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Gene Therapy Market, 6th Edition: Industry Trends and Global Forecasts to 2035 - Therapeutic Area, Type of Vector, Type of Therapy, Type of Gene Delivery Method, Route of Administration, Geographical Region, Leading Market Players and Sales Forecast

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    Report

  • 972 Pages
  • September 2024
  • Region: Global
  • Roots Analysis
  • ID: 5240465

The Global Gene Therapy Market is valued at USD 2.4 billion in 2024 growing at a CAGR of 19.25% during the forecast period 2024-2035.

A diverse range of disorders arising from congenital abnormalities and inherited genetic mutations, including autoimmune disorders, genetic disorders, neurological disorders and oncological disorders significantly impacts individuals’ health. These disorders are consequences of certain inherited genetic anomalies that disrupt the normal functioning of genes and adversely affect the process of translation. The statistics presented by the World Health Organization (WHO) states that 10 out of every 1,000 individuals are affected by genetic disorders, contributing to 70 million people globally. In addition, more than 40% of infant mortality globally is associated with various genetic disorders.  Therefore, to address such challenges, gene therapies have emerged as a revolutionary approach and have proven to be a promising modality in treating a wide array of diseases, focused on delivering targeted therapies directly to affected organs or cells. 

The objective of gene therapy is to comprehend the underlying genetic cause of a disease, followed by either introduction of a healthy version of the mutated gene or by inactivation of the faulty / disease-causing genes in the body. It is worth noticing that in 2003, Gendicine® (Sibiono GeneTech) became the world’s first gene therapy to receive regulatory approval from the China FDA. In the past two decades, this has led to several breakthroughs related to the development of gene therapies. For instance, in December 2017, the USFDA approved Luxturna® (Spark Therapeutics), the first gene therapy intended for the treatment of inherited retinal disorders.  Additionally, in May 2019, the USFDA approved ZOLGENSMA® (Novartis), the first gene therapy for the treatment of spinal muscular atrophy in pediatric patients.  It is noteworthy that the therapy received multiple designations, namely breakthrough therapy, fast track, orphan drug and priority review designations resulting in the accelerated approval of the drug in the US. Further, in 2023, five gene therapies received the USFDA market approval for various therapeutic indications.

In addition, the month of April 2024 witnessed a significant increase in the number of active clinical trials (1,100 active clinical trials) for evaluating gene therapies across various phases of clinical studies. Moreover, three gene therapies have already received regulatory approval in 2024, and according to predictions made by USFDA in 2019, it is estimated that 10 to 20 would gain market access, each year, till 2025. It is noteworthy that the favorable results from ongoing clinical research initiatives have spurred investments from both government and private sectors to support the development of these therapeutic products. With the progressing domain, gene therapies are anticipated to be utilized for the treatment of more than 65.6 million patients suffering from a myriad of disease indications, by 2034.

The rapidly evolving gene therapy product portfolio tends to attract an increasing number of startups and big pharma players, marking their presence in the gene therapy market. At present, 345 gene therapy companies are involved in the development of various early and late-stage therapies worldwide. The recent years have observed a significant increase in the integration of novel technologies, such as gene modification, genome editing, genome sequencing and manipulation technologies (molecular / gene switch), in conjugation with advanced gene delivery methods. Additionally, next-generation delivery platforms, including nanoparticles and hybrid vector systems, have been proven to facilitate an effective, precise and safe delivery of gene-based therapeutics, thereby enabling more targeted treatment approaches. With the integration of advancements in technology and growing preference for single-dose effective therapeutics, the gene therapy market is poised to witness substantial market growth during the forecast period. 

Key Market Segments

Therapeutic Area

  • Cardiovascular Disorders
  • Dermatological Disorders
  • Genetic Disorders
  • Hematological Disorders
  • Metabolic Disorders
  • Muscle Disorders
  • Oncological Disorders
  • Ophthalmic Disorders
  • Other Disorders

Type of Vector

  • Adeno-associated Virus Vectors
  • Adenovirus Vectors
  • Herpes Simplex Virus Vectors
  • Lentivirus Vectors
  • Non-viral Vectors
  • Retrovirus Vectors
  • Other Viral Vectors

Type of Therapy

  • Gene Augmentation
  • Gene Editing
  • Gene Regulation
  • Oncolytic Immunotherapies
  • Other Therapies

Type of Gene Delivery Method

  • Ex vivo Gene Delivery
  • In vivo Gene Delivery

Route of Administration

  • Intramuscular Route
  • Intratumoral Route
  • Intravenous Route
  • Subretinal Route
  • Other Routes of Administration

Key Geographical Regions 

  • North America
  • Europe 
  • Asia-Pacific 
  • Latin America
  • Rest of the World 

Leading Players 

  • Amgen
  • Krystal Biotech
  • Novartis
  • Orchard Therapeutics
  • Sarepta Therapeutics

Report Coverage:

The chapter discusses evolutionary studies on gene therapies, along with the classification based on various attributes. Additionally, the chapter features a detailed discussion on the mechanism of action of gene therapies, prominent routes of administration and highlights the various advantages and disadvantages associated with this form of therapeutic intervention, providing an insight on the genome editing techniques, their evolution and applications in the biopharmaceutical industry. 

  • A thorough 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 has been comprehended in the report.
  • The chapter provides a detailed input on the regulatory landscape related to gene therapies across various geographies, namely North America (US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing information on treatment-related reimbursement scenarios for gene therapies, highlighting existing challenges associated with such processes.
  • A cross-examined report comprising the marketed and clinical stage gene therapies, based on several relevant parameters, such as stage of development (marketed, registration, phase III, phase II and phase I), drug designations (advanced therapy medicinal products, breakthrough therapy, fast track, orphan drug, priority medicines, priority review, regenerative medicine advanced therapy and rare pediatric disease designation), therapeutic area (autoimmune disorders, cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, metabolic disorders, muscle disorders, neurological disorders, oncological disorders, ophthalmic disorders, rare disorders and others), target gene (GM-CSF, 4-1BBL gene, anti-VEGF gene, DMD gene, F8 gene, F9 gene, HGF gene, HPV antigens, IL-12 gene, PH20 hyaluronidase, SMN1 gene, TMZ-CD40L gene and others), type of vector used (adeno-associated virus vectors, adenovirus vectors, herpes simplex virus vectors, lentivirus vectors, non-viral vectors, retrovirus vectors and other viral vectors), type of therapy (gene augmentation, gene editing, gene regulation, immunotherapy and oncolytic immunotherapy), type of gene delivery method (ex vivo and in vivo), route of administration (intracisternal, intradermal, intramuscular, intratumoral, intravenous, intravitreal, subcutaneous, subretinal and other routes) and dosing frequency (single dose and multiple doses). Furthermore, the chapter presents a detailed analysis of the current landscape of discovery and preclinical stage gene therapies based on parameters. These parameters include stages of development (discovery and preclinical), therapeutic area (cardiovascular disorders, genetic disorders, hematological disorders, hepatic disorders, infectious diseases, metabolic disorders, muscle related disorders, neurological disorders, oncological disorders, ophthalmic disorders and others), target gene (ABCA4 gene, antigen, anti-VEGF gene, caveolin-1 gene, DMD gene, F8 gene, F9 gene, FGF 21 gene, FXN gene, JUMP70 gene and others), type of vector used (adeno-associated virus vectors, adenovirus vectors, herpes simplex virus vectors, lentivirus vectors, non-viral vectors, retrovirus vectors and other viral vectors), type of therapy (gene augmentation, gene editing, gene regulation, immunotherapy and oncolytic immunotherapy) and type of gene delivery method (ex vivo and in vivo).
  • A comprehensive assessment of the market landscape of gene therapy developers featuring information on year of establishment, company size (in terms of number of employees), location of headquarters and most active players (in terms of the number of gene therapies developed) has been provided in the report.
  • A comprehensive set of profiles of prominent players involved in developing gene therapies. Each profile features a brief overview of the company (including information on year of establishment, location of headquarters and number of employees, leadership team and annual revenues (if available)), gene therapy portfolio, recent developments and an informed future perspective.  
  • The chapter provides a detailed exploration of marketed gene therapies, along with information on the development timeline of the therapy, mechanism of action, type of vector used, dosage and manufacturing details, target indication, status of development, as well as details related to the developer company.
  • A comprehensive report comprising various commercialization strategies that have been adopted by drug developers engaged in the gene therapy domain across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing stage.
  • An intricate set of profiles of late stage (phase II / III and above) gene therapies, along with information on the development timeline of the therapy, mechanism of action, type of vector used, affiliated technology, dosage and manufacturing details, as well as details related to the developer company.
  • A cross-examined 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. Further, it also provides an insight on competitive benchmarking analysis of the patent portfolios of leading industry / non-industry players with an emphasis on patent valuation. We have a separate detailed study available on gene therapy and vectors IP Landscape. 
  • An elaborate discussion on the various mergers and acquisitions undertaken in the gene therapy domain, 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 has been provided in the report.
  • A brief context of the funding and investments made in the gene therapy market (seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings), at various stages of development in companies that are engaged in this domain has been offered in the report.
  • An analyzed report of the completed, ongoing and planned clinical studies, based on several relevant parameters, which includes, trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.
  • A detailed report focusing on the factors that are likely to affect the pricing of gene therapies, featuring different models / approaches that may be adopted by developers / manufacturers to decide the prices of these therapies.
  • The report provides an overview encompassing the startup companies engaged in this domain (since 2017) based on year of experience.
  • A brief review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting the trend across parameters, such as therapeutic area, type of vector used, type of therapy and type of gene delivery method used. Furthermore, it provides a detailed exploration of the benchmarking analysis across key parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength. 
  • An informed estimate of the global annual demand for gene therapies, considering the marketed gene-based therapies and clinical trials evaluating gene therapies, based on various parameters, such as target patient population, dosing frequency and dose strength.
  • A cross-examined report comprising the analysis of the factors that can impact the growth of the gene therapy market. It also features identification and analysis of key drivers, potential restraints, emerging opportunities, and existing challenges.
  • A detailed gene therapy market forecast to estimate the current market size and future opportunity over the next 11 years. It is based on multiple parameters, likely adoption trends and through primary validations, we have provided an informed estimate on the market size, till 2035. 
  • An in-depth insightful projection of the current size and future opportunity within the gene therapy industry across different therapeutic areas, namely cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle disorders, oncological disorders, ophthalmic disorders and other disorders. 
  • A comprehensive report carrying details about the projections of the current size and future opportunity within the gene therapies market across different types of vectors, namely adeno-associated virus vectors, adenovirus vectors, herpes simplex virus vectors, lentivirus vectors, non-viral vectors, retrovirus vectors and other viral vectors.
  • A substantial report focusing on the detailed projections of the current size and future opportunity within the gene therapies market across different types of therapy, namely gene augmentation, gene editing, gene regulation, oncolytic immunotherapies and other therapies.
  • An enumerated report having insights on the detailed projections of the current size and future opportunity within the gene therapy market across different types of gene delivery method, namely ex vivo gene delivery and in vivo gene delivery.
  • Detailed projections of the current size and future opportunity within the gene therapies market across different routes of administration, namely intramuscular, intratumoral, intravenous, subretinal and other routes of administration.
  • Comprehensive projections of the current size and future opportunity within the gene therapy market across geographical regions, namely North America, Europe, Asia-Pacific, Latin America and rest of the world.
  • The report includes detailed projections of the current and future revenues from the sales of gene therapies developed by the leading players, namely Novartis, Sarepta Therapeutics, and other players.
  • An insightful projection of the current and future revenues from the sales of marketed and phase III gene therapies developed by the leading players within the gene therapy market.
  • 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 are being used for the development of gene therapies.
  • A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing viral vectors used in gene therapy products. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.
  • A refined report discussing 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.

Key Benefits of Buying this Report

  • The report offers market leaders and newcomers valuable insights into revenue estimations for both the overall market and its sub-segments.
  • Stakeholders can utilize the report to enhance their understanding of the competitive landscape, allowing for improved business positioning and more effective go-to-market strategies.
  • The report provides stakeholders with a pulse on the Gene Therapy Market, furnishing them with essential information on significant market drivers, barriers, opportunities, and challenges.

Leading Market Companies

  • Amgen
  • Artgen Biotech
  • BioMarin Pharmaceutical
  • bluebird bio
  • CRISPR Therapeutics
  • CSL Behring
  • Ferring Pharmaceuticals
  • Kolon TissueGene
  • Krystal Biotech
  • Novartis
  • Orchard Therapeutics
  • Pfizer
  • PTC Therapeutics
  • Sarepta Therapeutics
  • Shanghai Sunway Biotech
  • Sibiono GeneTech
  • Spark Therapeutics

Please note: This report can be updated on request. Please contact our Customer Experience team using the Ask a Question widget on our website.

Table of Contents

1. PREFACE
1.1. Introduction
1.2. Market Share Insights
1.3. Key Market Insights
1.4 Report Coverage
1.5. Key Questions Answered
1.6. Chapter Outlines

2. RESEARCH METHODOLOGY
2.1. Chapter Overview

2.2. Research Assumptions
2.2.1. Market Landscape and Market Trends
2.2.2. Market Forecast and Opportunity Analysis
2.2.3. Comparative Analysis

2.3. Database Building
2.3.1. Data Collection
2.3.2. Data Validation
2.3.3. Data Analysis

2.4. Project Methodology
2.4.1. Secondary Research
2.4.1.1. Annual Reports
2.4.1.2. Academic Research Papers
2.4.1.3. Company Websites
2.4.1.4. Investor Presentations
2.4.1.5. Regulatory Filings
2.4.1.6. White Papers
2.4.1.7. Industry Publications
2.4.1.8. Conferences and Seminars
2.4.1.9. Government Portals
2.4.1.10. Media and Press Releases
2.4.1.11. Newsletters
2.4.1.12. Industry Databases
2.4.1.13. Roots Proprietary Databases
2.4.1.14. Paid Databases and Sources
2.4.1.15. Social Media Portals
2.4.1.16. Other Secondary Sources

2.4.2. Primary Research
2.4.2.1. Types of Primary Research
2.4.2.1.1. Qualitative Research
2.4.2.1.2. Quantitative Research
2.4.2.1.3. Hybrid Approach

2.4.2.2. Advantages of Primary Research

2.4.2.3. Techniques for Primary
2.4.2.3.1. Interviews
2.4.2.3.2. Surveys
2.4.2.3.3. Focus Groups
2.4.2.3.4. Observational Research
2.4.2.3.5. Social Media Interactions

2.4.2.4. Key Opinion Leaders Considered in Primary Research
2.4.2.4.1. Company Executives (CXOs)
2.4.2.4.2. Board of Directors
2.4.2.4.3. Company Presidents and Vice Presidents
2.4.2.4.4. Research and Development Heads
2.4.2.4.5. Technical Experts
2.4.2.4.6. Subject Matter Experts
2.4.2.4.7. Scientists
2.4.2.4.8. Doctors and Other Healthcare Providers

2.4.2.5. Ethics and Integrity
2.4.2.5.1. Research Ethics
2.4.2.5.2. Data Integrity

2.4.3. Analytical Tools and Databases

3. MARKET DYNAMICS
3.1. Chapter Overview
3.2. Forecast Methodology
3.2.1. Top-down Approach
3.2.2. Bottom-up Approach
3.2.3. Hybrid Approach

3.3. Market Assessment Framework
3.3.1. Total Addressable Market (TAM)
3.3.2. Serviceable Addressable Market (SAM)
3.3.3. Serviceable Obtainable Market (SOM)
3.3.4. Currently Acquired Market (CAM)

3.4. Forecasting Tools and Techniques
3.4.1. Qualitative Forecasting
3.4.2. Correlation
3.4.3. Regression
3.4.4. Extrapolation
3.4.5. Convergence
3.4.6. Sensitivity Analysis
3.4.7. Scenario Planning
3.4.8. Data Visualization
3.4.9. Time Series Analysis
3.4.10. Forecast Error Analysis

3.5. Key Considerations
3.5.1. Demographics
3.5.2. Government Regulations
3.5.3. Reimbursement Scenarios
3.5.4. Market Access
3.5.5. Supply Chain
3.5.6. Industry Consolidation
3.5.7. Pandemic / Unforeseen Disruptions Impact

3.6. Key Market Segmentation
3.7. Robust Quality Control
3.8. Limitations

4. ECONOMIC CONSIDERATONS
4.1. Chapter Overview
4.2. Market Dynamics
4.2.1. Time Period
4.2.1.1. Historical Trends
4.2.1.2. Current and Forecasted Estimates

4.2.2. Currency Coverage
4.2.2.1. Overview of Major Currencies Affecting the Market
4.2.2.2. Impact of Currency Fluctuations on the Industry

4.2.3. Foreign Exchange Impact
4.2.3.1. Evaluation of Foreign Exchange Rates and Their Impact on Market
4.2.3.2. Strategies for Mitigating Foreign Exchange Risk

4.2.4. Recession
4.2.4.1. Historical Analysis of Past Recessions and Lessons Learnt
4.2.4.2. Assessment of Current Economic Conditions and Potential Impact on the Market

4.2.5. Inflation
4.2.5.1. Measurement and Analysis of Inflationary Pressures in the Economy
4.2.5.2. Potential Impact of Inflation on the Market Evolution

5. EXECUTIVE SUMMARY
6. INTRODUCTION
6.1. Chapter Overview
6.2. Evolution of Gene Therapies

6.3. Classification of Gene Therapies
6.3.1. Based on Source of Origin
6.3.2. Based on Method of Delivery
6.3.2.1. Ex vivo Gene Therapies
6.3.2.2. In vivo Gene Therapies

6.4. Routes of Administration
6.5. Mechanism of Action of Gene Therapies
6.6. Advantages and Disadvantages of Gene Therapies

6.7. Challenges associated with Gene Therapies
6.7.1. Ethical and Social Challenges
6.7.2. Therapy Development Challenges
6.7.3. Manufacturing Challenges
6.7.4. Commercial Viability Challenges

6.8. Introduction to Genome Editing
6.8.1. Evolution of Genome Editing
6.8.2. Applications of Genome Editing
6.8.3. Types of Genome Editing Techniques

6.9. Concluding Remarks

7. GENE DELIVERY VECTORS
7.1. Chapter Overview
7.2. Methods of Gene Transfer

7.3. Viral Vectors for Genetically Modified Therapies
7.3.1. Types of Viral Vectors
7.3.1.1. Adeno-associated Viral Vectors
7.3.1.2. Adenoviral Vectors
7.3.1.3. Lentiviral Vectors
7.3.1.4. Retroviral Vectors
7.3.1.5. Other Viral Vectors

7.4. Non-Viral Vectors for Genetically Modified Therapies
7.4.1. Types of Non-Viral Vectors
7.4.1.1. Plasmid DNA
7.4.1.2. Liposomes, Lipoplexes and Polyplexes
7.4.1.3. Oligonucleotides
7.4.1.4. Nanoparticles
7.4.1.5. Hybrid System Vectors
7.4.1.6. Other Non-Viral Vectors

7.4.2. Gene Delivery using Non-Viral Vectors
7.4.2.1. Biolistic Methods
7.4.2.2. Electroporation
7.4.2.3. Receptor Mediated Gene Delivery
7.4.2.4. Gene Activated matrix (GAM)

8. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIOS
8.1. Chapter Overview
8.2. Regulatory Guidelines in North America
8.2.1. The US Scenario
8.2.2. Canadian Scenario

8.3. Regulatory Guidelines in Europe
8.3.1. Quality Documentation for Gene Therapy Products
8.3.2. Non-Clinical Development
8.3.3. Clinical Development

8.4. Regulatory Guidelines in Asia-Pacific
8.4.1. Chinese Scenario
8.4.1.1. Construction of DNA Expression Cassette and Gene Delivery Systems

8.4.1.2. Generation and Characterization of Cell Banks and Engineered Bacteria Banks
8.4.1.2.1. Cell Bank
8.4.1.2.2. Bacterial Cell Bank

8.4.1.3. Manufacturing of Gene Therapy Products
8.4.1.4. Quality Control
8.4.1.5. Evaluation of Efficacy of Gene Therapy Products
8.4.1.6. Evaluation of Safety of Gene Therapy Products
8.4.1.7. Clinical Trial of Gene Therapy Products
8.4.1.8. Ethics Study

8.4.2. Japanese Scenario
8.4.3. South Korean Scenario
8.4.4. Australian Scenario
8.4.5. Hong Kong Scenario

8.5. Reimbursement Scenario
8.5.1. Challenges Related to Reimbursement

8.6. Concluding Remarks and Future Outlook

9. MARKET LANDSCAPE
9.1. Chapter Overview
9.2. Marketed and Clinical Stage Gene Therapies: Market Landscape
9.2.1. Analysis by Stage of Development
9.2.2. Analysis by Drug Designation
9.2.3. Analysis by Therapeutic Area
9.2.4. Analysis by Target Gene
9.2.5. Analysis by Type of Vector Used
9.2.6. Analysis by Type of Therapy
9.2.7. Analysis by Type of Gene Delivery Method
9.2.8. Analysis by Route of Administration
9.2.9. Analysis by Dosing Frequency

9.3. Preclinical and Discovery Stage Gene Therapies: Market Landscape
9.3.1. Analysis by Stage of Development
9.3.2. Analysis by Therapeutic Area
9.3.3. Analysis by Target Gene
9.3.4. Analysis by Type of Vector Used
9.3.5. Analysis by Type of Therapy
9.3.6. Analysis by Type of Gene Delivery Method

10. DEVELOPER LANDSCAPE
10.1. Chapter Overview
10.2. Gene Therapy: Competitive Landscape
10.2.1. Analysis by Year of Establishment
10.2.2. Analysis by Company Size
10.2.3. Analysis by Location of Headquarters

10.2.4. Most Active Players: Analysis by Number of Gene Therapies Developed

11. COMPANY PROFILES
11.1. Chapter Overview
11.2. Gene Therapy Developers in North America
11.2.1. Amgen
11.2.1.1. Company Overview
11.2.1.2. Gene Therapy Portfolio
11.2.1.3. Recent Developments and Future Outlook

11.2.2. BioMarin Pharmaceutical
11.2.2.1. Company Overview
11.2.2.2. Gene Therapy Portfolio
11.2.2.3. Recent Developments and Future Outlook

11.2.3. bluebird bio
11.2.3.1. Company Overview
11.2.3.2. Gene Therapy Portfolio
11.2.3.3. Recent Developments and Future Outlook

11.2.4. CRISPR Therapeutics
11.2.4.1. Company Overview
11.2.4.2. Gene Therapy Portfolio
11.2.4.3. Recent Developments and Future Outlook

11.2.5. Kolon TissueGene
11.2.5.1. Company Overview
11.2.5.2. Gene Therapy Portfolio
11.2.5.3. Recent Developments and Future Outlook

11.2.6. Krystal Biotech
11.2.6.1. Company Overview
11.2.6.2. Gene Therapy Portfolio
11.2.6.3. Recent Developments and Future Outlook

11.2.7. Pfizer
11.2.7.1. Company Overview
11.2.7.2. Gene Therapy Portfolio
11.2.7.3. Recent Developments and Future Outlook

11.2.8. PTC Therapeutics
11.2.8.1. Company Overview
11.2.8.2. Gene Therapy Portfolio
11.2.8.3. Recent Developments and Future Outlook

11.2.9. Sarepta Therapeutics
11.2.9.1. Company Overview
11.2.9.2. Gene Therapy Portfolio
11.2.9.3. Recent Developments and Future Outlook

11.2.10. Spark Therapeutics
11.2.10.1. Company Overview
11.2.10.2. Gene Therapy Portfolio
11.2.10.3. Recent Developments and Future Outlook

11.3. Gene Therapy Developers in Europe
11.3.1. Artgen Biotech
11.3.1.1. Company Overview
11.3.1.2. Gene Therapy Portfolio
11.3.1.3. Recent Developments and Future Outlook

11.3.2. Ferring Pharmaceuticals
11.3.2.1. Company Overview
11.3.2.2. Gene Therapy Portfolio
11.3.2.3. Recent Developments and Future Outlook

11.3.3. Novartis
11.3.3.1. Company Overview
11.3.3.2. Gene Therapy Portfolio
11.3.3.3. Recent Developments and Future Outlook

11.3.4. Orchard Therapeutics
11.3.4.1. Company Overview
11.3.4.2. Gene Therapy Portfolio
11.3.4.3. Recent Developments and Future Outlook

11.4. Gene Therapy Developers in Asia-Pacific and Rest of the World
11.4.1. CSL Behring
11.4.1.1. Company Overview
11.4.1.2. Gene Therapy Portfolio
11.4.1.3. Recent Developments and Future Outlook

11.4.2. Shanghai Sunway Biotech
11.4.2.1. Company Overview
11.4.2.2. Gene Therapy Portfolio
11.4.2.3. Recent Developments and Future Outlook

11.4.3. Sibiono GeneTech
11.4.3.1. Company Overview
11.4.3.2. Gene Therapy Portfolio
11.4.3.3. Recent Developments and Future Outlook

12. MARKETED GENE THERAPIES
12.1. Chapter Overview
12.2. Gendicine® (Shenzhen Sibiono GeneTech)
12.2.1. Company Overview
12.2.2. Development Timeline
12.2.3. Mechanism of Action and Vector Used
12.2.4. Target Indication
12.2.5. Current Stage of Development
12.2.6. Manufacturing, Dosage and Sales

12.3. Oncorine® (Shanghai Sunway Biotech)
12.3.1. Company Overview
12.3.2. Development Timeline
12.3.3. Mechanism of Action and Vector Used
12.3.4. Target Indication
12.3.5. Current Stage of Development
12.3.6. Manufacturing, Dosage and Sales

12.4. Rexin-G® (Epeius Biotechnologies)
12.4.1. Company Overview
12.4.2. Development Timeline
12.4.3. Mechanism of Action and Vector Used
12.4.4. Target Indication
12.4.5. Current Stage of Development
12.4.6. Manufacturing, Dosage and Sales

12.5. Neovasculgen® (Human Stem Cells Institute)
12.5.1. Company Overview
12.5.2. Development Timeline
12.5.3. Mechanism of Action and Vector Used
12.5.4. Target Indication
12.5.5. Current Stage of Development
12.5.6. Manufacturing, Dosage and Sales

12.6. Imlygic® (Amgen)
12.6.1. Company Overview
12.6.2. Development Timeline
12.6.3. Mechanism of Action and Vector Used
12.6.4. Target Indication
12.6.5. Current Stage of Development
12.6.6. Manufacturing, Dosage and Sales

12.7. Strimvelis® (Orchard Therapeutics)
12.7.1. Company Overview
12.7.2. Development Timeline
12.7.3. Mechanism of Action and Vector Used
12.7.4. Target Indication
12.7.5. Current Stage of Development
12.7.6. Manufacturing, Dosage and Sales

12.8. Luxturna™ (Spark Therapeutics)
12.8.1. Company Overview
12.8.2. Development Timeline
12.8.3. Mechanism of Action and Vector Used
12.8.4. Target Indication
12.8.5. Current Stage of Development
12.8.6. Manufacturing, Dosage and Sales

12.9. Zolgensa™ (Novartis)
12.9.1. Company Overview
12.9.2. Development Timeline
12.9.3. Mechanism of Action and Vector Used
12.9.4. Target Indication
12.9.5. Current Stage of Development
12.9.6. Manufacturing, Dosage and Sales

12.10. Collategene® (AnGes)
12.10.1. Company Overview
12.10.2. Development Timeline
12.10.3. Mechanism of Action and Vector Used
12.10.4. Target Indication
12.10.5. Current Stage of Development
12.10.6. Manufacturing, Dosage and Sales

12.11. Zyntelgo™ (bluebird bio)
12.11.1. Company Overview
12.11.2. Development Timeline
12.11.3. Mechanism of Action and Vector Used
12.11.4. Target Indication
12.11.5. Current Stage of Development
12.11.6. Manufacturing, Dosage and Sales

12.12. Libmeldy™ (Orchard Therapeutics)
12.12.1. Company Overview
12.12.2. Development Timeline
12.12.3. Mechanism of Action and Vector Used
12.12.4. Target Indication
12.12.5. Current Stage of Development
12.12.6. Manufacturing, Dosage and Sales

13. KEY COMMERCIALIZATION STRATEGIES
13.1. Chapter Overview
13.2. Successful Drug Launch Strategy: ROOTS Framework
13.3. Successful Drug Launch Strategy: Product Differentiation
13.4. Commonly Adopted Commercialization Strategies based on Phase of Development
13.5. List of Approved Gene Therapies

13.6. Key Commercialization Strategies Adopted by Gene Therapy Developers
13.6.1. Strategies Adopted before Therapy Approval
13.6.1.1. Participation in Global Events
13.6.1.2. Collaborations with Stakeholders and Pharmaceutical Firms
13.6.1.3. Indication Expansion

13.6.2. Strategies adopted During / Post Therapy Approval
13.6.2.1. Geographical Expansion
13.6.2.2. Participation in Global Events
13.6.2.3. Patient Assistance Programs
13.6.2.4. Awareness through Product Websites
13.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms

13.7. Concluding Remarks

14. LATE-STAGE GENE THERAPIES
14.1. Chapter Overview
14.2. LUMEVOQ (GS010)
14.3. OTL-103
14.4. PTC-AADC
14.5. BMN 270
14.6. rAd-IFN/Syn3
14.7. beti-cel
14.8. eli-cel
14.9. lovo-cel
14.10. SRP-9001
14.11. EB-101
14.12. ProstAtak
14.13. D-Fi
14.14. CG0070
14.15. Vigil-EWS
14.16. Engensis
14.17. VGX-3100
14.18. INVOSSA (TG-C)
14.19. VYJUVEKT
14.20. PF-06939926
14.21. PF06838435
14.22. PF-07055480
14.23. SPK-8011
14.24. AMT-061
14.25. VB-111
14.26. Generx
14.27. ADXS-HPV
14.28. AGTC 501
14.29. LYS-SAF302
14.30. NFS-01
14.31. AG0302-COVID-19
14.32. RGX-314
14.33. Hologene 5

15. PATENT ANALYSIS
15.1. Chapter Overview

15.2. Gene Therapy: Patent Analysis
15.2.1. Scope and Methodology
15.2.1.1. Analysis by Publication Year
15.2.1.2. Analysis by Publication Year and Type of Patent

15.2.1.3. Analysis by Geography
15.2.1.3.1. Analysis by Geography (North America)
15.2.1.3.2. Analysis by Geography (Europe)
15.2.1.3.3. Analysis by Geography (Asia-Pacific)

15.2.1.4. Analysis by CPC Symbols
15.2.1.5. Analysis by Emerging Focus Areas
15.2.1.6. Leading Players: Analysis by Number of Patents
15.2.1.7. Patent Benchmarking Analysis
15.2.1.7.1. Analysis by Patent Characteristics
15.2.1.7.1.1. Sanofi and Genethon
15.2.1.7.1.2. Other Leading Patent Assignees

15.2.1.8. Patent Valuation Analysis

15.3. Gene Editing Market: Patent Analysis
15.3.1. Scope and Methodology
15.3.1.1. Analysis by Publication Year
15.3.1.2. Analysis by Publication Year and Type of Patent

15.3.1.3. Analysis by Geography
15.3.1.3.1. Analysis by Geography (North America)
15.3.1.3.2. Analysis by Geography (Europe)
15.3.1.3.3. Analysis by Geography (Asia-Pacific)

15.3.1.4. Analysis by CPC Symbols
15.3.1.5. Analysis by Emerging Focus Areas
15.3.1.6. Leading Players: Analysis by Number of Patents

15.3.1.7. Patent Benchmarking Analysis
15.3.1.7.1. Analysis by Patent Characteristics
15.3.1.7.1.1. Intellia Therapeutics and CRISPR Therapeutics
15.3.1.7.1.2. Other Leading Patent Assignees

15.3.1.8. Patent Valuation Analysis

15.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization

16. MERGERS AND ACQUISITIONS
16.1. Chapter Overview
16.2. Merger and Acquisition Models

16.3. Gene Therapy: Mergers and Acquisitions
16.3.1. Analysis by Year of Merger / Acquisition
16.3.2. Analysis by Type of Agreement

16.3.3. Analysis by Geography
16.3.3.1. Intercontinental and Intracontinental Deals
16.3.3.2. Local and International Deals

16.3.4. Analysis by Key Value Drivers
16.3.4.1. Analysis by Key Value Drivers and Year of Acquisition

16.3.5. Analysis by Stage of Development of the Acquired Company’s Product
16.3.6. Analysis by Therapeutic Area

16.4. Analysis by Deal Multiples

17. FUNDING AND INVESTMENTS
17.1. Chapter Overview
17.2. Types of Funding

17.3. Gene Therapy: Funding and Investment Analysis
17.3.1. Analysis by Year of Funding
17.3.2. Analysis by Amount Invested
17.3.3. Analysis by Funding Instance and Amount Invested by Type of Funding
17.3.4. Analysis by Funding Instances and Amount Invested by Year and Type of Funding
17.3.5. Analysis by Funding Instances and Amount Invested by Type of Therapy
17.3.6. Analysis by Funding Instances and Amount Invested by Geography
17.3.7. Analysis by Funding Instances and Amount Invested by Highest Phase of Development
17.3.8. Analysis by Funding Instances by Therapeutic Area
17.3.9. Most Active Players: Analysis by Funding Instances and Amount Invested
17.3.10. Key Investors: Analysis by Number of Funding Instances

17.4. Concluding Remarks

18. CLINICAL TRIAL ANALYSIS
18.1. Chapter Overview
18.2. Scope and Methodology

18.3. Gene Therapy: Clinical Trial Analysis
18.3.1. Analysis by Trial Registration Year
18.3.2. Analysis by Trial Status
18.3.3. Analysis by Trial Phase
18.3.4. Analysis by Therapeutic Area
18.3.5. Analysis by Geography
18.3.6. Analysis by Trial Registration Year and Geography
18.3.7. Analysis by Trial Status and Geography
18.3.8. Analysis by Trial Status, Trial Phase and Geography
18.3.9. Analysis by Therapeutic Area and Geography

18.4. Analysis by Type of Sponsor / Collaborator
18.5. Analysis by Prominent Treatment Sites

18.6. Gene Therapy: Analysis of Enrolled Patient Population
18.6.1. Analysis by Trial Registration Year
18.6.2. Analysis by Trial Status
18.6.3. Analysis by Trial Phase
18.6.4. Analysis by Therapeutic Area
18.6.5. Analysis by Location of Trial Site
18.6.6. Analysis by Trial Status and Location of Trial Site
18.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
18.6.8. Analysis by Therapeutic Area and Location of Trial Site

18.7. Concluding Remarks

19. COST PRICE ANALYSIS
19.1. Chapter Overview

19.2. Factors Contributing to the High Price of Gene Therapies

19.3. Gene Therapy Market: Price Models
19.3.1. Based on Associated Product / Component Costs
19.3.2. Based on Competition
19.3.3. Based on Patient Segment
19.3.4. Based Opinion of Industry Experts

20. START-UP VALUATION
20.1. Chapter Overview
20.2. Valuation by Years of Experience of Start-ups
20.2.1. Methodology
20.2.2. Results and Interpretation

21. BIG PHARMA INITIATIVES
21.1. Chapter Overview

21.2. Gene Therapy: List of Most Prominent Big Pharmaceutical Players
21.2.1. Analysis by Therapeutic Area
21.2.2. Analysis by Type of Vector Used
21.2.3. Analysis by Type of Therapy
21.2.4. Analysis by Type of Gene Delivery Method Used

21.3. Benchmarking Analysis of Key Parameters
21.3.1. Spider Web Analysis: Pipeline Strength
21.3.2. Spider Web Analysis: Mergers and Acquisitions
21.3.3. Spider Web Analysis: Funding and Investments
21.3.4. Spider Web Analysis: Clinical Trials
21.3.5. Spider Web Analysis: Technologies
21.3.6. Spider Web Analysis: Patents

21.4. Benchmarking Analysis of Big Pharmaceutical Players
21.4.1. Spider Web Analysis: Novartis
21.4.2. Spider Web Analysis: Takeda Pharmaceutical
21.4.3. Spider Web Analysis: Roche
21.4.4. Spider Web Analysis: Merck
21.4.5. Spider Web Analysis: Pfizer
21.4.6. Spider Web Analysis: Johnson & Johnson
21.4.7. Spider Web Analysis: AbbVie
21.4.8. Spider Web Analysis: Bristol Myers Squibb
21.4.9. Spider Web Analysis: AstraZeneca
21.4.10. Spider Web Analysis: Sanofi

22. DEMAND ANALYSIS
22.1 Chapter Overview
22.2. Assumptions and Methodology

22.3. Gene Therapy Market: Global Demand Analysis
22.3.1. Analysis by Stage of Development
22.3.2. Analysis by Therapeutic Area
22.3.3. Analysis by Type of Therapy
22.3.4. Analysis by Type of Gene Delivery Method
22.3.5. Analysis by Geography

23. MARKET IMPACT ANALYSIS: DRIVERS, RESTRAINTS, OPPORTUNITIES AND CHALLENGES
23.1. Chapter Overview
23.2. Market Drivers
23.3. Market Restraints
23.4. Market Opportunities
23.5. Market Challenges
23.6. Conclusion

24. GLOBAL GENE THERAPY MARKET
24.1. Chapter Overview
24.2. Key Assumptions and Methodology

24.3. Global Gene Therapy Market, till 2035
24.3.1. Scenario Analysis
24.3.1.1. Conservative Scenario
24.3.1.2. Optimistic Scenario

24.4. Key Market Segmentations

25. GENE THERAPIES MARKET, BY THERAPEUTIC AREA
25.1. Chapter Overview
25.2. Key Assumptions and Methodology

25.3. Gene Therapy Market: Distribution by Therapeutic Area, 2024, 2029 and 2035
25.3.1. Gene Therapy Market for Muscle Disorders, till 2035
25.3.2. Gene Therapy Market for Hematological Disorders, till 2035
25.3.3. Gene Therapy Market for Dermatological Disorders, till 2035
25.3.4. Gene Therapy Market for Oncological Disorders, till 2035
25.3.5. Gene Therapy Market for Genetic Disorders, till 2035
25.3.6. Gene Therapy Market for Ophthalmic Disorders, till 2035
25.3.7. Gene Therapy Market for Metabolic Disorders, till 2035
25.3.8. Gene Therapy Market for Cardiovascular Disorders, till 2035
25.3.9. Gene Therapy Market for Other Disorders, till 2035

25.4. Data Triangulation and Validation

26. GENE THERAPIES MARKET, BY TYPE OF VECTOR
26.1. Chapter Overview
26.2. Key Assumptions and Methodology

26.3. Gene Therapy Market: Distribution by Type of Vector, 2024, 2029 and 2035
26.3.1. Gene Therapy Market for Adeno-associated Virus Vectors, till 2035
26.3.2. Gene Therapy Market for Herpes Simplex Virus Vectors, till 2035
26.3.3. Gene Therapy Market for Lentivirus Vectors, till 2035
26.3.4. Gene Therapy Market for Adenovirus Vectors, till 2035
26.3.5. Gene Therapy Market for Non-viral Vectors, till 2035
26.3.6. Gene Therapy Market for Retrovirus Vectors, till 2035
26.3.7. Gene Therapy Market for Other Viral Vectors, till 2035

26.4. Data Triangulation and Validation

27. GENE THERAPIES MARKET, BY TYPE OF THERAPY
27.1. Chapter Overview
27.2. Key Assumptions and Methodology

27.3. Gene Therapy Market: Distribution by Type of Therapy, 2024, 2029 and 2035
27.3.1. Gene Therapy Market for Gene Augmentation, till 2035
27.3.2. Gene Therapy Market for Oncolytic Immunotherapies, till 2035
27.3.3. Gene Therapy Market for Gene Editing, till 2035
27.3.4. Gene Therapy Market for Gene Regulation, till 2035
27.3.5. Gene Therapy Market for Other Therapies, till 2035

27.4. Data Triangulation and Validation

28. GENE THERAPIES MARKET, BY TYPE OF GENE DELIVERY METHOD
28.1. Chapter Overview
28.2. Key Assumptions and Methodology

28.3. Gene Therapy Market: Distribution by Type of Gene Delivery Method, 2024, 2029 and 2035
28.3.1. Gene Therapy Market for In vivo Gene Delivery, till 2035
28.3.2. Gene Therapy Market for Ex vivo Gene Delivery, till 2035

28.4. Data Triangulation and Validation

29. GENE THERAPIES MARKET, BY ROUTE OF ADMINISTRATION
29.1. Chapter Overview
29.2. Key Assumptions and Methodology

29.3. Gene Therapy Market: Distribution by Route of Administration, 2024, 2029 and 2035
29.3.1. Gene Therapy Market for Intravenous Route, till 2035
29.3.2. Gene Therapy Market for Subretinal Route, till 2035
29.3.3. Gene Therapy Market for Intratumoral Route, till 2035
29.3.4. Gene Therapy Market for Intramuscular Route, till 2035
29.3.5. Gene Therapy Market for Other Routes of Administration, till 2035

29.4. Data Triangulation and Validation

30. GENE THERAPIES MARKET, BY GEOGRAPHICAL REGIONS
30.1. Chapter Overview
30.2. Key Assumptions and Methodology

30.3. Gene Therapy Market: Distribution by Geographical Regions, 2024, 2029 and 2035
30.3.1. Gene Therapy Market in North America, till 2035
30.3.2. Gene Therapy Market in Europe, till 2035
30.3.3. Gene Therapy Market in Asia-Pacific, till 2035
30.3.4. Gene Therapy Market in Latin America, till 2035
30.3.5. Gene Therapy Market in Rest of the World, till 2035

30.4. Data Triangulation and Validation

31. GENE THERAPIES MARKET, BY LEADING PLAYERS
31.1. Chapter Overview
31.2. Gene Therapy Market: Distribution by Leading Gene Therapy Developers
31.3. Data Triangulation and Validation

32. GENE THERAPIES MARKET, SALES FORECAST OF THERAPIES
32.1. Chapter Overview
32.2. Key Assumptions and Methodology

32.3. Commercialized Gene Therapies Market: Sales Forecast
32.3.1. Adstiladrin®
32.3.1.1. Sales Forecast
32.3.1.2. Net Present Value
32.3.1.3. Value Creation Analysis

32.3.2. BEQVEZ™
32.3.2.1. Sales Forecast
32.3.2.2. Net Present Value
32.3.2.3. Value Creation Analysis

32.3.3. Casgevy™
32.3.3.1. Sales Forecast
32.3.3.2. Net Present Value
32.3.3.3. Value Creation Analysis

32.3.4. Elevidys®
32.3.4.1. Sales Forecast
32.3.4.2. Net Present Value
32.3.4.3. Value Creation Analysis

32.3.5. Gendicine®
32.3.5.1. Sales Forecast
32.3.5.2. Net Present Value
32.3.5.3. Value Creation Analysis

32.3.6. Hemgenix®
32.3.6.1. Sales Forecast
32.3.6.2. Net Present Value
32.3.6.3. Value Creation Analysis

32.3.7. IMLYGIC®
32.3.7.1. Sales Forecast
32.3.7.2. Net Present Value
32.3.7.3. Value Creation Analysis

32.3.8. Libmeldy®
32.3.8.1. Sales Forecast
32.3.8.2. Net Present Value
32.3.8.3. Value Creation Analysis

32.3.9. Luxturna®
32.3.9.1. Sales Forecast
32.3.9.2. Net Present Value
32.3.9.3. Value Creation Analysis

32.3.10. LYFGENIA™
32.3.10.1. Sales Forecast
32.3.10.2. Net Present Value
32.3.10.3. Value Creation Analysis

32.3.11. Neovasculgen®
32.3.11.1. Sales Forecast
32.3.11.2. Net Present Value
32.3.11.3. Value Creation Analysis

32.3.12. Oncorine®
32.3.12.1. Sales Forecast
32.3.12.2. Net Present Value
32.3.12.3. Value Creation Analysis

32.3.13. Roctavian™
32.3.13.1. Sales Forecast
32.3.13.2. Net Present Value
32.3.13.3. Value Creation Analysis

32.3.14. SKYSONA®
32.3.14.1. Sales Forecast
32.3.14.2. Net Present Value
32.3.14.3. Value Creation Analysis

32.3.15. Strimvelis™
32.3.15.1. Sales Forecast
32.3.15.2. Net Present Value
32.3.15.3. Value Creation Analysis

32.3.16. Upstaza™
32.3.16.1. Sales Forecast
32.3.16.2. Net Present Value
32.3.16.3. Value Creation Analysis

32.3.17. VYJUVEKT®
32.3.17.1. Sales Forecast
32.3.17.2. Net Present Value
32.3.17.3. Value Creation Analysis

32.3.18. Zolgensma®
32.3.18.1. Sales Forecast
32.3.18.2. Net Present Value
32.3.18.3. Value Creation Analysis

32.3.19. Zynteglo™
32.3.19.1. Sales Forecast
32.3.19.2. Net Present Value
32.3.19.3. Value Creation Analysis

32.4. Phase III Gene Therapies Market: Sales Forecast
32.4.1. AAV-RPE65
32.4.1.1. Sales Forecast
32.4.1.2. Net Present Value
32.4.1.3. Value Creation Analysis

32.4.2. ABO-102 (UX111)
32.4.2.1. Sales Forecast
32.4.2.2. Net Present Value
32.4.2.3. Value Creation Analysis

32.4.3. AGTC 501 (rAAV2tYF-GRK1-RPGR)
32.4.3.1. Sales Forecast
32.4.3.2. Net Present Value
32.4.3.3. Value Creation Analysis

32.4.4. Cretostimogene Grenadenorepvec (CG0070)
32.4.4.1. Sales Forecast
32.4.4.2. Net Present Value
32.4.4.3. Value Creation Analysis

32.4.5. Dirloctocogene samoparvovec (SPK-8011)
32.4.5.1. Sales Forecast
32.4.5.2. Net Present Value
32.4.5.3. Value Creation Analysis

32.4.6. DTx-301
32.4.6.1. Sales Forecast
32.4.6.2. Net Present Value
32.4.6.3. Value Creation Analysis

32.4.7. DTx-401
32.4.7.1. Sales Forecast
32.4.7.2. Net Present Value
32.4.7.3. Value Creation Analysis

32.4.8. Generx® (Ad5FGF-4)
32.4.8.1. Sales Forecast
32.4.8.2. Net Present Value
32.4.8.3. Value Creation Analysis

32.4.9. Giroctocogene fitelparvovec (SB-525 / PF-07055480)
32.4.9.1. Sales Forecast
32.4.9.2. Net Present Value
32.4.9.3. Value Creation Analysis

32.4.10. Hologene 5
32.4.10.1. Sales Forecast
32.4.10.2. Net Present Value
32.4.10.3. Value Creation Analysis

32.4.11. INVOSSA™ (TG-C)
32.4.11.1. Sales Forecast
32.4.11.2. Net Present Value
32.4.11.3. Value Creation Analysis

32.4.12. LUMEVOQ®
32.4.12.1. Sales Forecast
32.4.12.2. Net Present Value
32.4.12.3. Value Creation Analysis

32.4.13. NFS-01 (rAAV2-ND4)
32.4.13.1. Sales Forecast
32.4.13.2. Net Present Value
32.4.13.3. Value Creation Analysis

32.4.14. NTLA-2001
32.4.14.1. Sales Forecast
32.4.14.2. Net Present Value
32.4.14.3. Value Creation Analysis

32.4.15. Olvi-Vec (Olvimulogene nanivacirepvec)
32.4.15.1. Sales Forecast
32.4.15.2. Net Present Value
32.4.15.3. Value Creation Analysis

32.4.16. ProstAtak®
32.4.16.1. Sales Forecast
32.4.16.2. Net Present Value
32.4.16.3. Value Creation Analysis

32.4.17. pz-cel (prademagene zamikeracel / EB-101)
32.4.17.1. Sales Forecast
32.4.17.2. Net Present Value
32.4.17.3. Value Creation Analysis

32.4.18. RGX-121
32.4.18.1. Sales Forecast
32.4.18.2. Net Present Value
32.4.18.3. Value Creation Analysis

32.4.19. RGX-314
32.4.19.1. Sales Forecast
32.4.19.2. Net Present Value
32.4.19.3. Value Creation Analysis

32.4.20. VGX-3100
32.4.20.1. Sales Forecast
32.4.20.2. Net Present Value
32.4.20.3. Value Creation Analysis

33. GENE THERAPY MARKET: EMERGING TECHNOLOGIES
33.1. Chapter Overview
33.2. Gene Editing Technologies
33.2.1. Applications of Gene Editing

33.3. Emerging Gene Editing Platforms
33.3.1. CRISPR / Cas9 System
33.3.1.1. Technology Providers

33.3.2. Zinc Finger Nuclease
33.3.2.1. Technology Providers

33.3.3. TALENs

33.3.4. megaTAL
33.3.4.1. Technology Providers

33.4. Gene Therapy Regulation Technologies (Gene Switch Technology)
33.4.1. Technology Providers

33.5. Other Emerging Technologies
33.6. Technology Platforms for Developing / Delivering Gene Therapies

33.7. Concluding Remarks

34. VECTOR MANUFACTURING FOR GENE THERAPIES
34.1. Chapter Overview
34.2. Viral Vector Manufacturing Process
34.2.1. Mode of Vector Production
34.2.2. Adherent and Suspension Cultures
34.2.3. Unit Processes and Multiple Parallel Processes

34.2.4. Cell Culture Systems for Production of Viral Vectors
34.2.4.1. Small-Scale / Laboratory Scale Cell Culture Systems

34.2.4.2. Large-Scale Cell Culture Systems
34.2.4.2.1. Stirred Tank Reactor Systems
34.2.4.2.2. Fixed Bed Reactor / Packed Bed Reactor
34.2.4.2.3. WAVE Bioreactor System

34.2.5. Culture Media Specifications

34.3. Bioprocessing of Viral Vectors
34.3.1. AAV Vector Production
34.3.2. Adenoviral Vector Production
34.3.3. Lentiviral Vector Production
34.3.4. ? - Retroviral Vector Production

34.4. Challenges Associated with Vector Manufacturing
34.5. Contract Manufacturing Organizations Involved in Viral Vector Production

35. CASE STUDY: GENE THERAPY SUPPLY CHAIN
35.1. Chapter Overview

35.2. Overview of Gene Therapy Supply Chain
35.3. Implementation of Supply Chain Models

35.4. Logistics in Gene Therapy
35.4.1. Logistic Processes for Autologous and Allogenic Therapies

35.5. Regulatory Supply Chain Across the Globe
35.6. Challenges Associated with Gene Therapy Supply Chain

35.7. Software Applications for Gene Therapy Supply Chain Management
35.7.1. Enterprise Manufacturing System
35.7.2. Laboratory Information Management System
35.7.3. Inventory Management System
35.7.4. Quality Management System
35.7.5. Logistics Management System
35.7.6. Patient Management System
35.7.7. Electronic Clinical Outcome Assessments System
35.7.8. Supply Chain Orchestration Platform

35.8. Recent Developments and Upcoming Trends

36. EXECUTIVE INSIGHTS
36.1. Chapter Overview

36.2. Orchard Therapeutics
36.2.1. Company Snapshot
36.2.2. Interview Transcript: Molly Cameron, Former Corporate Communications Manager

36.3. Vivet Therapeutics
36.3.1. Company Snapshot
36.3.2. Interview Transcript: Jean-Phillipe Combal, Co-Founder and Chief Executive Officer

36.4. Kubota Pharmaceutical
36.4.1. Company Snapshot
36.4.2. Interview Transcript: Ryo Kubota, Chief Executive Officer, Chairman and President

36.5. Eyevensys
36.5.1. Company Snapshot
36.5.2. Interview Transcript: Patricia Zilliox, Former Chief Executive Officer and President

36.6. AGTC
36.6.1. Company Snapshot
36.6.2. Interview Transcript: Sue Washer, Former Chief Executive Officer and President

36.7. LogicBio Therapeutics
36.7.1. Company Snapshot
36.7.2. Interview Transcript: Tom Wilton, Former Chief Business Officer

36.8. AAVogen
36.8.1. Company Snapshot
36.8.2. Interview Transcript: Buel Dan Rodgers, Founder and Chief Executive Officer

36.9. Hemera Biosciences
36.9.1. Company Snapshot
36.9.2. Interview Transcript: Adam Rogers, Chief Executive Officer

36.10. Myonexus Therapeutics
36.10.1. Company Snapshot
36.10.2. Interview Transcript: Michael Tripletti, Ex Co-Founder, Chief Executive Officer and President

36.11. Arthrogen
36.11.1. Company Snapshot
36.11.2. Interview Transcript: Robert Jan Lamers, Former Chief Executive Officer

36.12. PlasmidFactory
36.12.1. Company Snapshot
36.12.2. Interview Transcript: Marco Schmeer, Project Manager and Tatjana Buchholz, Former Marketing Manager

36.13. Delphi Genetics
36.13.1. Company Snapshot
36.13.2. Interview Transcript: Cedric Szpirer, Former Executive and Scientific Director

36.14. Vigene Biosciences
36.14.1. Company Snapshot
36.14.2. Interview Transcript: Jeffrey Hung, Former Chief Commercial Officer

36.15. Gene Biotherapeutics
36.15.1. Company Snapshot
36.15.2. Interview Transcript: Christopher Reinhard, Chief Executive Officer and Chairman

36.16. Milo Biotechnology
36.16.1. Company Snapshot
36.16.2. Interview Transcript: AL Hawkins, Chief Executive Officer

37. CONCLUSION38. APPENDIX I: TABULATED DATA39. APPENDIX II: LIST OF COMPANIES AND ORGANIZATIONS
LIST OF FIGURES
Figure 2.1 Research Methodology: Project Methodology
Figure 2.2 Research Methodology: Data Sources for Secondary Research
Figure 3.1 Market Dynamics: Forecast Methodology
Figure 3.2 Market Dynamics: Key Market Segmentation
Figure 3.3 Market Dynamics: Robust Quality Control
Figure 4.1 Lesson Learnt from Past Recessions
Figure 5.1 Executive Summary: Market Landscape
Figure 5.2 Executive Summary: Developer Landscape
Figure 5.3 Executive Summary: Patent Analysis
Figure 5.4 Executive Summary: Mergers and Acquisitions
Figure 5.5 Executive Summary: Funding and Investment Analysis
Figure 5.6 Executive Summary: Clinical Trial Analysis
Figure 5.7 Executive Summary: Demand Analysis
Figure 5.8 Executive Summary: Market Forecast and Opportunity Analysis
Figure 6.1 Evolution of Gene Therapies
Figure 6.2 Ex vivo Gene Therapies
Figure 6.3 In vivo Gene Therapies
Figure 6.4 Mechanism of Action of Gene Therapies
Figure 6.5 Genome Editing: Historical Milestones
Figure 6.6 Types of Genome Editing Techniques
Figure 7.1 Viral and Non-Viral Vectors Used in Gene Therapy
Figure 8.1 Centralized Regulatory Pathway for Market Approval in the US
Figure 8.2 Centralized Regulatory Pathway for Market Approval in Europe
Figure 8.3 Manufacturing Requirements for Gene Therapy Products in China
Figure 8.4 Key Elements of Quality Control in China
Figure 8.5 Centralized Regulatory Pathway for Market Approval in Japan
Figure 9.1 Marketed and Clinical Stage Gene Therapies: Distribution by Stage of Development
Figure 9.2 Marketed and Clinical Stage Gene Therapies: Distribution by Drug Designation
Figure 9.3 Marketed and Clinical Stage Gene Therapies: Distribution by Therapeutic Area
Figure 9.4 Marketed and Clinical Stage Gene Therapies: Distribution by Biological Target
Figure 9.5 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Vector Used
Figure 9.6 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Therapy
Figure 9.7 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Gene Delivery Method
Figure 9.8 Marketed and Clinical Stage Gene Therapies: Distribution by Route of Administration
Figure 9.9 Marketed and Clinical Stage Gene Therapies: Distribution by Dosage
Figure 9.10 Preclinical and Discovery Stage Gene Therapies: Distribution by Stage of Development
Figure 9.11 Preclinical and Discovery Stage Gene Therapies: Distribution by Therapeutic Area
Figure 9.12 Preclinical and Discovery Stage Gene Therapies: Distribution by Biological Target
Figure 9.13 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Vector Used
Figure 9.14 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Therapy
Figure 9.15 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Gene Delivery Method
Figure 10.1 Gene Therapy Developers: Distribution by Year of Establishment
Figure 10.2 Gene Therapy Developers: Distribution by Company Size
Figure 10.3 Gene Therapy Developers: Distribution by Location of Headquarters
Figure 10.4 Most Active Players: Distribution by Number of Gene Therapies
Figure 12.1 Gendicine: Development Timeline
Figure 12.2 Oncorine: Development Timeline
Figure 12.3 Oncorine: Mechanism of Action
Figure 12.4 Rexin-G: Development Timeline
Figure 12.5 Neovasculgen: Development Timeline
Figure 12.6 Imlygic: Development Timeline
Figure 12.7 Imlygic: Mechanism of Action
Figure 12.9 Luxturna: Development Timeline
Figure 12.10 Zolgensma: Development Timeline
Figure 12.11 Collategene: Development Timeline
Figure 12.12 Zynteglo: Development Timeline
Figure 12.13 Libmeldy: Development Timeline
Figure 13.1 Successful Drug Launch Strategy: ROOTS Framework
Figure 13.2 Successful Drug Launch Strategy: Product Differentiation
Figure 13.3 Commonly Adopted Commercialization Strategies based on Phase of Development of Product
Figure 13.4 Harvey Ball Analysis: Commercialization Strategies Adopted by Gene Therapy Developers
Figure 13.5 Approved Gene Therapies: Historical Timeline of Geographical Expansion
Figure 13.6 Approved Gene Therapies: Snapshot of Promotional Activities on Product Websites
Figure 13.7 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
Figure 15.1 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Figure 15.2 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Figure 15.3 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 15.4 Gene Therapy Patent Portfolio: Distribution by Geography
Figure 15.5 Gene Therapy Patent Portfolio: North America Scenario
Figure 15.6 Gene Therapy Patent Portfolio: Europe Scenario
Figure 15.7 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
Figure 15.8 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
Figure 15.9 Gene Therapy Patent Portfolio: Emerging Focus Areas
Figure 15.10 Leading Industry Players (Gene Therapy): Distribution by Number of Patents
Figure 15.11 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
Figure 15.12 Gene Therapy Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 15.13 Gene Therapy Patent Portfolio: Distribution by Patent Age (2017-2022)
Figure 15.14 Gene Therapy: Patent Valuation Analysis
Figure 15.15 Gene Editing Patent Portfolio: Distribution by Type of Patent
Figure 15.16 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Figure 15.17 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 15.18 Gene Editing Patent Portfolio: Distribution by Geography
Figure 15.19 Gene Editing Patent Portfolio: North America Scenario
Figure 15.20 Gene Editing Patent Portfolio: Europe Scenario
Figure 15.21 Gene Editing Patent Portfolio: Asia-Pacific Scenario
Figure 15.22 Gene Editing Patent Portfolio: Distribution by CPC Symbols
Figure 15.23 Gene Editing Patent Portfolio: Emerging Focus Areas
Figure 15.24 Leading Industry Players (Gene Editing): Distribution by Number of Patents
Figure 15.25 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
Figure 15.26 Gene Editing Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 15.27 Gene Editing Patent Portfolio: Distribution by Patent Age (2017-2022)
Figure 15.28 Gene Editing: Patent Valuation Analysis
Figure 15.29 Gene Therapy Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 15.30 Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 16.1 Mergers and Acquisitions: Cumulative Year-Wise Trend (2015-2022)
Figure 16.2 Mergers and Acquisitions: Distribution by Type of Agreement
Figure 16.3 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Figure 16.5 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
Figure 16.6 Mergers and Acquisitions: Local and International Deals
Figure 16.7 Acquisitions: Distribution by Key Value Drivers
Figure 16.8 Acquisitions: Distribution by Key Value Drivers and Year of Acquisition
Figure 16.9 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Figure 16.10 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product and Year of Acquisition
Figure 16.11 Mergers and Acquisitions: Distribution of Deal Amount by Phase of Development of the Acquired Company’s Product
Figure 16.12 Mergers and Acquisitions: Distribution by Therapeutic Area
Figure 17.1 Funding and Investments: Cumulative Year-wise Trend, 2015-2022
Figure 17.2 Funding and Investments: Cumulative Distribution by Amount Invested, 2015-2022 (USD Million)
Figure 17.3 Funding and Investments: Distribution of Instances by Type of Funding, 2015-2022
Figure 17.4 Funding and Investments: Distribution of Total Amount Invested by Type of Funding, 2015-2022 (USD Million)
Figure 17.5 Funding and Investments: Distribution of Instances by Year and Type of Funding, 2015-2022
Figure 17.8 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
Figure 17.9 Funding and Investments: Distribution of Instances and Amount Invested by Geography
Figure 17.10 Funding and Investments: Regional Distribution of Funding Instances
Figure 17.11 Funding and Investments: Distribution of Funding Instances, Amount Invested and Phase of Development
Figure 17.12 Funding and Investments: Distribution of Instances by Therapeutic Area
Figure 17.13 Most Active Players: Distribution by Number of Funding Instances, 2015-2022
Figure 17.14 Most Active Players: Distribution by Amount Invested, 2015-2022 (USD Million)
Figure 17.15 Funding and Investments: Distribution of Instances by Type of Investors
Figure 17.16 Leading Investors: Distribution by Number of Instances
Figure 17.17 Funding and Investment Summary, 2015-2022 (USD Million)
Figure 18.1 Clinical Trial Analysis: Cumulative Distribution by Trial Registration Year, 2017-2022
Figure 18.2 Clinical Trial Analysis: Distribution by Trial Status
Figure 18.3 Clinical Trial Analysis: Distribution by Trial Phase
Figure 18.4 Clinical Trial Analysis: Distribution by Therapeutic Area
Figure 18.5 Clinical Trial Analysis: Distribution by Geography
Figure 18.6 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
Figure 18.7 Clinical Trial Analysis: Distribution by Trial Status and Geography
Figure 18.8 Clinical Trial Analysis: Distribution by Trial Status, Trial Phase and Geography
Figure 18.9 Clinical Trial Analysis: Distribution by Therapeutic Area and Geography
Figure 18.10 Clinical Trial Analysis: Distribution by Type of Sponsor
Figure 18.11 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Figure 18.12 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, 2017-2022
Figure 18.13 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Figure 18.14 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Figure 18.15 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Figure 18.16 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
Figure 18.17 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
Figure 18.18 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status, Trial Phase and Location of Trial Site
Figure 18.19 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area and Location of Trial Site
Figure 19.1 Gene Therapy: Pricing Model Based on Patient Segment
Figure 20.1 Start-up Valuation: Valuation based on Experience of the Gene Therapy Developer
Figure 21.1 Big Pharma Players: Top Pharmaceutical Companies (Heat Map Representation)
Figure 21.2 Big Pharma Players: Analysis by Therapeutic Area
Figure 21.3 Big Pharma Players: Analysis by Type of Vector Used
Figure 21.4 Big Pharma Players: Analysis by Type of Therapy
Figure 21.5 Big Pharma Players: Analysis by Type of Gene Delivery Method Used
Figure 21.6 Spider Web Analysis: Pipeline Strength
Figure 21.7 Spider Web Analysis: Mergers and Acquisitions
Figure 21.8 Spider Web Analysis: Funding and Investments
Figure 21.9 Spider Web Analysis: Clinical Trials
Figure 21.10 Spider Web Analysis: Technologies
Figure 21.11 Spider Web Analysis: Patents
Figure 21.12 Spider Web Analysis: Novartis
Figure 21.13 Spider Web Analysis: Takeda Pharmaceutical
Figure 21.14 Spider Web Analysis: Roche
Figure 21.15 Spider Web Analysis: Merck
Figure 21.16 Spider Web Analysis: Pfizer
Figure 21.17 Spider Web Analysis: Johnson & Johnson
Figure 21.18 Spider Web Analysis: AbbVie
Figure 21.19 Spider Web Analysis: Bristol Myers Squibb
Figure 21.20 Spider Web Analysis: AstraZeneca
Figure 21.21 Spider Web Analysis: Sanofi
Figure 22.1 Global Demand for Gene Therapies, till 2035 (in terms of number of patients)
Figure 22.2 Global Demand for Gene Therapies: Distribution by Stage of Development (in terms of number of patients)
Figure 22.3 Global Demand for Gene Therapies: Distribution by Therapeutic Area (in terms of number of patients)
Figure 22.4 Global Demand for Gene Therapies: Distribution by Type of Therapy (in terms of number of patients)
Figure 22.5 Global Demand for Gene Therapies: Distribution by Type of Gene Delivery Method (in terms of number of patients)
Figure 22.6 Global Demand for Gene Therapies: Distribution by Geography (in terms of Number of Patients)
Figure 23.1 Gene Therapy: Market Drivers
Figure 23.2 Gene Therapy: Market Restraints
Figure 23.3 Gene Therapy: Market Opportunities
Figure 23.4 Gene Therapy: Market Challenges
Figure 24.1 Global Gene Therapy Market, till 2035 (USD Billion)
Figure 24.2 Global Gene Therapy Market, till 2035: Conservative Scenario (USD Billion)
Figure 24.3 Global Gene Therapy Market, till 2035: Optimistic Scenario (USD Billion)
Figure 25.1 Gene Therapy Market: Distribution by Therapeutic Area, 2024, 2029 and 2035
Figure 25.2 Gene Therapy Market for Muscle Disorders, till 2035 (USD Billion)
Figure 25.3 Gene Therapy Market for Hematological Disorders, till 2035 (USD Billion)
Figure 25.4 Gene Therapy Market for Dermatological Disorders, till 2035 (USD Billion)
Figure 25.5 Gene Therapy Market for Oncological Disorders, till 2035 (USD Billion)
Figure 25.6 Gene Therapy Market for Genetic Disorders, till 2035 (USD Billion)
Figure 25.7 Gene Therapy Market for Ophthalmic Disorders, till 2035 (USD Billion)
Figure 25.8 Gene Therapy Market for Metabolic Disorders, till 2035 (USD Billion)
Figure 25.9 Gene Therapy Market for Cardiovascular Disorders, till 2035 (USD Billion)
Figure 25.10 Gene Therapy Market for Other Disorders, till 2035 (USD Billion)
Figure 26.1 Gene Therapy Market: Distribution by Type of Vector, 2024, 2029 and 2035
Figure 26.2 Gene Therapy Market for Adeno-associated Virus Vectors, till 2035 (USD Billion)
Figure 26.3 Gene Therapy Market for Herpes Simplex Virus Vectors, till 2035 (USD Billion)
Figure 26.4 Gene Therapy Market for Lentivirus Vectors, till 2035 (USD Billion)
Figure 26.5 Gene Therapy Market for Adenovirus Vectors, till 2035 (USD Billion)
Figure 26.6 Gene Therapy Market for Non-viral Vectors, till 2035 (USD Billion)
Figure 26.7 Gene Therapy Market for Retrovirus Vectors, till 2035 (USD Billion)
Figure 26.8 Gene Therapy Market for Other Viral Vectors, till 2035 (USD Billion)
Figure 27.1 Gene Therapy Market: Distribution by Type of Therapy, 2024, 2029 and 2035
Figure 27.2 Gene Therapy Market for Gene Augmentation, till 2035 (USD Billion)
Figure 27.3 Gene Therapy Market for Oncolytic Immunotherapies, till 2035 (USD Billion)
Figure 27.4 Gene Therapy Market for Gene Editing, till 2035 (USD Billion)
Figure 27.5 Gene Therapy Market for Gene Regulation, till 2035 (USD Billion)
Figure 27.6 Gene Therapy Market for Other Therapies, till 2035 (USD Billion)
Figure 28.1 Gene Therapy Market: Distribution by Type of Gene Delivery Method, 2024, 2029 and 2035
Figure 28.2 Gene Therapy Market for In vivo Gene Delivery, till 2035 (USD Billion)
Figure 28.3 Gene Therapy Market for Ex vivo Gene Delivery, till 2035 (USD Billion)
Figure 29.1 Gene Therapy Market: Distribution by Route of Administration, 2024, 2029 and 2035
Figure 29.2 Gene Therapy Market for Intravenous Route, till 2035 (USD Billion)
Figure 29.3 Gene Therapy Market for Subretinal Route, till 2035 (USD Billion)
Figure 29.4 Gene Therapy Market for Intratumoral Route, till 2035 (USD Billion)
Figure 29.5 Gene Therapy Market for Intramuscular Route, till 2035 (USD Billion)
Figure 29.6 Gene Therapy Market for Other Routes of Administration, till 2035 (USD Billion)
Figure 30.1 Gene Therapy Market: Distribution by Geographical Regions, 2024, 2029 and 2035
Figure 30.2 Gene Therapy Market in North America, till 2035 (USD Billion)
Figure 30.3 Gene Therapy Market in Europe, till 2035 (USD Billion)
Figure 30.4 Gene Therapy Market in Asia-Pacific, till 2035 (USD Billion)
Figure 30.5 Gene Therapy Market in Latin America, till 2035 (USD Billion)
Figure 30.6 Gene Therapy Market in Rest of the World, till 2035 (USD Billion)
Figure 31.1 Gene Therapy Market: Distribution by Leading Gene Therapy Developers (based on sales of gene therapies in the year 2024 (USD Billion))
Figure 31.2 Gene Therapy Market: Distribution by Leading Gene Therapy Developers (based on the forecasted sales of gene therapies in the year 2035 (USD Billion))
Figure 32.1 Gene Therapy Market: Adstiladrin Sales Forecast, till 2035 (USD Million)
Figure 32.2 Gene Therapy Market: BEQVEZ Sales Forecast, till 2035 (USD Million)
Figure 32.3 Gene Therapy Market: Casgevy Sales Forecast, till 2035 (USD Million)
Figure 32.4 Gene Therapy Market: Elevidys Sales Forecast, till 2035 (USD Million)
Figure 32.5 Gene Therapy Market: Gendicine Sales Forecast, till 2035 (USD Million)
Figure 32.6 Gene Therapy Market: Hemgenix Sales Forecast, till 2035 (USD Million)
Figure 32.7 Gene Therapy Market: Imlygic Sales Forecast, till 2035 (USD Million)
Figure 32.8 Gene Therapy Market: Libmeldy Sales Forecast, till 2035 (USD Million)
Figure 32.9 Gene Therapy Market: Luxturna Sales Forecast, till 2035 (USD Million)
Figure 32.10 Gene Therapy Market: LYFGENIA Sales Forecast, till 2035 (USD Million)
Figure 32.11 Gene Therapy Market: Neovasculgen Sales Forecast, till 2035 (USD Million)
Figure 32.12 Gene Therapy Market: Oncorine Sales Forecast, till 2035 (USD Million)
Figure 32.13 Gene Therapy Market: Roctavian Sales Forecast, till 2035 (USD Million)
Figure 32.14 Gene Therapy Market: SKYSONA Sales Forecast, till 2035 (USD Million)
Figure 32.15 Gene Therapy Market: Strimvelis Sales Forecast, till 2035 (USD Million)
Figure 32.16 Gene Therapy Market: Upstaza Sales Forecast, till 2035 (USD Million)
Figure 32.17 Gene Therapy Market: VYJUVEKT Sales Forecast, till 2035 (USD Million)
Figure 32.18 Gene Therapy Market: Zolgensma Sales Forecast, till 2035 (USD Million)
Figure 32.19 Gene Therapy Market: Zynteglo Sales Forecast, till 2035 (USD Million)
Figure 32.20 Gene Therapy Market: AAV-RPE65 Sales Forecast, till 2035 (USD Million)
Figure 32.21 Gene Therapy Market: ABO-102 (UX111) Sales Forecast, till 2035 (USD Million)
Figure 32.22 Gene Therapy Market: AGTC 501 (rAAV2tYF-GRK1-RPGR) Sales Forecast, till 2035 (USD Million)
Figure 32.23 Gene Therapy Market: Crestostimogene Grenadenorepvec (CG0070) Sales Forecast, till 2035 (USD Million)
Figure 32.24 Gene Therapy Market: Dirloctocogene samoparvovec (SPK-8011) Sales Forecast, till 2035 (USD Million)
Figure 32.25 Gene Therapy Market: DTx-301 Sales Forecast, till 2035 (USD Million)
Figure 32.26 Gene Therapy Market: DTx-401 Sales Forecast, till 2035 (USD Million)
Figure 32.27 Gene Therapy Market: Generx (Ad5FGF-4) Sales Forecast, till 2035 (USD Million)
Figure 32.28 Gene Therapy Market: Giroctocogene fitelparvovec (SB-525 / PF-07055480) Sales Forecast, till 2035 (USD Million)
Figure 32.29 Gene Therapy Market: Hologene 5 Sales Forecast, till 2035 (USD Million)
Figure 32.30 Gene Therapy Market: INVOSSA (TG-C) Sales Forecast, till 2035 (USD Million)
Figure 32.31 Gene Therapy Market: LUMEVOQ Sales Forecast, till 2035 (USD Million)
Figure 32.32 Gene Therapy Market: NFS-01 (rAAV2-ND4) Sales Forecast, till 2035 (USD Million)
Figure 32.33 Gene Therapy Market: NTLA-2001 Sales Forecast, till 2035 (USD Million)
Figure 32.34 Gene Therapy Market: Olvi-Vec (Olvimulogene nanivacirepvec) Sales Forecast, till 2035 (USD Million)
Figure 32.35 Gene Therapy Market: ProstAtak Sales Forecast, till 2035 (USD Million)
Figure 32.36 Gene Therapy Market: pz-cel (Prademagene zamikeracel / EB-101) Sales Forecast, till 2035 (USD Million)
Figure 32.37 Gene Therapy Market: RGX-121 Sales Forecast, till 2035 (USD Million)
Figure 32.38 Gene Therapy Market: RGX-314 Sales Forecast, till 2035 (USD Million)
Figure 32.39 Gene Therapy Market: VGX-3100 Sales Forecast, till 2035 (USD Million)
Figure 33.1 Gene Editing: Key Application Areas
Figure 33.2 Emerging Gene Editing Platforms
Figure 34.1 Viral Vectors: Key Manufacturing Steps
Figure 35.1 Steps Involved in the Gene Therapy Supply Chain
Figure 35.2 Key Responsibilities of Stakeholders Involved in Gene Therapies Supply Chain
Figure 35.3 Key Steps Involved in the Implementation of a Supply Chain Strategy
Figure 35.4 Comparison of Logistics Processes for Autologous and Allogeneic Therapies
Figure 35.5 Global Regulatory Authorities for Gene Therapy Supply Chain
Figure 35.6 Overview of Supply Chain Orchestration Platform
Figure 37.1 Conclusion: Overall Market Landscape (I / II)
Figure 37.2 Conclusion: Overall Market Landscape (II / II)
Figure 37.3 Conclusion: Developer Landscape
Figure 37.4 Conclusion: Patent Analysis
Figure 37.5 Conclusion: Mergers and Acquisitions
Figure 37.6 Conclusion: Funding and Investment Analysis
Figure 37.7 Conclusion: Clinical Trial Analysis
Figure 37.8 Conclusion: Demand Analysis
Figure 37.9 Conclusion: Market Sizing and Opportunity Analysis (I / II)
Figure 37.10 Conclusion: Market Sizing and Opportunity Analysis (II / II)

LIST OF TABLES
Table 6.1 Comparison of Ex vivo and In vivo Techniques
Table 6.2 Routes of Administration of Gene Therapies: Advantages and Disadvantages
Table 6.3 Advantages and Disadvantages of Gene Therapies
Table 7.1 Key Features of Viral Vectors
Table 8.1 Payment Options Available for Gene Therapies
Table 8.2 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
Table 9.1 Marketed and Clinical Stage Gene Therapies: Information on Developer, Stage of Development and Drug Designation
Table 9.2 Marketed and Clinical Stage Gene Therapies: Information on Developer, Therapeutic Area and Biological Target
Table 9.3 Marketed and Clinical Stage Gene Therapies: Information on Developer and Type of Vector Used
Table 9.4 Marketed and Clinical Stage Gene Therapies: Information on Developer, Type of Therapy and Type of Gene Delivery Method
Table 9.5 Marketed and Clinical Stage Gene Therapies: Information on Developer, Route of Administration and Dosage
Table 9.6 Preclinical and Discovery Stage Gene Therapies: Information on Developer, Stage of Development, Therapeutic Area and Biological Target
Table 9.7 Preclinical and Discovery Stage Gene Therapies: Information on Developer and Type of Vector Used
Table 9.8 Preclinical and Discovery Stage Gene Therapies: Information on Developer, Type of Therapy and Type of Gene Delivery Method
Table 10.1 Gene Therapy Developers: Information on Year of Establishment, Company Size and Location of Headquarters
Table 11.1 Gene Therapy: List of Companies Profiled
Table 11.2 Amgen: Company Overview
Table 11.3 Amgen: Marketed Gene Therapy Portfolio
Table 11.4 Amgen: Clinical Stage Gene Therapy Portfolio
Table 11.5 bluebird bio: Company Overview
Table 11.6 bluebird bio: Marketed Gene Therapy Portfolio
Table 11.7 bluebird bio: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.8 bluebird bio: Recent Developments and Future Outlook
Table 11.9 BioMarin Pharmaceutical: Company Overview
Table 11.10 BioMarin Pharmaceutical: Marketed Gene Therapy Portfolio
Table 11.11 BioMarin Pharmaceutical: Preclinical Stage Gene Therapy Portfolio
Table 11.12 BioMarin Pharmaceutical: Recent Developments and Future Outlook
Table 11.13 CRISPR Therapeutics: Company Overview
Table 11.14 CRISPR Therapeutics: Marketed Gene Therapy Portfolio
Table 11.15 CRISPR Therapeutics: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.16 CRISPR Therapeutics: Recent Developments and Future Outlook
Table 11.17 Kolon TissueGene: Company Overview
Table 11.18 Kolon TissueGene: Marketed Gene Therapy Portfolio
Table 11.19 Kolon TissueGene: Clinical Stage Gene Therapy Portfolio
Table 11.20 Kolon TissueGene: Preclinical Stage Gene Therapy Portfolio
Table 11.21 Kolon TissueGene: Recent Developments and Future Outlook
Table 11.22 Krystal Biotech: Company Overview
Table 11.23 Krystal Biotech: Marketed Gene Therapy Portfolio
Table 11.24 Krystal Biotech: Clinical Stage Gene Therapy Portfolio
Table 11.25 Krystal Biotech: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.26 Krystal Biotech: Recent Developments and Future Outlook
Table 11.27 Pfizer: Company Overview
Table 11.28 Pfizer: Marketed Gene Therapy Portfolio
Table 11.29 Pfizer: Clinical Stage Gene Therapy Portfolio
Table 11.30 Pfizer: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.31 Pfizer: Recent Developments and Future Outlook
Table 11.32 PTC Therapeutics: Company Overview
Table 11.33 PTC Therapeutics: Marketed Gene Therapy Portfolio
Table 11.34 PTC Therapeutics: Preclinical Stage Gene Therapy Portfolio
Table 11.35 PTC Therapeutics: Recent Developments and Future Outlook
Table 11.36 Sarepta Therapeutics: Company Overview
Table 11.37 Sarepta Therapeutics: Marketed Gene Therapy Portfolio
Table 11.38 Sarepta Therapeutics: Clinical Stage Gene Therapy Portfolio
Table 11.39 Sarepta Therapeutics: Preclinical and Discovery Gene Therapy Portfolio
Table 11.40 Sarepta Therapeutics: Recent Developments and Future Outlook
Table 11.41 Spark Therapeutics: Company Overview
Table 11.42 Spark Therapeutics: Marketed Gene Therapy Portfolio
Table 11.43 Spark Therapeutics: Clinical Stage Gene Therapy Portfolio
Table 11.44 Spark Therapeutics: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.45 Spark Therapeutics: Recent Developments and Future Outlook
Table 11.46 Artgen Biotech: Company Overview
Table 11.47 Artgen Biotech: Marketed Gene Therapy Portfolio
Table 11.48 Artgen Biotech: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.49 Artgen Biotech: Recent Developments and Future Outlook
Table 11.50 Ferring Pharmaceuticals: Company Overview
Table 11.51 Ferring Pharmaceuticals: Marketed Gene Therapy Portfolio
Table 11.52 Ferring Pharmaceuticals: Clinical Stage Gene Therapy Portfolio
Table 11.53 Ferring Pharmaceuticals: Recent Developments and Future Outlook
Table 11.54 Novartis: Company Overview
Table 11.55 Novartis: Marketed Gene Therapy Portfolio
Table 11.56 Novartis: Clinical Stage Gene Therapy Portfolio
Table 11.57 Novartis: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.58 Novartis: Recent Developments and Future Outlook
Table 11.59 Orchard Therapeutics: Company Overview
Table 11.60 Orchard Therapeutics: Marketed Gene Therapy Portfolio
Table 11.61 Orchard Therapeutics: Clinical Stage Gene Therapy Portfolio
Table 11.62 Orchard Therapeutics: Preclinical Stage Gene Therapy Portfolio
Table 11.63 Orchard Therapeutics: Recent Developments and Future Outlook
Table 11.64 CSL Behring: Company Overview
Table 11.65 CSL Behring: Marketed Gene Therapy Portfolio
Table 11.66 CSL Behring: Discovery Stage Gene Therapy Portfolio
Table 11.67 CSL Behring: Recent Developments and Future Outlook
Table 11.68 Shanghai Sunway Biotech: Company Overview
Table 11.69 Shanghai Sunway Biotech: Marketed Gene Therapy Portfolio
Table 11.70 Sibiono GeneTech: Company Overview
Table 11.71 Sibiono GeneTech: Marketed Gene Therapy Portfolio
Table 12.1 Gene Therapies: Marketed and Approved Products
Table 12.2 Company Overview: Sibiono GeneTech
Table 12.3 Gendicine: Patent Portfolio
Table 12.4 Gendicine: Stage of Development
Table 12.5 Company Overview: Shanghai Sunway Biotech
Table 12.6 Oncorine: Stage of Development
Table 12.7 Company Overview: Human Stem Cell Institute
Table 12.8 Neovasculgen: Stage of Development
Table 12.9 Company Overview: Amgen
Table 12.10 Imlygic: Stage of Development
Table 12.11 Imlygic: Recommended Dose and Schedule
Table 12.12 Imlygic: Determination of Injection Volume based upon Lesion Size
Table 12.13 Company Overview: Orchard Therapeutics
Table 12.14 Strimvelis: Stage of Development
Table 12.15 Company Overview: Spark Therapeutics
Table 12.16 Luxturna: Stage of Development
Table 12.17 Company Overview: Novartis
Table 12.18 Zolgensma: Stage of Development
Table 12.19 Company Overview: bluebird bio
Table 12.20 Zynteglo: Stage of Development
Table 12.21 Libmeldy: Stage of Development
Table 13.1 Libmeldy: Presentations in Conferences Before Product Approval
Table 13.2 Luxturna: Presentations in Conferences Before Product Approval
Table 13.3 Zolgensma: Presentations in Conferences Before Product Approval
Table 13.4 Zynteglo: Presentations in Conferences Before Product Approval
Table 13.5 Imlygic: Presentations in Conferences After Product Approval
Table 13.6 Libmeldy: Presentations in Conferences After Product Approval
Table 13.7 Luxturna: Presentations in Conferences After Product Approval
Table 13.8 Strimvelis: Presentations in Conferences After Product Approval
Table 13.9 Zolgensma: Presentations in Conferences After Product Approval
Table 13.10 Zynteglo: Presentations in Conferences After Product Approval
Table 13.11 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
Table 14.1 Gene Therapies: Phase II / III and Above Drugs
Table 14.2 Drug Profile: LUMEVOQ (GS010)
Table 14.3 Drug Profile: OTL-103
Table 14.4 Drug Profile: PTC-AADC
Table 14.5 Drug Profile: BMN 270
Table 14.6 Drug Profile: nadofaragene firadenovec (rAd-IFN/Syn3)
Table 14.7 Drug Profile: beti-cel
Table 14.8 Drug Profile: eli-cel
Table 14.9 Drug Profile: lovo-cel
Table 14.10 Drug Profile: SRP-9001
Table 14.11 Drug Profile: EB-101
Table 14.12 Drug Profile: ProstAtak
Table 14.13 Drug Profile: D-Fi
Table 14.14 Drug Profile: CG0070
Table 14.15 Drug Profile: Vigil-EWS
Table 14.16 Drug Profile: Engensis
Table 14.17 Drug Profile: VGX-3100
Table 14.18 Drug Profile: INVOSSA (TG-C)
Table 14.19 Drug Profile: VYJUVEKT
Table 14.20 Drug Profile: PF-06939926
Table 14.21 Drug Profile: PF-06838435
Table 14.22 Drug Profile: PF-07055480
Table 14.23 Drug Profile: SPK-8011
Table 14.24 Drug Profile: AMT-061
Table 14.25 Drug Profile: VB-111
Table 14.26 Drug Profile: Generx
Table 14.27 Drug Profile: ADXS-HPV
Table 14.28 Drug Profile: AGTC 501
Table 14.29 Drug Profile: LYS-SAF302
Table 14.30 Drug Profile: NFS-01
Table 14.31 Drug Profile: AG0302-COVID-19
Table 14.32 Drug Profile: RGX-314
Table 14.33 Drug Profile: Hologene 5
Table 15.1 Gene Therapy Patent Portfolio: List of Top CPC Symbols
Table 15.2 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
Table 15.3 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 15.4 Gene Therapy Patent Portfolio: List of Leading Patents (In terms of Highest Relative Valuation)
Table 15.5 Gene Editing Patent Portfolio: List of Top CPC Classifications
Table 15.6 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
Table 15.7 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 15.8 Gene Editing Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
Table 16.1 Gene Therapy: List of Mergers and Acquisitions, 2015-2022
Table 16.2 Acquisitions: Key Value Drivers
Table 16.3 Mergers and Acquisitions: Information on Phase of Development and Therapeutic Area of the Acquired Company’s Gene Therapy Product
Table 16.4 Mergers and Acquisitions: Information on Deal Multiple based on Acquisition Amount
Table 17.1 Gene Therapy Market: Funding and Investments, 2015-2022
Table 17.2 Funding and Investments: Summary of Investments
Table 17.3 Funding and Investments: Summary of Venture Capital Funding
Table 18.1 Clinical Trial Analysis: Emerging Molecules to Watch
Table 18.2 Clinical Trial Analysis: Most Important Trials to Watch
Table 19.1 Pricing Model: Price of Marketed Gene / Cell Therapies
Table 19.2 Pricing Model: Price of Marketed Targeted Drugs (Antibody Drug Conjugates and Monoclonal Antibodies)
Table 19.3 Pricing Model: Opinions of Experts / Other Analysts
Table 20.1 Start-up Valuation: Estimates based on Overall Experience
Table 22.1 Global Clinical Demand for Gene Therapies: Information on Trial Phase, Distribution of Patient Enrolment and Average Number of Patients Enrolled
Table 24.1 Gene Therapy Market: List of Forecasted Therapies along with Information on Developer, Stage of Development and First Launch Year / Likely Approval Year
Table 32.1 Adstiladrin®: Net Present Value (USD Million)
Table 32.2 Adstilarin®: Value Creation Analysis (USD Million)
Table 32.3 BEQVEZ™: Net Present Value (USD Million)
Table 32.4 BEQVEZ™: Value Creation Analysis (USD Million)
Table 32.5 Casgevy™: Net Present Value (USD Million)
Table 32.6 Casgevy™: Value Creation Analysis (USD Million)
Table 32.7 Elevidys®: Net Present Value (USD Million)
Table 32.8 Elevidys®: Value Creation Analysis (USD Million)
Table 32.9 Gendicine®: Net Present Value (USD Million)
Table 32.10 Gendicine®: Value Creation Analysis (USD Million)
Table 32.11 Hemgenix®: Net Present Value (USD Million)
Table 32.12 Hemgenix®: Value Creation Analysis (USD Million)
Table 32.13 IMLYGIC®: Net Present Value (USD Million)
Table 32.14 IMLYGIC®: Value Creation Analysis (USD Million)
Table 32.15 Libmeldy®: Net Present Value (USD Million)
Table 32.16 Libmeldy®: Value Creation Analysis (USD Million)
Table 32.17 Luxturna®: Net Present Value (USD Million)
Table 32.18 Luxturna®: Value Creation Analysis (USD Million)
Table 32.19 LYFGENIA™: Net Present Value (USD Million)
Table 32.20 LYFGENIA™: Value Creation Analysis (USD Million)
Table 32.21 Neovasculgen®: Net Present Value (USD Million)
Table 32.22 Neovasculgen®: Value Creation Analysis (USD Million)
Table 32.23 Oncorine®: Net Present Value (USD Million)
Table 32.24 Oncorine®: Value Creation Analysis (USD Million)
Table 32.25 Roctavian™: Net Present Value (USD Million)
Table 32.26 Roctavian™: Value Creation Analysis (USD Million)
Table 32.27 SKYSONA®: Net Present Value (USD Million)
Table 32.28 SKYSONA®: Value Creation Analysis (USD Million)
Table 32.29 Strimvelis™: Net Present Value (USD Million)
Table 32.30 Strimvelis™: Value Creation Analysis (USD Million)
Table 32.31 Upstaza™: Net Present Value (USD Million)
Table 32.32 Upstaza™: Value Creation Analysis (USD Million)
Table 32.33 VYJUVEKT®: Net Present Value (USD Million)
Table 32.34 VYJUVEKT®: Value Creation Analysis (USD Million)
Table 32.35 Zolgensma®: Net Present Value (USD Million)
Table 32.36 Zolgensma®: Value Creation Analysis (USD Million)
Table 32.37 Zynteglo™: Net Present Value (USD Million)
Table 32.38 Zynteglo™: Value Creation Analysis (USD Million)
Table 32.39 AAV-RPE65: Net Present Value (USD Million)
Table 32.40 AAV-RPE65: Value Creation Analysis (USD Million)
Table 32.41 ABO-102 (UX111): Net Present Value (USD Million)
Table 32.42 ABO-102 (UX111): Value Creation Analysis (USD Million)
Table 32.43 AGTC 501 (rAAV2tYF-GRK1-RPGR): Net Present Value (USD Million)
Table 32.44 AGTC 501 (rAAV2tYF-GRK1-RPGR): Value Creation Analysis (USD Million)
Table 32.45 Cretostimogene Grenadenorepvec (CG0070): Net Present Value (USD Million)
Table 32.46 Cretostimogene Grenadenorepvec (CG0070: Value Creation Analysis (USD Million)
Table 32.47 Dirloctocogene samoparvovec (SPK-8011): Net Present Value (USD Million)
Table 32.48 Dirloctocogene samoparvovec (SPK-8011): Value Creation Analysis (USD Million)
Table 32.49 DTx-301: Net Present Value (USD Million)
Table 32.50 DTx-301: Value Creation Analysis (USD Million)
Table 32.51 DTx-401: Net Present Value (USD Million)
Table 32.52 DTx-401: Value Creation Analysis (USD Million)
Table 32.53 Generx® (Ad5FGF-4): Net Present Value (USD Million)
Table 32.54 Generx® (Ad5FGF-4): Value Creation Analysis (USD Million)
Table 32.55 Giroctocogene fitelparvovec (SB-525 / PF-07055480): Net Present Value (USD Million)
Table 32.56 Giroctocogene fitelparvovec (SB-525 / PF-07055480): Value Creation Analysis (USD Million)
Table 32.57 Hologene 5: Net Present Value (USD Million)
Table 32.58 Hologene 5: Value Creation Analysis (USD Million)
Table 32.59 INVOSSA™ (TG-C): Net Present Value (USD Million)
Table 32.60 INVOSSA™ (TG-C): Value Creation Analysis (USD Million)
Table 32.61 LUMEVOQ®: Net Present Value (USD Million)
Table 32.62 LUMEVOQ®: Value Creation Analysis (USD Million)
Table 32.63 NFS-01 (rAAV2-ND4): Net Present Value (USD Million)
Table 32.64 NFS-01 (rAAV2-ND4): Value Creation Analysis (USD Million)
Table 32.65 NTLA-2001: Net Present Value (USD Million)
Table 32.66 NTLA-2001: Value Creation Analysis (USD Million)
Table 32.67 Olvi-Vec (Olvimulogene nanivacirepvec): Net Present Value (USD Million)
Table 32.68 Olvi-Vec (Olvimulogene nanivacirepvec): Value Creation Analysis (USD Million)
Table 32.69 ProstAtak®: Net Present Value (USD Million)
Table 32.70 ProstAtak®: Value Creation Analysis (USD Million)
Table 32.71 pz-cel (prademagene zamikeracel / EB-101): Net Present Value (USD Million)
Table 32.72 pz-cel (prademagene zamikeracel / EB-101): Value Creation Analysis (USD Million)
Table 32.73 RGX-121: Net Present Value (USD Million)
Table 32.74 RGX-121: Value Creation Analysis (USD Million)
Table 32.75 RGX-314: Net Present Value (USD Million)
Table 32.76 RGX-314: Value Creation Analysis (USD Million)
Table 32.77 VGX-3100: Net Present Value (USD Million)
Table 32.78 VGX-3100: Value Creation Analysis (USD Million)
Table 32.79 Summary of Variations in Forecasted Sales Revenue of Gene Therapies (Compared to 5th Edition)
Table 33.1 Gene Editing Technologies: Information on Developer and Highest Phase of Drug Development Supported
Table 33.2 Key Features of Gene Editing Platforms
Table 33.3 ASC Therapeutics: Collaborations
Table 33.4 CRISPR Therapeutics: Funding Instances
Table 33.5 CRISPR Therapeutics: Collaborations
Table 33.6 Editas Medicine: Funding Instances
Table 33.7 Editas Medicine: Collaborations
Table 33.8 Emendo Biotherapeutics: Collaborations
Table 33.9 Excision Biotherapeutics: Funding Instances
Table 33.10 Intellia Therapeutics: Funding Instances
Table 33.11 Intellia Therapeutics: Collaborations
Table 33.12 Sarepta Therapeutics: Funding Instances
Table 33.13 Sarepta Therapeutics: Collaborations
Table 33.14 ToolGen: Collaborations
Table 33.15 Sangamo Therapeutics: Funding Instances
Table 33.16 Sangamo Therapeutics: Collaborations
Table 33.17 bluebird bio: Funding Instances
Table 34.18 bluebird bio: Collaborations
Table 34.19 Gene Switch Technology Platforms
Table 33.20 Precigen: Funding Instances
Table 33.21 MeiraGTx: Funding Instances
Table 33.22 MeiraGTx: Collaborations
Table 33.23 Gene Therapy Developers: Information on Headquarters, Technology / Platform and Type of Gene Delivery Method
Table 34.1 Key Features of Small-scale Cell Culture Systems
Table 34.2 List of Viral Vector Contract Manufacturing Organizations: Information on Type of Viral Vector Manufactured
Table 36.1 Orchard Therapeutics: Company Snapshot
Table 36.2 Vivet Therapeutics: Company Snapshot
Table 36.3 Kubota Pharmaceutical: Company Snapshot
Table 36.4 Eyevensys: Company Snapshot
Table 36.5 AGTC: Company Snapshot
Table 36.6 LogicBio Therapeutics: Company Snapshot
Table 36.7 AAVogen: Company Snapshot
Table 36.8 Hemera Biosciences: Company Snapshot
Table 36.9 Myonexus Therapeutics: Company Snapshot
Table 36.10 Arthrogen: Company Snapshot
Table 36.11 PlasmidFactory: Company Snapshot
Table 36.12 Delphi Genetics: Company Snapshot
Table 36.13 Vigene Biosciences: Company Snapshot
Table 36.14 Gene Biotherapeutics: Company Snapshot
Table 36.15 Milo Biotechnology: Company Snapshot
Table 38.1 Marketed and Clinical Stage Gene Therapies: Distribution by Stage of Development
Table 38.2 Marketed and Clinical Stage Gene Therapies: Distribution by Drug Designation
Table 38.3 Marketed and Clinical Stage Gene Therapies: Distribution by Therapeutic Area
Table 38.4 Marketed and Clinical Stage Gene Therapies: Distribution by Biological Target
Table 38.5 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Vector Used
Table 38.6 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Therapy
Table 38.7 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Gene Delivery Method
Table 38.8 Marketed and Clinical Stage Gene Therapies: Distribution by Route of Administration
Table 38.9 Marketed and Clinical Stage Gene Therapies: Distribution by Dosage
Table 38.10 Preclinical and Discovery Stage Gene Therapies: Distribution by Stage of Development
Table 38.11 Preclinical and Discovery Stage Gene Therapies: Distribution by Therapeutic Area
Table 38.12 Preclinical and Discovery Stage Gene Therapies: Distribution by Biological Target
Table 38.13 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Vector Used
Table 38.14 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Therapy
Table 38.15 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Gene Delivery Method Used
Table 38.16 Gene Therapy Developers: Distribution by Year of Establishment
Table 38.17 Gene Therapy Developers: Distribution by Company Size
Table 38.18 Gene Therapy Developers: Distribution by Location of Headquarters
Table 38.19 Most Active Players: Distribution by Number of Gene Therapies
Table 38.20 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Table 38.21 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Table 38.22 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 38.23 Gene Therapy Patent Portfolio: Distribution by Geography
Table 38.24 Gene Therapy Patent Portfolio: North America Scenario
Table 38.25 Gene Therapy Patent Portfolio: Europe Scenario
Table 38.26 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
Table 38.27 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
Table 38.28 Gene Therapy Patent Portfolio: Leading Industry Players: Distribution by Number of Players
Table 38.29 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
Table 38.30 Gene Editing Patent Portfolio: Distribution by Type of Patent
Table 38.31 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
Table 38.32 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 38.33 Gene Editing Patent Portfolio: Distribution by Geography
Table 38.34 Gene Editing Patent Portfolio: North America Scenario
Table 38.35 Gene Editing Patent Portfolio: Europe Scenario
Table 38.36 Gene Editing Patent Portfolio: Asia-Pacific Scenario
Table 38.37 Gene Editing Patent Portfolio: Distribution by CPC Symbols
Table 38.38 Leading Industry Players (Gene Editing): Distribution by Number of Patents
Table 38.39 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
Table 38.40 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2015-2022
Table 38.42 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Table 38.43 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
Table 38.44 Mergers and Acquisitions: Local and International Deals
Table 38.45 Acquisitions: Distribution by Key Value Drivers
Table 38.46 Acquisitions: Distribution by Year of Acquisition and Key Value
Table 38.47 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Table 38.48 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company’s Product
Table 38.49 Mergers and Acquisitions: Distribution by Therapeutic Area
Table 38.50 Funding and Investments: Cumulative Distribution of Instances by Year, 2015-2022
Table 38.51 Funding and Investments: Cumulative Distribution of Amount Invested by Year, 2015-2022 (USD Million)
Table 38.52 Funding and Investments: Distribution of Instances by Type of Funding, 2015-2022
Table 38.53 Funding and Investments: Distribution of Total Amount Invested by Type of Funding (USD Million)
Table 38.54 Funding and Investments: Summary of Investments, 2015-2022 (USD Million)
Table 38.55 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
Table 38.56 Funding and Investments: Distribution of Instances and Amount Invested by Geography
Table 38.57 Funding and Investments: Regional Distribution of Funding Instances
Table 38.58 Funding and Investments: Distribution of Instances and Amount Invested by Highest Phase of Development of the Company’s Product
Table 38.59 Funding and Investments: Distribution of Instances by Therapeutic Area
Table 38.60 Most Active Players: Distribution by Number of Funding Instances
Table 38.61 Most Active Players: Distribution by Amount Invested (USD Million)
Table 38.62 Funding and Investments: Distribution of Instances by Type of Investors
Table 38.63 Leading Investors: Distribution by Number of Instances
Table 38.64 Funding and Investment Summary, 2015-2022 (USD Million)
Table 38.65 Clinical Trial Analysis: Distribution by Trial Registration Year, 2017-2022
Table 38.66 Clinical Trial Analysis: Distribution by Trial Status
Table 38.67 Clinical Trial Analysis: Distribution by Trial Phase
Table 38.68 Clinical Trial Analysis: Distribution by Therapeutic Area
Table 38.69 Clinical Trial Analysis: Distribution by Geography
Table 38.70 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
Table 38.71 Clinical Trial Analysis: Distribution by Trial Status and Geography
Table 38.72 Clinical Trial Analysis: Distribution by Type of Sponsor
Table 38.73 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Table 38.74 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, 2017-2022
Table 38.75 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Table 38.76 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Table 38.77 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Table 38.78 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
Table 38.79 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
Table 38.80 Demand Analysis: Global Demand for Gene Therapies
Table 38.81 Demand Analysis: Distribution by Stage of Development
Table 38.82 Demand Analysis: Distribution by Therapeutic Area
Table 38.83 Demand Analysis: Distribution by Type of Therapy
Table 38.84 Demand Analysis: Distribution by Type of Gene Delivery Method
Table 38.85 Demand Analysis: Distribution by Geography
Table 38.86 Global Gene Therapy Market, till 2035, Base Scenario (USD Billion)
Table 38.87 Global Gene Therapy Market, till 2035, Conservative Scenario (USD Billion)
Table 38.88 Global Gene Therapy Market, till 2035, Optimistic Scenario (USD Billion)
Table 38.89 Gene Therapy Market: Distribution by Therapeutic Area, 2024, 2029 and 2035
Table 38.90 Gene Therapy Market for Muscle Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.91 Gene Therapy Market for Hematologic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.92 Gene Therapy Market for Dermatological Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.93 Gene Therapy Market for Oncological Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.94 Gene Therapy Market for Genetic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.95 Gene Therapy Market for Ophthalmic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.96 Gene Therapy Market for Metabolic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.97 Gene Therapy Market for Cardiovascular Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.98 Gene Therapy Market for Other Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.99 Gene Therapy Market: Distribution by Type of Vector Used, 2024, 2029 and 2035
Table 38.100 Gene Therapy Market for Adeno-associated Viral Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.101 Gene Therapy Market for Herpes Simplex Virus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.102 Gene Therapy Market for Lentivirus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.103 Gene Therapy Market for Adenovirus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.104 Gene Therapy Market for Non-Viral Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.105 Gene Therapy Market for Retrovirus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.106 Gene Therapy Market for Other-Viral Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.107 Gene Therapy Market: Distribution by Type of Therapy, 2024, 2029 and 2035
Table 38.108 Gene Therapy Market for Gene Augmentation, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.109 Gene Therapy Market for Oncolytic Immunotherapies, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.110 Gene Therapy Market for Gene Editing, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.111 Gene Therapy Market for Gene Regulation, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.112 Gene Therapy Market for Other Therapies, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.113 Gene Therapy Market: Distribution by Type of Gene Delivery Method Used, 2024, 2029 and 2035
Table 38.114 Gene Therapy Market for In vivo Gene Delivery, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.115 Gene Therapy Market for Ex vivo Gene Delivery, Forecasted till 2035, Conservative, Base and Optimistic Scenarios (USD
Table 38.116 Gene Therapy Market: Distribution by Route of Administration, 2024, 2029 and 2035
Table 38.117 Gene Therapy Market for Intravenous till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.118 Gene Therapy Market for Subretinal till 2024-2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.119 Gene Therapy Market for Intratumoral till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.120 Gene Therapy Market for Intramuscular till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.121 Gene Therapy Market for Other till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.122 Gene Therapy Market: Distribution by Geography, 2024, 2029 and 2035
Table 38.123 Gene Therapy Market for North America, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.124 Gene Therapy Market for Europe, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.125 Gene Therapy Market for Asia-Pacific, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.126 Gene Therapy Market for Latin America, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.127 Gene Therapy Market for Rest of the World, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.128 Adstiladrin® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.129 BEQVEZ™ Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.130 Casgevy™ Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.131 Elevidys® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.132 Gendicine® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.133 Hemgenix® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.134 IMLYGIC® (AMT-061) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.135 Libmeldy® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.136 Luxturna® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.137 LYFGENIA™ Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.138 Neovasculgen® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.139 Oncorine® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.140 Roctavian™ Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.141 SKYSONA® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.142 Strimvelis™ Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.143 Upstaza™ Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.144 VYJUVEKT® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.145 Zolgensma® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.146 Zynteglo™ Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.147 AAV-RPE65 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.148 ABO-102 (UX111) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.149 AGTC 501 (rAAV2tYF-GRK1-RPGR) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.150 Cretostimogene Grenadenorepvec (CG0070) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.151 Dirloctocogene samoparvovec (SPK-8011) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.152 DTx-301 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.153 DTx-401 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.154 Generx® (Ad5FGF-4) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.155 Giroctocogene fitelparvovec (SB-525 / PF-07055480) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.156 Hologene 5 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.157 INVOSSA™ (TG-C) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.158 LUMEVOQ® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.159 NFS-01 (rAAV2-ND4) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.160 NTLA-2001 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.161 Olvi-Vec (Olvimulogene nanivacirepvec) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.162 ProstAtak® Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.163 pz-cel (prademagene zamikeracel / EB-101) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.164 RGX-121 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.165 RGX-314 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.166 VGX-3100 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • Amgen
  • Artgen Biotech
  • BioMarin Pharmaceutical
  • bluebird bio
  • CRISPR Therapeutics
  • CSL Behring
  • Ferring Pharmaceuticals
  • Kolon TissueGene
  • Krystal Biotech
  • Novartis
  • Orchard Therapeutics
  • Pfizer
  • PTC Therapeutics
  • Sarepta Therapeutics
  • Shanghai Sunway Biotech
  • Sibiono GeneTech
  • Spark Therapeutics

Methodology

 

 

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