Product Code: RA100359
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(R) (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(R) (Spark Therapeutics), the first gene therapy intended for the treatment of inherited retinal disorders. Additionally, in May 2019, the USFDA approved ZOLGENSMA(R) (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
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(R) (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(R) (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(R) (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(R) (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(R) (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(R) (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(TM) (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(TM) (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(R) (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(TM) (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(TM) (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-COVID19
- 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(R)
- 32.3.1.1. Sales Forecast
- 32.3.1.2. Net Present Value
- 32.3.1.3. Value Creation Analysis
- 32.3.2. BEQVEZ(TM)
- 32.3.2.1. Sales Forecast
- 32.3.2.2. Net Present Value
- 32.3.2.3. Value Creation Analysis
- 32.3.3. Casgevy(TM)
- 32.3.3.1. Sales Forecast
- 32.3.3.2. Net Present Value
- 32.3.3.3. Value Creation Analysis
- 32.3.4. Elevidys(R)
- 32.3.4.1. Sales Forecast
- 32.3.4.2. Net Present Value
- 32.3.4.3. Value Creation Analysis
- 32.3.5. Gendicine(R)
- 32.3.5.1. Sales Forecast
- 32.3.5.2. Net Present Value
- 32.3.5.3. Value Creation Analysis
- 32.3.6. Hemgenix(R)
- 32.3.6.1. Sales Forecast
- 32.3.6.2. Net Present Value
- 32.3.6.3. Value Creation Analysis
- 32.3.7. IMLYGIC(R)
- 32.3.7.1. Sales Forecast
- 32.3.7.2. Net Present Value
- 32.3.7.3. Value Creation Analysis
- 32.3.8. Libmeldy(R)
- 32.3.8.1. Sales Forecast
- 32.3.8.2. Net Present Value
- 32.3.8.3. Value Creation Analysis
- 32.3.9. Luxturna(R)
- 32.3.9.1. Sales Forecast
- 32.3.9.2. Net Present Value
- 32.3.9.3. Value Creation Analysis
- 32.3.10. LYFGENIA(TM)
- 32.3.10.1. Sales Forecast
- 32.3.10.2. Net Present Value
- 32.3.10.3. Value Creation Analysis
- 32.3.11. Neovasculgen(R)
- 32.3.11.1. Sales Forecast
- 32.3.11.2. Net Present Value
- 32.3.11.3. Value Creation Analysis
- 32.3.12. Oncorine(R)
- 32.3.12.1. Sales Forecast
- 32.3.12.2. Net Present Value
- 32.3.12.3. Value Creation Analysis
- 32.3.13. Roctavian(TM)
- 32.3.13.1. Sales Forecast
- 32.3.13.2. Net Present Value
- 32.3.13.3. Value Creation Analysis
- 32.3.14. SKYSONA(R)
- 32.3.14.1. Sales Forecast
- 32.3.14.2. Net Present Value
- 32.3.14.3. Value Creation Analysis
- 32.3.15. Strimvelis(TM)
- 32.3.15.1. Sales Forecast
- 32.3.15.2. Net Present Value
- 32.3.15.3. Value Creation Analysis
- 32.3.16. Upstaza(TM)
- 32.3.16.1. Sales Forecast
- 32.3.16.2. Net Present Value
- 32.3.16.3. Value Creation Analysis
- 32.3.17. VYJUVEKT(R)
- 32.3.17.1. Sales Forecast
- 32.3.17.2. Net Present Value
- 32.3.17.3. Value Creation Analysis
- 32.3.18. Zolgensma(R)
- 32.3.18.1. Sales Forecast
- 32.3.18.2. Net Present Value
- 32.3.18.3. Value Creation Analysis
- 32.3.19. Zynteglo(TM)
- 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(R) (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(TM) (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(R)
- 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(R)
- 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. Y - 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. CONCLUSION
38. APPENDIX I: TABULATED DATA
39. APPENDIX II: LIST OF COMPANIES AND ORGANIZATIONS