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

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    Report

  • 972 Pages
  • December 2025
  • Region: Global
  • Roots Analysis
  • ID: 5240465
Description Jump to:
The global gene therapy market is estimated to grow from USD 3.4 billion in the current year to USD 16.5 billion by 2035, at a CAGR of 17.0% during the forecast period, till 2035.

GENE THERAPY MARKET: GROWTH AND TRENDS

Congenital abnormalities and inherited genetic mutations include a variety of conditions, such as cancer-related disorders, genetic conditions, autoimmune disorders, and neurological conditions. The World Health Organization (WHO) reports that genetic disorders affect 10 out of every 1,000 individuals, leading to more than 70 million people globally suffering from these conditions. Notably, more than 40% of worldwide infant fatalities relate to various genetic disorders. At present, various research initiatives are underway to develop safe and effective methods for selectively altering the genes that cause diseases in specific patients. Among these, gene therapies have emerged as a promising option. These therapies intend to tackle the underlying genetic cause of a disease by either providing a healthy copy of the mutated gene or by blocking the functions of genes that activate the disease in the organism. Significantly, over 1,100 clinical trials related to gene therapies have been registered in recent years. We anticipate the gene therapy market to grow at a notable growth rate in the next decade.

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:

  • 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.

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

  • Foreseeing lucrative opportunities, many public and private investors have made investments worth USD 36.4 billion, across more than 560 funding instances.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • The gene therapy market is anticipated to grow at an annualized rate (CAGR) of 17.0 %, 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, Company A
  • Co-Founder and Chief Executive Officer, Company B
  • Chief Executive Officer, Chairman and President, Company C
  • Former Chief Executive Officer and President, Company D
  • Former Chief Executive Officer and President, Company E
  • Former Chief Business Officer, Company F
  • Founder and Chief Executive Officer, Company G
  • Chief Executive Officer, Company H
  • Ex Co-Founder, Chief Executive Officer and President, Company I
  • Former Chief Executive Officer, Company J
  • Project Manager and Tatjana Buchholz, Former Marketing Manager, Company K
  • Former Executive and Scientific Director, Company L
  • Former Chief Commercial Officer, Company M
  • Chief Executive Officer and Chairman, Company N
  • Chief Executive Officer, Company O

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 overview, [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 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?

REASONS TO BUY THIS REPORT

  • The report provides a comprehensive market analysis, offering detailed revenue projections of the overall market and its specific sub-segments. This information is valuable to both established market leaders and emerging entrants.
  • 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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Table of Contents

1. PREFACE
1.1. Introduction
1.2. Market Share Insights
1.3. Key Market Insights
1.4. Report Coverage
1.5. Key Questions Answered
1.6. Chapter Outlines
2. RESEARCH METHODOLOGY
2.1. Chapter Overview
2.2. Research Assumptions
2.2.1. Market Landscape and Market Trends
2.2.2. Market Forecast and Opportunity Analysis
2.2.3. Comparative Analysis
2.3. Database Building
2.3.1. Data Collection
2.3.2. Data Validation
2.3.3. Data Analysis
2.4. Project Methodology
2.4.1. Secondary Research
2.4.1.1. Annual Reports
2.4.1.2. Academic Research Papers
2.4.1.3. Company Websites
2.4.1.4. Investor Presentations
2.4.1.5. Regulatory Filings
2.4.1.6. White Papers
2.4.1.7. Industry Publications
2.4.1.8. Conferences and Seminars
2.4.1.9. Government Portals
2.4.1.10. Media and Press Releases
2.4.1.11. Newsletters
2.4.1.12. Industry Databases
2.4.1.13. Roots Proprietary Databases
2.4.1.14. Paid Databases and Sources
2.4.1.15. Social Media Portals
2.4.1.16. Other Secondary Sources
2.4.2. Primary Research
2.4.2.1. Types of Primary Research
2.4.2.1.1. Qualitative Research
2.4.2.1.2. Quantitative Research
2.4.2.1.3. Hybrid Approach
2.4.2.2. Advantages of Primary Research
2.4.2.3. Techniques for Primary 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® (Shenzhen Sibiono GeneTech)
12.2.1. Company Overview
12.2.2. Development Timeline
12.2.3. Mechanism of Action and Vector Used
12.2.4. Target Indication
12.2.5. Current Stage of Development
12.2.6. Manufacturing, Dosage and Sales
12.3. Oncorine® (Shanghai Sunway Biotech)
12.3.1. Company Overview
12.3.2. Development Timeline
12.3.3. Mechanism of Action and Vector Used
12.3.4. Target Indication
12.3.5. Current Stage of Development
12.3.6. Manufacturing, Dosage and Sales
12.4. Rexin-G® (Epeius Biotechnologies)
12.4.1. Company Overview
12.4.2. Development Timeline
12.4.3. Mechanism of Action and Vector Used
12.4.4. Target Indication
12.4.5. Current Stage of Development
12.4.6. Manufacturing, Dosage and Sales
12.5. Neovasculgen® (Human Stem Cells Institute)
12.5.1. Company Overview
12.5.2. Development Timeline
12.5.3. Mechanism of Action and Vector Used
12.5.4. Target Indication
12.5.5. Current Stage of Development
12.5.6. Manufacturing, Dosage and Sales
12.6. Imlygic® (Amgen)
12.6.1. Company Overview
12.6.2. Development Timeline
12.6.3. Mechanism of Action and Vector Used
12.6.4. Target Indication
12.6.5. Current Stage of Development
12.6.6. Manufacturing, Dosage and Sales
12.7. Strimvelis® (Orchard Therapeutics)
12.7.1. Company Overview
12.7.2. Development Timeline
12.7.3. Mechanism of Action and Vector Used
12.7.4. Target Indication
12.7.5. Current Stage of Development
12.7.6. Manufacturing, Dosage and Sales
12.8. Luxturna™ (Spark Therapeutics)
12.8.1. Company Overview
12.8.2. Development Timeline
12.8.3. Mechanism of Action and Vector Used
12.8.4. Target Indication
12.8.5. Current Stage of Development
12.8.6. Manufacturing, Dosage and Sales
12.9. Zolgensa™ (Novartis)
12.9.1. Company Overview
12.9.2. Development Timeline
12.9.3. Mechanism of Action and Vector Used
12.9.4. Target Indication
12.9.5. Current Stage of Development
12.9.6. Manufacturing, Dosage and Sales
12.10. Collategene® (AnGes)
12.10.1. Company Overview
12.10.2. Development Timeline
12.10.3. Mechanism of Action and Vector Used
12.10.4. Target Indication
12.10.5. Current Stage of Development
12.10.6. Manufacturing, Dosage and Sales
12.11. Zyntelgo™ (bluebird bio)
12.11.1. Company Overview
12.11.2. Development Timeline
12.11.3. Mechanism of Action and Vector Used
12.11.4. Target Indication
12.11.5. Current Stage of Development
12.11.6. Manufacturing, Dosage and Sales
12.12. Libmeldy™ (Orchard Therapeutics)
12.12.1. Company Overview
12.12.2. Development Timeline
12.12.3. Mechanism of Action and Vector Used
12.12.4. Target Indication
12.12.5. Current Stage of Development
12.12.6. Manufacturing, Dosage and Sales
13. KEY COMMERCIALIZATION STRATEGIES
13.1. Chapter Overview
13.2. Successful Drug Launch Strategy: ROOTS Framework
13.3. Successful Drug Launch Strategy: Product Differentiation
13.4. Commonly Adopted Commercialization Strategies based on Phase of Development
13.5. List of Approved Gene Therapies
13.6. Key Commercialization Strategies Adopted by Gene Therapy Developers
13.6.1. Strategies Adopted before Therapy Approval
13.6.1.1. Participation in Global Events
13.6.1.2. Collaborations with Stakeholders and Pharmaceutical Firms
13.6.1.3. Indication Expansion
13.6.2. Strategies adopted During / Post Therapy Approval
13.6.2.1. Geographical Expansion
13.6.2.2. Participation in Global Events
13.6.2.3. Patient Assistance Programs
13.6.2.4. Awareness through Product Websites
13.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms
13.7. Concluding Remarks
14. LATE-STAGE GENE THERAPIES
14.1. Chapter Overview
14.2. LUMEVOQ (GS010)
14.3. OTL-103
14.4. PTC-AADC
14.5. BMN 270
14.6. rAd-IFN/Syn3
14.7. beti-cel
14.8. eli-cel
14.9. lovo-cel
14.10. SRP-9001
14.11. EB-101
14.12. ProstAtak
14.13. D-Fi
14.14. CG0070
14.15. Vigil-EWS
14.16. Engensis
14.17. VGX-3100
14.18. INVOSSA (TG-C)
14.19. VYJUVEKT
14.20. PF-06939926
14.21. PF06838435
14.22. PF-07055480
14.23. SPK-8011
14.24. AMT-061
14.25. VB-111
14.26. Generx
14.27. ADXS-HPV
14.28. AGTC 501
14.29. LYS-SAF302
14.30. NFS-01
14.31. AG0302-COVID-19
14.32. RGX-314
14.33. Hologene 5
15. PATENT ANALYSIS
15.1. Chapter Overview
15.2 Gene Therapy: Patent Analysis
15.2.1. Scope and Methodology
15.2.1.1. Analysis by Publication Year
15.2.1.2. Analysis by Publication Year and Type of Patent
15.2.1.3. Analysis by Geography
15.2.1.3.1. Analysis by Geography (North America)
15.2.1.3.2. Analysis by Geography (Europe)
15.2.1.3.3. Analysis by Geography (Asia-Pacific)
15.2.1.4. Analysis by CPC Symbols
15.2.1.5. Analysis by Emerging Focus Areas
15.2.1.6. Leading Players: Analysis by Number of Patents
15.2.1.7. Patent Benchmarking Analysis
15.2.1.7.1. Analysis by Patent Characteristics
15.2.1.7.1.1. Sanofi and Genethon
15.2.1.7.1.2. Other Leading Patent Assignees
15.2.1.8. Patent Valuation Analysis
15.3. Gene Editing Market: Patent Analysis
15.3.1. Scope and Methodology
15.3.1.1. Analysis by Publication Year
15.3.1.2. Analysis by Publication Year and Type of Patent
15.3.1.3. Analysis by Geography
15.3.1.3.1. Analysis by Geography (North America)
15.3.1.3.2. Analysis by Geography (Europe)
15.3.1.3.3. Analysis by Geography (Asia-Pacific)
15.3.1.4. Analysis by CPC Symbols
15.3.1.5. Analysis by Emerging Focus Areas
15.3.1.6. Leading Players: Analysis by Number of Patents
15.3.1.7. Patent Benchmarking Analysis
15.3.1.7.1. Analysis by Patent Characteristics
15.3.1.7.1.1. Intellia Therapeutics and CRISPR Therapeutics
15.3.1.7.1.2. Other Leading Patent Assignees
15.3.1.8. Patent Valuation Analysis
15.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization
16. MERGERS AND ACQUISITIONS
16.1. Chapter Overview
16.2. Merger and Acquisition Models
16.3. Gene Therapy: Mergers and Acquisitions
16.3.1. Analysis by Year of Merger / Acquisition
16.3.2. Analysis by Type of Agreement
16.3.3. Analysis by Geography
16.3.3.1. Intercontinental and Intracontinental Deals
16.3.3.2. Local and International Deals
16.3.4. Analysis by Key Value Drivers
16.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
16.3.5. Analysis by Stage of Development of the Acquired Company’s Product
16.3.6. Analysis by Therapeutic Area
16.4. Analysis by Deal Multiples
17. FUNDING AND INVESTMENTS
17.1. Chapter Overview
17.2. Types of Funding
17.3. Gene Therapy: Funding and Investment Analysis
17.3.1. Analysis by Year of Funding
17.3.2. Analysis by Amount Invested
17.3.3. Analysis by Funding Instance and Amount Invested by Type of Funding
17.3.4. Analysis by Funding Instances and Amount Invested by Year and Type of Funding
17.3.5. Analysis by Funding Instances and Amount Invested by Type of Therapy
17.3.6. Analysis by Funding Instances and Amount Invested by Geography
17.3.7. Analysis by Funding Instances and Amount Invested by Highest Phase of Development
17.3.8. Analysis by Funding Instances by Therapeutic Area
17.3.9. Most Active Players: Analysis by Funding Instances and Amount Invested
17.3.10. Key Investors: Analysis by Number of Funding Instances
17.4. Concluding Remarks
18. CLINICAL TRIAL ANALYSIS
18.1. Chapter Overview
18.2. Scope and Methodology
18.3. Gene Therapy: Clinical Trial Analysis
18.3.1. Analysis by Trial Registration Year
18.3.2. Analysis by Trial Status
18.3.3. Analysis by Trial Phase
18.3.4. Analysis by Therapeutic Area
18.3.5. Analysis by Geography
18.3.6. Analysis by Trial Registration Year and Geography
18.3.7. Analysis by Trial Status and Geography
18.3.8. Analysis by Trial Status, Trial Phase and Geography
18.3.9. Analysis by Therapeutic Area and Geography
18.4. Analysis by Type of Sponsor / Collaborator
18.5. Analysis by Prominent Treatment Sites
18.6. Gene Therapy: Analysis of Enrolled Patient Population
18.6.1. Analysis by Trial Registration Year
18.6.2. Analysis by Trial Status
18.6.3. Analysis by Trial Phase
18.6.4. Analysis by Therapeutic Area
18.6.5. Analysis by Location of Trial Site
18.6.6. Analysis by Trial Status and Location of Trial Site
18.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
18.6.8. Analysis by Therapeutic Area and Location of Trial Site
18.7. Concluding Remarks
19. COST PRICE ANALYSIS
19.1. Chapter Overview
19.2. Factors Contributing to the High Price of Gene Therapies
19.3. Gene Therapy Market: Price Models
19.3.1. Based on Associated Product / Component Costs
19.3.2. Based on Competition
19.3.3. Based on Patient Segment
19.3.4. Based Opinion of Industry Experts
20. START-UP VALUATION
20.1. Chapter Overview
20.2. Valuation by Years of Experience of Start-ups
20.2.1. Methodology
20.2.2. Results and Interpretation
21. BIG PHARMA INITIATIVES
21.1. Chapter Overview
21.2. Gene Therapy: List of Most Prominent Big Pharmaceutical Players
21.2.1. Analysis by Therapeutic Area
21.2.2. Analysis by Type of Vector Used
21.2.3. Analysis by Type of Therapy
21.2.4. Analysis by Type of Gene Delivery Method Used
21.3. Benchmarking Analysis of Key Parameters
21.3.1. Spider Web Analysis: Pipeline Strength
21.3.2. Spider Web Analysis: Mergers and Acquisitions
21.3.3. Spider Web Analysis: Funding and Investments
21.3.4. Spider Web Analysis: Clinical Trials
21.3.5. Spider Web Analysis: Technologies
21.3.6. Spider Web Analysis: Patents
21.4. Benchmarking Analysis of Big Pharmaceutical Players
21.4.1. Spider Web Analysis: Novartis
21.4.2. Spider Web Analysis: Takeda Pharmaceutical
21.4.3. Spider Web Analysis: Roche
21.4.4. Spider Web Analysis: Merck
21.4.5. Spider Web Analysis: Pfizer
21.4.6. Spider Web Analysis: Johnson & Johnson
21.4.7. Spider Web Analysis: AbbVie
21.4.8. Spider Web Analysis: Bristol Myers Squibb
21.4.9. Spider Web Analysis: AstraZeneca
21.4.10. Spider Web Analysis: Sanofi
22. DEMAND ANALYSIS
22.1. Chapter Overview
22.2. Assumptions and Methodology
22.3. Gene Therapy Market: Global Demand Analysis
22.3.1. Analysis by Stage of Development
22.3.2. Analysis by Therapeutic Area
22.3.3. Analysis by Type of Therapy
22.3.4. Analysis by Type of Gene Delivery Method
22.3.5. Analysis by Geography
23. MARKET IMPACT ANALYSIS: DRIVERS, RESTRAINTS, OPPORTUNITIES AND CHALLENGES
23.1. Chapter Overview
23.2. Market Drivers
23.3. Market Restraints
23.4. Market Opportunities
23.5. Market Challenges
23.6. Conclusion
24. GLOBAL GENE THERAPY MARKET
24.1. Chapter Overview
24.2. Key Assumptions and Methodology
24.3. Global Gene Therapy Market, till 2035
24.3.1. Scenario Analysis
24.3.1.1. Conservative Scenario
24.3.1.2. Optimistic Scenario
24.4. Key Market Segmentations
25. GENE THERAPIES MARKET, BY THERAPEUTIC AREA
25.1. Chapter Overview
25.2. Key Assumptions and Methodology
25.3. Gene Therapy Market: Distribution by Therapeutic Area, 2024, 2029 and 2035
25.3.1. Gene Therapy Market for Muscle Disorders, till 2035
25.3.2. Gene Therapy Market for Hematological Disorders, till 2035
25.3.3. Gene Therapy Market for Dermatological Disorders, till 2035
25.3.4. Gene Therapy Market for Oncological Disorders, till 2035
25.3.5. Gene Therapy Market for Genetic Disorders, till 2035
25.3.6. Gene Therapy Market for Ophthalmic Disorders, till 2035
25.3.7. Gene Therapy Market for Metabolic Disorders, till 2035
25.3.8. Gene Therapy Market for Cardiovascular Disorders, till 2035
25.3.9. Gene Therapy Market for Other Disorders, till 2035
25.4. Data Triangulation and Validation
26. GENE THERAPIES MARKET, BY TYPE OF VECTOR
26.1. Chapter Overview
26.2. Key Assumptions and Methodology
26.3. Gene Therapy Market: Distribution by Type of Vector, 2024, 2029 and 2035
26.3.1. Gene Therapy Market for Adeno-associated Virus Vectors, till 2035
26.3.2. Gene Therapy Market for Herpes Simplex Virus Vectors, till 2035
26.3.3. Gene Therapy Market for Lentivirus Vectors, till 2035
26.3.4. Gene Therapy Market for Adenovirus Vectors, till 2035
26.3.5. Gene Therapy Market for Non-viral Vectors, till 2035
26.3.6. Gene Therapy Market for Retrovirus Vectors, till 2035
26.3.7. Gene Therapy Market for Other Viral Vectors, till 2035
26.4. Data Triangulation and Validation
27. GENE THERAPIES MARKET, BY TYPE OF THERAPY
27.1. Chapter Overview
27.2. Key Assumptions and Methodology
27.3. Gene Therapy Market: Distribution by Type of Therapy, 2024, 2029 and 2035
27.3.1. Gene Therapy Market for Gene Augmentation, till 2035
27.3.2. Gene Therapy Market for Oncolytic Immunotherapies, till 2035
27.3.3. Gene Therapy Market for Gene Editing, till 2035
27.3.4. Gene Therapy Market for Gene Regulation, till 2035
27.3.5. Gene Therapy Market for Other Therapies, till 2035
27.4. Data Triangulation and Validation
28. GENE THERAPIES MARKET, BY TYPE OF GENE DELIVERY METHOD
28.1. Chapter Overview
28.2. Key Assumptions and Methodology
28.3. Gene Therapy Market: Distribution by Type of Gene Delivery Method, 2024, 2029 and 2035
28.3.1. Gene Therapy Market for In vivo Gene Delivery, till 2035
28.3.2. Gene Therapy Market for Ex vivo Gene Delivery, till 2035
28.4. Data Triangulation and Validation
29. GENE THERAPIES MARKET, BY ROUTE OF ADMINISTRATION
29.1. Chapter Overview
29.2. Key Assumptions and Methodology
29.3. Gene Therapy Market: Distribution by Route of Administration, 2024, 2029 and 2035
29.3.1. Gene Therapy Market for Intravenous Route, till 2035
29.3.2. Gene Therapy Market for Subretinal Route, till 2035
29.3.3. Gene Therapy Market for Intratumoral Route, till 2035
29.3.4. Gene Therapy Market for Intramuscular Route, till 2035
29.3.5. Gene Therapy Market for Other Routes of Administration, till 2035
29.4. Data Triangulation and Validation
30. GENE THERAPIES MARKET, BY GEOGRAPHICAL REGIONS
30.1. Chapter Overview
30.2. Key Assumptions and Methodology
30.3. Gene Therapy Market: Distribution by Geographical
Regions, 2024, 2029 and 2035
30.3.1. Gene Therapy Market in North America, till 2035
30.3.2. Gene Therapy Market in Europe, till 2035
30.3.3. Gene Therapy Market in Asia-Pacific, till 2035
30.3.4. Gene Therapy Market in Latin America, till 2035
30.3.5. Gene Therapy Market in Rest of the World, till 2035
30.4. Data Triangulation and Validation
31. GENE THERAPIES MARKET, BY LEADING PLAYERS
31.1. Chapter Overview
31.2. Gene Therapy Market: Distribution by Leading Gene Therapy Developers
31.3. Data Triangulation and Validation
32. GENE THERAPIES MARKET, SALES FORECAST OF THERAPIES
32.1. Chapter Overview
32.2. Key Assumptions and Methodology
32.3. Commercialized Gene Therapies Market: Sales Forecast
32.3.1. Adstiladrin®
32.3.1.1. Sales Forecast
32.3.1.2. Net Present Value
32.3.1.3. Value Creation Analysis
32.3.2. BEQVEZ™
32.3.2.1. Sales Forecast
32.3.2.2. Net Present Value
32.3.2.3. Value Creation Analysis
32.3.3. Casgevy™
32.3.3.1. Sales Forecast
32.3.3.2. Net Present Value
32.3.3.3. Value Creation Analysis
32.3.4. Elevidys®
32.3.4.1. Sales Forecast
32.3.4.2. Net Present Value
32.3.4.3. Value Creation Analysis
32.3.5. Gendicine®
32.3.5.1. Sales Forecast
32.3.5.2. Net Present Value
32.3.5.3. Value Creation Analysis
32.3.6. Hemgenix®
32.3.6.1. Sales Forecast
32.3.6.2. Net Present Value
32.3.6.3. Value Creation Analysis
32.3.7. IMLYGIC®
32.3.7.1. Sales Forecast
32.3.7.2. Net Present Value
32.3.7.3. Value Creation Analysis
32.3.8. Libmeldy®
32.3.8.1. Sales Forecast
32.3.8.2. Net Present Value
32.3.8.3. Value Creation Analysis
32.3.9. Luxturna®
32.3.9.1. Sales Forecast
32.3.9.2. Net Present Value
32.3.9.3. Value Creation Analysis
32.3.10. LYFGENIA™
32.3.10.1. Sales Forecast
32.3.10.2. Net Present Value
32.3.10.3. Value Creation Analysis
32.3.11. Neovasculgen®
32.3.11.1. Sales Forecast
32.3.11.2. Net Present Value
32.3.11.3. Value Creation Analysis
32.3.12. Oncorine®
32.3.12.1. Sales Forecast
32.3.12.2. Net Present Value
32.3.12.3. Value Creation Analysis
32.3.13. Roctavian™
32.3.13.1. Sales Forecast
32.3.13.2. Net Present Value
32.3.13.3. Value Creation Analysis
32.3.14. SKYSONA®
32.3.14.1. Sales Forecast
32.3.14.2. Net Present Value
32.3.14.3. Value Creation Analysis
32.3.15. Strimvelis™
32.3.15.1. Sales Forecast
32.3.15.2. Net Present Value
32.3.15.3. Value Creation Analysis
32.3.16. Upstaza™
32.3.16.1. Sales Forecast
32.3.16.2. Net Present Value
32.3.16.3. Value Creation Analysis
32.3.17. VYJUVEKT®
32.3.17.1. Sales Forecast
32.3.17.2. Net Present Value
32.3.17.3. Value Creation Analysis
32.3.18. Zolgensma®
32.3.18.1. Sales Forecast
32.3.18.2. Net Present Value
32.3.18.3. Value Creation Analysis
32.3.19. Zynteglo™
32.3.19.1. Sales Forecast
32.3.19.2. Net Present Value
32.3.19.3. Value Creation Analysis
32.4. Phase III Gene Therapies Market: Sales Forecast
32.4.1. AAV-RPE65
32.4.1.1. Sales Forecast
32.4.1.2. Net Present Value
32.4.1.3. Value Creation Analysis
32.4.2. ABO-102 (UX111)
32.4.2.1. Sales Forecast
32.4.2.2. Net Present Value
32.4.2.3. Value Creation Analysis
32.4.3. AGTC 501 (rAAV2tYF-GRK1-RPGR)
32.4.3.1. Sales Forecast
32.4.3.2. Net Present Value
32.4.3.3. Value Creation Analysis
32.4.4. Cretostimogene Grenadenorepvec (CG0070)
32.4.4.1. Sales Forecast
32.4.4.2. Net Present Value
32.4.4.3. Value Creation Analysis
32.4.5. Dirloctocogene samoparvovec (SPK-8011)
32.4.5.1. Sales Forecast
32.4.5.2. Net Present Value
32.4.5.3. Value Creation Analysis
32.4.6. DTx-301
32.4.6.1. Sales Forecast
32.4.6.2. Net Present Value
32.4.6.3. Value Creation Analysis
32.4.7. DTx-401
32.4.7.1. Sales Forecast
32.4.7.2. Net Present Value
32.4.7.3. Value Creation Analysis
32.4.8. Generx® (Ad5FGF-4)
32.4.8.1. Sales Forecast
32.4.8.2. Net Present Value
32.4.8.3. Value Creation Analysis
32.4.9. Giroctocogene fitelparvovec (SB-525 / PF-07055480)
32.4.9.1. Sales Forecast
32.4.9.2. Net Present Value
32.4.9.3. Value Creation Analysis
32.4.10. Hologene 5
32.4.10.1. Sales Forecast
32.4.10.2. Net Present Value
32.4.10.3. Value Creation Analysis
32.4.11. INVOSSA™ (TG-C)
32.4.11.1. Sales Forecast
32.4.11.2. Net Present Value
32.4.11.3. Value Creation Analysis
32.4.12. LUMEVOQ®
32.4.12.1. Sales Forecast
32.4.12.2. Net Present Value
32.4.12.3. Value Creation Analysis
32.4.13. NFS-01 (rAAV2-ND4)
32.4.13.1. Sales Forecast
32.4.13.2. Net Present Value
32.4.13.3. Value Creation Analysis
32.4.14. NTLA-2001
32.4.14.1. Sales Forecast
32.4.14.2. Net Present Value
32.4.14.3. Value Creation Analysis
32.4.15. Olvi-Vec (Olvimulogene nanivacirepvec)
32.4.15.1. Sales Forecast
32.4.15.2. Net Present Value
32.4.15.3. Value Creation Analysis
32.4.16. ProstAtak®
32.4.16.1. Sales Forecast
32.4.16.2. Net Present Value
32.4.16.3. Value Creation Analysis
32.4.17. pz-cel (prademagene zamikeracel / EB-101)
32.4.17.1. Sales Forecast
32.4.17.2. Net Present Value
32.4.17.3. Value Creation Analysis
32.4.18. RGX-121
32.4.18.1. Sales Forecast
32.4.18.2. Net Present Value
32.4.18.3. Value Creation Analysis
32.4.19. RGX-314
32.4.19.1. Sales Forecast
32.4.19.2. Net Present Value
32.4.19.3. Value Creation Analysis
32.4.20. VGX-3100
32.4.20.1. Sales Forecast
32.4.20.2. Net Present Value
32.4.20.3. Value Creation Analysis
33. GENE THERAPY MARKET: EMERGING TECHNOLOGIES
33.1. Chapter Overview
33.2. Gene Editing Technologies
33.2.1. Applications of Gene Editing
33.3. Emerging Gene Editing Platforms
33.3.1. CRISPR / Cas9 System
33.3.1.1. Technology Providers
33.3.2. Zinc Finger Nuclease
33.3.2.1. Technology Providers
33.3.3. TALENs
33.3.4. megaTAL
33.3.4.1. Technology Providers
33.4. Gene Therapy Regulation Technologies (Gene Switch Technology)
33.4.1. Technology Providers
33.5. Other Emerging Technologies
33.6. Technology Platforms for Developing / Delivering Gene Therapies
33.7. Concluding Remarks
34. VECTOR MANUFACTURING FOR GENE THERAPIES
34.1. Chapter Overview
34.2. Viral Vector Manufacturing Process
34.2.1. Mode of Vector Production
34.2.2. Adherent and Suspension Cultures
34.2.3. Unit Processes and Multiple Parallel Processes
34.2.4. Cell Culture Systems for Production of Viral Vectors
34.2.4.1. Small-Scale / Laboratory Scale Cell Culture Systems
34.2.4.2. Large-Scale Cell Culture Systems
34.2.4.2.1. Stirred Tank Reactor Systems
34.2.4.2.2. Fixed Bed Reactor / Packed Bed Reactor
34.2.4.2.3. WAVE Bioreactor System
34.2.5. Culture Media Specifications
34.3. Bioprocessing of Viral Vectors
34.3.1. AAV Vector Production
34.3.2. Adenoviral Vector Production
34.3.3. Lentiviral Vector Production
34.3.4. ? - Retroviral Vector Production
34.4. Challenges Associated with Vector Manufacturing
34.5. Contract Manufacturing Organizations Involved in Viral Vector Production
35. CASE STUDY: GENE THERAPY SUPPLY CHAIN
35.1. Chapter Overview
35.2. Overview of Gene Therapy Supply Chain
35.3. Implementation of Supply Chain Models
35.4. Logistics in Gene Therapy
35.4.1. Logistic Processes for Autologous and Allogenic Therapies
35.5. Regulatory Supply Chain Across the Globe
35.6. Challenges Associated with Gene Therapy Supply Chain
35.7. Software Applications for Gene Therapy Supply Chain Management
35.7.1. Enterprise Manufacturing System
35.7.2. Laboratory Information Management System
35.7.3. Inventory Management System
35.7.4. Quality Management System
35.7.5. Logistics Management System
35.7.6. Patient Management System
35.7.7. Electronic Clinical Outcome Assessments System
35.7.8. Supply Chain Orchestration Platform
35.8. Recent Developments and Upcoming Trends
36. EXECUTIVE INSIGHTS
36.1. Chapter Overview
36.1. Company A
36.1.1. Company Snapshot
36.1.2. Interview Transcript: Former Corporate Communications Manager
36.2. Company B
36.2.1. Company Snapshot
36.2.2. Interview Transcript: Co-Founder and Chief Executive Officer
36.3. Company C
36.3.1. Company Snapshot
36.3.2. Interview Transcript: Chief Executive Officer, Chairman and President
36.4. Company D
36.4.1. Company Snapshot
36.4.2. Interview Transcript: Former Chief Executive Officer and President
36.5. Company E
36.5.1. Company Snapshot
36.5.2. Interview Transcript: Former Chief Executive Officer and President
36.6. Company F
36.6.1. Company Snapshot
36.6.2. Interview Transcript: Former Chief Business Officer
36.7. Company G
36.7.1. Company Snapshot
36.7.2. Interview Transcript: Founder and Chief Executive Officer
36.8. Company H
36.8.1. Company Snapshot
36.8.2. Interview Transcript: Chief Executive Officer
36.9. Company I
36.9.1. Company Snapshot
36.9.2. Interview Transcript: Ex Co-Founder, Chief Executive Officer and President
36.10. Company J
36.10.1. Company Snapshot
36.10.2. Interview Transcript: Former Chief Executive Officer
36.11. Company K
36.11.1. Company Snapshot
36.11.2. Interview Transcript: Project Manager and Tatjana Buchholz, Former Marketing Manager
36.12. Company L
36.12.1. Company Snapshot
36.12.2. Interview Transcript: Former Executive and Scientific Director
36.13. Company M
36.13.1. Company Snapshot
36.13.2. Interview Transcript: Former Chief Commercial Officer
36.15. Company N
36.15.1. Company Snapshot
36.15.2. Interview Transcript: Chief Executive Officer and Chairman
36.16. Company O
36.16.1. Company Snapshot
36.16.2. Interview Transcript: AL Hawkins, Chief Executive Officer
37. CONCLUSION38. APPENDIX I: TABULATED DATA39. APPENDIX II: LIST OF COMPANIES AND ORGANIZATIONS
List of Figures
Figure 2.1 Research Methodology: Project Methodology
Figure 2.2 Research Methodology: Data Sources for Secondary Research
Figure 3.1 Market Dynamics: Forecast Methodology
Figure 3.2 Market Dynamics: Key Market Segmentation
Figure 3.3 Market Dynamics: Robust Quality Control
Figure 4.1 Lesson Learnt from Past Recessions
Figure 5.1 Executive Summary: Market Landscape
Figure 5.2 Executive Summary: Developer Landscape
Figure 5.3 Executive Summary: Patent Analysis
Figure 5.4 Executive Summary: Mergers and Acquisitions
Figure 5.5 Executive Summary: Funding and Investment Analysis
Figure 5.6 Executive Summary: Clinical Trial Analysis
Figure 5.7 Executive Summary: Demand Analysis
Figure 5.8 Executive Summary: Market Forecast and Opportunity Analysis
Figure 6.1 Evolution of Gene Therapies
Figure 6.2 Ex vivo Gene Therapies
Figure 6.3 In vivo Gene Therapies
Figure 6.4 Mechanism of Action of Gene Therapies
Figure 6.5 Genome Editing: Historical Milestones
Figure 6.6 Types of Genome Editing Techniques
Figure 7.1 Viral and Non-Viral Vectors Used in Gene Therapy
Figure 8.1 Centralized Regulatory Pathway for Market Approval in the US
Figure 8.2 Centralized Regulatory Pathway for Market Approval in Europe
Figure 8.3 Manufacturing Requirements for Gene Therapy Products in China
Figure 8.4 Key Elements of Quality Control in China
Figure 8.5 Centralized Regulatory Pathway for Market Approval in Japan
Figure 9.1 Marketed and Clinical Stage Gene Therapies: Distribution by Stage of Development
Figure 9.2 Marketed and Clinical Stage Gene Therapies: Distribution by Drug Designation
Figure 9.3 Marketed and Clinical Stage Gene Therapies: Distribution by Therapeutic Area
Figure 9.4 Marketed and Clinical Stage Gene Therapies: Distribution by Biological Target
Figure 9.5 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Vector Used
Figure 9.6 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Therapy
Figure 9.7 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Gene Delivery Method
Figure 9.8 Marketed and Clinical Stage Gene Therapies: Distribution by Route of Administration
Figure 9.9 Marketed and Clinical Stage Gene Therapies: Distribution by Dosage
Figure 9.10 Preclinical and Discovery Stage Gene Therapies: Distribution by Stage of Development
Figure 9.11 Preclinical and Discovery Stage Gene Therapies: Distribution by Therapeutic Area
Figure 9.12 Preclinical and Discovery Stage Gene Therapies: Distribution by Biological Target
Figure 9.13 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Vector Used
Figure 9.14 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Therapy
Figure 9.15 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Gene Delivery Method
Figure 10.1 Gene Therapy Developers: Distribution by Year of Establishment
Figure 10.2 Gene Therapy Developers: Distribution by Company Size
Figure 10.3 Gene Therapy Developers: Distribution by Location of Headquarters
Figure 10.4 Most Active Players: Distribution by Number of Gene Therapies
Figure 12.1 Gendicine: Development Timeline
Figure 12.2 Oncorine: Development Timeline
Figure 12.3 Oncorine: Mechanism of Action
Figure 12.4 Rexin-G: Development Timeline
Figure 12.5 Neovasculgen: Development Timeline
Figure 12.6 Imlygic: Development Timeline
Figure 12.7 Imlygic: Mechanism of Action
Figure 12.8 Luxturna: Development Timeline
Figure 12.9 Zolgensma: Development Timeline
Figure 12.10 Collategene: Development Timeline
Figure 12.11 Zynteglo: Development Timeline
Figure 12.12 Libmeldy: Development Timeline
Figure 13.1 Successful Drug Launch Strategy: ROOTS Framework
Figure 13.2 Successful Drug Launch Strategy: Product Differentiation
Figure 13.3 Commonly Adopted Commercialization Strategies based on Phase of Development of Product
Figure 13.4 Harvey Ball Analysis: Commercialization Strategies Adopted by Gene Therapy Developers
Figure 13.5 Approved Gene Therapies: Historical Timeline of Geographical Expansion
Figure 13.6 Approved Gene Therapies: Snapshot of Promotional Activities on Product Websites
Figure 13.7 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
Figure 15.1 Gene Therapy Patent Portfolio: Distribution by Type of Patent
Figure 15.2 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, since 2017
Figure 15.3 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 15.4 Gene Therapy Patent Portfolio: Distribution by Geography
Figure 15.5 Gene Therapy Patent Portfolio: North America Scenario
Figure 15.6 Gene Therapy Patent Portfolio: Europe Scenario
Figure 15.7 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
Figure 15.8 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
Figure 15.9 Gene Therapy Patent Portfolio: Emerging Focus Areas
Figure 15.10 Leading Industry Players (Gene Therapy): Distribution by Number of Patents
Figure 15.11 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
Figure 15.12 Gene Therapy Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 15.13 Gene Therapy Patent Portfolio: Distribution by Patent Age (since 2017)
Figure 15.14 Gene Therapy: Patent Valuation Analysis
Figure 15.15 Gene Editing Patent Portfolio: Distribution by Type of Patent
Figure 15.16 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, since 2017
Figure 15.17 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
Figure 15.18 Gene Editing Patent Portfolio: Distribution by Geography
Figure 15.19 Gene Editing Patent Portfolio: North America Scenario
Figure 15.20 Gene Editing Patent Portfolio: Europe Scenario
Figure 15.21 Gene Editing Patent Portfolio: Asia-Pacific Scenario
Figure 15.22 Gene Editing Patent Portfolio: Distribution by CPC Symbols
Figure 15.23 Gene Editing Patent Portfolio: Emerging Focus Areas
Figure 15.24 Leading Industry Players (Gene Editing): Distribution by Number of Patents
Figure 15.25 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
Figure 15.26 Gene Editing Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
Figure 15.27 Gene Editing Patent Portfolio: Distribution by Patent Age (since 2017)
Figure 15.28 Gene Editing: Patent Valuation Analysis
Figure 15.29 Gene Therapy Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 15.30 Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
Figure 16.1 Mergers and Acquisitions: Cumulative Year-Wise Trend (since 2015)
Figure 16.2 Mergers and Acquisitions: Distribution by Type of Agreement
Figure 16.3 Mergers and Acquisitions: Distribution by Year and Type of Agreement
Figure 16.4 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
Figure 16.5 Mergers and Acquisitions: Local and International Deals
Figure 16.6 Acquisitions: Distribution by Key Value Drivers
Figure 16.7 Acquisitions: Distribution by Key Value Drivers and Year of Acquisition
Figure 16.8 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product
Figure 16.9 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company’s Product and Year of Acquisition
Figure 16.10 Mergers and Acquisitions: Distribution of Deal Amount by Phase of Development of the Acquired Company’s Product
Figure 16.11 Mergers and Acquisitions: Distribution by Therapeutic Area
Figure 17.1 Funding and Investments: Cumulative Year-wise Trend, since 2015
Figure 17.2 Funding and Investments: Cumulative Distribution by Amount Invested, since 2015 (USD Million)
Figure 17.3 Funding and Investments: Distribution of Instances by Type of Funding, since 2015
Figure 17.4 Funding and Investments: Distribution of Total Amount Invested by Type of Funding, since 2015 (USD Million)
Figure 17.5 Funding and Investments: Distribution of Instances by Year and Type of Funding, since 2015
Figure 17.6 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
Figure 17.7 Funding and Investments: Distribution of Instances and Amount Invested by Geography
Figure 17.8 Funding and Investments: Regional Distribution of Funding Instances
Figure 17.9 Funding and Investments: Distribution of Funding Instances, Amount Invested and Phase of Development
Figure 17.10 Funding and Investments: Distribution of Instances by Therapeutic Area
Figure 17.11 Most Active Players: Distribution by Number of Funding Instances, since 2015
Figure 17.12 Most Active Players: Distribution by Amount Invested, since 2015 (USD Million)
Figure 17.13 Funding and Investments: Distribution of Instances by Type of Investors
Figure 17.14 Leading Investors: Distribution by Number of Instances
Figure 17.15 Funding and Investment Summary, since 2015 (USD Million)
Figure 18.1 Clinical Trial Analysis: Cumulative Distribution by Trial Registration Year, since 2017
Figure 18.2 Clinical Trial Analysis: Distribution by Trial Status
Figure 18.3 Clinical Trial Analysis: Distribution by Trial Phase
Figure 18.4 Clinical Trial Analysis: Distribution by Therapeutic Area
Figure 18.5 Clinical Trial Analysis: Distribution by Geography
Figure 18.6 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
Figure 18.7 Clinical Trial Analysis: Distribution by Trial Status and Geography
Figure 18.8 Clinical Trial Analysis: Distribution by Trial Status, Trial Phase and Geography
Figure 18.9 Clinical Trial Analysis: Distribution by Therapeutic Area and Geography
Figure 18.10 Clinical Trial Analysis: Distribution by Type of Sponsor
Figure 18.11 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
Figure 18.12 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, since 2017
Figure 18.13 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
Figure 18.14 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Figure 18.15 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
Figure 18.16 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
Figure 18.17 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
Figure 18.18 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status, Trial Phase and Location of Trial Site
Figure 18.19 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area and Location of Trial Site
Figure 19.1 Gene Therapy: Pricing Model Based on Patient Segment
Figure 20.1 Start-up Valuation: Valuation based on Experience of the Gene Therapy Developer
Figure 21.1 Big Pharma Players: Top Pharmaceutical Companies (Heat Map Representation)
Figure 21.2 Big Pharma Players: Analysis by Therapeutic Area
Figure 21.3 Big Pharma Players: Analysis by Type of Vector Used
Figure 21.4 Big Pharma Players: Analysis by Type of Therapy
Figure 21.5 Big Pharma Players: Analysis by Type of Gene Delivery Method Used
Figure 21.6 Spider Web Analysis: Pipeline Strength
Figure 21.7 Spider Web Analysis: Mergers and Acquisitions
Figure 21.8 Spider Web Analysis: Funding and Investments
Figure 21.9 Spider Web Analysis: Clinical Trials
Figure 21.10 Spider Web Analysis: Technologies
Figure 21.11 Spider Web Analysis: Patents
Figure 21.12 Spider Web Analysis: Novartis
Figure 21.13 Spider Web Analysis: Takeda Pharmaceutical
Figure 21.14 Spider Web Analysis: Roche
Figure 21.15 Spider Web Analysis: Merck
Figure 21.16 Spider Web Analysis: Pfizer
Figure 21.17 Spider Web Analysis: Johnson & Johnson
Figure 21.18 Spider Web Analysis: AbbVie
Figure 21.19 Spider Web Analysis: Bristol Myers Squibb
Figure 21.20 Spider Web Analysis: AstraZeneca
Figure 21.21 Spider Web Analysis:
Sanofi
Figure 22.1 Global Demand for Gene Therapies, till 2035 (in terms of number of patients)
Figure 22.2 Global Demand for Gene Therapies: Distribution by Stage of Development (in terms of number of patients)
Figure 22.3 Global Demand for Gene Therapies: Distribution by Therapeutic Area (in terms of number of patients)
Figure 22.4 Global Demand for Gene Therapies: Distribution by Type of Therapy (in terms of number of patients)
Figure 22.5 Global Demand for Gene Therapies: Distribution by Type of Gene Delivery Method (in terms of number of patients)
Figure 22.6 Global Demand for Gene Therapies: Distribution by Geography (in terms of Number of Patients)
Figure 23.1 Gene Therapy: Market Drivers
Figure 23.2 Gene Therapy: Market Restraints
Figure 23.3 Gene Therapy: Market Opportunities
Figure 23.4 Gene Therapy: Market Challenges
Figure 24.1 Global Gene Therapy Market, till 2035 (USD Billion)
Figure 24.2 Global Gene Therapy Market, till 2035: Conservative Scenario (USD Billion)
Figure 24.3 Global Gene Therapy Market, till 2035: Optimistic Scenario (USD Billion)
Figure 25.1 Gene Therapy Market: Distribution by Therapeutic Area, 2024, 2029 and 2035
Figure 25.2 Gene Therapy Market for Muscle Disorders, till 2035 (USD Billion)
Figure 25.3 Gene Therapy Market for Hematological Disorders, till 2035 (USD Billion)
Figure 25.4 Gene Therapy Market for Dermatological Disorders, till 2035 (USD Billion)
Figure 25.5 Gene Therapy Market for Oncological Disorders, till 2035 (USD Billion)
Figure 25.6 Gene Therapy Market for Genetic Disorders, till 2035 (USD Billion)
Figure 25.7 Gene Therapy Market for Ophthalmic Disorders, till 2035 (USD Billion)
Figure 25.8 Gene Therapy Market for Metabolic Disorders, till 2035 (USD Billion)
Figure 25.9 Gene Therapy Market for Cardiovascular Disorders, till 2035 (USD Billion)
Figure 25.10 Gene Therapy Market for Other Disorders, till 2035 (USD Billion)
Figure 26.1 Gene Therapy Market: Distribution by Type of Vector, 2024, 2029 and 2035
Figure 26.2 Gene Therapy Market for Adeno-associated Virus Vectors, till 2035 (USD Billion)
Figure 26.3 Gene Therapy Market for Herpes Simplex Virus Vectors, till 2035 (USD Billion)
Figure 26.4 Gene Therapy Market for Lentivirus Vectors, till 2035 (USD Billion)
Figure 26.5 Gene Therapy Market for Adenovirus Vectors, till 2035 (USD Billion)
Figure 26.6 Gene Therapy Market for Non-viral Vectors, till 2035 (USD Billion)
Figure 26.7 Gene Therapy Market for Retrovirus Vectors, till 2035 (USD Billion)
Figure 26.8 Gene Therapy Market for Other Viral Vectors, till 2035 (USD Billion)
Figure 27.1 Gene Therapy Market: Distribution by Type of Therapy, 2024, 2029 and 2035
Figure 27.2 Gene Therapy Market for Gene Augmentation, till 2035 (USD Billion)
Figure 27.3 Gene Therapy Market for Oncolytic Immunotherapies, till 2035 (USD Billion)
Figure 27.4 Gene Therapy Market for Gene Editing, till 2035 (USD Billion)
Figure 27.5 Gene Therapy Market for Gene Regulation, till 2035 (USD Billion)
Figure 27.6 Gene Therapy Market for Other Therapies, till 2035 (USD Billion)
Figure 28.1 Gene Therapy Market: Distribution by Type of Gene Delivery Method, 2024, 2029 and 2035
Figure 28.2 Gene Therapy Market for In vivo Gene Delivery, till 2035 (USD Billion)
Figure 28.3 Gene Therapy Market for Ex vivo Gene Delivery, till 2035 (USD Billion)
Figure 29.1 Gene Therapy Market: Distribution by Route of Administration, 2024, 2029 and 2035
Figure 29.2 Gene Therapy Market for Intravenous Route, till 2035 (USD Billion)
Figure 29.3 Gene Therapy Market for Subretinal Route, till 2035 (USD Billion)
Figure 29.4 Gene Therapy Market for Intratumoral Route, till 2035 (USD Billion)
Figure 29.5 Gene Therapy Market for Intramuscular Route, till 2035 (USD Billion)
Figure 29.6 Gene Therapy Market for Other Routes of Administration, till 2035 (USD Billion)
Figure 30.1 Gene Therapy Market: Distribution by Geographical Regions, 2024, 2029 and 2035
Figure 30.2 Gene Therapy Market in North America, till 2035 (USD Billion)
Figure 30.3 Gene Therapy Market in Europe, till 2035 (USD Billion)
Figure 30.4 Gene Therapy Market in Asia-Pacific, till 2035 (USD Billion)
Figure 30.5 Gene Therapy Market in Latin America, till 2035 (USD Billion)
Figure 30.6 Gene Therapy Market in Rest of the World, till 2035 (USD Billion)
Figure 31.1 Gene Therapy Market: Distribution by Leading Gene Therapy Developers (based on sales of gene therapies in the year 2024 (USD Billion))
Figure 31.2 Gene Therapy Market: Distribution by Leading Gene Therapy Developers (based on the forecasted sales of gene therapies in the year 2035 (USD Billion))
Figure 32.1 Gene Therapy Market: Adstiladrin Sales Forecast, till 2035 (USD Million)
Figure 32.2 Gene Therapy Market: BEQVEZ Sales Forecast, till 2035 (USD Million)
Figure 32.3 Gene Therapy Market: Casgevy Sales Forecast, till 2035 (USD Million)
Figure 32.4 Gene Therapy Market: Elevidys Sales Forecast, till 2035 (USD Million)
Figure 32.5 Gene Therapy Market: Gendicine Sales Forecast, till 2035 (USD Million)
Figure 32.6 Gene Therapy Market: Hemgenix Sales Forecast, till 2035 (USD Million)
Figure 32.7 Gene Therapy Market: Imlygic Sales Forecast, till 2035 (USD Million)
Figure 32.8 Gene Therapy Market: Libmeldy Sales Forecast, till 2035 (USD Million)
Figure 32.9 Gene Therapy Market: Luxturna Sales Forecast, till 2035 (USD Million)
Figure 32.10 Gene Therapy Market: LYFGENIA Sales Forecast, till 2035 (USD Million)
Figure 32.11 Gene Therapy Market: Neovasculgen Sales Forecast, till 2035 (USD Million)
Figure 32.12 Gene Therapy Market: Oncorine Sales Forecast, till 2035 (USD Million)
Figure 32.13 Gene Therapy Market: Roctavian Sales Forecast, till 2035 (USD Million)
Figure 32.14 Gene Therapy Market:
SKYSONA Sales Forecast, till 2035 (USD Million)
Figure 32.15 Gene Therapy Market: Strimvelis Sales Forecast, till 2035 (USD Million)
Figure 32.16 Gene Therapy Market: Upstaza Sales Forecast, till 2035 (USD Million)
Figure 32.17 Gene Therapy Market: VYJUVEKT Sales Forecast, till 2035 (USD Million)
Figure 32.18 Gene Therapy Market: Zolgensma Sales Forecast, till 2035 (USD Million)
Figure 32.19 Gene Therapy Market: Zynteglo Sales Forecast, till 2035 (USD Million)
Figure 32.20 Gene Therapy Market: AAV-RPE65 Sales Forecast, till 2035 (USD Million)
Figure 32.21 Gene Therapy Market: ABO-102 (UX111) Sales Forecast, till 2035 (USD Million)
Figure 32.22 Gene Therapy Market: AGTC 501 (rAAV2tYF-GRK1-RPGR) Sales Forecast, till 2035 (USD Million)
Figure 32.23 Gene Therapy Market: Crestostimogene Grenadenorepvec (CG0070) Sales Forecast, till 2035 (USD Million)
Figure 32.24 Gene Therapy Market: Dirloctocogene samoparvovec (SPK-8011) Sales Forecast, till 2035 (USD Million)
Figure 32.25 Gene Therapy Market: DTx-301 Sales Forecast, till 2035 (USD Million)
Figure 32.26 Gene Therapy Market: DTx-401 Sales Forecast, till 2035 (USD Million)
Figure 32.27 Gene Therapy Market: Generx (Ad5FGF-4) Sales Forecast, till 2035 (USD Million)
Figure 32.28 Gene Therapy Market: Giroctocogene fitelparvovec (SB-525 / PF-07055480) Sales Forecast, till 2035 (USD Million)
Figure 32.29 Gene Therapy Market:
Hologene 5 Sales Forecast, till 2035 (USD Million)
Figure 32.30 Gene Therapy Market: INVOSSA (TG-C) Sales Forecast, till 2035 (USD Million)
Figure 32.31 Gene Therapy Market: LUMEVOQ Sales Forecast, till 2035 (USD Million)
Figure 32.32 Gene Therapy Market: NFS-01 (rAAV2-ND4) Sales Forecast, till 2035 (USD Million)
Figure 32.33 Gene Therapy Market: NTLA-2001 Sales Forecast, till 2035 (USD Million)
Figure 32.34 Gene Therapy Market: Olvi-Vec (Olvimulogene nanivacirepvec) Sales Forecast, till 2035 (USD Million)
Figure 32.35 Gene Therapy Market: ProstAtak Sales Forecast, till 2035 (USD Million)
Figure 32.36 Gene Therapy Market: pz-cel (Prademagene zamikeracel / EB-101) Sales Forecast, till 2035 (USD Million)
Figure 32.37 Gene Therapy Market: RGX-121 Sales Forecast, till 2035 (USD Million)
Figure 32.38 Gene Therapy Market: RGX-314 Sales Forecast, till 2035 (USD Million)
Figure 32.39 Gene Therapy Market: VGX-3100 Sales Forecast, till 2035 (USD Million)
Figure 33.1 Gene Editing: Key Application Areas
Figure 33.2 Emerging Gene Editing Platforms
Figure 34.1 Viral Vectors: Key Manufacturing Steps
Figure 35.1 Steps Involved in the Gene Therapy Supply Chain
Figure 35.2 Key Responsibilities of Stakeholders Involved in Gene Therapies Supply Chain
Figure 35.3 Key Steps Involved in the Implementation of a Supply Chain Strategy
Figure 35.4 Comparison of Logistics Processes for Autologous and Allogeneic Therapies
Figure 35.5 Global Regulatory Authorities for Gene Therapy Supply Chain
Figure 35.6 Overview of Supply Chain Orchestration Platform
Figure 37.1 Conclusion: Overall Market Landscape (I / II)
Figure 37.2 Conclusion: Overall Market Landscape (II / II)
Figure 37.3 Conclusion: Developer Landscape
Figure 37.4 Conclusion: Patent Analysis
Figure 37.5 Conclusion: Mergers and Acquisitions
Figure 37.6 Conclusion: Funding and Investment Analysis
Figure 37.7 Conclusion: Clinical Trial Analysis
Figure 37.8 Conclusion: Demand Analysis
Figure 37.9 Conclusion: Market Sizing and Opportunity Analysis (I / II)
Figure 37.10 Conclusion: Market Sizing and Opportunity Analysis (II / II)

Companies Mentioned (Partial List)

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

  • 2seventy bio
  • 3SBio
  • 4basebio
  • 4BIO Capital
  • 4D Molecular Therapeutics
  • 5AM Ventures
  • AAVantgarde Bio
  • aaVective
  • AaviGen
  • AAVogen
  • AbbVie
  • Abby Grace Foundation
  • Abeona Therapeutics
  • Abingworth
  • Access Biotechnology
  • Acrigen Biosciences
  • Adage Capital Management
  • Addgene
  • Addimmune (Previously Known as American Gene Technologies)
  • Adrenas Therapeutics (A Subsidiary of BridgeBio)
  • Advanced BioScience Laboratories
  • Advaxia (Previously Known as Advent)
  • Advaxis
  • Adverum Biotechnologies
  • Aevitas Therapeutics
  • Affinia Therapeutics
  • Agathos Biologics
  • AGC Biologics
  • Agent Capital
  • Aisling Capital
  • AJU IB Investment
  • Akamis Bio
  • Akouos (A Subsidiary of Eli Lilly)
  • Alaska Permanent Fund
  • Alaunos Therapeutics
  • Alcyone Therapeutics
  • Aldevron
  • Alexandria Venture Investments
  • Alia Therapeutics
  • Allergan
  • AllStripes
  • Ally Bridge Group
  • Almirall
  • Alpha Cognition
  • Alpha Holdings
  • AlphaVax
  • Altheia Science
  • Altitude Life Science Ventures
  • Altruist Biologics
  • Alumni Ventures
  • Amarna Therapeutics
  • Ambulero
  • American Gene Technologies
  • American Gene Technologies
  • Amgen
  • Amicus Therapeutics
  • Amplo
  • Amplo Biotechnology
  • AMSBIO
  • Anaeropharma Science
  • Andelyn Biosciences
  • Andreessen Horowitz
  • AnGes
  • Angionetics (A Subsidiary of Gene Biotherapeutics)
  • Anjarium Biosciences
  • ANLBIO
  • Anlong Biopharmaceutical
  • Apellis Pharmaceuticals
  • Apertura Gene Therapy
  • ApolloBio
  • Apple Tree Partners
  • Applied Biological Materials
  • Applied StemCell
  • Applied Viromics
  • Arbor Biotechnologies
  • ARCH Overage Fund
  • ARCH Venture Partners
  • Arctic Light Venture Capital
  • Arcturus Therapeutics
  • Arix Bioscience
  • Arkin Bio Ventures
  • ArrowMark Partners
  • Artgen Biotech
  • Arthrogen
  • ARTIS Ventures
  • Aruvant Sciences
  • Asabys Partners
  • ASC Therapeutics
  • Ascidian Therapeutics
  • Asgard Therapeutics
  • Asia Alpha
  • Asklepios BioPharmaceutical (A Subsidiary by Bayer)
  • Aspa Therapeutics (A Subsidiary of BridgeBio)
  • Aspire Capital Fund
  • Astellas Gene Therapies (A Subsidiary of Astellas Pharma)
  • Astellas Pharma
  • AstraZeneca
  • Atamyo Therapeutics
  • Atlas Venture
  • Atsena Therapeutics
  • Auburn University
  • Audition Therapeutics
  • Auriga Partners
  • Australian Taxation Office
  • Autolus Therapeutics
  • Avellino Lab USA
  • AviadoBio
  • Avid Bioservices
  • Avidity Partners
  • Avrion Therapeutics
  • AVROBIO
  • Axovia Therapeutics (To be Acquired by ALSA Ventures)
  • Ayala Pharmaceuticals (Previously Known as Advaxis)
  • Baillie Gifford
  • Bain Capital Life Sciences
  • Banca Esperia
  • Barclays and Cantor
  • Batavia Biosciences
  • Bayer
  • Beacon Therapeutics (Previously known as AGTC)
  • Beam Therapeutics
  • Beijing Hetang Shenghua Medical Technology
  • Beijing innovec Pharmaceutical Technology
  • Beijing Jinlan Gene Technology
  • Beijing Northland Biotechnology
  • Belief BioMed
  • Berkeley Catalyst Fund
  • Bessemer Trust
  • Bezos Expeditions
  • Bill & Melinda Gates Foundation
  • Bio Palette
  • BioCentriq
  • Biogen
  • Biointaxis
  • BioInvent International
  • Biolocity
  • BioMarin Pharmaceutical
  • Bionic Sight
  • BioNTech Innovative Manufacturing Service
  • BioReliance
  • Bioscience Innovation Grant Program
  • Bio-Sourcing
  • Biotechnology Value Fund
  • BioVec Pharma
  • Biovian
  • Bloomsbury Genetic Therapies
  • Blue Bear Ventures
  • Blue Gen Therapeutics Foundation
  • bluebird bio
  • BlueRock Therapeutics
  • BlueWater Angels
  • Boehringer Ingelheim
  • Bonus BioGroup
  • Borea Therapeutics
  • Boxer Capital
  • Bpifrance
  • Brace Pharma Capital
  • Branca Bunús
  • Breton Capital
  • BridgeBio
  • BrightGene Bio-Medical Technology
  • Bristol Myers Squibb
  • Brookside Capital
  • BVF Partners
  • C4 Holding
  • California Institute for Regenerative Medicine
  • CAM Capital
  • Cambridge Enterprise
  • Cambridge Gene Therapy
  • Cambridge Innovation Capital
  • Camford Capital
  • Canada Pension Plan Investment Board
  • CANbridge Pharmaceuticals
  • Cancer Research Institute
  • Cancer Research UK
  • Candel Therapeutics
  • CanSino Biologics
  • CapDecisif Management
  • Capsida Biotherapeutics
  • Carbon Biosciences
  • Caribou Biosciences
  • Carmine Therapeutics (A Subsidiary of EVX Ventures)
  • CARsgen Therapeutics
  • Cartherics
  • Casdin Capital
  • Case Western Reserve University
  • Castle Creek Biosciences
  • Catalent
  • Catalent Biologics (A Subsidiary of Catalent)
  • Catalyst Biosciences
  • CavoGene LifeSciences
  • CCRM Enterprises
  • CDG Capital
  • Celgene
  • CELLeBRAIN
  • Cellectis
  • CellGenTech
  • Cellular Biomedicine Group
  • CellVec
  • Celonic
  • Celosia Therapeutics
  • Celsion
  • Cenova Capital
  • Center for Breakthrough Medicines
  • Centre for Process Innovation
  • Centre Léon Bérard
  • Century Therapeutics
  • CEVEC Pharmaceuticals
  • CG Oncology
  • CGP Investment
  • Charcot-Marie-Tooth Association
  • Charles River Laboratories
  • Chende Capital
  • Chengdu Genevector Biotechnology
  • Chengdu Origen Biotechnology (Acquired by Chengdu Kanghong Pharmaceutical)
  • Chiba University
  • Chiesi Ventures
  • Children’s Medical Research Foundation
  • Children’s Oncology Group
  • CICC
  • Cima Universidad de Navarra
  • CincyTech
  • Circio (Previously Known as Targovax)
  • CITIC
  • CLAIRIgene
  • Clarus Ventures
  • Clean Cells
  • Clearside Biomedical
  • Clínica Universidad de Navarra
  • CLINO
  • Clough Capital Partners
  • CMG-SDIC Capital
  • Coalition for Epidemic Preparedness Innovations
  • Coave Therapeutics
  • CODA Biotherapeutics
  • Code Biotherapeutics
  • Codexis
  • CoJourney
  • Columbia University
  • Columbus Venture Partners
  • CombiGene
  • Complement Therapeutics
  • Conectus
  • Consort Medical
  • Copernicus Therapeutics
  • Cormorant Asset Management
  • CorrectSequence Therapeutics
  • CorriXR Therapeutics
  • Couaragene
  • Cowen
  • Creative Biogene
  • Creative Biolabs
  • Crispr Stem & Therapeutics
  • CRISPR Therapeutics
  • CSL Behring
  • Cure Genetics
  • CureDuchenne Ventures
  • Curia
  • CyGenica
  • Cynvec
  • Cyprium Therapeutics (A Subsidiary of Fortress Biotech)
  • Cystic Fibrosis Foundation
  • Cytiva
  • D1 Capital Partners
  • Daiichi Sankyo
  • DARPA
  • Decheng Capital
  • Decibel Therapeutics (Acquired by Regeneron Pharmaceuticals)
  • Deerfield Management
  • Defense Advanced Research Projects Agency
  • Dexcel Pharma
  • DH-LT-Investments
  • DINAMIQS
  • DiNAQOR
  • Diorasis Therapeutics
  • DNAlite Therapeutics
  • DNAtrix
  • Dong-A ST
  • Driehaus Capital Management
  • Duke University
  • Duke University Medical Center
  • DyDo Pharma
  • Dyno Therapeutics
  • EB Medical Research Foundation
  • EB Research Partnership
  • EcoR1 Capital
  • EDBI
  • EdiGene
  • Editas Medicine
  • Edmond de Rothschild Investment Partners
  • eGenesis
  • Eight Roads Ventures
  • Eikonoklastes Therapeutics
  • Elaia
  • ElevateBio
  • Eleven Therapeutics
  • Eli Lilly
  • Emendo Biotherapeutics (A Subsidiary of AnGes)
  • Emergent BioSolutions
  • Emerging Technology Partners
  • Emory University
  • Encoded Therapeutics
  • Endsulin
  • enGene
  • Ennovation Ventures
  • Enochian BioSciences
  • Ensoma
  • Entos Pharmaceuticals
  • Epeius Biotechnologies
  • EPIQ Capital Group
  • EPS Holdings
  • Esco Aster
  • Esco Ventures
  • Essex Bio-Technology
  • Esteve
  • Eucalyptus Bio (Previously Known as Crisp-HR)
  • Eudora (A Subsidiary of Replay)
  • Eureka Eurostars
  • Eurocrine Vaccines
  • EUROFANCOLEN
  • European Innovation Council Accelerator
  • European Investment Bank
  • Eventide Asset Management
  • EVERCRISP BIOSCIENCES
  • Evox Therapeutics
  • EVX Ventures
  • ExcellGene
  • Excision BioTherapeutics
  • Exegenesis Bio
  • Exen Capital
  • Exothera
  • Expression Therapeutics
  • ExSight Capital
  • Eyevensys
  • F2 Ventures
  • Farallon Capital Management
  • FAUN Foundation
  • FEDER
  • FerGene
  • Ferring Pharmaceuticals
  • Fidelity Biosciences
  • Fidelity Management & Research Company
  • Fidim
  • Fin Posillipo
  • FinVector
  • Flagship Pioneering
  • Flash Therapeutics
  • Flerie Invest
  • Flexion Therapeutics
  • Fondation Sanfilippo Suisse
  • Fondazione Telethon
  • Forbion
  • Forecyte Bio
  • Foresite Capital
  • Forge Biologics
  • Forty Seven
  • Fosun Capital
  • Foundation Fighting Blindness
  • Fountain Healthcare Partners
  • FoxKiser
  • F-Prime Capital
  • Frametact
  • Franklin Templeton Investments
  • Frazier Healthcare Partners
  • Freeline Therapeutics
  • Friedreich’s Ataxia Research Alliance
  • Frontera Therapeutics
  • FUJIFILM Diosynth Biotechnologies
  • Fund+
  • Fundación Sanfilippo B
  • G&P Biosciences
  • Galia Gestion
  • GEG Tech
  • GEMoaB
  • Genascence
  • GenAssist Therapeutics
  • Gene Biotherapeutics
  • Gene Surgery
  • Gene Therapy Research Institution
  • GeneCopoeia
  • GeneCraft
  • GeneCure Biotechnologies
  • GeneDetect
  • GenEdit
  • GenEditBio
  • Genelux
  • GeneMedicine
  • Genenta Science
  • Genentech
  • GeneOne Life Science
  • GeneQuine Biotherapeutics
  • Generation Bio
  • Generoath
  • Genespire
  • Genethon
  • Genevant Sciences
  • GeneVentiv Therapeutics
  • Genexine
  • Genezen Laboratories
  • Genflow Biosciences
  • GenIbet Biopharmaceuticals
  • GenixCure
  • GenNBio
  • Genotarget
  • Genprex
  • GenScript
  • GenSight Biologics
  • GenVivo
  • Genzyme
  • Georgia Research Alliance
  • Gesetzliche Krankenversicherung Spitzenverband
  • Ghost Tree Capital Group
  • Gimv
  • GO CAPITAL
  • GOFAR
  • GOG Foundation
  • Google Ventures
  • GordonMD Global Investments
  • GQ Bio
  • Grace Science
  • Gradalis
  • Graphite Bio
  • GreatPoint Ventures
  • Green Cross Holdings
  • Gritgen Therapeutics
  • GrittGene Therapeutics
  • Gromit Therapeutics
  • Guangzhou Ruifeng Biotechnology
  • Guofang Capital
  • Gyroscope Therapeutics
  • H. Lee Moffitt Cancer Center and Research Institute
  • HALIX
  • Harvard University
  • Hatteras Venture Partners
  • Hayfin Capital Management
  • HBM Healthcare Investments
  • HealthCap
  • Helixmith
  • Hemera Biosciences
  • Hemogen
  • Herantis Pharma
  • Hercules Capital
  • Hillhouse Capital Group
  • Hiroshima Venture Capital
  • HIV Vaccine Trials Network
  • Holostem Terapie Avanzate
  • Homology Medicines
  • Horizon Technology Finance
  • Hovione Scientia
  • Huapont Life Sciences
  • Huida Shanghai Biotechnology
  • HuidaGene Therapeutics
  • Human Stem Cells Institute PJSC
  • Icahn School of Medicine at Mount Sinai
  • ICM
  • Idinvest Partners
  • IDT Biologika
  • iECURE
  • Ikarian Capital
  • Ikarovec
  • Illumina Ventures
  • Ilya Pharma
  • Imperial Innovations
  • IMUNON
  • Imvus
  • Inception Capital Management
  • Inhibrx
  • InnoSkel
  • Innostellar Biotherapeutics
  • Innovate UK
  • Innovation Angel Funds
  • Innovation Network Corporation of Japan (ICNJ)
  • Innovation Norway
  • InnovaVector
  • InnoVision Capital
  • Inomagen Therapeutics
  • Inovio Pharmaceuticals
  • Inscripta
  • Inserm Transfert
  • Institute of Myology
  • Intas Pharmaceuticals
  • Intellia Therapeutics
  • InverVest
  • Investitionsbank des Landes Brandenburg
  • InvivoGen
  • Invus
  • Israel Innovation Authority
  • IVERIC bio
  • Jaguar Gene Therapy
  • Janus Capital Management
  • Janus Henderson Investors
  • Jasper Therapeutics
  • JCR Pharmaceuticals
  • Jeito Capital
  • Jennison Associates
  • Jeune (A Subsidiary of Krystal Biotech)
  • Jiangsu Puxin Biomedicine
  • Jinwei
  • Johnson & Johnson
  • Johnson & Johnson Innovative Medicine (Previously known as Janssen Pharmaceuticals)
  • Juda Capital
  • K2 Venture Capital
  • Kanglin Biotechnology
  • Kate Therapeutics
  • Kendall Capital Partners
  • Kepler Cheuvreux
  • Keytone Ventures
  • Kinea Bio
  • Kissei Pharmaceutical
  • Kite Pharma
  • Kiwoom Securities
  • Kodikaz Therapeutic Solutions
  • Kohlberg Kravis Roberts
  • Kolon TissueGene
  • Korea Investment Partners
  • Kriya Therapeutics
  • Krystal Biotech
  • KSQ Therapeutics
  • Kubota Vision
  • Kurma Partners
  • Kyorin Pharmaceutical
  • Kyverna Therapeutics
  • Lacerta Therapeutics
  • Lantu Biopharma
  • Leerink Partners
  • Lee's Pharmaceutical
  • Legend Capital
  • Leiden University Medical Center
  • Leland Stanford Junior University
  • Lentistem Biotech
  • LENZ Therapeutics
  • Lepu Medical Technology
  • LEXEO Therapeutics
  • Life Sciences Partners
  • LifeArc
  • Lightstone Ventures
  • Lilly Asia Ventures
  • Lime Asset Management
  • Lipigon Pharmaceuticals
  • LogicBio Therapeutics (Acquired by Alexion)
  • Lokon Pharma
  • Longevity Biotech
  • Longitude Capital
  • Longmen Capital
  • Longwood Fund
  • LP Investments
  • Ludwig Cancer Research
  • Lumira Ventures
  • Lundbeckfond Ventures
  • LYFE Capital
  • Lysogene
  • Malin
  • Mangrove Partners
  • Maryland Technology Development
  • Matrix Capital Management
  • Matrix Partners China
  • Maverick Ventures
  • MaxCyte
  • Maze Therapeutics
  • University of Texas- MD Anderson Cancer Center
  • MDimune
  • MedImmune
  • Medinno
  • Mediphage Bioceuticals
  • Medison Ventures
  • MeiraGTx
  • Memorial Sloan Kettering Cancer Center
  • Mercia Technologies
  • Merck
  • MERITZ Securities
  • Metagenomi
  • Michigan Economic Development
  • MidCap Financial
  • Milo Biotechnology
  • Minicircle
  • Mirae Asset Global Investments
  • Mirum Pharmaceuticals
  • Mita Securities
  • Mitsubishi Tanabe Pharma
  • Mitsubishi UFJ Capital
  • Modalis Therapeutics
  • Moderna
  • Momotaro-Gene
  • Monograph Capital
  • Morningside
  • Movember
  • MPM Capital
  • MultiVir
  • Muscular Dystrophy Association
  • Mustang Bio
  • Myonexus Therapeutics
  • Myosana Therapeutics
  • Myrtelle
  • NanoCor Therapeutics
  • Nanoscope Therapeutics
  • Narya Capital
  • National Cancer Institute
  • National Education Association
  • National Eye Institute
  • National Health Service
  • National Institute of Allergy and Infectious Diseases
  • National Institute of Child Health and Human Development
  • National Institute of Dental and Craniofacial Research
  • National Institute of Neurological Disorders and Stroke
  • National Institutes of Health
  • National MPS Society
  • Nationwide Children’s Hospital
  • Navega Therapeutics
  • NEC
  • Neo Gene Pharm
  • NeoMed
  • Nerveda
  • Nervosave Therapeutics
  • Netherlands Enterprise Agency
  • NeuExcell Therapeutics
  • Neuracle Genetics
  • Neurochase
  • Neurocrine Biosciences
  • Neurogene
  • Neurophth Therapeutics
  • NeuShen Therapeutics
  • Neuvocor
  • New Energy and Industrial Technology Development Organization
  • New Enterprise Associates
  • New Leaf Venture Partners
  • New Therapies (NT)
Council
  • New York Stem Cell Foundation
  • Newsoara BioPharma
  • Next Generation Gene Therapeutics Biotechnology
  • Ninevah Therapeutics
  • Nissei Capital
  • Noga Therapeutics
  • Nolan Capital
  • Northern Light Venture Capital
  • Northpond Ventures
  • Novartis
  • Novo Holdings (A Subsidiary of Novo Nordisk)
  • Novo Nordisk
  • Nuntius Therapeutics
  • Oberland Capital
  • Obsidian Therapeutics
  • Ocugen
  • Odylia Therapeutics
  • Oisin Biotechnologies
  • Omega Funds
  • Omnes Capital
  • Oncolys BioPharma
  • Oncorus
  • OncoSec Immunotherapies
  • OncoSenX
  • ONK Therapeutics
  • Opus Genetics
  • OrbiMed
  • OrbiMed Advisors
  • ORCA Therapeutics
  • Orchard Therapeutics (Acquired by Kyowa Kirin)
  • ORI Capital
  • Orphina Biotechnology
  • Orphinic Scientific
  • OS Therapies
  • Osage University Partners
  • Osaka University
  • OSI Investment
Management
  • Otonomy
  • Otsuka Pharmaceutical
  • Oxford BioMedica
  • Oxford Finance
  • Oxular
  • Pacira BioSciences
  • Panmure Gordon
  • Pappas Capital
  • Paragon Biosciences
  • Parker Institute for Cancer Immunotherapy
  • Parkwalk
  • Particella
  • Partners Innovation Fund
  • Passage Bio
  • Patient Square Capital
  • Pattern Biosciences
  • Pavilion Capital
  • PBM Capital
  • Pentwater Capital Management
  • Perceptive Advisors
  • PeriphaGen
  • Perseverance Capital Management
  • Pfizer
  • PFM Health Sciences
  • Pharmakon Advisors
  • Pharming Group
  • Phlox Therapeutics
  • Phoenix Nest
  • PhorMed
  • Pivotal bioVenture Partners
  • PlasmidFactory
  • Pontifax
  • POSCO Capital
  • Poseida Therapeutics
  • Precigen
  • Precision BioSciences
  • Prevail Therapeutics (A Subsidiary of Eli Lilly)
  • ProBioGen
  • Prosight Management
  • Prostate Cancer Foundation
  • PsiOxus Therapeutics
  • PTC Therapeutics
  • Purespring Therapeutics
  • Qianhai Funds of Funds
  • Qianzhan Investment Management
  • Qiming Venture Partners
  • QVT Financial
  • RA Capital Management
  • Ray Therapeutics
  • RBV Capital
  • RD Fund
  • Recipharm
  • ReCode Therapeutics
  • Red Sanfilippo Foundation
  • Redbiotec
  • Redmile Group
  • Redpin Therapeutics
  • Reflection Biotechnologies
  • Reforgene Medicine
  • Regel Therapeutics
  • Regeneron Pharmaceuticals
  • REGENXBIO
  • Rejuvenate Bio
  • Remedium Bio
  • Renova Therapeutics
  • Renovaro
  • Research Council of Norway
  • Restore Vision
  • Rev1 Ventures
  • Reyon Pharmaceutical
  • Rznomics
  • Ridgeback Capital Investments
  • Ring Therapeutics
  • Roche
  • Rock Springs Capital Management
  • Rocket Pharmaceuticals
  • Roswell Park Comprehensive Cancer Center
  • Royal Holloway
University of London
  • Royal Melbourne Hospital
  • Royalty Pharma
  • RTW Investments
  • SAFE Pharmaceutical
  • Saifu Pharmaceutical
  • SalioGen Therapeutics
  • Samsara BioCapital
  • Samum Vermögensverwaltungs
  • San Rocco Therapeutics
  • Sana Biotechnology
  • Sanfilippo Children’s Foundation
  • Sangamo Therapeutics
  • Sanofi
  • Santen Pharmaceutical
  • Santhara Pharmaceuticals
  • Sardocor (A subsidiary of Medera)
  • Sarepta Therapeutics
  • SBI Japan-Israel Innovation Fund
  • Scancell
  • Schroder Adveq
  • Scottish Enterprise
  • Scribe Therapeutics
  • S-Cubed Capital
  • SDL Ventures
  • SEAL Therapeutics
  • Seattle Children’s Research Institute
  • Sectoral Asset Management
  • Seelos Therapeutics
  • Selecta Biosciences
  • Seneca Therapeutics
  • Sensorion
  • Sequoia Capital China
  • Seroba Life Sciences
  • Seven and Eight Biopharmaceuticals
  • Seventure Partners
  • Sham Innovation Santé
  • Shanghai BDgene Technology
  • Shanghai Jinwei Biotechnology
  • Shanghai Refreshgene Therapeutics
  • Shanghai Sunway Biotech
  • Shanghai Vitalgen BioPharma
  • Shantou University
  • Shanxi Medical University
  • Shape Therapeutics
  • Shenzhen Qianhai Taxus
  • Sherpa Healthcare Partners
  • Shinhan Capital
  • Shinsei Corporate Investment
  • Sibiono GeneTech
  • Sight Again
  • Silicon Valley Bank
  • SillaJen
  • SineuGene Therapeutics
  • Sio Gene Therapies
  • Sirtex Medical
  • SiSaf
  • SK pharmateco
  • Skyline Therapeutics
  • SMBC Venture Capital
  • SML Genetree
  • Sofinnova Ventures
  • SOLA Biosciences
  • Solid Biosciences
  • Song Hong Fang
  • Sonova
  • Sorrento Therapeutics
  • SOSV
  • Spanish Ministry of Science
  • SparingVision
  • Spark Therapeutics
  • Sphera Global Healthcare Fund
  • Spirovant Sciences
  • SpliceBio
  • SR One
  • SR-Tiget
  • St. Jude Children’s
Research Hospital
  • StemCardia
  • Stockholm University
  • Stop Sanfilippo Fundación
  • StrideBio (Acquired by Boston Company Ginkgo Bioworks)
  • STRM.BIO
  • Surveyor Capital
  • Suvretta Capital Management
  • SV Health Investors
  • SVB Leerink
  • SVE Capital
  • SwanBio Therapeutics
  • Swedbank Robur
  • Swiftgen
  • Syncona
  • T&R Biofab
  • T. Rowe Price Associates
  • Takeda Pharmaceutical
  • Tamid Bio (A Subsidiary of Fortress Biotech)
  • Target ALS Foundation
  • Targovax
  • Taysha Gene Therapies
  • Team Sanfilippo Foundation
  • TeamedOn International
  • Tech Coast Angels
  • TechLife Capital
  • Telaria (A Subsidiary of Replay)
  • Telomere Therapeutics
  • Temasek
  • Temple University
  • Tenaya Therapeutics
  • TeneoBio
  • Tetraneuron
  • The Column
  • The Invus
  • Théa Open Innovation
  • TheraBiologics
  • Theravectys
  • Theriva Biologics (Acquired by Synthetic Biologics)
  • Third Rock Ventures
  • ToolGen
  • TPG Capital
  • Transgene
  • Transhuman Capital
  • Trentino Invest
  • Triumph Venture Capital
  • Trizell
  • Tsinghua Holdings Capital
  • Tufts University
  • Turenne
  • Tyris Therapeutics (A Subsidiary of Columbus Venture Partners)
  • UCB
  • UCL Technology Fund
  • UK Cystic Fibrosis Gene Therapy Consortium
  • UK Innovation & Science Seed Fund
  • Ultragenyx Pharmaceutical
  • UMass Chan Medical School
  • UNC Gene Therapy Center
  • uniQure Biopharma
  • Universitat Autònoma de Barcelona
  • University College London
  • University Hospitals Cleveland Medical Center
  • University of Alabama at Birmingham
  • University of California
  • University of Florida
  • University of Leeds
  • University of Massachusetts
  • University of Minnesota
  • University of Modena and Reggio Emilia
  • University of North Carolina at Chapel Hill
  • University of Oxford
  • University of Padua
  • University of Pennsylvania
  • University of Pittsburgh
  • University of Utah
  • University of Washington
  • University of Washington School of Medicine
  • UPMC Enterprises
  • Uppsala University
  • Urovant Sciences
  • Usher 2020 Foundation
  • Valencian Innovation Agency
  • Valencian Institute of Finance
  • Valor Equity Partners
  • V-Bio Ventures
  • VBL Therapeutics
  • VCGT (A Subsidiary of Vision Care)
  • Vector Neurosciences
  • VectorY Therapeutics
  • venBio
  • Venrock
  • Versant Ventures
  • Vertex Pharmaceuticals
  • Verve Therapeutics
  • ViaCyte
  • Vickers Venture Partners
  • Vida Ventures
  • Vigene Biosciences
  • ViGeneron
  • Viking Global Investors
  • Viralgen
  • VivaZome Therapeutics
  • Vivet Therapeutics
  • Vivo Capital
  • Voyager Therapeutics
  • VREX Therapeutics
  • Washington Research Foundation
  • Washington State University
  • Waverly Capital
  • Weill Cornell Medicine
  • Weizmann Institute of Science
  • Wellcome Trust
  • Wellington Management
  • Westlake Village BioPartners
  • Whitesun Healthcare Ventures
  • WI Harper
  • Wize Pharma
  • Woodford Investment Management
  • Woodline Partners
  • Wu Capital
  • Wuhan Binhui Biotechnology
  • WuXi Advanced Therapies
  • WuXi AppTec
  • Wyvern Pharmaceuticals
  • Xalud Therapeutics
  • XyloCor Therapeutics
  • Yangtze River Pharmaceutical
  • Yonsei University
  • Ysios Capital
  • Yuhan
  • Zhongmou Therapeutics Technology
  • ZIOPHARM Oncology
  • ZYNEYRO

Methodology

 

 

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About the Gene Therapy Market

Gene therapy is a form of genomics that involves the introduction of genetic material into a patient's cells to treat or prevent a disease. It is a rapidly growing field of medicine, with the potential to revolutionize the treatment of genetic diseases. Gene therapy works by introducing a healthy gene into a patient's cells, replacing a defective gene or providing a missing gene. This can be done through a variety of methods, such as viral vectors, gene editing, and stem cell therapy. Gene therapy has the potential to provide long-term, curative treatments for a wide range of genetic diseases, including cancer, cystic fibrosis, and hemophilia. It is also being explored as a potential treatment for other conditions, such as Alzheimer's disease and Parkinson's disease. Gene therapy is a rapidly growing field, with many companies investing in research and development. Companies in the gene therapy market include Editas Medicine, Intellia Therapeutics, CRISPR Therapeutics, and Spark Therapeutics. Show Less Read more

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