Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts (5th Edition), 2022-2035
Gene Therapy Market (5th Edition) by Type of Therapy (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Delivery Method Used (Ex vivo and In vivo), Type of Vector Used (Adeno-associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Non-Viral Vectors, Retrovirus and Others), Target Therapeutic Areas (Cardiovascular Diseases, Dermatological Diseases, Genetic Diseases, Hematological Diseases, Infectious Diseases, Metabolic Diseases, Muscle-related Diseases, Oncological Diseases, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebral, Intracoronary, Intradermal, Intralesional, Intramuscular, Intrapleural, Intrathecal, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal, Topical and Others), and Key Geographical Regions (US, Europe, Asia-Pacific and rest of the world): Industry Trends and Global Forecasts, 2022-2035
Report Link: https://www.rootsanalysis.com/reports/gene-therapies-market/268.html
The gene therapy market is valued at approximately $2.6 billion in 2023, with an expected compounded annual growth rate (CAGR) of 18.0% during the forecast period.
In August 2017, a significant milestone was achieved with the FDA approval of Tisagenlecleucel as the first gene therapy for the treatment of relapsed or refractory pediatric and young-adult B-cell acute lymphoblastic leukemia. Over the past two decades, there have been numerous breakthroughs in the development of gene therapies. In 2020, Libmeldy™, an ex vivo gene therapy, received approval for the treatment of metachromatic leukodystrophy. These therapies, encompassing gene replacement and gene-editing modalities, aim to correct mutated genes in patients using molecular carriers, including both viral and non-viral vectors.
Following the onset of the COVID-19 pandemic, there has been a steady increase in investigational new drug (IND) applications filed for cell and gene therapies. In 2021, more than 200 gene therapies were in phase II and III studies, and it is expected that six gene therapies will receive USFDA market approval in 2022. Promising results from ongoing clinical research have encouraged both government and private firms to invest in therapy product development in this field. In 2021, gene therapy companies raised approximately $9.5 billion in capital investments. With the continuous progress in this domain, gene therapies are anticipated to benefit 1.1 million patients with a wide range of disease indications by 2035.
Currently, over 250 gene therapy companies are actively involved in the development of early and late-stage gene therapies on a global scale. Recent years have witnessed a significant surge in the integration of cutting-edge technologies, including gene modification, gene editing, genome sequencing, and manipulation technologies (such as molecular switches), in conjunction with gene delivery methods. The CRISPR-Cas9-based gene-editing tool is a notable technological advancement that enables precise alterations to transgenes. It's important to note that new-generation delivery platforms, such as nanoparticles and hybrid vector systems, have proven effective and safe for delivering gene-based therapeutics. Furthermore, various consolidation efforts are underway in the industry, primarily focused on expanding and strengthening existing development efforts. A clear indicator of this is the fact that 56% of the total acquisitions in this domain have centered around consolidating drug classes. Driven by the collective and persistent efforts of gene therapy companies and the growing demand for effective therapeutic options requiring a single dose, the gene therapy market is poised for significant growth during the forecast period.
Key Market Segments
Type of Therapy
Report Link: https://www.rootsanalysis.com/reports/gene-therapies-market/268.html
The gene therapy market is valued at approximately $2.6 billion in 2023, with an expected compounded annual growth rate (CAGR) of 18.0% during the forecast period.
In August 2017, a significant milestone was achieved with the FDA approval of Tisagenlecleucel as the first gene therapy for the treatment of relapsed or refractory pediatric and young-adult B-cell acute lymphoblastic leukemia. Over the past two decades, there have been numerous breakthroughs in the development of gene therapies. In 2020, Libmeldy™, an ex vivo gene therapy, received approval for the treatment of metachromatic leukodystrophy. These therapies, encompassing gene replacement and gene-editing modalities, aim to correct mutated genes in patients using molecular carriers, including both viral and non-viral vectors.
Following the onset of the COVID-19 pandemic, there has been a steady increase in investigational new drug (IND) applications filed for cell and gene therapies. In 2021, more than 200 gene therapies were in phase II and III studies, and it is expected that six gene therapies will receive USFDA market approval in 2022. Promising results from ongoing clinical research have encouraged both government and private firms to invest in therapy product development in this field. In 2021, gene therapy companies raised approximately $9.5 billion in capital investments. With the continuous progress in this domain, gene therapies are anticipated to benefit 1.1 million patients with a wide range of disease indications by 2035.
Currently, over 250 gene therapy companies are actively involved in the development of early and late-stage gene therapies on a global scale. Recent years have witnessed a significant surge in the integration of cutting-edge technologies, including gene modification, gene editing, genome sequencing, and manipulation technologies (such as molecular switches), in conjunction with gene delivery methods. The CRISPR-Cas9-based gene-editing tool is a notable technological advancement that enables precise alterations to transgenes. It's important to note that new-generation delivery platforms, such as nanoparticles and hybrid vector systems, have proven effective and safe for delivering gene-based therapeutics. Furthermore, various consolidation efforts are underway in the industry, primarily focused on expanding and strengthening existing development efforts. A clear indicator of this is the fact that 56% of the total acquisitions in this domain have centered around consolidating drug classes. Driven by the collective and persistent efforts of gene therapy companies and the growing demand for effective therapeutic options requiring a single dose, the gene therapy market is poised for significant growth during the forecast period.
Key Market Segments
Type of Therapy
- Gene Augmentation
- Oncolytic Viral Therapy
- Immunotherapy
- Others
- Ex vivo
- In vivo
- Adeno-associated Virus
- Adenovirus
- Herpes Simplex Virus
- Lentivirus
- Non-Viral Vectors
- Retrovirus
- Others
- Cardiovascular Diseases
- Dermatological Diseases
- Genetic Diseases
- Hematological Diseases
- Infectious Diseases
- Metabolic Diseases
- Muscle-related Diseases
- Oncological Diseases
- Ophthalmic Diseases
- Others
- Intraarticular
- Intracerebral
- Intracoronary
- Intradermal
- Intralesional
- Intramuscular
- Intrapleural
- Intrathecal
- Intratumoral
- Intravenous
- Intravesical
- Intravitreal
- Subretinal
- Topical
- Others
- US
- Europe
- Asia-Pacific
- Rest of the World
- The report studies the gene therapy market by type of therapy, type of gene delivery method used, type of vector used, target therapeutic areas, route of administration, and key geographical regions.
- The report analyzes factors (such as drivers, restraints, opportunities, and challenges) affecting the market growth.
- The report assesses the potential advantages and obstacles within the market for those involved and offers information on the competitive environment for top players in the market.
- The report forecasts the revenue of market segments with respect to major regions.
- An overview of key findings from our research on the gene therapy market, offering insights into its current state and likely evolution in the short, mid, and long term.
- A comprehensive overview of the gene therapy market, detailing the phase of development (marketed, clinical, preclinical, and discovery), therapeutic areas, target disease indications, types of vectors, gene/molecule targets, types of therapy, gene delivery methods, routes of administration, and special drug designations.
- An overview of companies involved in gene therapy development, including establishment year, company size, headquarters location, regional landscape, and key players in the domain.
- Discussion on viral and non-viral vectors, covering their design, manufacturing requirements, advantages, and limitations.
- Analysis of gene therapy regulations in North America, Europe, and Asia-Pacific, focusing on challenges related to reimbursement.
- Examination of commercialization strategies adopted by gene therapy companies at different development stages: pre-launch, during launch, and post-marketing.
- Profiles of marketed and late-stage gene therapies, including development timeline, current status, mechanism of action, technology used, patent portfolio, dosage, manufacturing details, and affiliated companies.
- Review of emerging technologies and therapy development platforms used for manufacturing gene therapies, with a focus on technologies used in four or more products/candidates.
- In-depth analysis of gene therapy and gene editing therapy patents filed/granted since 2017, including patent type, publication year, regional applicability, CPC symbols, leading industry players in terms of patents, and patent valuation.
- Analysis of mergers and acquisitions in the gene therapy domain from 2015-2022, based on parameters like year, deal type, geographical location, value drivers, product development phase, therapeutic area, and deal multiples.
- Analysis of investments at various stages (seed financing, venture capital, IPOs, secondary offerings, debt financing, grants, and equity offerings) by gene therapy companies.
- Analysis of completed, ongoing, and planned clinical studies, considering parameters like registration year, trial status, phase, therapeutic area, geography, sponsor type, treatment sites, and patient population.
- Examination of factors influencing gene therapy pricing, with various models/approaches used by manufacturers.
- Analysis of startup companies in the gene therapy domain established between 2017-2022, based on years of experience.
- A case study on vector manufacturing trends and companies offering contract vector manufacturing services, along with details on vector manufacturing processes for different types.
- 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.
- You will get access to complimentary PPT insights and excel data packs / dynamic dashboards to easily navigate through complex analyses / charts.
- Shenzhen Sibiono GeneTech
- Shanghai Sunway Biotech
- Epeius Biotechnologies
- Human Stem Cells Institute
- Amgen
- Orchard Therapeutics
- Spark Therapeutics
- Novartis
- AnGes
- bluebird bio
- Orchard Therapeutics
1. PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Key Questions Answered
1.4. Chapter Outlines
2. EXECUTIVE SUMMARY
2.1. Chapter Overview
3. INTRODUCTION
3.1. Context and Background
3.2. Evolution of Gene Therapies
3.3. Classification of Gene Therapies
3.3.1. Somatic and Germline Gene Therapies
3.3.2. Ex Vivo and In Vivo Gene Therapies
3.4. Routes of Administration
3.5. Mechanism of Action
3.6. Overview of Gene Editing
3.6.1. Evolution of Genome Editing
3.6.2. Applications of Genome Editing
3.6.3. Available Genome Editing Techniques
3.7. Advantages and Disadvantages of Gene Therapies
3.7.1 Ethical and Social Concerns Related to Gene Therapies
3.7.2. Constraints and Challenges Related to Gene Therapies
3.7.3. Therapy Development Concerns
3.7.4. Manufacturing Concerns
3.7.5. Commercial Viability Concerns
4. GENE DELIVERY VECTORS
4.1. Chapter Overview
4.2. Viral and Non-Viral Methods of Gene Transfer
4.3. Viral Vectors for Genetically Modified Therapies
4.4. Types of Viral Vectors
4.4.1. Adeno-associated Viral Vectors
4.4.1.1. Overview
4.4.1.2. Design and Manufacturing
4.4.1.3. Advantages Offered
4.4.1.4. Associated Limitations
4.4.2. Adenoviral Vectors
4.4.2.1. Overview
4.4.2.2. Design and Manufacturing
4.4.2.3. Advantages Offered
4.4.2.4. Associated Limitations
4.4.3. Lentiviral Vectors
4.4.3.1. Overview
4.4.3.2. Design and Manufacturing
4.4.3.3. Advantages Offered
4.4.3.4. Associated Limitations
4.4.4. Retroviral Vectors
4.4.4.1. Overview
4.4.4.2. Design and Manufacturing
4.4.4.3. Advantages Offered
4.4.4.4. Associated Limitations
4.4.5. Other Viral Vectors
4.4.5.1. Alphavirus
4.4.5.2. Foamy Virus
4.4.5.3. Herpes Simplex Virus
4.4.5.4. Sendai Virus
4.4.5.5. Simian Virus
4.4.5.6. Vaccinia Virus
4.5. Types of Non-Viral Vectors
4.5.1. Plasmid DNA
4.5.2. Liposomes, Lipoplexes and Polyplexes
4.5.3. Oligonucleotides
4.5.4. Nanoparticles
4.5.5. Hybrid Vector Systems
4.5.6. Other Non-Viral Vectors
4.5.7. Gene Delivery Using Non-Viral Vectors
4.5.7.1. Biolistic Methods
4.5.7.2. Electroporation
4.5.7.3. Receptor Mediated Gene Delivery
4.5.7.4. Gene Activated Matrix (GAM)
5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO
5.1. Chapter Overview
5.2. Regulatory Guidelines in North America
5.2.1. The US Scenario
5.2.2. Canadian Scenario
5.3. Regulatory Guidelines in Europe
5.3.1. Quality of Gene Therapy Products
5.3.2. Non-Clinical Development
5.3.3. Clinical Development
5.4. Regulatory Guidelines in Asia-Pacific
5.4.1. Chinese Scenario
5.4.1.1. Construction of DNA Expression Cassette and Gene Delivery Systems
5.4.1.2. Generation and Characterization of Cell Banks and Engineered Bacteria Banks
5.4.1.3. Manufacturing of Gene Therapy Products
5.4.1.4. Quality Control
5.4.1.5. Evaluation of Efficacy of Gene Therapy Products
5.4.1.6. Safety Evaluation of Gene Therapy Products
5.4.1.7. Clinical Trial of Gene Therapy Products
5.4.1.8. Ethics Study
5.4.2. Japanese Scenario
5.4.3. South Korean Scenario
5.4.4. Australian Scenario
5.4.5. Hong Kong Scenario
5.5. Reimbursement Scenario
5.5.1. Challenges Related to Reimbursement
5.6. Commonly Offered Payment Models for Gene Therapies
6. MARKET OVERVIEW
6.1. Chapter Overview
6.1.1. Analysis of Gene Therapy Candidates by Stage of Development
6.2. Gene Therapy Market: Clinical and Commercial Pipeline
6.2.1. Analysis by Phase of Development
6.2.2. Analysis by Therapeutic Area
6.2.3. Analysis by Type of Vector Used
6.2.4. Analysis by Type of Gene / Molecule Targeted
6.2.5. Analysis by Type of Therapy
6.2.6. Analysis by Type of Gene Delivery Method Used
6.2.7. Analysis by Route of Administration
6.3. Gene Therapy Market: Early-Stage Pipeline
6.3.1. Analysis by Phase of Development
6.3.2. Analysis by Therapeutic Area
6.3.3. Analysis by Type of Vector Used
6.3.4. Analysis by Type of Therapy
6.3.5. Analysis by Type of Gene Delivery Method Used
6.3.6. Analysis by Route of Administration
6.4. Gene Therapy Market: Special Drug Designations
6.4.1. Analysis by Special Designation(s) Awarded
6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)
7. COMPETITIVE LANDSCAPE
7.1. Chapter Overview
7.2. Gene Therapy Market: List of Developers
7.2.1. Analysis by Year of Establishment
7.2.2. Analysis by Company Size
7.2.3. Analysis by Location of Headquarters
7.2.3.1. Analysis by Year of Establishment, Company Size and Location of Headquarters
7.2.3.2. Regional Landscape
7.3. Key Players: Analysis by Number of Pipeline Candidates
8. MARKETED GENE THERAPIES
8.1. Chapter Overview
8.2. Gendicine® (Shenzhen Sibiono GeneTech)
8.2.1. Company Overview
8.2.2. Development Timeline
8.2.3. Mechanism of Action and Vector Used
8.2.4. Target Indication(s)
8.2.5. Current Status of Development
8.2.6. Manufacturing, Dosage and Sales
8.3. Oncorine® (Shanghai Sunway Biotech)
8.3.1. Company Overview
8.3.2. Development Timeline
8.3.3. Mechanism of Action and Vector Used
8.3.4. Target Indication(s)
8.3.5. Current Status of Development
8.3.6. Manufacturing, Dosage and Sales
8.4. Rexin-G® (Epeius Biotechnologies)
8.4.1. Company Overview
8.4.2. Development Timeline
8.4.3. Mechanism of Action and Vector Used
8.4.4. Target Indication(s)
8.4.5. Current Status of Development
8.4.6. Manufacturing, Dosage and Sales
8.5. Neovasculgen® (Human Stem Cells Institute)
8.5.1. Company Overview
8.5.2. Development Timeline
8.5.3. Mechanism of Action and Vector Used
8.5.4. Target Indication(s)
8.5.5. Current Status of Development
8.5.6. Manufacturing, Dosage and Sales
8.6. Imlygic® (Amgen)
8.6.1. Company Overview
8.6.2. Development Timeline
8.6.3. Mechanism of Action and Vector Used
8.6.4. Target Indication(s)
8.6.5. Current Status of Development
8.6.6. Manufacturing, Dosage and Sales
8.7. Strimvelis® (Orchard Therapeutics)
8.7.1. Company Overview
8.7.2. Development Timeline
8.7.3. Mechanism of Action and Vector Used
8.7.4. Target Indication(s)
8.7.5. Current Status of Development
8.7.6. Manufacturing, Dosage and Sales
8.8. Luxturna™ (Spark Therapeutics)
8.8.1. Company Overview
8.8.2. Development Timeline
8.8.3. Mechanism of Action and Vector Used
8.8.4. Target Indication(s)
8.8.5. Current Status of Development
8.8.6. Manufacturing, Dosage and Sales
8.9. Zolgensma™ (Novartis)
8.9.1. Company Overview
8.9.2. Development Timeline
8.9.3. Mechanism of Action and Vector Used
8.9.4. Target Indication(s)
8.9.5. Current Status of Development
8.9.6. Manufacturing, Dosage and Sales
8.10. Collategene® (AnGes)
8.10.1. Company Overview
8.10.2. Development Timeline
8.10.3. Mechanism of Action and Vector Used
8.10.4. Target Indication(s)
8.10.5. Current Status of Development
8.10.6. Manufacturing, Dosage and Sales
8.11. Zyntelgo™ (bluebird bio)
8.11.1. Company Overview
8.11.2. Development Timeline
8.11.3. Mechanism of Action and Vector Used
8.11.4. Target Indication(s)
8.11.5. Current Status of Development
8.11.6. Manufacturing, Dosage and Sales
8.12. Libmeldy™ (Orchard Therapeutics)
8.12.1. Company Overview
8.12.2. Development Timeline
8.12.3. Mechanism of Action and Vector Used
8.12.4. Target Indication(s)
8.12.5. Current Status of Development
8.12.6. Manufacturing, Dosage and Sales
9. KEY COMMERCIALIZATION STRATEGIES
9.1. Chapter Overview
9.2. Successful Drug Launch Strategy: ROOTS Framework
9.3. Successful Drug Launch Strategy: Product Differentiation
9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product
9.5. List of Currently Approved Gene Therapies
9.6. Key Commercialization Strategies Adopted by Gene Therapy Developers
9.6.1. Strategies Adopted Before Therapy Approval
9.6.1.1. Participation in Global Events
9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms
9.6.1.3. Indication Expansion
9.6.2. Strategies Adopted During / Post Therapy Approval
9.6.2.1. Geographical Expansion
9.6.2.2. Participation in Global Events
9.6.2.3. Patience Assistance Programs
9.6.2.4. Awareness through Product Websites
9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms
9.7. Concluding Remarks
10. LATE STAGE (PHASE II / III AND ABOVE) GENE THERAPIES
10.1. Chapter Overview
10.2. Lumevoq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.3. OTL-103: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.4. PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.5. BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.6. rAd-IFN/Syn3: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.7. beti-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.8. eli-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.9. lovo-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.10. SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.11. EB-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.12. ProstAtak®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.13. D-Fi: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.14. CG0070: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.15. Vigil™-EWS: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.16. Engensis®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.17. VGX-3100: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.18. Invossa™ (TG-C): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.19. VYJUVEKT™: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.20. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.21. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.22. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.23. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.24. AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.25. VB-111: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.26. Generx®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.27. ADXS-HPV: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.28. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.29. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.30. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.31. AG0302-COVID19: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.32. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.33. Hologene 5: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
11. EMERGING TECHNOLOGIES
11.1. Chapter Overview
11.2. Gene Editing Technologies
11.2.1. Overview
11.2.2. Applications
11.3. Emerging Gene Editing Platforms
11.3.1. CRISPR / Cas9 System
11.3.1.1. Key Components and Functions
11.3.1.2. Mechanism of Action
11.3.1.3. Targeting Efficiency and Challenges
11.3.1.4. Next-GEN CRISPR Technology
11.3.1.5. Technology Providers
11.3.2. TALENs
11.3.2.1. Structural Features
11.3.2.2. Mechanism of Action
11.3.2.3. Advantages and Challenges
11.3.3. megaTAL
11.3.3.1. Technology Providers
11.3.4. Zinc Finger Nuclease
11.3.4.1. Technology Providers
11.4. Gene Expression Regulation Technologies
11.4.1. Technology Providers
11.5. Technology Platforms for Developing / Delivering Gene Therapies
12. KEY THERAPEUTICS AREAS
12.1. Chapter Overview
12.2. Analysis by Therapeutic Area and Special Designation(s) Awarded
12.3. Oncological Diseases
12.3.1. Analysis by Target Indication
12.3.2. Analysis by Type of Vector Used
12.4. Neurological Diseases
12.4.1. Analysis by Target Indication
12.4.2. Analysis by Type of Vector Used
12.5. Ophthalmic Diseases
12.5.1. Analysis by Target Indication
12.5.2. Analysis by Type of Vector Used
12.6. Metabolic Diseases
12.6.1. Analysis by Target Indication
12.6.2. Analysis by Type of Vector Used
12.7. Genetic Diseases
12.7.1. Analysis by Target Indication
12.7.2. Analysis by Type of Vector Used
13. PATENT ANALYSIS
13.1. Chapter Overview
13.2. Gene Therapy Market: Patent Analysis
13.2.1. Scope and Methodology
13.2.1.1. Analysis by Publication Year
13.2.1.2. Analysis by Publication Year and Type of Patent
13.2.1.3. Analysis by Geography
13.2.1.4. Analysis by CPC Symbols
13.2.1.5. Analysis by Emerging Focus Areas
13.2.1.6. Leading Players: Analysis by Number of Patents
13.2.1.7. Patent Benchmark Analysis
13.2.1.8. Patent Valuation Analysis
13.3. Gene Editing Market: Patent Analysis
13.3.1. Scope and Methodology
13.3.1.1. Analysis by Publication Year
13.3.1.2. Analysis by Publication Year and Type of Patent
13.3.1.3. Analysis by Geography
13.3.1.4. Analysis by CPC Symbols
13.3.1.5. Analysis by Emerging Focus Areas
13.3.1.6. Leading Players: Analysis by Number of Patents
13.3.1.7. Patent Benchmark Analysis
13.3.1.8. Patent Valuation Analysis
13.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization
14. MERGERS AND ACQUISITIONS
14.1. Chapter Overview
14.2. Merger and Acquisition Models
14.3. Gene Therapy Market: Mergers and Acquisitions
14.3.1. Analysis by Year of Merger / Acquisition
14.3.2. Analysis by Type of Agreement
14.3.3. Analysis by Geography
14.3.3.1. Continent-wise Distribution
14.3.3.2. Intercontinental and Intracontinental Deals
14.3.3.3. Local and International Deals
14.3.4. Analysis by Key Value Drivers
14.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
14.3.5. Analysis by Phase of Development of the Acquired Company’s Product
14.3.6. Analysis by Therapeutic Area
14.3.7. Analysis by Deal Multiples
15. FUNDING AND INVESTMENT ANALYSIS
15.1. Chapter Overview
15.2. Types of Funding
15.3. Gene Therapy Market: Funding and Investment Analysis
15.3.1. Analysis of Funding Instances by Year
15.3.2. Analysis of Amount Invested by Year
15.3.3. Analysis of Funding Instances and Amount Invested by Type of Funding
15.3.4. Analysis of Funding Instances and Amount Invested by Year and Type of Funding
15.3.5. Analysis of Funding Instances and Amount Invested by Type of Therapy
15.3.6. Analysis of Funding Instances and Amount Invested by Geography
15.3.7. Analysis of Funding Instances and Amount Invested by Highest Phase of Development
15.3.8. Analysis of Funding Instances by Therapeutic Area
15.3.9. Most Active Players: Analysis by Number of Funding Instances and Amount Invested
15.3.10. Key Investors: Analysis by Number of Funding Instances
15.4. Concluding Remarks
16. CLINICAL TRIAL ANALYSIS
16.1. Chapter Overview
16.2. Scope and Methodology
16.3. Gene Therapy Market: Clinical Trial Analysis
16.3.1. Analysis by Trial Registration Year
16.3.2. Analysis by Trial Status
16.3.3. Analysis by Trial Phase
16.3.4. Analysis by Therapeutic Area
16.3.5. Analysis by Geography
16.3.6. Analysis by Trial Registration Year and Geography
16.3.7. Analysis by Trial Status and Geography
16.3.8. Analysis by Trial Status, Trial Phase and Geography
16.3.9. Analysis by Therapeutic Area and Geography
16.4. Analysis by Type of Sponsor
16.5. Analysis by Prominent Treatment Sites
16.6. Gene Therapy Market: Analysis of Enrolled Patient Population
16.6.1. Analysis by Trial Registration Year
16.6.2. Analysis by Trial Status
16.6.3. Analysis by Trial Phase
16.6.4. Analysis by Therapeutic Area
16.6.5. Analysis by Location of Trial Site
16.6.6. Analysis by Trial Status and Location of Trial Site
16.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
16.6.8. Analysis by Therapeutic Area and Location of Trial Site
16.7. Concluding Remarks
16.7.1. Emerging Molecules to Watch
16.7.2. Most Important Trials to Watch
17. COST PRICE ANALYSIS
17.1. Chapter Overview
17.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
17.3. Gene Therapy Market: Pricing Models
17.3.1. Based on Associated Product / Component Costs
17.3.2. Based on Competition
17.3.3. Based on Patient Segment
17.3.4. Based on Opinions of Industry Experts
18. START-UP VALUATION
18.1. Chapter Overview
18.2. Valuation by Year of Experience
18.2.1. Methodology
18.2.2. Results and Interpretation
19. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES
19.1. Chapter Overview
19.2. Gene Therapy Market: List of Most Prominent Big Pharmaceutical Players
19.2.1. Analysis by Therapeutic Area
19.2.2. Analysis by Type of Vector Used
19.2.3. Analysis by Type of Therapy
19.2.4. Analysis by Type of Gene Delivery Method
19.3. Benchmark Analysis of Key Parameters
19.3.1. Spider Web Analysis: Pipeline Strength
19.3.2. Spider Web Analysis: Merger / Acquisitions
19.3.3. Spider Web Analysis: Funding and Investments
19.3.4. Spider Web Analysis: Clinical Trials
19.3.5. Spider Web Analysis: Technologies
19.3.6. Spider Web Analysis: Patents
19.4. Benchmark Analysis of Big Pharmaceutical Players
20. DEMAND ANALYSIS
20.1. Chapter Overview
20.2. Methodology
20.3. Global Demand for Gene Therapies, 2022-2035
20.3.1. Analysis by Type of Therapy
20.3.2. Analysis by Therapeutic Area
20.3.3. Analysis by Geography
21. MARKET FORECAST AND OPPORTUNITY ANALYSIS
21.1. Chapter Overview
21.2. Scope and Limitations
21.3. Key Assumptions and Forecast Methodology
21.4. Global Gene Therapy Market, 2022-2035
21.4.1. Gene Therapy Market: Analysis by Therapeutic Area
21.4.2. Gene Therapy Market: Analysis by Type of Vector Used
21.4.3. Gene Therapy Market: Analysis by Type of Therapy
21.4.4. Gene Therapy Market: Analysis by Gene Delivery Method Used
21.4.5. Gene Therapy Market: Analysis by Route of Administration
21.4.6. Gene Therapy Market: Analysis by Geography
21.5. Gene Therapy Market: Value Creation Analysis
21.6. Gene Therapy Market: Product-wise Sales Forecasts
21.6.1. Gendicine®
21.6.1.1. Target Patient Population
21.6.1.2. Sales Forecast
21.6.1.3. Net Present Value
21.6.1.4. Value Creation Analysis
21.6.2. Oncorine®
21.6.2.1. Target Patient Population
21.6.2.2. Sales Forecast
21.6.2.3. Net Present Value
21.6.2.4. Value Creation Analysis
21.6.3. Rexin-G®
21.6.3.1. Target Patient Population
21.6.3.2. Sales Forecast
21.6.3.3. Net Present Value
21.6.3.4. Value Creation Analysis
21.6.4. Neovasculgen®
21.6.4.1. Target Patient Population
21.6.4.2. Sales Forecast
21.6.4.3. Net Present Value
21.6.4.4. Value Creation Analysis
21.6.5. Strimvelis®
21.6.5.1. Target Patient Population
21.6.5.2. Sales Forecast
21.6.5.3. Net Present Value
21.6.5.4. Value Creation Analysis
21.6.6. Imlygic®
21.6.6.1. Target Patient Population
21.6.6.2. Sales Forecast
21.6.6.3. Net Present Value
21.6.6.4. Value Creation Analysis
21.6.7. Luxturna™
21.6.7.1. Target Patient Population
21.6.7.2. Sales Forecast
21.6.7.3. Net Present Value
21.6.7.4. Value Creation Analysis
21.6.8. Zolgensma™
21.6.8.1. Target Patient Population
21.6.8.2. Sales Forecast
21.6.8.3. Net Present Value
21.6.8.4. Value Creation Analysis
21.6.9. Collategene®
21.6.9.1. Target Patient Population
21.6.9.2. Sales Forecast
21.6.9.3. Net Present Value
21.6.9.4. Value Creation Analysis
21.6.10. Libmeldy™
21.6.10.1. Target Patient Population
21.6.10.2. Sales Forecast
21.6.10.3. Net Present Value
21.6.10.4. Value Creation Analysis
21.6.11. Lumevoq® (GS010)
21.6.11.1. Target Patient Population
21.6.11.2. Sales Forecast
21.6.11.3. Net Present Value
21.6.11.4. Value Creation Analysis
21.6.12. OTL-103
21.6.12.1. Target Patient Population
21.6.12.2. Sales Forecast
21.6.12.3. Net Present Value
21.6.12.4. Value Creation Analysis
21.6.13. PTC-AADC
21.6.13.1. Target Patient Population
21.6.13.2. Sales Forecast
21.6.13.3. Net Present Value
21.6.13.4. Value Creation Analysis
21.6.14. BMN
21.6.14.1. Target Patient Population
21.6.14.2. Sales Forecast
21.6.14.3. Net Present Value
21.6.14.4. Value Creation Analysis
21.6.15. rAd-IFN/Syn3
21.6.15.1. Target Patient Population
21.6.15.2. Sales Forecast
21.6.15.3. Net Present Value
21.6.15.4. Value Creation Analysis
21.6.16. beti-cel
21.6.16.1. Target Patient Population
21.6.16.2. Sales Forecast
21.6.16.3. Net Present Value
21.6.16.4. Value Creation Analysis
21.6.17. eli-cel
21.6.17.1. Target Patient Population
21.6.17.2. Sales Forecast
21.6.17.3. Net Present Value
21.6.17.4. Value Creation Analysis
21.6.18. lovo-cel
21.6.18.1. Target Patient Population
21.6.18.2. Sales Forecast
21.6.18.3. Net Present Value
21.6.18.4. Value Creation Analysis
21.6.19. SRP-9001
21.6.19.1. Target Patient Population
21.6.19.2. Sales Forecast
21.6.19.3. Net Present Value
21.6.19.4. Value Creation Analysis
21.6.20. EB-101
21.6.20.1. Target Patient Population
21.6.20.2. Sales Forecast
21.6.20.3. Net Present Value
21.6.20.4. Value Creation Analysis
21.6.21. ProstAtak®
21.6.21.1. Target Patient Population
21.6.21.2. Sales Forecast
21.6.21.3. Net Present Value
21.6.21.4. Value Creation Analysis
21.6.22. D-Fi
21.6.22.1. Target Patient Population
21.6.22.2. Sales Forecast
21.6.22.3. Net Present Value
21.6.22.4. Value Creation Analysis
21.6.23. CG0070
21.6.23.1. Target Patient Population
21.6.23.2. Sales Forecast
21.6.23.3. Net Present Value
21.6.23.4. Value Creation Analysis
21.6.24. Vigil™-EWS
21.6.24.1. Target Patient Population
21.6.24.2. Sales Forecast
21.6.24.3. Net Present Value
21.6.24.4. Value Creation Analysis
21.6.25. Engensis®
21.6.25.1. Target Patient Population
21.6.25.2. Sales Forecast
21.6.25.3. Net Present Value
21.6.25.4. Value Creation Analysis
21.6.26. VGX-3100
21.6.26.1. Target Patient Population
21.6.26.2. Sales Forecast
21.6. 26.3. Net Present Value
21.6.26.4. Value Creation Analysis
21.6.27. Invossa™ (TG-C)
21.6.27.1. Target Patient Population
21.6.27.2. Sales Forecast
21.6.27.3. Net Present Value
21.6.27.4. Value Creation Analysis
21.6.28. VYJUVEKT™
21.6.28.1. Target Patient Population
21.6.28.2. Sales Forecast
21.6.28.3. Net Present Value
21.6.28.4. Value Creation Analysis
21.6.29. PF-06939926
21.6.29.1. Target Patient Population
21.6.29.2. Sales Forecast
21.6.29.3. Net Present Value
21.6.29.4. Value Creation Analysis
21.6.30. PF-06838435
21.6.30.1. Target Patient Population
21.6.30.2. Sales Forecast
21.6.30.3. Net Present Value
21.6.30.4. Value Creation Analysis
21.6.31. PF-07055480
21.6.31.1. Target Patient Population
21.6.31.2. Sales Forecast
21.6.31.3. Net Present Value
21.6.31.4. Value Creation Analysis
21.6.32. SPK-8011
21.6.32.1. Target Patient Population
21.6.32.2. Sales Forecast
21.6.32.3. Net Present Value
21.6.32.4. Value Creation Analysis
21.6.33. AMT-061
21.6.33.1. Target Patient Population
21.6.33.2. Sales Forecast
21.6.33.3. Net Present Value
21.6.33.4. Value Creation Analysis
21.6.34. VB-111
21.6.34.1. Target Patient Population
21.6.34.2. Sales Forecast
21.6.34.3. Net Present Value
21.6.34.4. Value Creation Analysis
21.6.35. Generx®
21.6.35.1. Target Patient Population
21.6.35.2. Sales Forecast
21.6.35.3. Net Present Value
21.6.35.4. Value Creation Analysis
21.6.36. AMG001
21.6.36.1. Target Patient Population
21.6.36.2. Sales Forecast
21.6.36.3. Net Present Value
21.6.36.4. Value Creation Analysis
21.6.37. OAV-101
21.6.37.1. Target Patient Population
21.6.37.2. Sales Forecast
21.6.37.3. Net Present Value
21.6.37.4. Value Creation Analysis
21.6.38. ADXS-HPV
21.6.38.1. Target Patient Population
21.6.38.2. Sales Forecast
21.6.38.3. Net Present Value
21.6.38.4. Value Creation Analysis
21.6.39. AGTC
21.6.39.1. Target Patient Population
21.6.39.2. Sales Forecast
21.6.39.3. Net Present Value
21.6.39.4. Value Creation Analysis
21.6.40. LYS-SAF302
21.6.40.1. Target Patient Population
21.6.40.2. Sales Forecast
21.6.40.3. Net Present Value
21.6.40.4. Value Creation Analysis
21.6.41. NFS-01
21.6.41.1. Target Patient Population
21.6.41.2. Sales Forecast
21.6.41.3. Net Present Value
21.6.41.4. Value Creation Analysis
21.6.42. AG0302-COVID19
21.6.42.1. Target Patient Population
21.6.42.2. Sales Forecast
21.6.42.3. Net Present Value
21.6.42.4. Value Creation Analysis
21.6.43. RGX-314
21.6.43.1. Target Patient Population
21.6.43.2. Sales Forecast
21.6.43.3. Net Present Value
21.6.43.4. Value Creation Analysis
21.6.44. Hologene
21.6.44.1. Target Patient Population
21.6.44.2. Sales Forecast
21.6.44.3. Net Present Value
21.6.44.4. Value Creation Analysis
22. VECTOR MANUFACTURING
22.1. Chapter Overview
22.2. Overview of Viral Vector Manufacturing
22.3. Viral Vector Manufacturing Process
22.3.1. Mode of Vector Production
22.3.2. Adherent and Suspension Cultures
22.3.3. Unit Processes and Multiple Parallel Processes
22.3.4. Cell Culture Systems for Production of Viral Vectors
22.3.4.1. Small Scale / Laboratory Scale Cell Culture Systems
22.3.4.2. Large Scale Cell Culture Systems
22.3.5. Culture Media Specifications
22.4. Bioprocessing of Viral Vectors
22.4.1. AAV Vector Production
22.4.2. Adenoviral Vector Production
22.4.3. Lentiviral Vector Production
22.4.4. ? -Retroviral Vector Production
22.5. Challenges Associated with Vector Manufacturing
22.6. Contract Service Providers for Viral and Plasmid Vectors
23. CASE STUDY: GENE THERAPY SUPPLY CHAIN
23.1. Chapter Overview
23.2. Overview of Gene Therapy Supply Chain
23.3. Implementation of Supply Chain Models
23.4. Logistics in Gene Therapies
23.4.1. Logistic Processes for Autologous and Allogeneic Therapies
23.5. Regulatory Supply Chain Across the Globe
23.6. Challenges Associated with Gene Therapy Supply Chain
23.7. Optimizing Cell and Advanced Therapies Supply Chain Management
23.7.1. Enterprise Manufacturing System
23.7.2. Laboratory Information Management System
23.7.3. Inventory Management System
23.7.4. Quality Management System
23.7.5. Logistics Management System
23.7.6. Patient Management System
23.7.7. Electronic Clinical Outcome Assessments System
23.7.8. Supply Chain Orchestration Platform
23.8. Recent Developments and Upcoming Trends
24. CONCLUSION
24.1. Chapter Overview
25. INTERVIEW TRANSCRIPTS
25.1. Chapter Overview
25.2. Buel Dan Rodgers (Founder and CEO, AAVogen)
25.3. Sue Washer (President and CEO, AGTC)
25.4. Patricia Zilliox (President and CEO, Eyevensys)
25.5. Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))
25.6. Adam Rogers (CEO, Hemera Biosciences)
25.7. Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))
25.8. Al Hawkins (CEO, Milo Biotechnology)
25.9. Jean-Phillipe Combal (CEO, Vivet Therapeutics)
25.10. Robert Jan Lamers (former CEO, Arthrogen)
25.11. Tom Wilton (former CBO, LogicBio Therapeutics)
25.12. Michael Tripletti (former CEO, Myonexus Therapeutics)
25.13. Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)
25.14. Cedric Szpirer (former Executive and Scientific Director, Delphi Genetics)
25.15. Marco Schmeer (Project Manager) and Tatjana Buchholz, (former Marketing Manager, PlasmidFactory)
25.16. Jeffrey Hung (CCO, Vigene Biosciences)
26. APPENDIX 1: TABULATED DATA
27. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
1.1. Scope of the Report
1.2. Research Methodology
1.3. Key Questions Answered
1.4. Chapter Outlines
2. EXECUTIVE SUMMARY
2.1. Chapter Overview
3. INTRODUCTION
3.1. Context and Background
3.2. Evolution of Gene Therapies
3.3. Classification of Gene Therapies
3.3.1. Somatic and Germline Gene Therapies
3.3.2. Ex Vivo and In Vivo Gene Therapies
3.4. Routes of Administration
3.5. Mechanism of Action
3.6. Overview of Gene Editing
3.6.1. Evolution of Genome Editing
3.6.2. Applications of Genome Editing
3.6.3. Available Genome Editing Techniques
3.7. Advantages and Disadvantages of Gene Therapies
3.7.1 Ethical and Social Concerns Related to Gene Therapies
3.7.2. Constraints and Challenges Related to Gene Therapies
3.7.3. Therapy Development Concerns
3.7.4. Manufacturing Concerns
3.7.5. Commercial Viability Concerns
4. GENE DELIVERY VECTORS
4.1. Chapter Overview
4.2. Viral and Non-Viral Methods of Gene Transfer
4.3. Viral Vectors for Genetically Modified Therapies
4.4. Types of Viral Vectors
4.4.1. Adeno-associated Viral Vectors
4.4.1.1. Overview
4.4.1.2. Design and Manufacturing
4.4.1.3. Advantages Offered
4.4.1.4. Associated Limitations
4.4.2. Adenoviral Vectors
4.4.2.1. Overview
4.4.2.2. Design and Manufacturing
4.4.2.3. Advantages Offered
4.4.2.4. Associated Limitations
4.4.3. Lentiviral Vectors
4.4.3.1. Overview
4.4.3.2. Design and Manufacturing
4.4.3.3. Advantages Offered
4.4.3.4. Associated Limitations
4.4.4. Retroviral Vectors
4.4.4.1. Overview
4.4.4.2. Design and Manufacturing
4.4.4.3. Advantages Offered
4.4.4.4. Associated Limitations
4.4.5. Other Viral Vectors
4.4.5.1. Alphavirus
4.4.5.2. Foamy Virus
4.4.5.3. Herpes Simplex Virus
4.4.5.4. Sendai Virus
4.4.5.5. Simian Virus
4.4.5.6. Vaccinia Virus
4.5. Types of Non-Viral Vectors
4.5.1. Plasmid DNA
4.5.2. Liposomes, Lipoplexes and Polyplexes
4.5.3. Oligonucleotides
4.5.4. Nanoparticles
4.5.5. Hybrid Vector Systems
4.5.6. Other Non-Viral Vectors
4.5.7. Gene Delivery Using Non-Viral Vectors
4.5.7.1. Biolistic Methods
4.5.7.2. Electroporation
4.5.7.3. Receptor Mediated Gene Delivery
4.5.7.4. Gene Activated Matrix (GAM)
5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO
5.1. Chapter Overview
5.2. Regulatory Guidelines in North America
5.2.1. The US Scenario
5.2.2. Canadian Scenario
5.3. Regulatory Guidelines in Europe
5.3.1. Quality of Gene Therapy Products
5.3.2. Non-Clinical Development
5.3.3. Clinical Development
5.4. Regulatory Guidelines in Asia-Pacific
5.4.1. Chinese Scenario
5.4.1.1. Construction of DNA Expression Cassette and Gene Delivery Systems
5.4.1.2. Generation and Characterization of Cell Banks and Engineered Bacteria Banks
5.4.1.3. Manufacturing of Gene Therapy Products
5.4.1.4. Quality Control
5.4.1.5. Evaluation of Efficacy of Gene Therapy Products
5.4.1.6. Safety Evaluation of Gene Therapy Products
5.4.1.7. Clinical Trial of Gene Therapy Products
5.4.1.8. Ethics Study
5.4.2. Japanese Scenario
5.4.3. South Korean Scenario
5.4.4. Australian Scenario
5.4.5. Hong Kong Scenario
5.5. Reimbursement Scenario
5.5.1. Challenges Related to Reimbursement
5.6. Commonly Offered Payment Models for Gene Therapies
6. MARKET OVERVIEW
6.1. Chapter Overview
6.1.1. Analysis of Gene Therapy Candidates by Stage of Development
6.2. Gene Therapy Market: Clinical and Commercial Pipeline
6.2.1. Analysis by Phase of Development
6.2.2. Analysis by Therapeutic Area
6.2.3. Analysis by Type of Vector Used
6.2.4. Analysis by Type of Gene / Molecule Targeted
6.2.5. Analysis by Type of Therapy
6.2.6. Analysis by Type of Gene Delivery Method Used
6.2.7. Analysis by Route of Administration
6.3. Gene Therapy Market: Early-Stage Pipeline
6.3.1. Analysis by Phase of Development
6.3.2. Analysis by Therapeutic Area
6.3.3. Analysis by Type of Vector Used
6.3.4. Analysis by Type of Therapy
6.3.5. Analysis by Type of Gene Delivery Method Used
6.3.6. Analysis by Route of Administration
6.4. Gene Therapy Market: Special Drug Designations
6.4.1. Analysis by Special Designation(s) Awarded
6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)
7. COMPETITIVE LANDSCAPE
7.1. Chapter Overview
7.2. Gene Therapy Market: List of Developers
7.2.1. Analysis by Year of Establishment
7.2.2. Analysis by Company Size
7.2.3. Analysis by Location of Headquarters
7.2.3.1. Analysis by Year of Establishment, Company Size and Location of Headquarters
7.2.3.2. Regional Landscape
7.3. Key Players: Analysis by Number of Pipeline Candidates
8. MARKETED GENE THERAPIES
8.1. Chapter Overview
8.2. Gendicine® (Shenzhen Sibiono GeneTech)
8.2.1. Company Overview
8.2.2. Development Timeline
8.2.3. Mechanism of Action and Vector Used
8.2.4. Target Indication(s)
8.2.5. Current Status of Development
8.2.6. Manufacturing, Dosage and Sales
8.3. Oncorine® (Shanghai Sunway Biotech)
8.3.1. Company Overview
8.3.2. Development Timeline
8.3.3. Mechanism of Action and Vector Used
8.3.4. Target Indication(s)
8.3.5. Current Status of Development
8.3.6. Manufacturing, Dosage and Sales
8.4. Rexin-G® (Epeius Biotechnologies)
8.4.1. Company Overview
8.4.2. Development Timeline
8.4.3. Mechanism of Action and Vector Used
8.4.4. Target Indication(s)
8.4.5. Current Status of Development
8.4.6. Manufacturing, Dosage and Sales
8.5. Neovasculgen® (Human Stem Cells Institute)
8.5.1. Company Overview
8.5.2. Development Timeline
8.5.3. Mechanism of Action and Vector Used
8.5.4. Target Indication(s)
8.5.5. Current Status of Development
8.5.6. Manufacturing, Dosage and Sales
8.6. Imlygic® (Amgen)
8.6.1. Company Overview
8.6.2. Development Timeline
8.6.3. Mechanism of Action and Vector Used
8.6.4. Target Indication(s)
8.6.5. Current Status of Development
8.6.6. Manufacturing, Dosage and Sales
8.7. Strimvelis® (Orchard Therapeutics)
8.7.1. Company Overview
8.7.2. Development Timeline
8.7.3. Mechanism of Action and Vector Used
8.7.4. Target Indication(s)
8.7.5. Current Status of Development
8.7.6. Manufacturing, Dosage and Sales
8.8. Luxturna™ (Spark Therapeutics)
8.8.1. Company Overview
8.8.2. Development Timeline
8.8.3. Mechanism of Action and Vector Used
8.8.4. Target Indication(s)
8.8.5. Current Status of Development
8.8.6. Manufacturing, Dosage and Sales
8.9. Zolgensma™ (Novartis)
8.9.1. Company Overview
8.9.2. Development Timeline
8.9.3. Mechanism of Action and Vector Used
8.9.4. Target Indication(s)
8.9.5. Current Status of Development
8.9.6. Manufacturing, Dosage and Sales
8.10. Collategene® (AnGes)
8.10.1. Company Overview
8.10.2. Development Timeline
8.10.3. Mechanism of Action and Vector Used
8.10.4. Target Indication(s)
8.10.5. Current Status of Development
8.10.6. Manufacturing, Dosage and Sales
8.11. Zyntelgo™ (bluebird bio)
8.11.1. Company Overview
8.11.2. Development Timeline
8.11.3. Mechanism of Action and Vector Used
8.11.4. Target Indication(s)
8.11.5. Current Status of Development
8.11.6. Manufacturing, Dosage and Sales
8.12. Libmeldy™ (Orchard Therapeutics)
8.12.1. Company Overview
8.12.2. Development Timeline
8.12.3. Mechanism of Action and Vector Used
8.12.4. Target Indication(s)
8.12.5. Current Status of Development
8.12.6. Manufacturing, Dosage and Sales
9. KEY COMMERCIALIZATION STRATEGIES
9.1. Chapter Overview
9.2. Successful Drug Launch Strategy: ROOTS Framework
9.3. Successful Drug Launch Strategy: Product Differentiation
9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product
9.5. List of Currently Approved Gene Therapies
9.6. Key Commercialization Strategies Adopted by Gene Therapy Developers
9.6.1. Strategies Adopted Before Therapy Approval
9.6.1.1. Participation in Global Events
9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms
9.6.1.3. Indication Expansion
9.6.2. Strategies Adopted During / Post Therapy Approval
9.6.2.1. Geographical Expansion
9.6.2.2. Participation in Global Events
9.6.2.3. Patience Assistance Programs
9.6.2.4. Awareness through Product Websites
9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms
9.7. Concluding Remarks
10. LATE STAGE (PHASE II / III AND ABOVE) GENE THERAPIES
10.1. Chapter Overview
10.2. Lumevoq® (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.3. OTL-103: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.4. PTC-AADC: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.5. BMN 270: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.6. rAd-IFN/Syn3: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.7. beti-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.8. eli-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.9. lovo-cel: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.10. SRP-9001: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.11. EB-101: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.12. ProstAtak®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.13. D-Fi: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.14. CG0070: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.15. Vigil™-EWS: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.16. Engensis®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.17. VGX-3100: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.18. Invossa™ (TG-C): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.19. VYJUVEKT™: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.20. PF-06939926: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.21. PF-06838435: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.22. PF-07055480: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.23. SPK-8011: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.24. AMT-061: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.25. VB-111: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.26. Generx®: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.27. ADXS-HPV: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.28. AGTC 501: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.29. LYS-SAF302: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.30. NFS-01: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.31. AG0302-COVID19: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.32. RGX-314: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
10.33. Hologene 5: Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results
11. EMERGING TECHNOLOGIES
11.1. Chapter Overview
11.2. Gene Editing Technologies
11.2.1. Overview
11.2.2. Applications
11.3. Emerging Gene Editing Platforms
11.3.1. CRISPR / Cas9 System
11.3.1.1. Key Components and Functions
11.3.1.2. Mechanism of Action
11.3.1.3. Targeting Efficiency and Challenges
11.3.1.4. Next-GEN CRISPR Technology
11.3.1.5. Technology Providers
11.3.2. TALENs
11.3.2.1. Structural Features
11.3.2.2. Mechanism of Action
11.3.2.3. Advantages and Challenges
11.3.3. megaTAL
11.3.3.1. Technology Providers
11.3.4. Zinc Finger Nuclease
11.3.4.1. Technology Providers
11.4. Gene Expression Regulation Technologies
11.4.1. Technology Providers
11.5. Technology Platforms for Developing / Delivering Gene Therapies
12. KEY THERAPEUTICS AREAS
12.1. Chapter Overview
12.2. Analysis by Therapeutic Area and Special Designation(s) Awarded
12.3. Oncological Diseases
12.3.1. Analysis by Target Indication
12.3.2. Analysis by Type of Vector Used
12.4. Neurological Diseases
12.4.1. Analysis by Target Indication
12.4.2. Analysis by Type of Vector Used
12.5. Ophthalmic Diseases
12.5.1. Analysis by Target Indication
12.5.2. Analysis by Type of Vector Used
12.6. Metabolic Diseases
12.6.1. Analysis by Target Indication
12.6.2. Analysis by Type of Vector Used
12.7. Genetic Diseases
12.7.1. Analysis by Target Indication
12.7.2. Analysis by Type of Vector Used
13. PATENT ANALYSIS
13.1. Chapter Overview
13.2. Gene Therapy Market: Patent Analysis
13.2.1. Scope and Methodology
13.2.1.1. Analysis by Publication Year
13.2.1.2. Analysis by Publication Year and Type of Patent
13.2.1.3. Analysis by Geography
13.2.1.4. Analysis by CPC Symbols
13.2.1.5. Analysis by Emerging Focus Areas
13.2.1.6. Leading Players: Analysis by Number of Patents
13.2.1.7. Patent Benchmark Analysis
13.2.1.8. Patent Valuation Analysis
13.3. Gene Editing Market: Patent Analysis
13.3.1. Scope and Methodology
13.3.1.1. Analysis by Publication Year
13.3.1.2. Analysis by Publication Year and Type of Patent
13.3.1.3. Analysis by Geography
13.3.1.4. Analysis by CPC Symbols
13.3.1.5. Analysis by Emerging Focus Areas
13.3.1.6. Leading Players: Analysis by Number of Patents
13.3.1.7. Patent Benchmark Analysis
13.3.1.8. Patent Valuation Analysis
13.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization
14. MERGERS AND ACQUISITIONS
14.1. Chapter Overview
14.2. Merger and Acquisition Models
14.3. Gene Therapy Market: Mergers and Acquisitions
14.3.1. Analysis by Year of Merger / Acquisition
14.3.2. Analysis by Type of Agreement
14.3.3. Analysis by Geography
14.3.3.1. Continent-wise Distribution
14.3.3.2. Intercontinental and Intracontinental Deals
14.3.3.3. Local and International Deals
14.3.4. Analysis by Key Value Drivers
14.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
14.3.5. Analysis by Phase of Development of the Acquired Company’s Product
14.3.6. Analysis by Therapeutic Area
14.3.7. Analysis by Deal Multiples
15. FUNDING AND INVESTMENT ANALYSIS
15.1. Chapter Overview
15.2. Types of Funding
15.3. Gene Therapy Market: Funding and Investment Analysis
15.3.1. Analysis of Funding Instances by Year
15.3.2. Analysis of Amount Invested by Year
15.3.3. Analysis of Funding Instances and Amount Invested by Type of Funding
15.3.4. Analysis of Funding Instances and Amount Invested by Year and Type of Funding
15.3.5. Analysis of Funding Instances and Amount Invested by Type of Therapy
15.3.6. Analysis of Funding Instances and Amount Invested by Geography
15.3.7. Analysis of Funding Instances and Amount Invested by Highest Phase of Development
15.3.8. Analysis of Funding Instances by Therapeutic Area
15.3.9. Most Active Players: Analysis by Number of Funding Instances and Amount Invested
15.3.10. Key Investors: Analysis by Number of Funding Instances
15.4. Concluding Remarks
16. CLINICAL TRIAL ANALYSIS
16.1. Chapter Overview
16.2. Scope and Methodology
16.3. Gene Therapy Market: Clinical Trial Analysis
16.3.1. Analysis by Trial Registration Year
16.3.2. Analysis by Trial Status
16.3.3. Analysis by Trial Phase
16.3.4. Analysis by Therapeutic Area
16.3.5. Analysis by Geography
16.3.6. Analysis by Trial Registration Year and Geography
16.3.7. Analysis by Trial Status and Geography
16.3.8. Analysis by Trial Status, Trial Phase and Geography
16.3.9. Analysis by Therapeutic Area and Geography
16.4. Analysis by Type of Sponsor
16.5. Analysis by Prominent Treatment Sites
16.6. Gene Therapy Market: Analysis of Enrolled Patient Population
16.6.1. Analysis by Trial Registration Year
16.6.2. Analysis by Trial Status
16.6.3. Analysis by Trial Phase
16.6.4. Analysis by Therapeutic Area
16.6.5. Analysis by Location of Trial Site
16.6.6. Analysis by Trial Status and Location of Trial Site
16.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
16.6.8. Analysis by Therapeutic Area and Location of Trial Site
16.7. Concluding Remarks
16.7.1. Emerging Molecules to Watch
16.7.2. Most Important Trials to Watch
17. COST PRICE ANALYSIS
17.1. Chapter Overview
17.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
17.3. Gene Therapy Market: Pricing Models
17.3.1. Based on Associated Product / Component Costs
17.3.2. Based on Competition
17.3.3. Based on Patient Segment
17.3.4. Based on Opinions of Industry Experts
18. START-UP VALUATION
18.1. Chapter Overview
18.2. Valuation by Year of Experience
18.2.1. Methodology
18.2.2. Results and Interpretation
19. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES
19.1. Chapter Overview
19.2. Gene Therapy Market: List of Most Prominent Big Pharmaceutical Players
19.2.1. Analysis by Therapeutic Area
19.2.2. Analysis by Type of Vector Used
19.2.3. Analysis by Type of Therapy
19.2.4. Analysis by Type of Gene Delivery Method
19.3. Benchmark Analysis of Key Parameters
19.3.1. Spider Web Analysis: Pipeline Strength
19.3.2. Spider Web Analysis: Merger / Acquisitions
19.3.3. Spider Web Analysis: Funding and Investments
19.3.4. Spider Web Analysis: Clinical Trials
19.3.5. Spider Web Analysis: Technologies
19.3.6. Spider Web Analysis: Patents
19.4. Benchmark Analysis of Big Pharmaceutical Players
20. DEMAND ANALYSIS
20.1. Chapter Overview
20.2. Methodology
20.3. Global Demand for Gene Therapies, 2022-2035
20.3.1. Analysis by Type of Therapy
20.3.2. Analysis by Therapeutic Area
20.3.3. Analysis by Geography
21. MARKET FORECAST AND OPPORTUNITY ANALYSIS
21.1. Chapter Overview
21.2. Scope and Limitations
21.3. Key Assumptions and Forecast Methodology
21.4. Global Gene Therapy Market, 2022-2035
21.4.1. Gene Therapy Market: Analysis by Therapeutic Area
21.4.2. Gene Therapy Market: Analysis by Type of Vector Used
21.4.3. Gene Therapy Market: Analysis by Type of Therapy
21.4.4. Gene Therapy Market: Analysis by Gene Delivery Method Used
21.4.5. Gene Therapy Market: Analysis by Route of Administration
21.4.6. Gene Therapy Market: Analysis by Geography
21.5. Gene Therapy Market: Value Creation Analysis
21.6. Gene Therapy Market: Product-wise Sales Forecasts
21.6.1. Gendicine®
21.6.1.1. Target Patient Population
21.6.1.2. Sales Forecast
21.6.1.3. Net Present Value
21.6.1.4. Value Creation Analysis
21.6.2. Oncorine®
21.6.2.1. Target Patient Population
21.6.2.2. Sales Forecast
21.6.2.3. Net Present Value
21.6.2.4. Value Creation Analysis
21.6.3. Rexin-G®
21.6.3.1. Target Patient Population
21.6.3.2. Sales Forecast
21.6.3.3. Net Present Value
21.6.3.4. Value Creation Analysis
21.6.4. Neovasculgen®
21.6.4.1. Target Patient Population
21.6.4.2. Sales Forecast
21.6.4.3. Net Present Value
21.6.4.4. Value Creation Analysis
21.6.5. Strimvelis®
21.6.5.1. Target Patient Population
21.6.5.2. Sales Forecast
21.6.5.3. Net Present Value
21.6.5.4. Value Creation Analysis
21.6.6. Imlygic®
21.6.6.1. Target Patient Population
21.6.6.2. Sales Forecast
21.6.6.3. Net Present Value
21.6.6.4. Value Creation Analysis
21.6.7. Luxturna™
21.6.7.1. Target Patient Population
21.6.7.2. Sales Forecast
21.6.7.3. Net Present Value
21.6.7.4. Value Creation Analysis
21.6.8. Zolgensma™
21.6.8.1. Target Patient Population
21.6.8.2. Sales Forecast
21.6.8.3. Net Present Value
21.6.8.4. Value Creation Analysis
21.6.9. Collategene®
21.6.9.1. Target Patient Population
21.6.9.2. Sales Forecast
21.6.9.3. Net Present Value
21.6.9.4. Value Creation Analysis
21.6.10. Libmeldy™
21.6.10.1. Target Patient Population
21.6.10.2. Sales Forecast
21.6.10.3. Net Present Value
21.6.10.4. Value Creation Analysis
21.6.11. Lumevoq® (GS010)
21.6.11.1. Target Patient Population
21.6.11.2. Sales Forecast
21.6.11.3. Net Present Value
21.6.11.4. Value Creation Analysis
21.6.12. OTL-103
21.6.12.1. Target Patient Population
21.6.12.2. Sales Forecast
21.6.12.3. Net Present Value
21.6.12.4. Value Creation Analysis
21.6.13. PTC-AADC
21.6.13.1. Target Patient Population
21.6.13.2. Sales Forecast
21.6.13.3. Net Present Value
21.6.13.4. Value Creation Analysis
21.6.14. BMN
21.6.14.1. Target Patient Population
21.6.14.2. Sales Forecast
21.6.14.3. Net Present Value
21.6.14.4. Value Creation Analysis
21.6.15. rAd-IFN/Syn3
21.6.15.1. Target Patient Population
21.6.15.2. Sales Forecast
21.6.15.3. Net Present Value
21.6.15.4. Value Creation Analysis
21.6.16. beti-cel
21.6.16.1. Target Patient Population
21.6.16.2. Sales Forecast
21.6.16.3. Net Present Value
21.6.16.4. Value Creation Analysis
21.6.17. eli-cel
21.6.17.1. Target Patient Population
21.6.17.2. Sales Forecast
21.6.17.3. Net Present Value
21.6.17.4. Value Creation Analysis
21.6.18. lovo-cel
21.6.18.1. Target Patient Population
21.6.18.2. Sales Forecast
21.6.18.3. Net Present Value
21.6.18.4. Value Creation Analysis
21.6.19. SRP-9001
21.6.19.1. Target Patient Population
21.6.19.2. Sales Forecast
21.6.19.3. Net Present Value
21.6.19.4. Value Creation Analysis
21.6.20. EB-101
21.6.20.1. Target Patient Population
21.6.20.2. Sales Forecast
21.6.20.3. Net Present Value
21.6.20.4. Value Creation Analysis
21.6.21. ProstAtak®
21.6.21.1. Target Patient Population
21.6.21.2. Sales Forecast
21.6.21.3. Net Present Value
21.6.21.4. Value Creation Analysis
21.6.22. D-Fi
21.6.22.1. Target Patient Population
21.6.22.2. Sales Forecast
21.6.22.3. Net Present Value
21.6.22.4. Value Creation Analysis
21.6.23. CG0070
21.6.23.1. Target Patient Population
21.6.23.2. Sales Forecast
21.6.23.3. Net Present Value
21.6.23.4. Value Creation Analysis
21.6.24. Vigil™-EWS
21.6.24.1. Target Patient Population
21.6.24.2. Sales Forecast
21.6.24.3. Net Present Value
21.6.24.4. Value Creation Analysis
21.6.25. Engensis®
21.6.25.1. Target Patient Population
21.6.25.2. Sales Forecast
21.6.25.3. Net Present Value
21.6.25.4. Value Creation Analysis
21.6.26. VGX-3100
21.6.26.1. Target Patient Population
21.6.26.2. Sales Forecast
21.6. 26.3. Net Present Value
21.6.26.4. Value Creation Analysis
21.6.27. Invossa™ (TG-C)
21.6.27.1. Target Patient Population
21.6.27.2. Sales Forecast
21.6.27.3. Net Present Value
21.6.27.4. Value Creation Analysis
21.6.28. VYJUVEKT™
21.6.28.1. Target Patient Population
21.6.28.2. Sales Forecast
21.6.28.3. Net Present Value
21.6.28.4. Value Creation Analysis
21.6.29. PF-06939926
21.6.29.1. Target Patient Population
21.6.29.2. Sales Forecast
21.6.29.3. Net Present Value
21.6.29.4. Value Creation Analysis
21.6.30. PF-06838435
21.6.30.1. Target Patient Population
21.6.30.2. Sales Forecast
21.6.30.3. Net Present Value
21.6.30.4. Value Creation Analysis
21.6.31. PF-07055480
21.6.31.1. Target Patient Population
21.6.31.2. Sales Forecast
21.6.31.3. Net Present Value
21.6.31.4. Value Creation Analysis
21.6.32. SPK-8011
21.6.32.1. Target Patient Population
21.6.32.2. Sales Forecast
21.6.32.3. Net Present Value
21.6.32.4. Value Creation Analysis
21.6.33. AMT-061
21.6.33.1. Target Patient Population
21.6.33.2. Sales Forecast
21.6.33.3. Net Present Value
21.6.33.4. Value Creation Analysis
21.6.34. VB-111
21.6.34.1. Target Patient Population
21.6.34.2. Sales Forecast
21.6.34.3. Net Present Value
21.6.34.4. Value Creation Analysis
21.6.35. Generx®
21.6.35.1. Target Patient Population
21.6.35.2. Sales Forecast
21.6.35.3. Net Present Value
21.6.35.4. Value Creation Analysis
21.6.36. AMG001
21.6.36.1. Target Patient Population
21.6.36.2. Sales Forecast
21.6.36.3. Net Present Value
21.6.36.4. Value Creation Analysis
21.6.37. OAV-101
21.6.37.1. Target Patient Population
21.6.37.2. Sales Forecast
21.6.37.3. Net Present Value
21.6.37.4. Value Creation Analysis
21.6.38. ADXS-HPV
21.6.38.1. Target Patient Population
21.6.38.2. Sales Forecast
21.6.38.3. Net Present Value
21.6.38.4. Value Creation Analysis
21.6.39. AGTC
21.6.39.1. Target Patient Population
21.6.39.2. Sales Forecast
21.6.39.3. Net Present Value
21.6.39.4. Value Creation Analysis
21.6.40. LYS-SAF302
21.6.40.1. Target Patient Population
21.6.40.2. Sales Forecast
21.6.40.3. Net Present Value
21.6.40.4. Value Creation Analysis
21.6.41. NFS-01
21.6.41.1. Target Patient Population
21.6.41.2. Sales Forecast
21.6.41.3. Net Present Value
21.6.41.4. Value Creation Analysis
21.6.42. AG0302-COVID19
21.6.42.1. Target Patient Population
21.6.42.2. Sales Forecast
21.6.42.3. Net Present Value
21.6.42.4. Value Creation Analysis
21.6.43. RGX-314
21.6.43.1. Target Patient Population
21.6.43.2. Sales Forecast
21.6.43.3. Net Present Value
21.6.43.4. Value Creation Analysis
21.6.44. Hologene
21.6.44.1. Target Patient Population
21.6.44.2. Sales Forecast
21.6.44.3. Net Present Value
21.6.44.4. Value Creation Analysis
22. VECTOR MANUFACTURING
22.1. Chapter Overview
22.2. Overview of Viral Vector Manufacturing
22.3. Viral Vector Manufacturing Process
22.3.1. Mode of Vector Production
22.3.2. Adherent and Suspension Cultures
22.3.3. Unit Processes and Multiple Parallel Processes
22.3.4. Cell Culture Systems for Production of Viral Vectors
22.3.4.1. Small Scale / Laboratory Scale Cell Culture Systems
22.3.4.2. Large Scale Cell Culture Systems
22.3.5. Culture Media Specifications
22.4. Bioprocessing of Viral Vectors
22.4.1. AAV Vector Production
22.4.2. Adenoviral Vector Production
22.4.3. Lentiviral Vector Production
22.4.4. ? -Retroviral Vector Production
22.5. Challenges Associated with Vector Manufacturing
22.6. Contract Service Providers for Viral and Plasmid Vectors
23. CASE STUDY: GENE THERAPY SUPPLY CHAIN
23.1. Chapter Overview
23.2. Overview of Gene Therapy Supply Chain
23.3. Implementation of Supply Chain Models
23.4. Logistics in Gene Therapies
23.4.1. Logistic Processes for Autologous and Allogeneic Therapies
23.5. Regulatory Supply Chain Across the Globe
23.6. Challenges Associated with Gene Therapy Supply Chain
23.7. Optimizing Cell and Advanced Therapies Supply Chain Management
23.7.1. Enterprise Manufacturing System
23.7.2. Laboratory Information Management System
23.7.3. Inventory Management System
23.7.4. Quality Management System
23.7.5. Logistics Management System
23.7.6. Patient Management System
23.7.7. Electronic Clinical Outcome Assessments System
23.7.8. Supply Chain Orchestration Platform
23.8. Recent Developments and Upcoming Trends
24. CONCLUSION
24.1. Chapter Overview
25. INTERVIEW TRANSCRIPTS
25.1. Chapter Overview
25.2. Buel Dan Rodgers (Founder and CEO, AAVogen)
25.3. Sue Washer (President and CEO, AGTC)
25.4. Patricia Zilliox (President and CEO, Eyevensys)
25.5. Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))
25.6. Adam Rogers (CEO, Hemera Biosciences)
25.7. Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))
25.8. Al Hawkins (CEO, Milo Biotechnology)
25.9. Jean-Phillipe Combal (CEO, Vivet Therapeutics)
25.10. Robert Jan Lamers (former CEO, Arthrogen)
25.11. Tom Wilton (former CBO, LogicBio Therapeutics)
25.12. Michael Tripletti (former CEO, Myonexus Therapeutics)
25.13. Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)
25.14. Cedric Szpirer (former Executive and Scientific Director, Delphi Genetics)
25.15. Marco Schmeer (Project Manager) and Tatjana Buchholz, (former Marketing Manager, PlasmidFactory)
25.16. Jeffrey Hung (CCO, Vigene Biosciences)
26. APPENDIX 1: TABULATED DATA
27. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
