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PUBLISHER: Roots Analysis | PRODUCT CODE: 1101052

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PUBLISHER: Roots Analysis | PRODUCT CODE: 1101052

Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions : Industry Trends and Global Forecasts, 2022-2035

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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.
Product Code: RA100359

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

  • 2.1. Chapter Overview

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

  • 24.1. Chapter Overview

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

Product Code: RA100359

LIST OF TABLES

  • Table 3.1 Comparison of Ex Vivo and In Vivo Techniques
  • Table 3.2 Vectors Used for Targeted Gene Delivery to Tissues / Organs
  • Table 3.3 Routes of Administration of Gene Therapies: Advantages and Disadvantages
  • Table 4.1 Viral Vectors: Key Features
  • Table 5.1 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
  • Table 5.2 Payment Options Available for Gene Therapies
  • Table 6.1 Gene Therapy Market: Clinical and Commercial Pipeline
  • Table 6.2 Gene Therapy Market: Early-Stage Pipeline
  • Table 6.3 Gene Therapy Market: Special Designation(s) Awarded
  • Table 7.1 Gene Therapy Market: List of Developers
  • Table 8.1 Gene Therapies: Marketed and Approved Products
  • Table 8.2 Shenzhen Sibiono GeneTech: Company Overview
  • Table 8.3 Gendicine®: Patent Portfolio
  • Table 8.4 Gendicine®: Status of Development
  • Table 8.5 Shanghai Sunway Biotech: Company Overview
  • Table 8.6 Oncorine®: Status of Development
  • Table 8.7 Epeius Biotechnologies: Company Overview
  • Table 8.8 Rexin-G®: Status of Development
  • Table 8.9 Human Stem Cell Institute: Company Overview
  • Table 8.10 Neovasculgen®: Status of Development
  • Table 8.11 Amgen: Company Overview
  • Table 8.12 Imlygic®: Status of Development
  • Table 8.13 Imlygic®: Recommended Dose and Schedule
  • Table 8.14 Imlygic®: Determination of Injection Volume based upon Lesion Size
  • Table 8.15 Orchard Therapeutics: Company Overview
  • Table 8.16 Strimvelis®: Status of Development
  • Table 8.17 Spark Therapeutics: Company Overview
  • Table 8.18 Luxturna™: Status of Development
  • Table 8.19 Novartis Gene Therapies: Company Overview
  • Table 8.20 Zolgensma™: Status of Development
  • Table 8.21 AnGes: Company Overview
  • Table 8.22 Collategene®: Status of Development
  • Table 8.23 bluebird bio: Company Overview
  • Table 8.24 Zyntelgo™: Status of Development
  • Table 8.25 Libmeldy™: Status of Development
  • Table 9.1 Libmeldy™: Presentations in Conferences Before Product Approval
  • Table 9.2 Luxturna™: Presentations in Conferences Before Product Approval
  • Table 9.3 Zolgensma™: Presentations in Conferences Before Product Approval
  • Table 9.4 Zyntelgo™: Presentations in Conferences Before Product Approval
  • Table 9.5 Imlygic®: Presentations in Conferences After Product Approval
  • Table 9.6 Libmeldy™: Presentations in Conferences After Product Approval
  • Table 9.7 Luxturna™: Presentations in Conferences After Product Approval
  • Table 9.8 Strimvelis™: Presentations in Conferences After Product Approval
  • Table 9.9 Zolgensma™: Presentations in Conferences After Product Approval
  • Table 9.10 Zyntelgo™: Presentations in Conferences After Product Approval
  • Table 9.11 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
  • Table 10.1 Gene Therapies: Late Stage (Phase II / III and Above) Drug Candidates
  • Table 10.2 Lumevaq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.3 OTL-103: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.4 PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.5 BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.6 rAd-IFN/Syn3: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.7 beti-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.8 eli-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.9 lovo-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.10 SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.11 EB-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.12 ProstAtak®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.13 D-Fi: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.14 CG0070: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.15 Vigil™-EWS: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.16 Engensis®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.17 VGX-3100: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.18 Invossa™ (TG-C): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.19 VYJUVEKT™: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.20 PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.21 PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.22 PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.23 SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.24 AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.25 VB-111: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.26 Generx®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.27 ADXS-HPV: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.28 AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.29 LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.30 NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.31 AG0302-COVID19: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.32 RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 10.32 Hologene 5: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
  • Table 11.1 Gene Editing Technology Platforms
  • Table 11.2 ASC Therapeutics: Collaborations
  • Table 11.3 CRISPR Therapeutics: Funding Instances
  • Table 11.4 CRISPR Therapeutics: Collaborations
  • Table 11.5 Editas Medicine: Funding Instances
  • Table 11.6 Editas Medicine: Collaborations
  • Table 11.7 Emendo Biotherapeutics: Collaborations
  • Table 11.8 Excision Biotherapeutics: Funding Instances
  • Table 11.9 Intellia Therapeutics: Funding Instances
  • Table 11.10 Intellia Therapeutics: Collaborations
  • Table 11.11 Sarepta Therapeutics: Funding Instances
  • Table 11.12 Sarepta Therapeutics: Collaborations
  • Table 11.13 ToolGen: Collaborations
  • Table 11.14 bluebird bio: Funding Instances
  • Table 11.15 bluebird bio: Collaborations
  • Table 11.16 Sangamo Therapeutics: Funding Instances
  • Table 11.17 Sangamo Therapeutics: Collaborations
  • Table 11.18 Gene Switch Technology Platforms
  • Table 11.19 Precigen: Funding Instances
  • Table 11.20 Precigen: Collaborations
  • Table 11.21 MeiraGTx: Funding Instances
  • Table 11.22 MeiraGTx: Collaborations
  • Table 11.23 Gene Therapy: Technology Platforms
  • Table 12.1 Oncological Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
  • Table 12.2 Neurological Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
  • Table 12.3 Ophthalmic Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
  • Table 12.4 Metabolic Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
  • Table 12.5 Genetic Diseases: Information on Highest Phase of Development of Gene Therapy and Type of Vector Used
  • Table 13.1 Gene Therapy Patent Portfolio: List of Top CPC Symbols
  • Table 13.2 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
  • Table 13.3 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
  • Table 13.4 Gene Therapy Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
  • Table 13.5 Gene Editing Patent Portfolio: List of Top CPC Symbols
  • Table 13.6 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
  • Table 13.7 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
  • Table 13.8 Gene Editing Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
  • Table 14.1 Gene Therapy: List of Mergers and Acquisitions, 2015-2022
  • Table 14.2 Acquisitions: Key Value Drivers
  • Table 14.3 Mergers and Acquisitions: Information on Phase of Development and Therapeutic Area of the Acquired Company's Gene Therapy Product
  • Table 14.4 Mergers and Acquisitions: Information on Deal Multiple based on Acquisition Amount
  • Table 15.1 Gene Therapy Market: Funding and Investments, 2015-2022
  • Table 15.2 Funding and Investments: Summary of Investments
  • Table 15.3 Funding and Investments: Summary of Venture Capital Funding
  • Table 16.1 Clinical Trial Analysis: Emerging Molecules to Watch
  • Table 16.2 Clinical Trial Analysis: Most Important Trials to Watch
  • Table 17.1 Pricing Model: Price of Marketed Gene / Cell Therapies
  • Table 17.2 Pricing Model: Price of Marketed Targeted Drugs (Antibody Drug Conjugates and Monoclonal Antibodies)
  • Table 17.3 Pricing Model: Opinions of Experts / Other Analysts
  • Table 18.1 Start-up Valuation: Estimates based on Overall Experience
  • Table 21.1 Gene Therapies: Expected Launch Years of Advanced Stage Drug Candidates
  • Table 21.2 Gendicine®: Target Patient Population
  • Table 21.3 Gendicine®: Net Present Value (USD Million)
  • Table 21.4 Gendicine®: Value Creation Analysis (USD Million)
  • Table 21.5 Oncorine®: Target Patient Population
  • Table 21.6 Oncorine®: Net Present Value (USD Million)
  • Table 21.7 Oncorine®: Value Creation Analysis (USD Million)
  • Table 21.8 Rexin-G®: Target Patient Population
  • Table 21.9 Rexin-G®: Net Present Value (USD Million)
  • Table 21.10 Rexin-G®: Value Creation Analysis (USD Million)
  • Table 21.11 Neovasculgen®: Target Patient Population
  • Table 21.12 Neovasculgen®: Net Present Value (USD Million)
  • Table 21.13 Neovasculgen®: Value Creation Analysis (USD Million)
  • Table 21.14 Strimvelis®: Target Patient Population
  • Table 21.15 Strimvelis®: Net Present Value (USD Million)
  • Table 21.16 Strimvelis®: Value Creation Analysis (USD Million)
  • Table 21.17 Imlygic®: Target Patient Population
  • Table 21.18 Imlygic®: Net Present Value (USD Million)
  • Table 21.19 Imlygic®: Value Creation Analysis (USD Million)
  • Table 21.20 Luxturna™: Target Patient Population
  • Table 21.21 Luxturna™: Net Present Value (USD Million)
  • Table 21.22 Luxturna™: Value Creation Analysis (USD Million)
  • Table 21.23 Zolgensma™: Target Patient Population
  • Table 21.24 Zolgensma™: Net Present Value (USD Million)
  • Table 21.25 Zolgensma™: Value Creation Analysis (USD Million)
  • Table 21.26 Collategene®: Target Patient Population
  • Table 21.27 Collategene®: Net Present Value (USD Million)
  • Table 21.28 Collategene®: Value Creation Analysis (USD Million)
  • Table 21.29 Libmedly™: Target Patient Population
  • Table 21.30 Libmedly™: Net Present Value (USD Million)
  • Table 21.31 Libmedly™: Value Creation Analysis (USD Million)
  • Table 21.32 Lumevaq® (GS010): Target Patient Population
  • Table 21.33 Lumevaq® (GS010): Net Present Value (USD Million)
  • Table 21.34 Lumevaq® (GS010): Value Creation Analysis (USD Million)
  • Table 21.35 OTL-103: Target Patient Population
  • Table 21.36 OTL-103: Net Present Value (USD Million)
  • Table 21.37 OTL-103: Value Creation Analysis (USD Million
  • Table 21.38 PTC-AADC: Target Patient Population
  • Table 21.39 PTC-AADC: Net Present Value (USD Million)
  • Table 21.40 PTC-AADC: Value Creation Analysis (USD Million)
  • Table 21.41 BMN 270: Target Patient Population
  • Table 21.42 BMN 270: Net Present Value (USD Million)
  • Table 21.43 BMN 270: Value Creation Analysis (USD Million)
  • Table 21.44 rAd-IFN/Syn3: Target Patient Population
  • Table 21.45 rAd-IFN/Syn3: Net Present Value (USD Million)
  • Table 21.46 rAd-IFN/Syn3: Value Creation Analysis (USD Million)
  • Table 21.47 beti-cel: Target Patient Population
  • Table 21.48 beti-cel: Net Present Value (USD Million)
  • Table 21.49 beti-cel: Value Creation Analysis (USD Million)
  • Table 21.50 eli-cel: Target Patient Population
  • Table 21.51 eli-cel: Net Present Value (USD Million)
  • Table 21.52 eli-cel: Value Creation Analysis (USD Million)
  • Table 21.53 lovo-cel: Target Patient Population
  • Table 21.54 lovo-cel: Net Present Value (USD Million)
  • Table 21.55 lovo-cel: Value Creation Analysis (USD Million)
  • Table 21.56 SRP-9001: Target Patient Population
  • Table 21.57 SRP-9001: Net Present Value (USD Million)
  • Table 21.58 SRP-9001: Value Creation Analysis (USD Million)
  • Table 21.59 EB-101: Target Patient Population
  • Table 21.60 EB-101: Net Present Value (USD Million)
  • Table 21.61 EB-101: Value Creation Analysis (USD Million)
  • Table 21.62 ProstAtak®: Target Patient Population
  • Table 21.63 ProstAtak®: Net Present Value (USD Million)
  • Table 21.64 ProstAtak®: Value Creation Analysis (USD Million)
  • Table 21.65 D-Fi: Target Patient Population
  • Table 21.66 D-Fi: Net Present Value (USD Million)
  • Table 21.67 D-Fi: Value Creation Analysis (USD Million)
  • Table 21.68 CG0070: Target Patient Population
  • Table 21.69 CG0070: Net Present Value (USD Million)
  • Table 21.70 CG0070: Value Creation Analysis (USD Million)
  • Table 21.71 Vigil™-EWS: Target Patient Population
  • Table 21.72 Vigil™-EWS: Net Present Value (USD Million)
  • Table 21.73 Vigil™-EWS: Value Creation Analysis (USD Million)
  • Table 21.74 Engensis®: Target Patient Population
  • Table 21.75 Engensis®: Net Present Value (USD Million)
  • Table 21.76 Engensis®: Value Creation Analysis (USD Million)
  • Table 21.77 VGX-3100: Target Patient Population
  • Table 21.78 VGX-3100: Net Present Value (USD Million)
  • Table 21.79 VGX-3100: Value Creation Analysis (USD Million)
  • Table 21.80 Invossa™ (TG-C): Target Patient Population
  • Table 21.81 Invossa™ (TG-C): Net Present Value (USD Million)
  • Table 21.82 Invossa™ (TG-C): Value Creation Analysis (USD Million)
  • Table 21.83 VYJUVEKT™: Target Patient Population
  • Table 21.84 VYJUVEKT™: Net Present Value (USD Million)
  • Table 21.85 VYJUVEKT™: Value Creation Analysis (USD Million)
  • Table 21.86 PF-06939926: Target Patient Population
  • Table 21.87 PF-06939926: Net Present Value (USD Million)
  • Table 21.88 PF-06939926: Value Creation Analysis (USD Million)
  • Table 21.89 PF-06838435: Target Patient Population
  • Table 21.90 PF-06838435: Net Present Value (USD Million)
  • Table 21.91 PF-06838435: Value Creation Analysis (USD Million)
  • Table 21.92 PF-07055480: Target Patient Population
  • Table 21.93 PF-07055480: Net Present Value (USD Million)
  • Table 21.94 PF-07055480: Value Creation Analysis (USD Million)
  • Table 21.95 SPK-8011: Target Patient Population
  • Table 21.96 SPK-8011: Net Present Value (USD Million)
  • Table 21.97 SPK-8011: Value Creation Analysis (USD Million)
  • Table 21.98 AMT-061: Target Patient Population
  • Table 21.99 AMT-061: Net Present Value (USD Million)
  • Table 21.100 AMT-061: Value Creation Analysis (USD Million)
  • Table 21.101 VB-111: Target Patient Population
  • Table 21.102 VB-111: Net Present Value (USD Million)
  • Table 21.103 VB-111: Value Creation Analysis (USD Million)
  • Table 21.104 Generx®: Target Patient Population
  • Table 21.105 Generx®: Net Present Value (USD Million)
  • Table 21.106 Generx®: Value Creation Analysis (USD Million)
  • Table 21.107 AMG001: Target Patient Population
  • Table 21.108 AMG001: Net Present Value (USD Million)
  • Table 21.109 AMG001: Value Creation Analysis (USD Million)
  • Table 21.110 OAV-101: Target Patient Population
  • Table 21.111 OAV-101: Net Present Value (USD Million)
  • Table 21.112 OAV-101: Value Creation Analysis (USD Million)
  • Table 21.113 ADXS-HPV: Target Patient Population
  • Table 21.114 ADXS-HPV: Net Present Value (USD Million)
  • Table 21.115 ADXS-HPV: Value Creation Analysis (USD Million)
  • Table 21.116 AGTC 501: Target Patient Population
  • Table 21.117 AGTC 501: Net Present Value (USD Million)
  • Table 21.118 AGTC 501: Value Creation Analysis (USD Million)
  • Table 21.119 LYS-SAF302: Target Patient Population
  • Table 21.120 LYS-SAF302: Net Present Value (USD Million)
  • Table 21.121 LYS-SAF302: Value Creation Analysis (USD Million)
  • Table 21.122 NFS-01: Target Patient Population
  • Table 21.123 NFS-01: Net Present Value (USD Million)
  • Table 21.124 NFS-01: Value Creation Analysis (USD Million)
  • Table 21.125 AG0302-COVID19: Target Patient Population
  • Table 21.126 AG0302-COVID19: Net Present Value (USD Million)
  • Table 21.127 AG0302-COVID19: Value Creation Analysis (USD Million)
  • Table 21.128 RGX-314: Target Patient Population
  • Table 21.129 RGX-314: Net Present Value (USD Million)
  • Table 21.130 RGX-314: Value Creation Analysis (USD Million)
  • Table 21.131 Hologene 5: Target Patient Population
  • Table 21.132 Hologene 5: Net Present Value (USD Million)
  • Table 21.133 Hologene 5: Value Creation Analysis (USD Million)
  • Table 22.1 Small-scale Cell Culture Systems
  • Table 22.2 List of Contract Manufacturing Service Providers for Viral Vectors and Plasmid DNA
  • Table 26.1 Gene Therapies: Distribution by Stage of Development
  • Table 26.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
  • Table 26.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
  • Table 26.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
  • Table 26.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
  • Table 26.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene / Molecule Targeted
  • Table 26.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Therapy
  • Table 26.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
  • Table 26.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
  • Table 26.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of Administration
  • Table 26.11 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development
  • Table 26.12 Early-Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
  • Table 26.13 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
  • Table 26.14 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
  • Table 26.15 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Therapy
  • Table 26.16 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
  • Table 26.17 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
  • Table 26.18 Early-Stage Pipeline of Gene Therapies: Distribution by Route of Administration
  • Table 26.19 Gene Therapies: Distribution by Special Designation(s) Awarded
  • Table 26.20 Gene Therapy Developers: Distribution by Year of Establishment
  • Table 26.21 Gene Therapy Developers: Distribution by Company Size
  • Table 26.22 Gene Therapy Developers: Distribution by Location of Headquarters
  • Table 26.23 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
  • Table 26.24 Most Active Players: Distribution by Number of Gene Therapies
  • Table 26.25 Most Active Players: Distribution by Number of Gene Therapies and Phase of Development
  • Table 26.26 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
  • Table 26.27 Gene Therapies for Oncological Diseases: Distribution by Target Indication and Phase of Development
  • Table 26.28 Gene Therapies for Oncological Diseases: Distribution by Type of Vector Used
  • Table 26.29 Gene Therapies for Neurological Diseases: Distribution by Target Indication and Phase of Development
  • Table 26.30 Gene Therapies for Neurological Diseases: Distribution by Type of Vector Used
  • Table 26.31 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
  • Table 26.32 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
  • Table 26.33 Gene Therapies for Metabolic Diseases: Distribution by Target Indication and Phase of Development
  • Table 26.34 Gene Therapies for Metabolic Diseases: Distribution by Type of Vector Used
  • Table 26.35 Gene Therapies for Genetic Diseases: Distribution by Target Indication and Phase of Development
  • Table 26.36 Gene Therapies for Genetic Diseases: Distribution by Type of Vector Used
  • Table 26.37 Gene Therapy Patent Portfolio: Distribution by Type of Patent
  • Table 26.38 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
  • Table 26.39 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
  • Table 26.40 Gene Therapy Patent Portfolio: Distribution by Geography
  • Table 26.41 Gene Therapy Patent Portfolio: North America Scenario
  • Table 26.42 Gene Therapy Patent Portfolio: Europe Scenario
  • Table 26.43 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
  • Table 26.44 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
  • Table 26.45 Gene Therapy Patent Portfolio: Leading Industry Players: Distribution by Number of Patents
  • Table 26.46 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
  • Table 26.47 Gene Editing Patent Portfolio: Distribution by Type of Patent
  • Table 26.48 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
  • Table 26.49 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
  • Table 26.50 Gene Editing Patent Portfolio: Distribution by Geography
  • Table 26.51 Gene Editing Patent Portfolio: North America Scenario
  • Table 26.52 Gene Editing Patent Portfolio: Europe Scenario
  • Table 26.53 Gene Editing Patent Portfolio: Asia-Pacific Scenario
  • Table 26.54 Gene Editing Patent Portfolio: Distribution by CPC Symbols
  • Table 26.55 Gene Editing Patent Portfolio: Leading Industry Players: Distribution by Number of Patents
  • Table 26.56 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
  • Table 26.57 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2015-2022
  • Table 26.58 Mergers and Acquisitions: Distribution by Type of Agreement
  • Table 26.59 Mergers and Acquisitions: Distribution by Year and Type of Agreement
  • Table 26.60 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
  • Table 26.61 Mergers and Acquisitions: Local and International Deals
  • Table 26.62 Acquisitions: Distribution by Key Value Drivers
  • Table 26.63 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
  • Table 26.64 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product
  • Table 26.65 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company's Product
  • Table 26.66 Mergers and Acquisitions: Distribution by Therapeutic Area
  • Table 26.67 Funding and Investments: Cumulative Distribution of Instances by Year, 2015-2022
  • Table 26.68 Funding and Investments: Cumulative Distribution of Amount Invested by Year, 2015-2022 (USD Million)
  • Table 26.69 Funding and Investments: Distribution of Instances by Type of Funding, 2015-2022
  • Table 26.70 Funding and Investments: Distribution of Total Amount Invested by Type of Funding, 2015-2022 (USD Million)
  • Table 26.71 Funding and Investments: Summary of Investments, 2015-2022 (USD Million)
  • Table 26.72 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
  • Table 26.73 Funding and Investments: Distribution of Instances and Amount Invested by Geography
  • Table 26.74 Funding and Investments: Regional Distribution of Funding Instances
  • Table 26.75 Funding and Investments: Distribution of Instances and Amount Invested by Highest Phase of Development of the Company's Product
  • Table 26.76 Funding and Investments: Distribution of Instances by Therapeutic Area
  • Table 26.77 Most Active Players: Distribution by Number of Funding Instances, 2015-2022
  • Table 26.78 Most Active Players: Distribution by Amount Invested, 2015-2022 (USD Million)
  • Table 26.79 Funding and Investments: Distribution of Instances by Type of Investors
  • Table 26.80 Leading Investors: Distribution by Number of Instances
  • Table 26.81 Funding and Investment Summary, 2015-2022 (USD Million)
  • Table 26.82 Clinical Trial Analysis: Distribution by Trial Registration Year, 2017-2022
  • Table 26.83 Clinical Trial Analysis: Distribution by Trial Status
  • Table 26.84 Clinical Trial Analysis: Distribution by Trial Phase
  • Table 26.85 Clinical Trial Analysis: Distribution by Therapeutic Area
  • Table 26.86 Clinical Trial Analysis: Distribution by Geography
  • Table 26.87 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
  • Table 26.88 Clinical Trial Analysis: Distribution by Trial Status and Geography
  • Table 26.89 Clinical Trial Analysis: Distribution by Type of Sponsor
  • Table 26.90 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
  • Table 26.91 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, 2017-2022
  • Table 26.92 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
  • Table 26.93 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
  • Table 26.94 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
  • Table 26.95 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
  • Table 26.96 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
  • Table 26.97 Demand Analysis: Overall Demand for Gene Therapies
  • Table 26.98 Demand Analysis: Distribution by Type of Therapy, 2022, 2030 and 2035
  • Table 26.99 Demand Analysis: Distribution by Therapeutic Area, 2022, 2030 and 2035
  • Table 26.100 Demand Analysis: Distribution by Geography, 2022, 2030 and 2035
  • Table 26.101 Demand Analysis: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
  • Table 26.102 Global Gene Therapy Market, 2022-2035 (USD Billion)
  • Table 26.103 Gene Therapy Market: Distribution by Therapeutic Area, 2022, 2030 and 2035
  • Table 26.104 Gene Therapy Market: Distribution by Type of Vector Used, 2022, 2030 and 2035
  • Table 26.105 Gene Therapy Market: Distribution by Type of Therapy, 2022, 2030 and 2035
  • Table 26.106 Gene Therapy Market: Distribution by Type of Gene Delivery Method Used, 2022, 2030 and 2035
  • Table 26.107 Gene Therapy Market: Distribution by Route of Administration, 2022, 2030 and 2035
  • Table 26.108 Gene Therapy Market: Distribution by Geography, 2022, 2030 and 2035
  • Table 26.109 Gene Therapy Market: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
  • Table 26.110 Gendicine® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.111 Oncorine® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.112 Rexin-G® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.113 Neovasculgen® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.114 Strimvelis® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.115 Imlygic® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.116 Luxturna™ Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.117 Zolgensma™ Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.118 Collategene® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.119 Libmeldy™ Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.120 Lumevaq® (GS010) Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.121 OTL-103 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.122 PTC-AADC Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.123 BMN 270 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.124 rAd-IFN/Syn3 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.125 beti-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.126 eli-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.127 lovo-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.128 SRP-9001 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.129 EB-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.130 ProstAtak® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.131 D-Fi Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.132 CG0070 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.133 Vigil™-EWS Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.134 Engensis® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.135 VGX-3100 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.136 Invossa™ (TG-C) Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.137 VYJUVEKT™ Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.138 PF-06939926 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.139 PF-06838435 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.140 PF-07055480 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.141 SPK-8011 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.142 AMT-061 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.143 VB-111 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.144 Generx® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.145 AMG001 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.146 OAV-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.147 ADXS-HPV Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.148 AGTC 501 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.149 LYS-SAF302 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.150 NFS-01 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.151 AG0302-COVID19 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.152 RGX-314 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Table 26.153 Hologene 5 Sales Forecast (Till 2035): Base Scenario (USD Million)

LIST OF FIGURES

  • Figure 2.1 Executive Summary: Overall Market Landscape of Gene Therapies
  • Figure 2.2 Executive Summary: Competitive Landscape
  • Figure 2.3 Executive Summary: Key Therapeutic Areas
  • Figure 2.4 Executive Summary: Patent Analysis
  • Figure 2.5 Executive Summary: Mergers and Acquisitions
  • Figure 2.6 Executive Summary: Funding and Investment Analysis
  • Figure 2.7 Executive Summary: Clinical Trial Analysis
  • Figure 2.8 Executive Summary: Demand Analysis
  • Figure 2.9 Executive Summary: Market Forecast and Opportunity Analysis
  • Figure 3.1 Evolution of Gene Therapies
  • Figure 3.2 Ex Vivo Gene Therapies
  • Figure 3.3 In Vivo Gene Therapies
  • Figure 3.4 Mechanism of Action of Gene Therapies
  • Figure 3.5 Genome Editing: Historical Milestones
  • Figure 3.6 Genome Editing Techniques
  • Figure 3.7 Advantages and Disadvantages of Gene Therapies
  • Figure 4.1 Gene Transfer: Viral and Non-Viral Methods
  • Figure 5.1 Centralized Regulatory Pathway for Market Approval in the US
  • Figure 5.2 Centralized Regulatory Pathway for Market Approval in Europe
  • Figure 5.3 Manufacturing Requirements for Gene Therapy Products in China
  • Figure 5.4 Key Elements of Quality Control in China
  • Figure 5.5 Centralized Regulatory Pathway for Market Approval in Japan
  • Figure 6.1 Gene Therapies: Distribution by Stage of Development
  • Figure 6.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
  • Figure 6.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
  • Figure 6.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
  • Figure 6.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
  • Figure 6.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene / Molecule Targeted
  • Figure 6.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Therapy
  • Figure 6.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
  • Figure 6.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
  • Figure 6.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of Administration
  • Figure 6.11 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development
  • Figure 6.12 Early-Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
  • Figure 6.13 Early-Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
  • Figure 6.14 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
  • Figure 6.15 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Therapy
  • Figure 6.16 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used and Type of Therapy
  • Figure 6.17 Early-Stage Pipeline of Gene Therapies: Distribution by Type of Gene Delivery Method Used
  • Figure 6.18 Early-Stage Pipeline of Gene Therapies: Distribution by Route of Administration
  • Figure 6.19 Gene Therapies: Distribution by Special Designation(s) Awarded
  • Figure 6.20 Gene Therapies: Distribution by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)
  • Figure 7.1 Gene Therapy Developers: Distribution by Year of Establishment
  • Figure 7.2 Gene Therapy Developers: Distribution by Company Size
  • Figure 7.3 Gene Therapy Developers: Distribution by Location of Headquarters
  • Figure 7.4 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
  • Figure 7.5 Gene Therapy Developers: Distribution of Players based in North America
  • Figure 7.6 Gene Therapy Developers: Distribution of Players based in Europe
  • Figure 7.7 Gene Therapy Developers: Distribution of Players based in Asia-Pacific
  • Figure 7.8 Gene Therapy Developers: Distribution of Players based in Middle East and North Africa
  • Figure 7.9 Key Players: Distribution by Number of Gene Therapies
  • Figure 7.10 Key Players: Distribution by Number of Gene Therapies and Phase of Development
  • Figure 8.1 Gendicine®: Development Timeline
  • Figure 8.2 Oncorine®: Development Timeline
  • Figure 8.3 Oncorine®: Mechanism of Action
  • Figure 8.4 Rexin-G®: Development Timeline
  • Figure 8.5 Neovasculgen®: Development Timeline
  • Figure 8.6 Imlygic®: Development Timeline
  • Figure 8.7 Imlygic®: Mechanism of Action
  • Figure 8.8 Strimvelis®: Development Timeline
  • Figure 8.9 Luxturna™: Development Timeline
  • Figure 8.10 Zolgensma™: Development Timeline
  • Figure 8.11 Collategene®: Development Timeline
  • Figure 8.12 Zyntelgo™: Development Timeline
  • Figure 8.13 Libmeldy™: Development Timeline
  • Figure 9.1 Successful Drug Launch Strategy: ROOTS Framework
  • Figure 9.2 Successful Drug Launch Strategy: Product Differentiation
  • Figure 9.3 Commonly Adopted Commercialization Strategies based on Phase of Development of Product
  • Figure 9.4 Harvey Ball Analysis: Key Commercialization Strategies Adopted by Gene Therapy Developers
  • Figure 9.5 Approved Gene Therapies: Historical Timeline of Geographical Expansion
  • Figure 9.6 Approved Gene Therapies: Snapshot of Promotional Activities on Product Websites
  • Figure 9.7 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
  • Figure 11.1 Gene Editing: Key Application Areas
  • Figure 11.2 Gene Editing: Emerging Technology Platforms
  • Figure 12.1 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
  • Figure 12.2 Gene Therapies for Oncological Diseases: Distribution by Target Indication and Phase of Development
  • Figure 12.3 Gene Therapies for Oncological Diseases: Distribution by Type of Vector Used
  • Figure 12.4 Gene Therapies for Neurological Diseases: Distribution by Target Indication and Phase of Development
  • Figure 12.5 Gene Therapies for Neurological Diseases: Distribution by Type of Vector Used
  • Figure 12.6 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
  • Figure 12.7 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
  • Figure 12.8 Gene Therapies for Metabolic Diseases: Distribution by Target Indication and Phase of Development
  • Figure 12.9 Gene Therapies for Metabolic Diseases: Distribution by Type of Vector Used
  • Figure 12.10 Gene Therapies for Genetic Diseases: Distribution by Target Indication and Phase of Development
  • Figure 12.11 Gene Therapies for Genetic Diseases: Distribution by Type of Vector Used
  • Figure 13.1 Gene Therapy Patent Portfolio: Distribution by Type of Patent
  • Figure 13.2 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
  • Figure 13.3 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
  • Figure 13.4 Gene Therapy Patent Portfolio: Distribution by Geography
  • Figure 13.5 Gene Therapy Patent Portfolio: North America Scenario
  • Figure 13.6 Gene Therapy Patent Portfolio: Europe Scenario
  • Figure 13.7 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
  • Figure 13.8 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
  • Figure 13.9 Gene Therapy Patent Portfolio: Emerging Focus Areas
  • Figure 13.10 Leading Industry Players (Gene Therapies): Distribution by Number of Patents
  • Figure 13.11 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
  • Figure 13.12 Gene Therapy Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
  • Figure 13.13 Gene Therapy Patent Portfolio: Distribution by Patent Age (2017-2022)
  • Figure 13.14 Gene Therapy Patent Portfolio: Valuation Analysis
  • Figure 13.15 Gene Editing Patent Portfolio: Distribution by Type of Patent
  • Figure 13.16 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
  • Figure 13.17 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
  • Figure 13.18 Gene Editing Patent Portfolio: Distribution by Geography
  • Figure 13.19 Gene Editing Patent Portfolio: North America Scenario
  • Figure 13.20 Gene Editing Patent Portfolio: Europe Scenario
  • Figure 13.21 Gene Editing Patent Portfolio: Asia-Pacific Scenario
  • Figure 13.22 Gene Editing Patent Portfolio: Distribution by CPC Symbols
  • Figure 13.23 Gene Editing Patent Portfolio: Emerging Focus Areas
  • Figure 13.24 Leading Industry Players (Gene Editing): Distribution by Number of Patents
  • Figure 13.25 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
  • Figure 13.26 Gene Editing Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
  • Figure 13.27 Gene Editing Patent Portfolio: Distribution by Patent Age (2017-2022)
  • Figure 13.28 Gene Editing Patent Portfolio: Valuation Analysis
  • Figure 13.29 Gene Therapy Patent Portfolio: Cumulative Distribution by Type of Organization
  • Figure 13.30 Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
  • Figure 14.1 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2015-2022
  • Figure 14.2 Mergers and Acquisitions: Distribution by Type of Agreement
  • Figure 14.3 Mergers and Acquisitions: Distribution by Year and Type of Agreement
  • Figure 14.4 Mergers and Acquisitions: Continent-wise Distribution
  • Figure 14.5 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
  • Figure 14.6 Mergers and Acquisitions: Local and International Deals
  • Figure 14.7 Acquisitions: Distribution by Key Value Drivers
  • Figure 14.8 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
  • Figure 14.9 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product
  • Figure 14.10 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company's Product
  • Figure 14.11 Mergers and Acquisitions: Distribution of Deal Amount by Phase of Development of the Acquired Company's Product
  • Figure 14.12 Mergers and Acquisitions: Distribution by Therapeutic Area
  • Figure 15.1 Funding and Investments: Cumulative Distribution of Instances by Year, 2015-2022
  • Figure 15.2 Funding and Investments: Cumulative Distribution of Amount Invested by Year, 2015-2022 (USD Million)
  • Figure 15.3 Funding and Investments: Distribution of Instances by Type of Funding, 2015-2022
  • Figure 15.4 Funding and Investments: Distribution of Total Amount Invested by Type of Funding, 2015-2022 (USD Million)
  • Figure 15.5 Funding and Investments: Distribution of Instances by Year and Type of Funding, 2015-2022
  • Figure 15.6 Funding and Investments: Distribution of Amount Invested by Year and Type of Funding, 2015-2022 (USD Million)
  • Figure 15.7 Funding and Investments: Summary of Investments, 2015-2022 (USD Million)
  • Figure 15.8 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
  • Figure 15.9 Funding and Investments: Distribution of Instances and Amount Invested by Geography
  • Figure 15.10 Funding and Investments: Regional Distribution of Funding Instances
  • Figure 15.11 Funding and Investments: Distribution of Instances and Amount Invested by Highest Phase of Development of the Company's Product
  • Figure 15.12 Funding and Investments: Distribution of Instances by Therapeutic Area
  • Figure 15.13 Most Active Players: Distribution by Number of Funding Instances, 2015-2022
  • Figure 15.14 Most Active Players: Distribution by Amount Invested, 2015-2022 (USD Million)
  • Figure 15.15 Funding and Investment Analysis: Distribution of Instances by Type of Investors
  • Figure 15.16 Leading Investors: Distribution by Number of Instances
  • Figure 15.17 Funding and Investment Summary, 2015-2022 (USD Million)
  • Figure 16.1 Clinical Trial Analysis: Distribution by Trial Registration Year, 2017-2022
  • Figure 16.2 Clinical Trial Analysis: Distribution by Trial Status
  • Figure 16.3 Clinical Trial Analysis: Distribution by Trial Phase
  • Figure 16.4 Clinical Trial Analysis: Distribution by Therapeutic Area
  • Figure 16.5 Clinical Trial Analysis: Distribution by Geography
  • Figure 16.6 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
  • Figure 16.7 Clinical Trial Analysis: Distribution by Trial Status and Geography
  • Figure 16.8 Clinical Trial Analysis: Distribution by Trial Status, Trial Phase and Geography
  • Figure 16.9 Clinical Trial Analysis: Distribution by Therapeutic Area and Geography
  • Figure 16.10 Clinical Trial Analysis: Distribution by Type of Sponsor
  • Figure 16.11 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
  • Figure 16.12 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, 2017-2022
  • Figure 16.13 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
  • Figure 16.14 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
  • Figure 16.15 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
  • Figure 16.16 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
  • Figure 16.17 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
  • Figure 16.18 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status, Trial Phase and Location of Trial Site
  • Figure 16.19 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area and Location of Trial Site
  • Figure 17.1 Gene Therapy: Pricing Model Based on Patient Segment
  • Figure 18.1 Start-up Valuation based on Experience of the Gene Therapy Developer
  • Figure 19.1 Big Pharma Players: Top Pharmaceutical Companies (Heat Map Representation)
  • Figure 19.2 Big Pharma Players: Analysis by Therapeutic Area
  • Figure 19.3 Big Pharma Players: Analysis by Type of Vector Used
  • Figure 19.4 Big Pharma Players: Analysis by Type of Therapy
  • Figure 19.5 Big Pharma Players: Analysis by Type of Gene Delivery Method Used
  • Figure 20.1 Global Demand for Gene Therapies, 2022-2035
  • Figure 20.2 Demand Analysis: Distribution by Type of Therapy, 2022, 2030 and 2035
  • Figure 20.3 Demand Analysis: Distribution by Therapeutic Area, 2022, 2030 and 2035
  • Figure 20.4 Demand Analysis: Distribution by Geography, 2022, 2030 and 2035
  • Figure 20.5 Demand Analysis: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035
  • Figure 21.1 Global Gene Therapy Market, 2022-2035 (USD Billion)
  • Figure 21.2 Gene Therapy Market: Distribution by Therapeutic Area, 2022, 2030 and 2035
  • Figure 21.3 Gene Therapy Market: Distribution by Type of Vector Used, 2022, 2030 and 2035
  • Figure 21.4 Gene Therapy Market: Distribution by Type of Therapy, 2022, 2030 and 2035
  • Figure 21.5 Gene Therapy Market: Distribution by Type of Gene Delivery Method Used, 2022, 2030 and 2035
  • Figure 21.6 Gene Therapy Market: Distribution by Route of Administration, 2022, 2030 and 2035
  • Figure 21.7 Gene Therapy Market: Distribution by Geography, 2022, 2030 and 2035
  • Figure 21.8 Gene Therapy Market: Country-wise Distribution (Asia-Pacific), 2022, 2030 and 2035

Figure 21. 9 Gendicine® Sales Forecast (Till 2035): Base Scenario (USD Million)

  • Figure 21.10 Oncorine® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.11 Rexin-G® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.12 Neovasculgen® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.13 Strimvelis® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.14 Imlygic® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.15 Luxturna™ Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.16 Zolgensma™ Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.17 Collategene® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.18 Libmeldy™ Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.19 Lumevaq® (GS010) Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.20 OTL-103 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.21 PTC-AADC Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.22 BMN 270 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.23 rAd-IFN/Syn3 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.24 beti-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.25 eli-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.26 lovo-cel Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.27 SRP-9001 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.28 EB-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.29 ProstAtak® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.30 D-Fi Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.31 CG0070 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.32 Vigil™-EWS Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.33 Engensis® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.34 VGX-3100 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.35 Invossa™ (TG-C) Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.36 VYJUVEKT™ Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.37 PF-06939926 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.38 PF-06838435 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.39 PF-07055480 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.40 SPK-8011 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.41 AMT-061 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.42 VB-111 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.43 Generx® Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.44 AMG001 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.45 OAV-101 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.46 ADXS-HPV Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.47 AGTC 501 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.48 LYS-SAF302 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.49 NFS-01 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.50 AG0302-COVID19 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.51 RGX-314 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 21.52 Hologene 5 Sales Forecast (Till 2035): Base Scenario (USD Million)
  • Figure 22.1 Viral Vectors: Key Manufacturing Steps
  • Figure 23.1 Steps Involved in the Development of Gene Therapies
  • Figure 23.2 Overall Development Process of Cell-based Gene Therapies
  • Figure 23.3 Key Responsibilities of Stakeholders Involved in Advanced Therapies Supply Chain
  • Figure 23.4 Key Steps Involved in the Implementation of a Supply Chain Strategy
  • Figure 23.5 Keys Steps Involved in the Logistics Process of Autologous Therapies
  • Figure 23.6 Comparison of Logistics Processes for Autologous and Allogeneic Therapies
  • Figure 23.7 Global Regulatory Authorities for Gene Therapy Supply Chain
  • Figure 23.8 Overview of Supply Chain Orchestration Platform
  • Figure 23.9 Functions of Key Technological Solutions for Cell and Advanced Therapies Supply Chain Management
  • Figure 24.1 Concluding Remarks: Overall Market Overview
  • Figure 24.2 Concluding Remarks: Patent Analysis
  • Figure 24.3 Concluding Remarks: Mergers and Acquisitions
  • Figure 24.4 Concluding Remarks: Funding and Investments
  • Figure 24.5 Concluding Remarks: Clinical Trials Analysis
  • Figure 24.6 Concluding Remarks: Big Pharmaceutical Gene Therapy Related Initiatives
  • Figure 24.7 Concluding Remarks: Market Forecast and Opportunity Analysis
  • Figure 24.8 Concluding Remarks: Market Forecast and Opportunity Analysis
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Manager - EMEA

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Manager - Americas

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