 Funding and Investment Analysis: Distribution by Type of Investors
Table 22.93 Funding and Investment Analysis: Distribution by Big Pharma Players (Investors)
Table 22.94 Funding and Investment Analysis: Distribution by Government Institutes (Investors)
Table 22.95 Funding and Investment Analysis: Leading Investors
Table 22.96 Funding and Investment Analysis: Distribution of Investments by Stages of Development
Table 22.97 Funding and Investment Analysis: Distribution by Highest Phase of Development of the Company’s Product for Funding
Table 22.98 Funding and Investment Summary, 2014-2019 (USD Million)
Table 22.99 Overall Gene Therapy Market, 2018-2030: Base Scenario (USD Million)
Table 22.100 Gene Therapy Market: Distribution by Type of Gene Modification, 2019, 2025 and 2030
Table 22.101 Gene Therapy Market: Distribution by Type of Therapy, 2019, 2025 and 2030
Table 22.102 Gene Therapy Market: Distribution by Type of Vector Used, 2019, 2025 and 2030
Table 22.103 Gene Therapy Market: Distribution by Therapeutic Area, 2019, 2025 and 2030
Table 22.104 Gene Therapy Market: Distribution by Route of Administration, 2019, 2025 and 2030
Table 22.105 Gene Therapy Market: Distribution by Geography, 2019, 2025 and 2030
Table 22.106 Gene Therapy Market: Distribution by Geography (Rest of the World), 2019, 2025 and 2030
Table 22.107 Gene Therapy Market: Key Players, 2019, 2025 and 2030
Table 22.108 Gendicine® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.109 Oncorine® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.110 Rexin-G® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.111 Neovasculgen® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.112 Strimvelis® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.113 Imlygic® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.114 Invossa™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.115 Luxturna™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.116 Zolgensma™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.117 Collategene® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.118 Zyntelgo™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.119 Axalimogene Filolisbac Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.120 AMT-061 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.121 BIIB111 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.122 BIIB112 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.123 Donaperminogene Seltoplasmid (VM202) Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.124 E10A Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.125 Fidanacogene Elaparvovec (PF-06838435) Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.126 FLT180a Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.127 GS010 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.128 Instiladrin® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.129 Lenti-D™ Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.130 LYS-SAF302 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.131 Ofranergene Obadenovec (VB-111) Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.132 OTL-101Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.133 OTL-103 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.134 OTL-200 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.135 Pexastimogene Devacirepvec (PEXA-VEC) Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.136 ProstAtak® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.137 SPK-8011 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.138 Unnamed Therapy Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.139 Valoctocogene Roxaparvovec (BMN 270) Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.140 Vigil® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.141 VGX-3100 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 22.142 Vocimagene Amiretrorepvec (Toca-511) Sales Forecast (Till 2030): Base Scenario (USD Million)
The following companies / institutes / government bodies and organizations have been mentioned in this report.
The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:
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Personalized medicine has brought about a paradigm shift within the healthcare sector. However, therapies tailored to specific disease-related molecular signatures require appropriate companion diagnostics in order to make physicians aware of patients’ unique genetic profiles, enabling them to make informed treatment related decisions. In fact, a clinical study of nearly 200 unique pharmacological interventions, which were evaluated across more than 670 clinical trials, suggests that the likelihood of a lead compound passing through various phases of clinical development and eventually getting approved is only 11%. The same study pointed out that using disease-specific biomarker information (indicative of susceptibility to particular types of therapeutics) to recruit patients for clinical research has been associated with a manifold increase in trial success rates. In addition, it is worth noting that companion diagnostics guided drug development efforts have demonstrated to effectively reduce clinical trial costs by almost 60%. Given the aforementioned advantages, the industry is gradually shifting from the traditional, one-drug-for-all, paradigm to using tailored pharmacological interventions. This shift is subsequently expected to increase the demand for companion diagnostics. However, given the complexity associated with the co-development of a drug and a corresponding companion diagnostic test, pharmaceutical developers have shown preference to outsource the diagnostics development operations. In fact, nearly 80% of the companies are known to rely on external diagnostics developers for companion diagnostics development, mostly owing to the lack of in-house expertise. As a result, numerous contract service providers are striving to expand their respective portfolios and developing the capabilities to offer end-to-end services to sponsor companies in this domain. Amidst tough competition, the availability of cutting-edge tools and technologies (such as in situ hybridization (ISH), immunohistochemistry (IHC), next generation sequencing (NGS), polymerase chain reaction (PCR)) has emerged as a differentiating factor and is likely to grant a competitive advantage to certain service providers over other players in the industry. Scope of the Report The “Companion Diagnostics Development Services Market, 2020-2030” report features an extensive study of the current market landscape, offering an informed opinion on the likely adoption of diagnostic development services over the next decade. It features an in-depth analysis, highlighting the capabilities of the various stakeholders in this domain. In addition to other elements, the study includes: A detailed assessment of the current market landscape of companies offering companion diagnostics services, including information on the type of services offered, type of analytical technique used and regulatory certifications / accreditations, and other company-specific details (such as year of establishment, company size and geographical location). Tabulated profiles of companion diagnostics service providers (shortlisted on the basis of the number of services offered), featuring an overview of the company, its financial information (if available), and companion diagnostics-related service portfolio details. In addition, each profile includes a list of the likely strategies that may be adopted by these players to support future growth. An analysis of the partnerships and collaborations pertaining to companion diagnostics services from 2017 to 2019, featuring a detailed set of analyses based on various parameters, such as the type of partnership, year of partnership, analytical technique used and the most active players. A list of stakeholders generated based on a detailed analysis of a set of relevant parameters (namely number of clinical trials sponsored by a developer and the time to market for proprietary personalized medicine products), which are anticipated to partner with companion diagnostics services providers in the foreseen future. A detailed competitiveness analysis of companion diagnostics services providers, taking into consideration the supplier power (based on the year of establishment of developer) and key specifications, such as portfolio strength, type of available technology platform, number of deals signed between 2017-2019. A comparative analysis of the needs of different stakeholders (drug developers, diagnostic developers, testing laboratories, physicians, payers and patients) involved in this domain. A discussion on various steps of the development operations, namely research and development, clinical assessment of the product, manufacturing and assembly, payer negotiation and marketing / sales activities, of a companion diagnostic and the cost requirements across each of the aforementioned stages. An analysis of completed, ongoing and planned clinical trials featuring disease-specific biomarkers. The analysis highlights the key trends associated with these clinical studies across various parameters, such as trial start year, trial status, phase of development, key indications, type of therapy, biomarkers evaluated, enrolled patient population and regional distribution of trials. One of the key objectives of the report was to estimate the existing market size and the future opportunity for companion diagnostic services providers, over the next decade. Based on multiple parameters, 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] key services offered (biomarker discovery, assay development, clinical validation, analytical validation and manufacturing), [B] analytical techniques used (ISH, IHC, NGS, PCR and others), and [C] key geographical regions (North America, Europe, Asia 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 research, analysis and insights presented in this report are backed by a deep understanding of key insights gathered from both secondary and primary research. The opinions and insights presented in the report were influenced by discussions held with senior stakeholders in the industry. The report features detailed transcripts of discussions held with the following industry stakeholders: Pablo Ortiz (Chief Executive Officer, OWL Metabolomics) Paul Kortschak (Senior Vice President, Novodiax) Lawrence M. Weiss (Chief Scientific Officer, NeoGenomics Laboratories) 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. ...read more
[COVID-19 SERIES] Advances in DNA sequencing technologies have led to significant developments in a variety of healthcare-focused research fields, such as precision medicine and diagnostics. Particularly, the impact of next generation sequencing (NGS) methods, enabling whole genome and whole exome sequencing, has been the most profound. This high throughput, parallel genome sequencing technology has greatly reduced the overall cost and time investment. In fact, compared to the Human Genome Project (~USD 3 billion), the cost of sequencing a single genome has decreased to USD 1,000, using currently available technologies. Owing to the ongoing innovation in this field, stakeholders believe that the aforementioned cost may get further reduced to USD 100 over the next decade. This decrease in genome sequencing costs has led to a marked increase in the number of genomes being sequenced around the world. In fact, several large scale efforts, such as UK Biobank and GenomeAsia 100k, have been initiated in order to collect genomic data for use in medical research. Big pharma players, including AstraZeneca, GSK, Pfizer, Merck and Roche, are actively on the lookout for collaborating with such data repositories in order to access the aforementioned information. Despite the progress made in this field of research, there are several existing challenges related to the NGS process affiliated workflow and data analysis. The lack of versatile in silico tools is considered to be the major rate-limiting step in NGS data analysis and interpretations. At present, industry stakeholders are actively collaborating in order to integrate their respective resources for mining these large and complex datasets to generate clinically relevant, actionable insights. Additionally, there is a need for better genomic library preparation protocols, which required less starting material, and are capable of generating libraries with more precisely estimated insert sizes and longer reads at reduced error rates. More efficient genome assembly algorithms and better processors (increased computational power) for genomic data processing are also likely to get developed. We are led to believe that, once the aforementioned challenges are addressed, this segment of the biopharmaceutical industry will witness significant growth. Scope of the Report The ‘Next Generation Sequencing (NGS) Market, 2020-2030: Service Providers (Whole Genome, Whole Exome and Targeted Sequencing) and Technology Platforms’ report features an extensive study of the current landscape and the future opportunities associated with service / technologies providers. Amongst other elements, the report features: An overview of the genome sequencing service providers landscape, featuring information on year of establishment, company size, geographical location and types of services offered (sanger sequencing, genotyping, whole genome sequencing, whole exome sequencing targeted sequencing and bioinformatics). Further, it provides details on the cost of services, sequencing systems used, average turn-around time and sequencing coverage, for certain types of sequencing-related services (whole genome, whole exome and targeted sequencing) offered by contract service providers. An overview of genome sequencing technologies landscape, featuring information on type of applications, run time, maximum reads per run, maximum sequencing output, maximum read length, type of sequencing technique, quality score and cost. It also provides information on the technology providers involved in this domain, including information on year of establishment, company size and geographical location. An informed competitiveness analysis of the genome sequencing technologies captured in our database, taking into consideration relevant parameters, such as supplier power (based on company size of technology provider) and other important technology-related specifications, such as types of applications, maximum sequencing output, maximum reads per run, maximum read length, quality score and cost of sequencer. An in-depth analysis of intellectual property related to this field of research, in order to generate an opinion on how the industry has evolved from the R&D perspective. The analysis takes into consideration genome sequencing-related patents that have been filed / granted since 2015, highlighting publication year, issuing authority / patent offices involved, CPC symbols, emerging focus areas, leading players, patent characteristics and geography. An analysis of completed, ongoing and planned clinical studies related to genome sequencing, featuring details on registration year, type of sponsors / collaborators, current status of trials, type of study design, target therapeutic area, type of application, regional distribution of clinical trials and enrolled patient population. An analysis of the various genome sequencing-focused initiatives of the ten big pharma players (shortlisted based on extent of activity in genome sequencing domain), highlighting the key focus areas of such companies along with information on funding, collaboration and acquisition activity. A case study on the various national and international, government sponsored initiatives related to genome sequencing, analyzed on the basis of year of initiation, type of investors, type of participant organization, research objectives, geographical distribution, region-specific data access policies and key focus areas of research. One of the key objectives of the report was to understand the primary growth drivers and estimate the future opportunity within the genome sequencing services and technologies market. Based on several parameters, such as number of genomes sequenced annually, average cost of sequencing, revenues generated by major players and expected annual growth rate, we have provided an informed estimate of the likely evolution of the market, for the period 2020-2030. The chapter also presents a detailed segmentation of the aforementioned opportunity across [A] key application areas (diagnostics, drug discovery, precision medicine and others), [B] end-users (hospitals and clinics, academics and research institutes, pharmaceuticals companies and others), [C] types of technologies (sequencing by synthesis, ion semiconductor, single-molecule real-time sequencing, nanopore and others), [D] types of services (whole genome sequencing, whole exome sequencing and targeted sequencing) and [E] key geographical regions (North America, Asia-Pacific, Europe, 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. In addition, the report features detailed transcripts of interviews held with the following individuals (in alphabetical order of company names): Michael Powell (Chief Scientific Officer, DiaCarta) Mike Klein (Chief Executive Officer, Genomenon) All actual figures have been sourced and analyzed from publicly available information forums. Financial figures mentioned in this report are in USD, unless otherwise specified. ...read more
The 1970s witnessed the introduction of one of the most revolutionary advances in biotechnology, when Hamilton Smith (a molecular biologist at Johns Hopkins University School of Medicine) purified the first site-specific restriction enzyme, called Hind II. This development enabled the scientific community to devise the means to manipulate living organisms at the genetic level, opening up a plethora of opportunities in fundamental and applied life science research. Soon after, in the 1980s, the US FDA approved the world’s first genetically engineered drug, which was human insulin marketed under the brand name HUMULIN®. Genetic engineering and genome editing concepts have evolved significantly over the last two decades, with the development of a variety of versatile DNA modulation technologies including zinc finger nucleases, transcription activator-like effector-based nucleases (TALENs), and the clustered regularly interspaced short palindromic repeats (CRISPR). These developments have enabled medical researchers to perform an array of gene / cell line engineering experiments, including gene knockdowns, gene overexpressions and single base editing, for a variety of R&D applications. It is worth highlighting that, of all the available genome manipulation technologies, CRISPR is currently considered to be the most popular tool, owing to its fast, accurate, and cost-effective approach. In fact, in 2018, scientists Emmanuelle Charpentier, Jennifer Doudna, and Virginijus Siksnys were felicitated for their pioneering efforts on the CRISPR-Cas9 technology, with the prestigious Kavli Prize in Nanoscience. Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The biopharmaceutical pipeline includes close to 500 gene therapies, several of which are being developed based on the CRISPR technology. Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. However, successful gene manipulation efforts involve complex experimental protocols and advanced molecular biology centered infrastructure. Therefore, many biopharmaceutical researchers and developers have demonstrated a preference to outsource such operations to capable contract service providers. Consequently, the genome editing contract services market was established and has grown to become an indispensable segment of the modern healthcare industry, offering a range of services, such as gRNA design and construction, cell line development (involving gene knockout, gene knockin, tagging and others) and transgenic animal model generation (such as knockout mice). Additionally, there are several players focused on developing advanced technology platforms that are intended to improve / augment existing gene editing tools, especially the CRISPR-based genome editing processes. Given the rising interest in personalized medicine, a number of strategic investors are presently willing to back genetic engineering focused initiatives. Prevalent trends indicate that the market for CRISPR-based genome editing services is likely to grow at a significant pace in the foreseen future. The “Genome Editing Services Market: Focus on CRISPR, 2019-2030” report features an extensive study of the current landscape of CRISPR-based genome editing service providers. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographical regions. Amongst other elements, the report includes: A detailed assessment of the current market landscape, featuring an elaborate list of over 80 companies that offer CRISPR-based genome editing services, and analyses based on a number of relevant parameters, such as type of gRNA service, availability of gRNA format, type of endonuclease, type of Cas9 endonuclease format, type of cell line engineering offering, type of cell line, type of animal model generation offering, availability of CRISPR libraries and important service provider details (year of establishment, company size and location of headquarters). An insightful 2X2 representation, highlighting the competitiveness of various CRISPR-based genome editing service providers captured in our database (segregated across various peer groups based on company size), taking into consideration the supplier power and the specific genome editing capabilities (which include gRNA service(s), endonuclease service(s), cell line engineering service(s), animal model generation service(s) and availability of CRISPR library(s)) of different companies. Elaborate profiles of key players (shortlisted based on strength of service portfolio), featuring a brief overview of the company, its financial performance (if available), a detailed description of its genome editing service offerings, recent developments and an informed future outlook. An in-depth analysis of over 10,000 patents related to CRISPR technology that have been filed / granted, since 2006, highlighting key trends associated with these patents, across type of patent, publication year and application year, regional applicability, CPC symbols, emerging focus areas, leading patent assignees (in terms of number of patents filed / granted), patent benhcmarking and valuation. A detailed analysis of close to 2,000 grants that have been awarded to support research projects related to CRISPR, between 2015 and 2019 (till September), highlighting important parameters, such as year of award, amount awarded, administring institute center, support period, funding mechanism, type of grant application, grant activity, type of recipient organization, regional distribution of recipient organization, prominent project leaders and emerging focus areas. It also features a detailed multivariate grant attractiveness analysis based on the amount awarded, support period, grant type and funding mechanism. A discussion on the advanced technologies and systems that have been developed to improve CRISPR-related processes. It includes a list of companies that have developed such innovative technology platforms, along with details on a number of relevant parameters, such as year of establishment, company size, core expertise, location of headquarters and important technology specifications (including technology name, focus area and key features). In addition, it includes short profiles of key technology providers. Further, the chapter highlights a list of companies that offer CRISPR kits and CRISPR design tools. An analysis highlighting potential strategic partners, segregated based on likelihood of entering into collaboration with CRISPR-based genome editing services providers. The analysis takes into consideration multiple relevant parameters, such as type of therapy, pipeline strength, pipeline maturity, company strength and therapeutic area. A discussion on important, industry-specific trends, key market drivers and challenges, under a SWOT framework, featuring a qualitative Harvey ball analysis that highlights the relative impact of each SWOT parameter on the overall market. One of the key objectives of the report was to evaluate the current opportunity and the future potential of CRISPR-based genome editing services market. We have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2019-2030. In addition, we have segmented the future opportunity across [A] type of services offered (gRNA construction, cell line engineering and animal model generation), [B] type of cell line used (mammalian, microbial, insect and others) and [C] different geographical regions (North America, Europe, Asia Pacific and rest of the world). To account for the uncertainties associated with the CRISPR-based genome editing services market and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the market’s evolution. The research, analysis and insights presented in this report are backed by a deep understanding of key insights generated from both secondary and primary research. 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. ...read more