Product Code: RA100359
INTRODUCTION
Over the last two decades, there have been several breakthroughs related to the development of gene therapies. In 2020, Libmeldy™, an ex vivo gene therapy received approval for the treatment of metachromatic leukodystrophy. To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors). Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain. In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.
Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods. For instance, the CRISPR-Cas9 based gene-editing tool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics. Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation. Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.
SCOPE OF THE REPORT
The Gene Therapy Market (5th Edition) by Type of Therapy (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Delivery Method Used (Ex vivo and In vivo), Type of Vector Used (Adeno-associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Non-Viral Vectors, Retrovirus and Others), Target Therapeutic Areas (Cardiovascular Diseases, Dermatological Diseases, Genetic Diseases, Hematological Diseases, Infectious Diseases, Metabolic Diseases, Muscle-related Diseases, Oncological Diseases, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebral, Intracoronary, Intradermal, Intralesional, Intramuscular, Intrapleural, Intrathecal, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal, Topical and Others), and Key Geographical Regions (US, Europe, Asia-Pacific and rest of the world): Industry Trends and Global Forecasts, 2022-2035 report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. Amongst other elements, the report features:
- A detailed overview of the overall market landscape of gene therapies, including information on their phase of development (marketed, clinical, preclinical and discovery), key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene / molecule targeted, type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), type of gene delivery method used (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).
- A detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain.
- An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.
- A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.
- An elaborate discussion on the various commercialization strategies that have been adopted by drug developers engaged in this domain across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing stage.
- Detailed profiles of marketed and late stage (phase II / III and above) gene therapies, along with information on the development timeline of the therapy, current development status, mechanism of action, affiliated technology, patent portfolio strength, dosage and manufacturing details, as well as details related to the developer company.
- A review of the various emerging technologies and therapy development platforms that are being used to manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates.
- An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation.
- A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company' product, target therapeutic area and deal multiples.
- An analysis of the investments made at various stages, such as seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings, by companies that are engaged in this domain.
- An analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.
- An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.
- An analysis of the startup companies engaged in this domain (established between 2017-2022) based on year of experience.
- A detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength.
- An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.
- A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.
A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.
One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over 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 2022-2035. Our year-wise projections of the current and future opportunity have further been segmented on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic areas (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific 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:
- Buel Dan Rodgers (Founder and CEO, AAVogen)
- Sue Washer (President and CEO, AGTC)
- Patricia Zilliox (President and CEO, Eyevensys)
- Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))
- Adam Rogers (CEO, Hemera Biosciences)
- Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))
- Al Hawkins (CEO, Milo Biotechnology)
- Jean-Phillipe Combal (CEO, Vivet Therapeutics)
- Robert Jan Lamers (former CEO, Arthrogen)
- Tom Wilton (former CBO, LogicBio Therapeutics)
- Michael Triplett (former CEO, Myonexus Therapeutics)
- Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)
- Cedric Szpirer (Executive and Scientific Director, Delphi Genetics)
- Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory)
- Jeffrey Hung (CCO, Vigene Biosciences)
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.
RESEARCH METHODOLOGY
The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.
Th secondary sources of information include:
- Annual reports
- Investor presentations
- SEC filings
- Industry databases
- News releases from company websites
- Government policy documents
- Industry analysts' views
While the focus has been on forecasting the market till 2035, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.
KEY QUESTIONS ANSWERED
- Who are the key industry players engaged in the development of gene therapies?
- How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products?
- Which types of vectors are most commonly used for effective delivery of gene therapies?
- What are the key regulatory requirements for gene therapy approval, across various geographies?
- Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?
- What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?
- What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?
- Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development?
- What are the key value drivers of the merger and acquisition activity in the gene therapy industry?
- Who are the key stakeholders that have actively made investments in the gene therapy domain?
- Which are the most active trial sites (in terms of number of clinical studies being conducted) in this domain?
- How is the current and future market opportunity likely to be distributed across key market segments?
CHAPTER OUTLINES
- Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.
- Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and ex vivo and in vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the various advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.
- Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.
- Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.
- Chapter 6 includes information on over 1150 gene therapies that are currently approved or are in different stages of development. It features a detailed analysis of the therapies, based on several relevant parameters, such as key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene / molecule targeted, type of gene delivery method used (ex vivo and in vivo), type of therapy (gene augmentation, oncolytic viral therapy, immunotherapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and type of therapy.
- Chapter 7 provides a detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe, Asia-Pacific, and Middle East and North Africa region on the basis of company size.
- Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the company's patent portfolio and collaborations focused on its gene therapy product / technology.
- Chapter 9 features an elaborate discussion on the various strategies that have been adopted by therapy developers engaged in this domain across key commercialization stages, including prior to drug launch, during drug launch and post-launch stage. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2022.
- Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II / III and above). Each drug profile provides information on the development timeline of the therapy, current developmental status, route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, affiliated technology, patent portfolio strength, clinical trials and collaborations (if any).
- Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline candidates that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.
- Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic diseases, metabolic diseases, neurological diseases, oncological diseases and ophthalmic diseases.
- Chapter 13 provides an overview of the various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed / granted). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation.
- Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company' product, target therapeutic area and deal multiples.
- Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and secondary offerings) received by the companies between 2015 and 2022, highlighting the growing interest of the venture capital community and other strategic investors in this market.
- Chapter 16 presents an analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.
- Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.
- Chapter 18 presents a detailed analysis of the start-up companies engaged in the field of gene therapy, established between 2017-2022, based on year of experience.
- Chapter 19 provides a detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength. In addition, it also a detailed analysis of the big pharma players based on several parameters, such as therapeutic area, type of vector used, type of therapy and type of gene delivery method used.
- Chapter 20 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.
- Chapter 21 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2035. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II / III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic area (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world).
- Chapter 22 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.
- Chapter 23 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.
- Chapter 24 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.
- Chapter 25 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 Buel Dan Rodgers (Founder and CEO, AAVogen), Sue Washer (President and CEO, AGTC), Patricia Zilliox (President and CEO, Eyevensys), Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics)), Adam Rogers (CEO, Hemera Biosciences), Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela)), Al Hawkins (CEO, Milo Biotechnology), Jean-Phillipe Combal (CEO, Vivet Therapeutics), Robert Jan Lamers (former CEO, Arthrogen), Tom Wilton (former CBO, LogicBio Therapeutics), Michael Triplett (former CEO, Myonexus Therapeutics), Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics), Cedric Szpirer (Executive and Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory), and Jeffrey Hung (CCO, Vigene Biosciences). In addition, a brief profile of each company has been provided.
- Chapter 26 is an appendix, which provides tabulated data and numbers for all the figures included in the report.
- Chapter 27 is an appendix, which contains a list of companies and organizations mentioned in this report.
TABLE OF CONTENTS
1. PREFACE
- 1.1. Scope of the Report
- 1.2. Research Methodology
- 1.3. Key Questions Answered
- 1.4. Chapter Outlines
2. EXECUTIVE SUMMARY
3. INTRODUCTION
- 3.1. Context and Background
- 3.2. Evolution of Gene Therapies
- 3.3. Classification of Gene Therapies
- 3.3.1. Somatic and Germline Gene Therapies
- 3.3.2. Ex Vivo and In Vivo Gene Therapies
- 3.4. Routes of Administration
- 3.5. Mechanism of Action
- 3.6. Overview of Gene Editing
- 3.6.1. Evolution of Genome Editing
- 3.6.2. Applications of Genome Editing
- 3.6.3. Available Genome Editing Techniques
- 3.7. Advantages and Disadvantages of Gene Therapies
- 3.7.1 Ethical and Social Concerns Related to Gene Therapies
- 3.7.2. Constraints and Challenges Related to Gene Therapies
- 3.7.3. Therapy Development Concerns
- 3.7.4. Manufacturing Concerns
- 3.7.5. Commercial Viability Concerns
4. GENE DELIVERY VECTORS
- 4.1. Chapter Overview
- 4.2. Viral and Non-Viral Methods of Gene Transfer
- 4.3. Viral Vectors for Genetically Modified Therapies
- 4.4. Types of Viral Vectors
- 4.4.1. Adeno-associated Viral Vectors
- 4.4.1.1. Overview
- 4.4.1.2. Design and Manufacturing
- 4.4.1.3. Advantages Offered
- 4.4.1.4. Associated Limitations
- 4.4.2. Adenoviral Vectors
- 4.4.2.1. Overview
- 4.4.2.2. Design and Manufacturing
- 4.4.2.3. Advantages Offered
- 4.4.2.4. Associated Limitations
- 4.4.3. Lentiviral Vectors
- 4.4.3.1. Overview
- 4.4.3.2. Design and Manufacturing
- 4.4.3.3. Advantages Offered
- 4.4.3.4. Associated Limitations
- 4.4.4. Retroviral Vectors
- 4.4.4.1. Overview
- 4.4.4.2. Design and Manufacturing
- 4.4.4.3. Advantages Offered
- 4.4.4.4. Associated Limitations
- 4.4.5. Other Viral Vectors
- 4.4.5.1. Alphavirus
- 4.4.5.2. Foamy Virus
- 4.4.5.3. Herpes Simplex Virus
- 4.4.5.4. Sendai Virus
- 4.4.5.5. Simian Virus
- 4.4.5.6. Vaccinia Virus
- 4.5. Types of Non-Viral Vectors
- 4.5.1. Plasmid DNA
- 4.5.2. Liposomes, Lipoplexes and Polyplexes
- 4.5.3. Oligonucleotides
- 4.5.4. Nanoparticles
- 4.5.5. Hybrid Vector Systems
- 4.5.6. Other Non-Viral Vectors
- 4.5.7. Gene Delivery Using Non-Viral Vectors
- 4.5.7.1. Biolistic Methods
- 4.5.7.2. Electroporation
- 4.5.7.3. Receptor Mediated Gene Delivery
- 4.5.7.4. 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. Canadian Scenario
- 5.3. Regulatory Guidelines in Europe
- 5.3.1. Quality of Gene Therapy Products
- 5.3.2. Non-Clinical Development
- 5.3.3. Clinical Development
- 5.4. Regulatory Guidelines in Asia-Pacific
- 5.4.1. Chinese Scenario
- 5.4.1.1. Construction of DNA Expression Cassette and Gene Delivery Systems
- 5.4.1.2. Generation and Characterization of Cell Banks and Engineered Bacteria Banks
- 5.4.1.3. Manufacturing of Gene Therapy Products
- 5.4.1.4. Quality Control
- 5.4.1.5. Evaluation of Efficacy of Gene Therapy Products
- 5.4.1.6. Safety Evaluation of Gene Therapy Products
- 5.4.1.7. Clinical Trial of Gene Therapy Products
- 5.4.1.8. Ethics Study
- 5.4.2. Japanese Scenario
- 5.4.3. South Korean Scenario
- 5.4.4. Australian Scenario
- 5.4.5. Hong Kong Scenario
- 5.5. Reimbursement Scenario
- 5.5.1. Challenges Related to Reimbursement
- 5.6. Commonly Offered Payment Models for Gene Therapies
6. MARKET OVERVIEW
- 6.1. Chapter Overview
- 6.1.1. Analysis of Gene Therapy Candidates by Stage of Development
- 6.2. Gene Therapy Market: Clinical and Commercial Pipeline
- 6.2.1. Analysis by Phase of Development
- 6.2.2. Analysis by Therapeutic Area
- 6.2.3. Analysis by Type of Vector Used
- 6.2.4. Analysis by Type of Gene / Molecule Targeted
- 6.2.5. Analysis by Type of Therapy
- 6.2.6. Analysis by Type of Gene Delivery Method Used
- 6.2.7. Analysis by Route of Administration
- 6.3. Gene Therapy Market: Early-Stage Pipeline
- 6.3.1. Analysis by Phase of Development
- 6.3.2. Analysis by Therapeutic Area
- 6.3.3. Analysis by Type of Vector Used
- 6.3.4. Analysis by Type of Therapy
- 6.3.5. Analysis by Type of Gene Delivery Method Used
- 6.3.6. Analysis by Route of Administration
- 6.4. Gene Therapy Market: Special Drug Designations
- 6.4.1. Analysis by Special Designation(s) Awarded
- 6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)
7. COMPETITIVE LANDSCAPE
- 7.1. Chapter Overview
- 7.2. Gene Therapy Market: List of Developers
- 7.2.1. Analysis by Year of Establishment
- 7.2.2. Analysis by Company Size
- 7.2.3. Analysis by Location of Headquarters
- 7.2.3.1. Analysis by Year of Establishment, Company Size and Location of Headquarters
- 7.2.3.2. Regional Landscape
- 7.3. Key Players: Analysis by Number of Pipeline Candidates
8. MARKETED GENE THERAPIES
- 8.1. Chapter Overview
- 8.2. Gendicine® (Shenzhen Sibiono GeneTech)
- 8.2.1. Company Overview
- 8.2.2. Development Timeline
- 8.2.3. Mechanism of Action and Vector Used
- 8.2.4. Target Indication(s)
- 8.2.5. Current Status of Development
- 8.2.6. Manufacturing, Dosage and Sales
- 8.3. Oncorine® (Shanghai Sunway Biotech)
- 8.3.1. Company Overview
- 8.3.2. Development Timeline
- 8.3.3. Mechanism of Action and Vector Used
- 8.3.4. Target Indication(s)
- 8.3.5. Current Status of Development
- 8.3.6. Manufacturing, Dosage and Sales
- 8.4. Rexin-G® (Epeius Biotechnologies)
- 8.4.1. Company Overview
- 8.4.2. Development Timeline
- 8.4.3. Mechanism of Action and Vector Used
- 8.4.4. Target Indication(s)
- 8.4.5. Current Status of Development
- 8.4.6. Manufacturing, Dosage and Sales
- 8.5. Neovasculgen® (Human Stem Cells Institute)
- 8.5.1. Company Overview
- 8.5.2. Development Timeline
- 8.5.3. Mechanism of Action and Vector Used
- 8.5.4. Target Indication(s)
- 8.5.5. Current Status of Development
- 8.5.6. Manufacturing, Dosage and Sales
- 8.6. Imlygic® (Amgen)
- 8.6.1. Company Overview
- 8.6.2. Development Timeline
- 8.6.3. Mechanism of Action and Vector Used
- 8.6.4. Target Indication(s)
- 8.6.5. Current Status of Development
- 8.6.6. Manufacturing, Dosage and Sales
- 8.7. Strimvelis® (Orchard Therapeutics)
- 8.7.1. Company Overview
- 8.7.2. Development Timeline
- 8.7.3. Mechanism of Action and Vector Used
- 8.7.4. Target Indication(s)
- 8.7.5. Current Status of Development
- 8.7.6. Manufacturing, Dosage and Sales
- 8.8. Luxturna™ (Spark Therapeutics)
- 8.8.1. Company Overview
- 8.8.2. Development Timeline
- 8.8.3. Mechanism of Action and Vector Used
- 8.8.4. Target Indication(s)
- 8.8.5. Current Status of Development
- 8.8.6. Manufacturing, Dosage and Sales
- 8.9. Zolgensma™ (Novartis)
- 8.9.1. Company Overview
- 8.9.2. Development Timeline
- 8.9.3. Mechanism of Action and Vector Used
- 8.9.4. Target Indication(s)
- 8.9.5. Current Status of Development
- 8.9.6. Manufacturing, Dosage and Sales
- 8.10. Collategene® (AnGes)
- 8.10.1. Company Overview
- 8.10.2. Development Timeline
- 8.10.3. Mechanism of Action and Vector Used
- 8.10.4. Target Indication(s)
- 8.10.5. Current Status of Development
- 8.10.6. Manufacturing, Dosage and Sales
- 8.11. Zyntelgo™ (bluebird bio)
- 8.11.1. Company Overview
- 8.11.2. Development Timeline
- 8.11.3. Mechanism of Action and Vector Used
- 8.11.4. Target Indication(s)
- 8.11.5. Current Status of Development
- 8.11.6. Manufacturing, Dosage and Sales
- 8.12. Libmeldy™ (Orchard Therapeutics)
- 8.12.1. Company Overview
- 8.12.2. Development Timeline
- 8.12.3. Mechanism of Action and Vector Used
- 8.12.4. Target Indication(s)
- 8.12.5. Current Status of Development
- 8.12.6. Manufacturing, Dosage and Sales
9. KEY COMMERCIALIZATION STRATEGIES
- 9.1. Chapter Overview
- 9.2. Successful Drug Launch Strategy: ROOTS Framework
- 9.3. Successful Drug Launch Strategy: Product Differentiation
- 9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product
- 9.5. List of Currently Approved Gene Therapies
- 9.6. Key Commercialization Strategies Adopted by Gene Therapy Developers
- 9.6.1. Strategies Adopted Before Therapy Approval
- 9.6.1.1. Participation in Global Events
- 9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms
- 9.6.1.3. Indication Expansion
- 9.6.2. Strategies Adopted During / Post Therapy Approval
- 9.6.2.1. Geographical Expansion
- 9.6.2.2. Participation in Global Events
- 9.6.2.3. Patience Assistance Programs
- 9.6.2.4. Awareness through Product Websites
- 9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms
- 9.7. Concluding Remarks
10. LATE STAGE (PHASE II / III AND ABOVE) GENE THERAPIES
- 10.1. Chapter Overview
- 10.2. Lumevoq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.3. OTL-103: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.4. PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.5. BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.6. rAd-IFN/Syn3: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.7. beti-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.8. eli-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.9. lovo-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.10. SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.11. EB-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.12. ProstAtak®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.13. D-Fi: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.14. CG0070: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.15. Vigil™-EWS: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.16. Engensis®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.17. VGX-3100: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.18. Invossa™ (TG-C): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.19. VYJUVEKT™: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.20. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.21. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.22. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.23. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.24. AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.25. VB-111: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.26. Generx®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.27. ADXS-HPV: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.28. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.29. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.30. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.31. AG0302-COVID19: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.32. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
- 10.33. Hologene 5: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
11. EMERGING TECHNOLOGIES
- 11.1. Chapter Overview
- 11.2. Gene Editing Technologies
- 11.2.1. Overview
- 11.2.2. Applications
- 11.3. Emerging Gene Editing Platforms
- 11.3.1. CRISPR / Cas9 System
- 11.3.1.1. Key Components and Functions
- 11.3.1.2. Mechanism of Action
- 11.3.1.3. Targeting Efficiency and Challenges
- 11.3.1.4. Next-GEN CRISPR Technology
- 11.3.1.5. Technology Providers
- 11.3.2. TALENs
- 11.3.2.1. Structural Features
- 11.3.2.2. Mechanism of Action
- 11.3.2.3. Advantages and Challenges
- 11.3.3. megaTAL
- 11.3.3.1. Technology Providers
- 11.3.4. Zinc Finger Nuclease
- 11.3.4.1. Technology Providers
- 11.4. Gene Expression Regulation Technologies
- 11.4.1. Technology Providers
- 11.5. Technology Platforms for Developing / Delivering Gene Therapies
12. KEY THERAPEUTICS AREAS
- 12.1. Chapter Overview
- 12.2. Analysis by Therapeutic Area and Special Designation(s) Awarded
- 12.3. Oncological Diseases
- 12.3.1. Analysis by Target Indication
- 12.3.2. Analysis by Type of Vector Used
- 12.4. Neurological Diseases
- 12.4.1. Analysis by Target Indication
- 12.4.2. Analysis by Type of Vector Used
- 12.5. Ophthalmic Diseases
- 12.5.1. Analysis by Target Indication
- 12.5.2. Analysis by Type of Vector Used
- 12.6. Metabolic Diseases
- 12.6.1. Analysis by Target Indication
- 12.6.2. Analysis by Type of Vector Used
- 12.7. Genetic Diseases
- 12.7.1. Analysis by Target Indication
- 12.7.2. Analysis by Type of Vector Used
13. PATENT ANALYSIS
- 13.1. Chapter Overview
- 13.2. Gene Therapy Market: Patent Analysis
- 13.2.1. Scope and Methodology
- 13.2.1.1. Analysis by Publication Year
- 13.2.1.2. Analysis by Publication Year and Type of Patent
- 13.2.1.3. Analysis by Geography
- 13.2.1.4. Analysis by CPC Symbols
- 13.2.1.5. Analysis by Emerging Focus Areas
- 13.2.1.6. Leading Players: Analysis by Number of Patents
- 13.2.1.7. Patent Benchmark Analysis
- 13.2.1.8. Patent Valuation Analysis
- 13.3. Gene Editing Market: Patent Analysis
- 13.3.1. Scope and Methodology
- 13.3.1.1. Analysis by Publication Year
- 13.3.1.2. Analysis by Publication Year and Type of Patent
- 13.3.1.3. Analysis by Geography
- 13.3.1.4. Analysis by CPC Symbols
- 13.3.1.5. Analysis by Emerging Focus Areas
- 13.3.1.6. Leading Players: Analysis by Number of Patents
- 13.3.1.7. Patent Benchmark Analysis
- 13.3.1.8. Patent Valuation Analysis
- 13.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization
14. MERGERS AND ACQUISITIONS
- 14.1. Chapter Overview
- 14.2. Merger and Acquisition Models
- 14.3. Gene Therapy Market: Mergers and Acquisitions
- 14.3.1. Analysis by Year of Merger / Acquisition
- 14.3.2. Analysis by Type of Agreement
- 14.3.3. Analysis by Geography
- 14.3.3.1. Continent-wise Distribution
- 14.3.3.2. Intercontinental and Intracontinental Deals
- 14.3.3.3. Local and International Deals
- 14.3.4. Analysis by Key Value Drivers
- 14.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
- 14.3.5. Analysis by Phase of Development of the Acquired Company's Product
- 14.3.6. Analysis by Therapeutic Area
- 14.3.7. Analysis by Deal Multiples
15. FUNDING AND INVESTMENT ANALYSIS
- 15.1. Chapter Overview
- 15.2. Types of Funding
- 15.3. Gene Therapy Market: Funding and Investment Analysis
- 15.3.1. Analysis of Funding Instances by Year
- 15.3.2. Analysis of Amount Invested by Year
- 15.3.3. Analysis of Funding Instances and Amount Invested by Type of Funding
- 15.3.4. Analysis of Funding Instances and Amount Invested by Year and Type of Funding
- 15.3.5. Analysis of Funding Instances and Amount Invested by Type of Therapy
- 15.3.6. Analysis of Funding Instances and Amount Invested by Geography
- 15.3.7. Analysis of Funding Instances and Amount Invested by Highest Phase of Development
- 15.3.8. Analysis of Funding Instances by Therapeutic Area
- 15.3.9. Most Active Players: Analysis by Number of Funding Instances and Amount Invested
- 15.3.10. Key Investors: Analysis by Number of Funding Instances
- 15.4. Concluding Remarks
16. CLINICAL TRIAL ANALYSIS
- 16.1. Chapter Overview
- 16.2. Scope and Methodology
- 16.3. Gene Therapy Market: Clinical Trial Analysis
- 16.3.1. Analysis by Trial Registration Year
- 16.3.2. Analysis by Trial Status
- 16.3.3. Analysis by Trial Phase
- 16.3.4. Analysis by Therapeutic Area
- 16.3.5. Analysis by Geography
- 16.3.6. Analysis by Trial Registration Year and Geography
- 16.3.7. Analysis by Trial Status and Geography
- 16.3.8. Analysis by Trial Status, Trial Phase and Geography
- 16.3.9. Analysis by Therapeutic Area and Geography
- 16.4. Analysis by Type of Sponsor
- 16.5. Analysis by Prominent Treatment Sites
- 16.6. Gene Therapy Market: Analysis of Enrolled Patient Population
- 16.6.1. Analysis by Trial Registration Year
- 16.6.2. Analysis by Trial Status
- 16.6.3. Analysis by Trial Phase
- 16.6.4. Analysis by Therapeutic Area
- 16.6.5. Analysis by Location of Trial Site
- 16.6.6. Analysis by Trial Status and Location of Trial Site
- 16.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
- 16.6.8. Analysis by Therapeutic Area and Location of Trial Site
- 16.7. Concluding Remarks
- 16.7.1. Emerging Molecules to Watch
- 16.7.2. Most Important Trials to Watch
17. COST PRICE ANALYSIS
- 17.1. Chapter Overview
- 17.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
- 17.3. Gene Therapy Market: Pricing Models
- 17.3.1. Based on Associated Product / Component Costs
- 17.3.2. Based on Competition
- 17.3.3. Based on Patient Segment
- 17.3.4. Based on Opinions of Industry Experts
18. START-UP VALUATION
- 18.1. Chapter Overview
- 18.2. Valuation by Year of Experience
- 18.2.1. Methodology
- 18.2.2. Results and Interpretation
19. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES
- 19.1. Chapter Overview
- 19.2. Gene Therapy Market: List of Most Prominent Big Pharmaceutical Players
- 19.2.1. Analysis by Therapeutic Area
- 19.2.2. Analysis by Type of Vector Used
- 19.2.3. Analysis by Type of Therapy
- 19.2.4. Analysis by Type of Gene Delivery Method
- 19.3. Benchmark Analysis of Key Parameters
- 19.3.1. Spider Web Analysis: Pipeline Strength
- 19.3.2. Spider Web Analysis: Merger / Acquisitions
- 19.3.3. Spider Web Analysis: Funding and Investments
- 19.3.4. Spider Web Analysis: Clinical Trials
- 19.3.5. Spider Web Analysis: Technologies
- 19.3.6. Spider Web Analysis: Patents
- 19.4. Benchmark Analysis of Big Pharmaceutical Players
20. DEMAND ANALYSIS
- 20.1. Chapter Overview
- 20.2. Methodology
- 20.3. Global Demand for Gene Therapies, 2022-2035
- 20.3.1. Analysis by Type of Therapy
- 20.3.2. Analysis by Therapeutic Area
- 20.3.3. Analysis by Geography
21. MARKET FORECAST AND OPPORTUNITY ANALYSIS
- 21.1. Chapter Overview
- 21.2. Scope and Limitations
- 21.3. Key Assumptions and Forecast Methodology
- 21.4. Global Gene Therapy Market, 2022-2035
- 21.4.1. Gene Therapy Market: Analysis by Therapeutic Area
- 21.4.2. Gene Therapy Market: Analysis by Type of Vector Used
- 21.4.3. Gene Therapy Market: Analysis by Type of Therapy
- 21.4.4. Gene Therapy Market: Analysis by Gene Delivery Method Used
- 21.4.5. Gene Therapy Market: Analysis by Route of Administration
- 21.4.6. Gene Therapy Market: Analysis by Geography
- 21.5. Gene Therapy Market: Value Creation Analysis
- 21.6. Gene Therapy Market: Product-wise Sales Forecasts
- 21.6.1. Gendicine®
- 21.6.1.1. Target Patient Population
- 21.6.1.2. Sales Forecast
- 21.6.1.3. Net Present Value
- 21.6.1.4. Value Creation Analysis
- 21.6.2. Oncorine®
- 21.6.2.1. Target Patient Population
- 21.6.2.2. Sales Forecast
- 21.6.2.3. Net Present Value
- 21.6.2.4. Value Creation Analysis
- 21.6.3. Rexin-G®
- 21.6.3.1. Target Patient Population
- 21.6.3.2. Sales Forecast
- 21.6.3.3. Net Present Value
- 21.6.3.4. Value Creation Analysis
- 21.6.4. Neovasculgen®
- 21.6.4.1. Target Patient Population
- 21.6.4.2. Sales Forecast
- 21.6.4.3. Net Present Value
- 21.6.4.4. Value Creation Analysis
- 21.6.5. Strimvelis®
- 21.6.5.1. Target Patient Population
- 21.6.5.2. Sales Forecast
- 21.6.5.3. Net Present Value
- 21.6.5.4. Value Creation Analysis
- 21.6.6. Imlygic®
- 21.6.6.1. Target Patient Population
- 21.6.6.2. Sales Forecast
- 21.6.6.3. Net Present Value
- 21.6.6.4. Value Creation Analysis
- 21.6.7. Luxturna™
- 21.6.7.1. Target Patient Population
- 21.6.7.2. Sales Forecast
- 21.6.7.3. Net Present Value
- 21.6.7.4. Value Creation Analysis
- 21.6.8. Zolgensma™
- 21.6.8.1. Target Patient Population
- 21.6.8.2. Sales Forecast
- 21.6.8.3. Net Present Value
- 21.6.8.4. Value Creation Analysis
- 21.6.9. Collategene®
- 21.6.9.1. Target Patient Population
- 21.6.9.2. Sales Forecast
- 21.6.9.3. Net Present Value
- 21.6.9.4. Value Creation Analysis
- 21.6.10. Libmeldy™
- 21.6.10.1. Target Patient Population
- 21.6.10.2. Sales Forecast
- 21.6.10.3. Net Present Value
- 21.6.10.4. Value Creation Analysis
- 21.6.11. Lumevoq® (GS010)
- 21.6.11.1. Target Patient Population
- 21.6.11.2. Sales Forecast
- 21.6.11.3. Net Present Value
- 21.6.11.4. Value Creation Analysis
- 21.6.12. OTL-103
- 21.6.12.1. Target Patient Population
- 21.6.12.2. Sales Forecast
- 21.6.12.3. Net Present Value
- 21.6.12.4. Value Creation Analysis
- 21.6.13. PTC-AADC
- 21.6.13.1. Target Patient Population
- 21.6.13.2. Sales Forecast
- 21.6.13.3. Net Present Value
- 21.6.13.4. Value Creation Analysis
- 21.6.14. BMN 270
- 21.6.14.1. Target Patient Population
- 21.6.14.2. Sales Forecast
- 21.6.14.3. Net Present Value
- 21.6.14.4. Value Creation Analysis
- 21.6.15. rAd-IFN/Syn3
- 21.6.15.1. Target Patient Population
- 21.6.15.2. Sales Forecast
- 21.6.15.3. Net Present Value
- 21.6.15.4. Value Creation Analysis
- 21.6.16. beti-cel
- 21.6.16.1. Target Patient Population
- 21.6.16.2. Sales Forecast
- 21.6.16.3. Net Present Value
- 21.6.16.4. Value Creation Analysis
- 21.6.17. eli-cel
- 21.6.17.1. Target Patient Population
- 21.6.17.2. Sales Forecast
- 21.6.17.3. Net Present Value
- 21.6.17.4. Value Creation Analysis
- 21.6.18. lovo-cel
- 21.6.18.1. Target Patient Population
- 21.6.18.2. Sales Forecast
- 21.6.18.3. Net Present Value
- 21.6.18.4. Value Creation Analysis
- 21.6.19. SRP-9001
- 21.6.19.1. Target Patient Population
- 21.6.19.2. Sales Forecast
- 21.6.19.3. Net Present Value
- 21.6.19.4. Value Creation Analysis
- 21.6.20. EB-101
- 21.6.20.1. Target Patient Population
- 21.6.20.2. Sales Forecast
- 21.6.20.3. Net Present Value
- 21.6.20.4. Value Creation Analysis
- 21.6.21. ProstAtak®
- 21.6.21.1. Target Patient Population
- 21.6.21.2. Sales Forecast
- 21.6.21.3. Net Present Value
- 21.6.21.4. Value Creation Analysis
- 21.6.22. D-Fi
- 21.6.22.1. Target Patient Population
- 21.6.22.2. Sales Forecast
- 21.6.22.3. Net Present Value
- 21.6.22.4. Value Creation Analysis
- 21.6.23. CG0070
- 21.6.23.1. Target Patient Population
- 21.6.23.2. Sales Forecast
- 21.6.23.3. Net Present Value
- 21.6.23.4. Value Creation Analysis
- 21.6.24. Vigil™-EWS
- 21.6.24.1. Target Patient Population
- 21.6.24.2. Sales Forecast
- 21.6.24.3. Net Present Value
- 21.6.24.4. Value Creation Analysis
- 21.6.25. Engensis®
- 21.6.25.1. Target Patient Population
- 21.6.25.2. Sales Forecast
- 21.6.25.3. Net Present Value
- 21.6.25.4. Value Creation Analysis
- 21.6.26. VGX-3100
- 21.6.26.1. Target Patient Population
- 21.6.26.2. Sales Forecast
- 21.6. 26.3. Net Present Value
- 21.6.26.4. Value Creation Analysis
- 21.6.27. Invossa™ (TG-C)
- 21.6.27.1. Target Patient Population
- 21.6.27.2. Sales Forecast
- 21.6.27.3. Net Present Value
- 21.6.27.4. Value Creation Analysis
- 21.6.28. VYJUVEKT™
- 21.6.28.1. Target Patient Population
- 21.6.28.2. Sales Forecast
- 21.6.28.3. Net Present Value
- 21.6.28.4. Value Creation Analysis
- 21.6.29. PF-06939926
- 21.6.29.1. Target Patient Population
- 21.6.29.2. Sales Forecast
- 21.6.29.3. Net Present Value
- 21.6.29.4. Value Creation Analysis
- 21.6.30. PF-06838435
- 21.6.30.1. Target Patient Population
- 21.6.30.2. Sales Forecast
- 21.6.30.3. Net Present Value
- 21.6.30.4. Value Creation Analysis
- 21.6.31. PF-07055480
- 21.6.31.1. Target Patient Population
- 21.6.31.2. Sales Forecast
- 21.6.31.3. Net Present Value
- 21.6.31.4. Value Creation Analysis
- 21.6.32. SPK-8011
- 21.6.32.1. Target Patient Population
- 21.6.32.2. Sales Forecast
- 21.6.32.3. Net Present Value
- 21.6.32.4. Value Creation Analysis
- 21.6.33. AMT-061
- 21.6.33.1. Target Patient Population
- 21.6.33.2. Sales Forecast
- 21.6.33.3. Net Present Value
- 21.6.33.4. Value Creation Analysis
- 21.6.34. VB-111
- 21.6.34.1. Target Patient Population
- 21.6.34.2. Sales Forecast
- 21.6.34.3. Net Present Value
- 21.6.34.4. Value Creation Analysis
- 21.6.35. Generx®
- 21.6.35.1. Target Patient Population
- 21.6.35.2. Sales Forecast
- 21.6.35.3. Net Present Value
- 21.6.35.4. Value Creation Analysis
- 21.6.36. AMG001
- 21.6.36.1. Target Patient Population
- 21.6.36.2. Sales Forecast
- 21.6.36.3. Net Present Value
- 21.6.36.4. Value Creation Analysis
- 21.6.37. OAV-101
- 21.6.37.1. Target Patient Population
- 21.6.37.2. Sales Forecast
- 21.6.37.3. Net Present Value
- 21.6.37.4. Value Creation Analysis
- 21.6.38. ADXS-HPV
- 21.6.38.1. Target Patient Population
- 21.6.38.2. Sales Forecast
- 21.6.38.3. Net Present Value
- 21.6.38.4. Value Creation Analysis
- 21.6.39. AGTC 501
- 21.6.39.1. Target Patient Population
- 21.6.39.2. Sales Forecast
- 21.6.39.3. Net Present Value
- 21.6.39.4. Value Creation Analysis
- 21.6.40. LYS-SAF302
- 21.6.40.1. Target Patient Population
- 21.6.40.2. Sales Forecast
- 21.6.40.3. Net Present Value
- 21.6.40.4. Value Creation Analysis
- 21.6.41. NFS-01
- 21.6.41.1. Target Patient Population
- 21.6.41.2. Sales Forecast
- 21.6.41.3. Net Present Value
- 21.6.41.4. Value Creation Analysis
- 21.6.42. AG0302-COVID19
- 21.6.42.1. Target Patient Population
- 21.6.42.2. Sales Forecast
- 21.6.42.3. Net Present Value
- 21.6.42.4. Value Creation Analysis
- 21.6.43. RGX-314
- 21.6.43.1. Target Patient Population
- 21.6.43.2. Sales Forecast
- 21.6.43.3. Net Present Value
- 21.6.43.4. Value Creation Analysis
- 21.6.44. Hologene 5
- 21.6.44.1. Target Patient Population
- 21.6.44.2. Sales Forecast
- 21.6.44.3. Net Present Value
- 21.6.44.4. Value Creation Analysis
22. VECTOR MANUFACTURING
- 22.1. Chapter Overview
- 22.2. Overview of Viral Vector Manufacturing
- 22.3. Viral Vector Manufacturing Process
- 22.3.1. Mode of Vector Production
- 22.3.2. Adherent and Suspension Cultures
- 22.3.3. Unit Processes and Multiple Parallel Processes
- 22.3.4. Cell Culture Systems for Production of Viral Vectors
- 22.3.4.1. Small Scale / Laboratory Scale Cell Culture Systems
- 22.3.4.2. Large Scale Cell Culture Systems
- 22.3.5. Culture Media Specifications
- 22.4. Bioprocessing of Viral Vectors
- 22.4.1. AAV Vector Production
- 22.4.2. Adenoviral Vector Production
- 22.4.3. Lentiviral Vector Production
- 22.4.4. γ -Retroviral Vector Production
- 22.5. Challenges Associated with Vector Manufacturing
- 22.6. Contract Service Providers for Viral and Plasmid Vectors
23. CASE STUDY: GENE THERAPY SUPPLY CHAIN
- 23.1. Chapter Overview
- 23.2. Overview of Gene Therapy Supply Chain
- 23.3. Implementation of Supply Chain Models
- 23.4. Logistics in Gene Therapies
- 23.4.1. Logistic Processes for Autologous and Allogeneic Therapies
- 23.5. Regulatory Supply Chain Across the Globe
- 23.6. Challenges Associated with Gene Therapy Supply Chain
- 23.7. Optimizing Cell and Advanced Therapies Supply Chain Management
- 23.7.1. Enterprise Manufacturing System
- 23.7.2. Laboratory Information Management System
- 23.7.3. Inventory Management System
- 23.7.4. Quality Management System
- 23.7.5. Logistics Management System
- 23.7.6. Patient Management System
- 23.7.7. Electronic Clinical Outcome Assessments System
- 23.7.8. Supply Chain Orchestration Platform
- 23.8. Recent Developments and Upcoming Trends
24. CONCLUSION
25. INTERVIEW TRANSCRIPTS
- 25.1. Chapter Overview
- 25.2. Buel Dan Rodgers (Founder and CEO, AAVogen)
- 25.3. Sue Washer (President and CEO, AGTC)
- 25.4. Patricia Zilliox (President and CEO, Eyevensys)
- 25.5. Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))
- 25.6. Adam Rogers (CEO, Hemera Biosciences)
- 25.7. Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))
- 25.8. Al Hawkins (CEO, Milo Biotechnology)
- 25.9. Jean-Phillipe Combal (CEO, Vivet Therapeutics)
- 25.10. Robert Jan Lamers (former CEO, Arthrogen)
- 25.11. Tom Wilton (former CBO, LogicBio Therapeutics)
- 25.12. Michael Tripletti (former CEO, Myonexus Therapeutics)
- 25.13. Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)
- 25.14. Cedric Szpirer (former Executive and Scientific Director, Delphi Genetics)
- 25.15. Marco Schmeer (Project Manager) and Tatjana Buchholz, (former Marketing Manager, PlasmidFactory)
- 25.16. Jeffrey Hung (CCO, Vigene Biosciences)
26. APPENDIX 1: TABULATED DATA
27. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS