Gene Therapy Market by Therapeutic Area, Type of Vector, Type of Therapy, Type of Gene Delivery Method, Route of Administration, Geographical Regions, Leading Players and Sales Forecast: Industry Trends and Global Forecasts, Till 2035

As per Roots Analysis, the global gene therapy market is estimated to grow from USD 2.4 billion in the current year to USD 16.5 billion by 2035, at a CAGR of 19.25% during the forecast period, till 2035.

The market sizing and opportunity analysis has been segmented across the following parameters:

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

GENE THERAPY MARKET: GROWTH AND TRENDS

Congenital abnormalities and inherited genetic mutations encompass a diverse range of disorders, including oncological disorders, genetic disorders, autoimmune disorders and neurological disorders. According to the World Health Organization (WHO), genetic disorders affect 10 out of every 1,000 individuals, resulting in over 70 million people living with these conditions globally. Notably, more than 40% of infant mortality worldwide is linked to various genetic disorders. At present, several research initiatives are underway to develop safe and effective methods to selectively correct (the mutated, disease-causing) genes of individual patients. Of these, gene therapies have emerged as a promising option. These therapies are designed to target the underlying genetic cause of a disease, either by introducing a healthy version of the mutated gene or by disabling the functions of genes that cause the disease within the body. It is worth highlighting that over 1,100 clinical trials related to gene therapies have been registered in the past few years, indicating substantial research activity. Additionally, as per the USFDA, 10 to 20 therapies are expected to gain market access, each year, till 2025. Driven by the collaborative efforts of gene therapy companies and an increasing preference for effective single-dose treatments, the gene therapy market is set to experience significant growth during the forecast period.

GENE THERAPY MARKET: KEY INSIGHTS

The report delves into the current state of the gene therapy market and identifies potential growth opportunities within the industry. Some key findings from the report include:

1. Presently, 345 developers, worldwide, are engaged in the development of gene therapies targeting various disorders; of these, more than 50% of the stakeholders are headquartered in the US, alone.

2. 70% of the gene therapies being evaluated in phase III clinical trials are gene augmentation therapies; it is worth noting that more than 35% of the candidates have received fast track designation.

3. Close to 95% of the preclinical stage gene therapies are in vivo therapies; more than 20% of the gene therapy candidates are being developed for the treatment of neurological disorders.

4. Over the years, the intellectual capital related to gene therapies has grown at a commendable pace; several patents have been filed by both industry stakeholders and academic players.

5. Over 500 patents focused on gene editing have been filed in the past five years; till date, 6% of such patents have been granted.

6. Rising interest of the stakeholders in this domain is reflected by the number of mergers and acquisitions reported over the last few years; 26% of these initiatives were focused on gaining access to novel platforms.

7. Foreseeing lucrative opportunities, many public and private investors have made investments worth USD 36.4 billion, across more than 560 funding instances.

8. In the recent past, more than 18,000 patients have been enrolled in clinical trials focused on gene therapies; close to 45% trials are evaluating gene therapies for the treatment of oncological disorders.

9. Start-ups are spearheading the innovation in the gene therapy domain; the valuation of a start-up is not solely dependent on profits, instead, it depends on experience and amount previously invested by industry stakeholders.

10. In order to tap into the lucrative opportunity associated with gene therapies, big pharma players have adopted various approaches, from proprietary product development to strategic investments, to advance their portfolios.

11. Given the growing incidence of inherited disorders, the demand for gene therapies (in terms of number of patients) has risen; by 2035, it is anticipated to grow at an annualized rate of 11.67%, across various geographies.

12. Owing to the growing interest towards personalized medicines and high specificity, the market for gene therapies is expected to rise steadily in the foreseeable future.

13. The gene therapy market is anticipated to grow at an annualized rate (CAGR) of 19.25%, till 2035; North America is expected to capture the majority share (close to 65%) of the market by 2035.

GENE THERAPY MARKET: KEY SEGMENTS

Currently, Muscle Disorders Segment Occupies the Largest Share of the Gene Therapy Market

Based on the therapeutic area, the market is segmented into cardiovascular disorders, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle disorders, oncological diseases, ophthalmic disorders and other disorders. At present, muscle disorders segment holds the maximum share of the gene therapy market. This can be attributed to the growing prevalence of muscle disorders and proven efficacy of gene therapies in treating such conditions.

Adeno-Associated Virus (AAV) Vector-Based Gene Therapies is Likely to Dominate the Gene Therapies Market During the Forecast Period

Based on the type of vector, the market is segmented into adeno-associated virus vectors, adenovirus vectors, herpes simplex virus vectors, lentivirus vectors, non-viral vectors, retrovirus vectors and other viral vectors. At present, AAV vector-based gene therapies holds the maximum share within the gene therapies market. It is worth highlighting that, owing to the numerous advantages of AAV vector, such as high target specificity, greater efficacy and infectivity, AAV vector-based gene therapies are likely to capture larger share in the coming decade.

Currently, Gene Augmentation Segment Occupies the Largest Share of the Gene Therapies Market

Based on the type of therapy, the market is segmented into gene augmentation, gene editing, gene regulation, oncolytic immunotherapies and other therapies. Owing to their ability to deliver therapeutic genes to the patient's genome, fewer side effects and precisely correcting the genomic mutations, the gene therapy market is currently dominated by gene augmentation therapies. This trend is likely to remain the same in the mid-to-long term.

In Vivo Gene Delivery Method is Likely to Dominate the Gene Therapies Market During the Forecast Period

Based on the type of gene delivery method, the market is segmented into ex vivo and in vivo gene delivery methods. It is worth highlighting that, at present, in vivo gene delivery method holds a larger share of the gene therapy market. This trend is likely to remain the same in the coming decade.

Intravenous Route of Administration is Likely to Dominate the Gene Therapy Market During the Forecast Period

Based on the route of administration, the market is segmented into intramuscular, intratumoral, intravenous, subretinal and other routes of administration. It is worth highlighting that majority of the current gene therapy market is captured by the intravenous route of administration and this trend is unlikely to change in mid-long term. This can be attributed to the fact that gene therapies allow widespread distribution of the gene-carrying therapeutic vector to multiple target organs. In addition, intravenous administration is easier to administer and minimally invasive compared to other methods like intralesional or intratumoral injection.

North America Accounts for the Largest Share of the Market

Based on the key geographical regions, the market is segmented into North America, Europe, Asia-Pacific, Latin America and Rest of the World. Majority share is expected to be captured by drug developers based in North America. It is worth highlighting that, over the years, the market for Europe is expected to grow at a higher CAGR.

Example Players in the Gene Therapy Market

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

Primary Research Overview

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders. The research report features detailed transcripts of interviews held with the following industry stakeholders:

Former Corporate Communications Manager, Orchard Therapeutics
Co-Founder and Chief Executive Officer, Vivet Therapeutics
Chief Executive Officer, Chairman and President, Kubota Pharmaceutical
Former Chief Executive Officer and President, Eyevensys
Former Chief Executive Officer and President, AGTC
Former Chief Business Officer, LogicBio Therapeutics
Founder and Chief Executive Officer, AAVogen
Chief Executive Officer, Hemera Biosciences
Ex Co-Founder, Chief Executive Officer and President, Myonexus Therapeutics
Former Chief Executive Officer, Arthrogen
Project Manager and Tatjana Buchholz, Former Marketing Manager, PlasmidFactory
Former Executive and Scientific Director, Delphi Genetics
Former Chief Commercial Officer, Vigene Biosciences
Chief Executive Officer and Chairman, Gene Biotherapeutics
Chief Executive Officer, Milo Biotechnology

GENE THERAPY MARKET: RESEARCH COVERAGE

Market Sizing and Opportunity Analysis: The report features an in-depth analysis of the gene therapy market, focusing on key market segments, including [A] therapeutic area, [B] type of vector, [C] type of therapy, [D] type of gene delivery method, [E] route of administration, [F] geographical regions and [H] leading players.
Market Landscape (Gene Therapies): A comprehensive evaluation of marketed and clinical stage gene therapies, considering various parameters, such as [A] stage of development, [B] drug designations, [C] therapeutic area, [D] target gene, [E] type of vector used, [F] type of therapy, [G] type of gene delivery method, [H] route of administration and [I] dosing frequency. The chapter also provides a detailed analysis of the current landscape of discovery and preclinical stage gene therapies based on parameters, such as [J] stage of development, [K] therapeutic area, [L] target gene, [M] type of vector used, [N] type of therapy and [O] type of gene delivery method.
Market Landscape (Gene Therapy Developers): A comprehensive evaluation of gene therapy developers, considering various parameters, such as [A] year of establishment, [B] company size (in terms of number of employees), [C] location of headquarters and [D] most active players (in terms of the number of gene therapies developed).
Company Profiles: In-depth profiles of key industry players engaged in the development of gene therapies, focusing on [A] company overviews, [B] financial information (if available), [C] gene therapy portfolio, [D] recent developments and [E] an informed future outlook.
Drug Profiles: In-depth profiles of marketed and late stage (phase II / III and above) gene therapies, along with information on the [A] development timeline of the therapy, [B] mechanism of action, [C] type of vector used, [D] dosage and manufacturing details, [E] target indication, as well as [F] details related to the developer company.
Commercialization Strategy: An elaborate discussion on the 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.
Patent Analysis: Detailed analysis of various patents filed / granted related to gene therapies and gene editing therapies based on [A] type of patent, [B] publication year, [C] regional applicability, [D] CPC symbols, [E] emerging focus areas, [F] leading industry players (in terms of the number of patents filed / granted), and [G] patent valuation. It also includes a [H] patent benchmarking analysis and [I] a detailed valuation analysis.
Merger and Acquisitions: A comprehensive examination of the various mergers and acquisitions, focusing on multiple relevant parameters, including [A] year of agreement, [B] type of deal, [C] geographical location of the companies involved, [D] key value drivers, [E] highest phase of development of the acquired company' product, [F] target therapeutic area and [G] deal multiples.
Funding and Investment Analysis: A detailed evaluation of the investments made in the gene therapy domain, encompassing seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings.
Clinical Trial Analysis: Examination of completed, ongoing, and planned clinical studies of various gene therapies, based on parameters, such as [A] trial registration year, [B] trial status, [C] trial phase, [D] target therapeutic area, [E] geography, [F] type of sponsor, [G] prominent treatment sites and [H] enrolled patient population.
Pricing Analysis: An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by developers / manufacturers to decide the prices of these therapies.
Start-up Valuation Analysis: An analysis of the startup companies engaged in this domain (since 2017) based on year of experience.
Big Pharma Analysis: A comprehensive examination of various gene therapy-based initiatives undertaken by major pharmaceutical companies, based on several relevant parameters, such as [A] therapeutic area, [B] type of vector used, [C] type of therapy and [D] type of gene delivery method used. In addition, it includes a detailed benchmarking analysis across key parameters, such as [E] number of gene therapies under development, [F] funding information, [G] partnership activity and [H] patent portfolio strength.
Demand Analysis: Informed estimates of the annual commercial and clinical demand for gene therapies based on several relevant parameters, such as [A] target patient population, [B] dosing frequency and [C] dose strength.
Additional Insight 1: 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.
Additional Insight 2: A case study examining the current and emerging trends in vector manufacturing, along with details on companies that provide contract services for producing viral vectors utilized in gene therapy products. The analysis also includes an in-depth discussion of the manufacturing processes associated with different types of vectors.
Additional Insight 3: Assessment of the supply chain model adopted by gene therapy developers 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.
Market Impact Analysis: The report analyzes various factors such as drivers, restraints, opportunities, and challenges affecting the market growth.

KEY QUESTIONS ANSWERED IN THIS REPORT

How many companies are currently engaged in this market?
Which are the leading companies in this market?
What is the annual demand for gene therapies?
What factors are likely to influence the evolution of this market?
What is the current and future market size?
What is the CAGR of this market?
How is the current and future market opportunity likely to be distributed across key market segments?

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Stakeholders can leverage the report to gain a deeper understanding of the competitive dynamics within the market. By analyzing the competitive landscape, businesses can make informed decisions to optimize their market positioning and develop effective go-to-market strategies.
The report offers stakeholders a comprehensive overview of the market, including key drivers, barriers, opportunities, and challenges. This information empowers stakeholders to stay abreast of market trends and make data-driven decisions to capitalize on growth prospects.

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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 Research
    - 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

37. CONCLUSION

38. APPENDIX I: TABULATED DATA

39. APPENDIX II: LIST OF COMPANIES AND ORGANIZATIONS

Product Code: RA100359

List of Tables

Table 6.1 Comparison of Ex vivo and In vivo Techniques
Table 6.2 Routes of Administration of Gene Therapies: Advantages and Disadvantages
Table 6.3 Advantages and Disadvantages of Gene Therapies
Table 7.1 Key Features of Viral Vectors
Table 8.1 Payment Options Available for Gene Therapies
Table 8.2 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
Table 9.1 Marketed and Clinical Stage Gene Therapies: Information on Developer, Stage of Development and Drug Designation
Table 9.2 Marketed and Clinical Stage Gene Therapies: Information on Developer, Therapeutic Area and Biological Target
Table 9.3 Marketed and Clinical Stage Gene Therapies: Information on Developer and Type of Vector Used
Table 9.4 Marketed and Clinical Stage Gene Therapies: Information on Developer, Type of Therapy and Type of Gene Delivery Method
Table 9.5 Marketed and Clinical Stage Gene Therapies: Information on Developer, Route of Administration and Dosage
Table 9.6 Preclinical and Discovery Stage Gene Therapies: Information on Developer, Stage of Development, Therapeutic Area and Biological Target
Table 9.7 Preclinical and Discovery Stage Gene Therapies: Information on Developer and Type of Vector Used
Table 9.8 Preclinical and Discovery Stage Gene Therapies: Information on Developer, Type of Therapy and Type of Gene Delivery Method
Table 10.1 Gene Therapy Developers: Information on Year of Establishment, Company Size and Location of Headquarters
Table 11.1 Gene Therapy: List of Companies Profiled
Table 11.2 Amgen: Company Overview
Table 11.3 Amgen: Marketed Gene Therapy Portfolio
Table 11.4 Amgen: Clinical Stage Gene Therapy Portfolio
Table 11.5 bluebird bio: Company Overview
Table 11.6 bluebird bio: Marketed Gene Therapy Portfolio
Table 11.7 bluebird bio: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.8 bluebird bio: Recent Developments and Future Outlook
Table 11.9 BioMarin Pharmaceutical: Company Overview
Table 11.10 BioMarin Pharmaceutical: Marketed Gene Therapy Portfolio
Table 11.11 BioMarin Pharmaceutical: Preclinical Stage Gene Therapy Portfolio
Table 11.12 BioMarin Pharmaceutical: Recent Developments and Future Outlook
Table 11.13 CRISPR Therapeutics: Company Overview
Table 11.14 CRISPR Therapeutics: Marketed Gene Therapy Portfolio
Table 11.15 CRISPR Therapeutics: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.16 CRISPR Therapeutics: Recent Developments and Future Outlook
Table 11.17 Kolon TissueGene: Company Overview
Table 11.18 Kolon TissueGene: Marketed Gene Therapy Portfolio
Table 11.19 Kolon TissueGene: Clinical Stage Gene Therapy Portfolio
Table 11.20 Kolon TissueGene: Prelinical Stage Gene Therapy Portfolio
Table 11.21 Kolon TissueGene: Recent Developments and Future Outlook
Table 11.21 Krystal Biotech: Company Overview
Table 11.22 Krystal Biotech: Marketed Gene Therapy Portfolio
Table 11.23 Krystal Biotech: Clinical Stage Gene Therapy Portfolio
Table 11.24 Krystal Biotech: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.25 Krystal Biotech: Recent Developments and Future Outlook
Table 11.26 Pfizer: Company Overview
Table 11.27 Pfizer: Marketed Gene Therapy Portfolio
Table 11.28 Pfizer: Clinical Stage Gene Therapy Portfolio
Table 11.29 Pfizer: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.30 Pfizer: Recent Developments and Future Outlook
Table 11.31 PTC Therapeutics: Company Overview
Table 11.32 PTC Therapeutics: Marketed Gene Therapy Portfolio
Table 11.33 PTC Therapeutics: Preclinical Stage Gene Therapy Portfolio
Table 11.34 PTC Therapeutics: Recent Developments and Future Outlook
Table 11.35 Sarepta Therapeutics: Company Overview
Table 11.36 Sarepta Therapeutics: Marketed Gene Therapy Portfolio
Table 11.37 Sarepta Therapeutics: Clinical Stage Gene Therapy Portfolio
Table 11.38 Sarepta Therapeutics: Preclinical and Discovery Gene Therapy Portfolio
Table 11.39 Sarepta Therapeutics: Recent Developments and Future Outlook
Table 11.40 Spark Therapeutics: Company Overview
Table 11.41 Spark Therapeutics: Marketed Gene Therapy Portfolio
Table 11.42 Spark Therapeutics: Clinical Stage Gene Therapy Portfolio
Table 11.43 Spark Therapeutics: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.44 Spark Therapeutics: Recent Developments and Future Outlook
Table 11.45 Artgen Biotech: Company Overview
Table 11.46 Artgen Biotech: Marketed Gene Therapy Portfolio
Table 11.47 Artgen Biotech: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.48 Artgen Biotech: Recent Developments and Future Outlook
Table 11.49 Ferring Pharmaceuticals: Company Overview
Table 11.50 Ferring Pharmaceuticals: Marketed Gene Therapy Portfolio
Table 11.51 Ferring Pharmaceuticals: Clinical Stage Gene Therapy Portfolio
Table 11.52 Ferring Pharmaceuticals: Recent Developments and Future Outlook
Table 11.53 Novartis: Company Overview
Table 11.54 Novartis: Marketed Gene Therapy Portfolio
Table 11.55 Novartis: Clinical Stage Gene Therapy Portfolio
Table 11.56 Novartis: Preclinical and Discovery Stage Gene Therapy Portfolio
Table 11.57 Novartis: Recent Developments and Future Outlook
Table 11.58 Orchard Therapeutics: Company Overview
Table 11.59 Orchard Therapeutics: Marketed Gene Therapy Portfolio
Table 11.60 Orchard Therapeutics: Clinical Stage Gene Therapy Portfolio
Table 11.61 Orchard Therapeutics: Preclinical Stage Gene Therapy Portfolio
Table 11.62 Orchard Therapeutics: Recent Developments and Future Outlook
Table 11.63 CSL Behring: Company Overview
Table 11.64 CSL Behring: Marketed Gene Therapy Portfolio
Table 11.65 CSL Behring: Discovery Stage Gene Therapy Portfolio
Table 11.66 CSL Behring: Recent Developments and Future Outlook
Table 11.67 Shanghai Sunway Biotech: Company Overview
Table 11.68 Shanghai Sunway Biotech: Marketed Gene Therapy Portfolio
Table 11.69 Sibiono GeneTech: Company Overview
Table 11.70 Sibiono GeneTech: Marketed Gene Therapy Portfolio
Table 12.1 Gene Therapies: Marketed and Approved Products
Table 12.2 Company Overview: Sibiono GeneTech
Table 12.3 Gendicine: Patent Portfolio
Table 12.4 Gendicine: Stage of Development
Table 12.5 Company Overview: Shanghai Sunway Biotech
Table 12.6 Oncorine: Stage of Development
Table 12.7 Company Overview: Human Stem Cell Institute
Table 12.8 Neovasculgen: Stage of Development
Table 12.9 Company Overview: Amgen
Table 12.10 Imlygic: Stage of Development
Table 12.11 Imlygic: Recommended Dose and Schedule
Table 12.12 Imlygic: Determination of Injection Volume based upon Lesion Size
Table 12.13 Company Overview: Orchard Therapeutics
Table 12.14 Strimvelis: Stage of Development
Table 12.15 Company Overview: Spark Therapeutics
Table 12.16 Luxturna: Stage of Development
Table 12.17 Company Overview: Novartis
Table 12.18 Zolgensma: Stage of Development
Table 12.19 Company Overview: bluebird bio
Table 12.20 Zynteglo: Stage of Development
Table 12.21 Libmeldy: Stage of Development
Table 13.1 Libmeldy: Presentations in Conferences Before Product Approval
Table 13.2 Luxturna: Presentations in Conferences Before Product Approval
Table 13.3 Zolgensma: Presentations in Conferences Before Product Approval
Table 13.4 Zynteglo: Presentations in Conferences Before Product Approval
Table 13.5 Imlygic: Presentations in Conferences After Product Approval
Table 13.6 Libmeldy: Presentations in Conferences After Product Approval
Table 13.7 Luxturna: Presentations in Conferences After Product Approval
Table 13.8 Strimvelis: Presentations in Conferences After Product Approval
Table 13.9 Zolgensma: Presentations in Conferences After Product Approval
Table 13.10 Zynteglo: Presentations in Conferences After Product Approval
Table 13.11 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
Table 14.1 Gene Therapies: Phase II / III and Above Drugs
Table 14.2 Drug Profile: LUMEVOQ (GS010)
Table 14.3 Drug Profile: OTL-103
Table 14.4 Drug Profile: PTC-AADC
Table 14.5 Drug Profile: BMN 270
Table 14.6 Drug Profile: nadofaragene firadenovec (rAd-IFN/Syn3)
Table 14.7 Drug Profile: beti-cel
Table 14.8 Drug Profile: eli-cel
Table 14.9 Drug Profile: lovo-cel
Table 14.10 Drug Profile: SRP-9001
Table 14.11 Drug Profile: EB-101
Table 14.12 Drug Profile: ProstAtak
Table 14.13 Drug Profile: D-Fi
Table 14.14 Drug Profile: CG0070
Table 14.15 Drug Profile: Vigil-EWS
Table 14.16 Drug Profile: Engensis
Table 14.17 Drug Profile: VGX-3100
Table 14.18 Drug Profile: INVOSSA (TG-C)
Table 14.19 Drug Profile: VYJUVEKT
Table 14.20 Drug Profile: PF-06939926
Table 14.21 Drug Profile: PF-06838435
Table 14.22 Drug Profile: PF-07055480
Table 14.23 Drug Profile: SPK-8011
Table 14.24 Drug Profile: AMT-061
Table 14.25 Drug Profile: VB-111
Table 14.26 Drug Profile: Generx
Table 14.27 Drug Profile: ADXS-HPV
Table 14.28 Drug Profile: AGTC 501
Table 14.29 Drug Profile: LYS-SAF302
Table 14.30 Drug Profile: NFS-01
Table 14.31 Drug Profile: AG0302-COVID19
Table 14.32 Drug Profile: RGX-314
Table 14.33 Drug Profile: Hologene 5
Table 15.1 Gene Therapy Patent Portfolio: List of Top CPC Symbols
Table 15.2 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
Table 15.3 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 15.4 Gene Therapy Patent Portfolio: List of Leading Patents (In terms of Highest Relative Valuation)
Table 15.5 Gene Editing Patent Portfolio: List of Top CPC Classifications
Table 15.6 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
Table 15.7 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
Table 15.8 Gene Editing Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
Table 16.1 Gene Therapy: List of Mergers and Acquisitions, since 2015
Table 16.2 Acquisitions: Key Value Drivers
Table 16.3 Mergers and Acquisitions: Information on Phase of Development and Therapeutic Area of the Acquired Company's Gene Therapy Product
Table 16.4 Mergers and Acquisitions: Information on Deal Multiple based on Acquisition Amount
Table 17.1 Gene Therapy Market: Funding and Investments, since 2015
Table 17.2 Funding and Investments: Summary of Investments
Table 17.3 Funding and Investments: Summary of Venture Capital Funding
Table 18.1 Clinical Trial Analysis: Emerging Molecules to Watch
Table 18.2 Clinical Trial Analysis: Most Important Trials to Watch
Table 19.1 Pricing Model: Price of Marketed Gene / Cell Therapies
Table 19.2 Pricing Model: Price of Marketed Targeted Drugs (Antibody Drug Conjugates and Monoclonal Antibodies)
Table 19.3 Pricing Model: Opinions of Experts / Other Analysts
Table 20.1 Start-up Valuation: Estimates based on Overall Experience
Table 22.1 Global Clinical Demand for Gene Therapies: Information on Trial Phase, Distribution of Patient Enrolment and Average Number of Patients Enrolled
Table 24.1 Gene Therapy Market: List of Forecasted Therapies along with Information on Developer, Stage of Development and First Launch Year / Likely Approval Year
Table 32.1 Adstiladrin(R): Net Present Value (USD Million)
Table 32.2 Adstilarin(R): Value Creation Analysis (USD Million)
Table 32.3 BEQVEZ(TM): Net Present Value (USD Million)
Table 32.4 BEQVEZ(TM): Value Creation Analysis (USD Million)
Table 32.5 Casgevy(TM): Net Present Value (USD Million)
Table 32.6 Casgevy(TM): Value Creation Analysis (USD Million)
Table 32.7 Elevidys(R): Net Present Value (USD Million)
Table 32.8 Elevidys(R): Value Creation Analysis (USD Million)
Table 32.9 Gendicine(R): Net Present Value (USD Million)
Table 32.10 Gendicine(R): Value Creation Analysis (USD Million)
Table 32.11 Hemgenix(R): Net Present Value (USD Million)
Table 32.12 Hemgenix(R): Value Creation Analysis (USD Million)
Table 32.13 IMLYGIC(R): Net Present Value (USD Million)
Table 32.14 IMLYGIC(R): Value Creation Analysis (USD Million)
Table 32.15 Libmeldy(R): Net Present Value (USD Million)
Table 32.16 Libmeldy(R): Value Creation Analysis (USD Million)
Table 32.17 Luxturna(R): Net Present Value (USD Million)
Table 32.18 Luxturna(R): Value Creation Analysis (USD Million)
Table 32.19 LYFGENIA(TM): Net Present Value (USD Million)
Table 32.20 LYFGENIA(TM): Value Creation Analysis (USD Million)
Table 32.21 Neovasculgen(R): Net Present Value (USD Million)
Table 32.22 Neovasculgen(R): Value Creation Analysis (USD Million)
Table 32.23 Oncorine(R): Net Present Value (USD Million)
Table 32.24 Oncorine(R): Value Creation Analysis (USD Million)
Table 32.25 Roctavian(TM): Net Present Value (USD Million)
Table 32.26 Roctavian(TM): Value Creation Analysis (USD Million)
Table 32.27 SKYSONA(R): Net Present Value (USD Million)
Table 32.28 SKYSONA(R): Value Creation Analysis (USD Million)
Table 32.29 Strimvelis(TM): Net Present Value (USD Million)
Table 32.30 Strimvelis(TM): Value Creation Analysis (USD Million)
Table 32.31 Upstaza(TM): Net Present Value (USD Million)
Table 32.32 Upstaza(TM): Value Creation Analysis (USD Million)
Table 32.33 VYJUVEKT(R): Net Present Value (USD Million)
Table 32.34 VYJUVEKT(R): Value Creation Analysis (USD Million)
Table 32.35 Zolgensma(R): Net Present Value (USD Million)
Table 32.36 Zolgensma(R): Value Creation Analysis (USD Million)
Table 32.37 Zynteglo(TM): Net Present Value (USD Million)
Table 32.38 Zynteglo(TM): Value Creation Analysis (USD Million)
Table 32.39 AAV-RPE65: Net Present Value (USD Million)
Table 32.40 AAV-RPE65: Value Creation Analysis (USD Million)
Table 32.41 ABO-102 (UX111): Net Present Value (USD Million)
Table 32.42 ABO-102 (UX111): Value Creation Analysis (USD Million)
Table 32.43 AGTC 501 (rAAV2tYF-GRK1-RPGR): Net Present Value (USD Million)
Table 32.44 AGTC 501 (rAAV2tYF-GRK1-RPGR): Value Creation Analysis (USD Million)
Table 32.45 Cretostimogene Grenadenorepvec (CG0070): Net Present Value (USD Million)
Table 32.46 Cretostimogene Grenadenorepvec (CG0070: Value Creation Analysis (USD Million)
Table 32.47 Dirloctocogene samoparvovec (SPK-8011): Net Present Value (USD Million)
Table 32.48 Dirloctocogene samoparvovec (SPK-8011): Value Creation Analysis (USD Million)
Table 32.49 DTx-301: Net Present Value (USD Million)
Table 32.50 DTx-301: Value Creation Analysis (USD Million)
Table 32.51 DTx-401: Net Present Value (USD Million)
Table 32.52 DTx-401: Value Creation Analysis (USD Million)
Table 32.53 Generx(R) (Ad5FGF-4): Net Present Value (USD Million)
Table 32.54 Generx(R) (Ad5FGF-4): Value Creation Analysis (USD Million)
Table 32.55 Giroctocogene fitelparvovec (SB-525 / PF-07055480): Net Present Value (USD Million)
Table 32.56 Giroctocogene fitelparvovec (SB-525 / PF-07055480): Value Creation Analysis (USD Million)
Table 32.57 Hologene 5: Net Present Value (USD Million)
Table 32.58 Hologene 5: Value Creation Analysis (USD Million)
Table 32.59 INVOSSA(TM) (TG-C): Net Present Value (USD Million)
Table 32.60 INVOSSA(TM) (TG-C): Value Creation Analysis (USD Million)
Table 32.61 LUMEVOQ(R): Net Present Value (USD Million)
Table 32.62 LUMEVOQ(R): Value Creation Analysis (USD Million)
Table 32.63 NFS-01 (rAAV2-ND4): Net Present Value (USD Million)
Table 32.64 NFS-01 (rAAV2-ND4): Value Creation Analysis (USD Million)
Table 32.65 NTLA-2001: Net Present Value (USD Million)
Table 32.66 NTLA-2001: Value Creation Analysis (USD Million)
Table 32.67 Olvi-Vec (Olvimulogene nanivacirepvec): Net Present Value (USD Million)
Table 32.68 Olvi-Vec (Olvimulogene nanivacirepvec): Value Creation Analysis (USD Million)
Table 32.69 ProstAtak(R): Net Present Value (USD Million)
Table 32.70 ProstAtak(R): Value Creation Analysis (USD Million)
Table 32.71 pz-cel (prademagene zamikeracel / EB-101): Net Present Value (USD Million)
Table 32.72 pz-cel (prademagene zamikeracel / EB-101): Value Creation Analysis (USD Million)
Table 32.73 RGX-121: Net Present Value (USD Million)
Table 32.74 RGX-121: Value Creation Analysis (USD Million)
Table 32.75 RGX-314: Net Present Value (USD Million)
Table 32.76 RGX-314: Value Creation Analysis (USD Million)
Table 32.77 VGX-3100: Net Present Value (USD Million)
Table 32.78 VGX-3100: Value Creation Analysis (USD Million)
Table 32.79 Summary of Variations in Forecasted Sales Revenue of Gene Therapies (Compared to 5th Edition)
Table 33.1 Gene Editing Technologies: Information on Developer and Highest Phase of Drug Development Supported
Table 33.2 Key Features of Gene Editing Platforms
Table 33.3 ASC Therapeutics: Collaborations
Table 33.4 CRISPR Therapeutics: Funding Instances
Table 33.5 CRISPR Therapeutics: Collaborations
Table 33.6 Editas Medicine: Funding Instances
Table 33.7 Editas Medicine: Collaborations
Table 33.8 Emendo Biotherapeutics: Collaborations
Table 33.9 Excision Biotherapeutics: Funding Instances
Table 33.10 Intellia Therapeutics: Funding Instances
Table 33.11 Intellia Therapeutics: Collaborations
Table 33.12 Sarepta Therapeutics: Funding Instances
Table 33.13 Sarepta Therapeutics: Collaborations
Table 33.14 ToolGen: Collaborations
Table 33.15 Sangamo Therapeutics: Funding Instances
Table 33.16 Sangamo Therapeutics: Collaborations
Table 33.17 bluebird bio: Funding Instances
Table 34.18 bluebird bio: Collaborations
Table 34.19 Gene Switch Technology Platforms
Table 33.20 Precigen: Funding Instances
Table 33.21 MeiraGTx: Funding Instances
Table 33.22 MeiraGTx: Collaborations
Table 33.23 Gene Therapy Developers: Information on Headquarters, Technology / Platform and Type of Gene Delivery Method
Table 34.1 Key Features of Small-scale Cell Culture Systems
Table 34.2 List of Viral Vector Contract Manufacturing Organizations: Information on Type of Viral Vector Manufactured
Table 36.1 Orchard Therapeutics: Company Snapshot
Table 36.2 Vivet Therapeutics: Company Snapshot
Table 36.3 Kubota Pharmaceutical: Company Snapshot
Table 36.4 Eyevensys: Company Snapshot
Table 36.5 AGTC: Company Snapshot
Table 36.6 LogicBio Therapeutics: Company Snapshot
Table 36.7 AAVogen: Company Snapshot
Table 36.8 Hemera Biosciences: Company Snapshot
Table 36.9 Myonexus Therapeutics: Company Snapshot
Table 36.10 Arthrogen: Company Snapshot
Table 36.11 PlasmidFactory: Company Snapshot
Table 36.12 Delphi Genetics: Company Snapshot
Table 36.13 Vigene Biosciences: Company Snapshot
Table 36.14 Gene Biotherapeutics: Company Snapshot
Table 36.15 Milo Biotechnology: Company Snapshot
Table 38.1 Marketed and Clinical Stage Gene Therapies: Distribution by Stage of Development
Table 38.2 Marketed and Clinical Stage Gene Therapies: Distribution by Drug Designation
Table 38.3 Marketed and Clinical Stage Gene Therapies: Distribution by Therapeutic Area
Table 38.4 Marketed and Clinical Stage Gene Therapies: Distribution by Biological Target
Table 38.5 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Vector Used
Table 38.6 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Therapy
Table 38.7 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Gene Delivery Method
Table 38.8 Marketed and Clinical Stage Gene Therapies: Distribution by Route of Administration
Table 38.9 Marketed and Clinical Stage Gene Therapies: Distribution by Dosage
Table 38.10 Preclinical and Discovery Stage Gene Therapies: Distribution by Stage of Development
Table 38.11 Preclinical and Discovery Stage Gene Therapies: Distribution by Therapeutic Area
Table 38.12 Preclinical and Discovery Stage Gene Therapies: Distribution by Biological Target
Table 38.13 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Vector Used
Table 38.14 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Therapy
Table 38.15 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Gene Delivery Method Used
Table 38.16 Gene Therapy Developers: Distribution by Year of Establishment
Table 38.17 Gene Therapy Developers: Distribution by Company Size
Table 38.18 Gene Therapy Developers: Distribution by Location of Headquarters
Table 38.19 Most Active Players: Distribution by Number of Gene Therapies
Table 38.20 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Table 38.21 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, since 2017
Table 38.22 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 38.23 Gene Therapy Patent Portfolio: Distribution by Geography
Table 38.24 Gene Therapy Patent Portfolio: North America Scenario
Table 38.25 Gene Therapy Patent Portfolio: Europe Scenario
Table 38.26 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
Table 38.27 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
Table 38.28 Gene Therapy Patent Portfolio: Leading Industry Players: Distribution by Number of Players
Table 38.29 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
Table 38.30 Gene Editing Patent Portfolio: Distribution by Type of Patent
Table 38.31 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, since 2017
Table 38.32 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
Table 38.33 Gene Editing Patent Portfolio: Distribution by Geography
Table 38.34 Gene Editing Patent Portfolio: North America Scenario
Table 38.35 Gene Editing Patent Portfolio: Europe Scenario
Table 38.36 Gene Editing Patent Portfolio: Asia-Pacific Scenario
Table 38.37 Gene Editing Patent Portfolio: Distribution by CPC Symbols
Table 38.38 Leading Industry Players (Gene Editing): Distribution by Number of Patents
Table 38.39 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
Table 38.40 Mergers and Acquisitions: Cumulative Year-Wise Trend, since 2015
Table 38.42 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Table 38.43 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
Table 38.44 Mergers and Acquisitions: Local and International Deals
Table 38.45 Acquisitions: Distribution by Key Value Drivers
Table 38.46 Acquisitions: Distribution by Year of Acquisition and Key Value
Table 38.47 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product
Table 38.48 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company's Product
Table 38.49 Mergers and Acquisitions: Distribution by Therapeutic Area
Table 38.50 Funding and Investments: Cumulative Distribution of Instances by Year, since 2015
Table 38.51 Funding and Investments: Cumulative Distribution of Amount Invested by Year, since 2015 (USD Million)
Table 38.52 Funding and Investments: Distribution of Instances by Type of Funding, since 2015
Table 38.53 Funding and Investments: Distribution of Total Amount Invested by Type of Funding (USD Million)
Table 38.54 Funding and Investments: Summary of Investments, since 2015 (USD Million)
Table 38.55 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
Table 38.56 Funding and Investments: Distribution of Instances and Amount Invested by Geography
Table 38.57 Funding and Investments: Regional Distribution of Funding Instances
Table 38.58 Funding and Investments: Distribution of Instances and Amount Invested by Highest Phase of Development of the Company's Product
Table 38.59 Funding and Investments: Distribution of Instances by Therapeutic Area
Table 38.60 Most Active Players: Distribution by Number of Funding Instances
Table 38.61 Most Active Players: Distribution by Amount Invested (USD Million)
Table 38.62 Funding and Investments: Distribution of Instances by Type of Investors
Table 38.63 Leading Investors: Distribution by Number of Instances
Table 38.64 Funding and Investment Summary, since 2015 (USD Million)
Table 38.65 Clinical Trial Analysis: Distribution by Trial Registration Year, since 2017
Table 38.66 Clinical Trial Analysis: Distribution by Trial Status
Table 38.67 Clinical Trial Analysis: Distribution by Trial Phase
Table 38.68 Clinical Trial Analysis: Distribution by Therapeutic Area
Table 38.69 Clinical Trial Analysis: Distribution by Geography
Table 38.70 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
Table 38.71 Clinical Trial Analysis: Distribution by Trial Status and Geography
Table 38.72 Clinical Trial Analysis: Distribution by Type of Sponsor
Table 38.73 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Table 38.74 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, since 2017
Table 38.75 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Table 38.76 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Table 38.77 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Table 38.78 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
Table 38.79 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
Table 38.80 Demand Analysis: Global Demand for Gene Therapies
Table 38.81 Demand Analysis: Distribution by Stage of Development
Table 38.82 Demand Analysis: Distribution by Therapeutic Area
Table 38.83 Demand Analysis: Distribution by Type of Therapy
Table 38.84 Demand Analysis: Distribution by Type of Gene Delivery Method
Table 38.85 Demand Analysis: Distribution by Geography
Table 38.86 Global Gene Therapy Market, till 2035, Base Scenario (USD Billion)
Table 38.87 Global Gene Therapy Market, till 2035, Conservative Scenario (USD Billion)
Table 38.88 Global Gene Therapy Market, till 2035, Optimistic Scenario (USD Billion)
Table 38.89 Gene Therapy Market: Distribution by Therapeutic Area, 2024, 2029 and 2035
Table 38.90 Gene Therapy Market for Muscle Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.91 Gene Therapy Market for Hematologic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.92 Gene Therapy Market for Dermatological Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.93 Gene Therapy Market for Oncological Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.94 Gene Therapy Market for Genetic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.95 Gene Therapy Market for Ophthalmic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.96 Gene Therapy Market for Metabolic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.97 Gene Therapy Market for Cardiovascular Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.98 Gene Therapy Market for Other Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.99 Gene Therapy Market: Distribution by Type of Vector Used, 2024, 2029 and 2035
Table 38.99 Gene Therapy Market for Adeno-associated Viral Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.101 Gene Therapy Market for Herpes Simplex Virus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.102 Gene Therapy Market for Lentivirus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.103 Gene Therapy Market for Adenovirus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.104 Gene Therapy Market for Non-Viral Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.105 Gene Therapy Market for Retrovirus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.106 Gene Therapy Market for Other-Viral Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.107 Gene Therapy Market: Distribution by Type of Therapy, 2024, 2029 and 2035
Table 38.108 Gene Therapy Market for Gene Augmentation, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.109 Gene Therapy Market for Oncolytic Immunotherapies, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.110 Gene Therapy Market for Gene Editing, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.111 Gene Therapy Market for Gene Regulation, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.112 Gene Therapy Market for Other Therapies, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.113 Gene Therapy Market: Distribution by Type of Gene Delivery Method Used, 2024, 2029 and 2035
Table 38.114 Gene Therapy Market for In vivo Gene Delivery, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.115 Gene Therapy Market for Ex vivo Gene Delivery, Forecasted till 2035, Conservative, Base and Optimistic Scenarios (USD
Table 38.116 Gene Therapy Market: Distribution by Route of Administration, 2024, 2029 and 2035
Table 38.117 Gene Therapy Market for Intravenous till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.118 Gene Therapy Market for Subretinal till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.119 Gene Therapy Market for Intratumoral till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.120 Gene Therapy Market for Intramuscular till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.121 Gene Therapy Market for Other till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.122 Gene Therapy Market: Distribution by Geography, 2024, 2029 and 2035
Table 38.123 Gene Therapy Market for North America, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.124 Gene Therapy Market for Europe, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.125 Gene Therapy Market for Asia-Pacific, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.126 Gene Therapy Market for Latin America, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.127 Gene Therapy Market for Rest of the World, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
Table 38.128 Adstiladrin(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.129 BEQVEZ(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.130 Casgevy(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.131 Elevidys(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.132 Gendicine(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.133 Hemgenix(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.134 IMLYGIC(R) (AMT-061) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.135 Libmeldy(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.136 Luxturna(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.137 LYFGENIA(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.138 Neovasculgen(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.139 Oncorine(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.140 Roctavian(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.141 SKYSONA(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.142 Strimvelis(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.143 Upstaza(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.144 VYJUVEKT(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.145 Zolgensma(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.146 Zynteglo(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.147 AAV-RPE65 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)

List of Figures

Table 38.148 ABO-102 (UX111) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.149 AGTC 501 (rAAV2tYF-GRK1-RPGR) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.150 Cretostimogene Grenadenorepvec (CG0070) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.151 Dirloctocogene samoparvovec (SPK-8011) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.152 DTx-301 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.153 DTx-401 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.154 Generx(R) (Ad5FGF-4) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.155 Giroctocogene fitelparvovec (SB-525 / PF-07055480) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.156 Hologene 5 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.157 INVOSSA(TM) (TG-C) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.158 LUMEVOQ(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.159 NFS-01 (rAAV2-ND4) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.160 NTLA-2001 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.161 Olvi-Vec (Olvimulogene nanivacirepvec) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.162 ProstAtak(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.163 pz-cel (prademagene zamikeracel / EB-101) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.164 RGX-121 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.165 RGX-314 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
Table 38.166 VGX-3100 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)

Gene Therapy Market by Therapeutic Area, Type of Vector, Type of Therapy, Type of Gene Delivery Method, Route of Administration, Geographical Regions, Leading Players and Sales Forecast: Industry Trends and Global Forecasts, Till 2035

Description

Table of Contents

List of Tables

GENE THERAPY MARKET: OVERVIEW

GENE THERAPY MARKET: GROWTH AND TRENDS

GENE THERAPY MARKET: KEY INSIGHTS

1. Presently, 345 developers, worldwide, are engaged in the development of gene therapies targeting various disorders; of these, more than 50% of the stakeholders are headquartered in the US, alone.

2. 70% of the gene therapies being evaluated in phase III clinical trials are gene augmentation therapies; it is worth noting that more than 35% of the candidates have received fast track designation.

3. Close to 95% of the preclinical stage gene therapies are in vivo therapies; more than 20% of the gene therapy candidates are being developed for the treatment of neurological disorders.

4. Over the years, the intellectual capital related to gene therapies has grown at a commendable pace; several patents have been filed by both industry stakeholders and academic players.

5. Over 500 patents focused on gene editing have been filed in the past five years; till date, 6% of such patents have been granted.

6. Rising interest of the stakeholders in this domain is reflected by the number of mergers and acquisitions reported over the last few years; 26% of these initiatives were focused on gaining access to novel platforms.

7. Foreseeing lucrative opportunities, many public and private investors have made investments worth USD 36.4 billion, across more than 560 funding instances.

8. In the recent past, more than 18,000 patients have been enrolled in clinical trials focused on gene therapies; close to 45% trials are evaluating gene therapies for the treatment of oncological disorders.

9. Start-ups are spearheading the innovation in the gene therapy domain; the valuation of a start-up is not solely dependent on profits, instead, it depends on experience and amount previously invested by industry stakeholders.

10. In order to tap into the lucrative opportunity associated with gene therapies, big pharma players have adopted various approaches, from proprietary product development to strategic investments, to advance their portfolios.

11. Given the growing incidence of inherited disorders, the demand for gene therapies (in terms of number of patients) has risen; by 2035, it is anticipated to grow at an annualized rate of 11.67%, across various geographies.

12. Owing to the growing interest towards personalized medicines and high specificity, the market for gene therapies is expected to rise steadily in the foreseeable future.

13. The gene therapy market is anticipated to grow at an annualized rate (CAGR) of 19.25%, till 2035; North America is expected to capture the majority share (close to 65%) of the market by 2035.

GENE THERAPY MARKET: KEY SEGMENTS

Example Players in the Gene Therapy Market

Primary Research Overview

GENE THERAPY MARKET: RESEARCH COVERAGE

KEY QUESTIONS ANSWERED IN THIS REPORT

REASONS TO BUY THIS REPORT

ADDITIONAL BENEFITS

TABLE OF CONTENTS

1. PREFACE

2. RESEARCH METHODOLOGY

3. MARKET DYNAMICS

4. ECONOMIC CONSIDERATONS

5. EXECUTIVE SUMMARY

6. INTRODUCTION

7. GENE DELIVERY VECTORS

8. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIOS

9. MARKET LANDSCAPE

10. DEVELOPER LANDSCAPE

11. COMPANY PROFILES

12. MARKETED GENE THERAPIES

13. KEY COMMERCIALIZATION STRATEGIES

14. LATE-STAGE GENE THERAPIES

15. PATENT ANALYSIS

16. MERGERS AND ACQUISITIONS

17. FUNDING AND INVESTMENTS

18. CLINICAL TRIAL ANALYSIS

19. COST PRICE ANALYSIS

20. START-UP VALUATION

21. BIG PHARMA INITIATIVES

22. DEMAND ANALYSIS

23. MARKET IMPACT ANALYSIS: DRIVERS, RESTRAINTS, OPPORTUNITIES AND CHALLENGES

24. GLOBAL GENE THERAPY MARKET

25. GENE THERAPIES MARKET, BY THERAPEUTIC AREA

26. GENE THERAPIES MARKET, BY TYPE OF VECTOR

27. GENE THERAPIES MARKET, BY TYPE OF THERAPY

28. GENE THERAPIES MARKET, BY TYPE OF GENE DELIVERY METHOD

29. GENE THERAPIES MARKET, BY ROUTE OF ADMINISTRATION

30. GENE THERAPIES MARKET, BY GEOGRAPHICAL REGIONS

31. GENE THERAPIES MARKET, BY LEADING PLAYERS

32. GENE THERAPIES MARKET, SALES FORECAST OF THERAPIES

33. GENE THERAPY MARKET: EMERGING TECHNOLOGIES

34. VECTOR MANUFACTURING FOR GENE THERAPIES

35. CASE STUDY: GENE THERAPY SUPPLY CHAIN

36. EXECUTIVE INSIGHTS

37. CONCLUSION

38. APPENDIX I: TABULATED DATA

39. APPENDIX II: LIST OF COMPANIES AND ORGANIZATIONS

List of Tables

List of Figures