基因治療市場:產業趨勢與世界預測 (～2035年) - 各治療領域，各媒介類型，各治療類型，各基因轉移方法類型，各給藥途徑，各地區，主要企業，銷售預測

2024年全球基因治療市場規模為24億美元，預計在2024-2035年預測期間內複合年增長率為19.25%。

先天性異常和遺傳性基因突變引起的多種疾病，如自體免疫疾病、遺傳性疾病、神經系統疾病、腫瘤疾病等，對個體健康有重大影響。這些疾病是某些遺傳異常的結果，這些異常會幹擾基因的正常功能並對翻譯過程產生不利影響。根據世界衛生組織（WHO）公佈的統計數據，全球每1,000人中就有10人患有遺傳疾病，全球約有7,000萬人。此外，全球 40% 以上的嬰兒死亡率與各種遺傳疾病有關。因此，為了應對這些挑戰，基因治療已成為一種突破性方法，有望治療多種疾病，重點是直接向受影響的器官和細胞提供標靶治療。

基因治療的目標是揭示疾病的潛在遺傳原因，然後在體內引入突變基因的健康版本或缺陷/致病基因以使其失去活性。值得注意的是，2003年，Gendicine（R）（Civiono Genetech）成為全球第一個獲得中國FDA批准的基因療法。過去二十年帶來了與基因治療發展相關的多項突破。例如，2017年12月，美國FDA批准了Luxturna®（Spark Therapeutics），這是第一個治療遺傳性視網膜疾病的基因療法。此外，2019年5月，美國FDA批准了ZOLGENSMA®（諾華），這是第一個用於治療兒科患者脊髓性肌肉萎縮症的基因療法。值得注意的是，該療法獲得了多項認定：突破性療法、快速通道、孤兒藥和優先審查認定，從而在美國加速獲得批准。此外，2023 年，五種基因治療藥物獲得美國 FDA 批准用於各種治療適應症。

快速發展的基因治療產品組合正在推動新創公司和大型製藥公司進入基因治療市場的成長趨勢。目前，全球有 345 家基因治療公司參與開發各種早期和晚期治療方法。近年來，基因修飾、基因組編輯、基因組定序和操作技術（分子/基因開關）等新技術與先進基因轉移方法的融合顯著增加。此外，包括奈米粒子和混合載體系統在內的下一代遞送平台已被證明可以促進基於基因的治療的有效、精確和安全的遞送，從而使更有針對性的治療方法成為可能。由於技術進步的融合和對單劑量有效療法的日益偏好，預計基因治療市場在預測期內將出現顯著的市場成長。

本報告提供全球基因治療市場相關調查，提供市場概要，以及各治療領域，各媒介類型，各治療類型，各基因轉移方法類型，各給藥途徑趨勢，各地區的趨勢，及加入此市場的主要企業簡介等資訊。

目錄

第1章 序文

第2章 調查手法

第3章 市場動態

第4章 經濟考慮

第5章 摘要整理

第6章 簡介

• 概要
• 基因治療的演進
• 基因治療的分類
• 給藥途徑
• 基因治療的作用機制
• 基因治療的優點和缺點
• 基因治療伴隨的課題
• 基因編輯概要
• 結論

第7章 基因轉移媒介

• 概要
• 基因轉移的方法
• 基因改造治療的病毒載體
• 基因改造治療的非病毒載體

第8章 與法規情形償付Scenario

第9章 市場形勢

第10章 開發商的形勢

• 概要
• 基因治療:競爭情形

第11章 企業簡介

• 概要
• 北美
• 歐洲
• 亞太地區，其他地區

第12章 已上市基因治療

• 概要
• Gendicine(R)(深圳 Sibiono GeneTech)
• Oncorine(R) (Shanghai Sunway Biotech)
• Rexin-G(R) (Epeius Biotechnologies)
• Neovasculgen(R) (Human Stem Cells Institute)
• Imlygic(R) (Amgen)
• Strimvelis(R) (Orchard Therapeutics)
• Luxturna(TM) (Spark Therapeutics)
• Zolgensa(TM) (Novartis)
• Collategene(R) (AnGes)
• Zyntelgo(TM)(bluebird bio)
• Libmeldy(TM)(Orchard Therapeutics)

第13章 主要商業化策略

第14章 後期基因治療

第15章 專利分析

• 概要
• 基因治療:專利分析
• 遺傳基因編輯市場:專利分析
• 智慧財產權組合全體:組織類型分別分析

第16章 合併和收購

• 概要
• 合併和收購模式
• 基因治療:合併和收購
• 交易倍率分析

第17章 資金籌措與投資

第18章 臨床試驗的分析

第19章 原價分析

• 概要
• 為基因治療的高額化貢獻的要素
• 基因治療市場:定價模式

第20章 Start-Ups的評估

第21章 大型製藥公司的配合措施

第22章 需求分析

• 概要
• 前提調查手法
• 基因治療市場:全球需求分析

第23章 市場影響分析:促進因素，阻礙因素，機會，課題

第24章 全球基因治療市場

第25章 基因治療市場(各治療領域)

第26章 基因治療市場(各媒介類型)

第27章 基因治療市場(各治療類型)

第28章 基因治療市場(各基因轉移方法類型)

第29章 基因治療市場(各給藥途徑)

第30章 基因治療市場(各地區)

第31章 基因治療市場，各主要企業

第32章 基因治療市場，治療藥的銷售預測

第33章 基因治療市場:新興技術

第34章 基因治療用媒介的製造

第35章 案例研究:基因治療供應鏈

第36章 執行洞察

• 概要
• Orchard Therapeutics
• Vivet Therapeutics
• Kubota Pharmaceutical
• Eyevensys
• AGTC
• LogicBio Therapeutics
• AAVogen
• Hemera Biosciences
• Myonexus Therapeutics
• Arthrogen
• PlasmidFactory
• Delphi Genetics
• Vigene Biosciences
• Gene Biotherapeutics
• Milo Biotechnology

第37章 結論

第38章 附錄1:表格形式的資料

第39章 附錄2:企業·團體一覽

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

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

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

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

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

Key Market Segments

Therapeutic Area

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

Type of Vector

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

Type of Therapy

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

Type of Gene Delivery Method

• Ex vivo Gene Delivery
• In vivo Gene Delivery

Route of Administration

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

Key Geographical Regions

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

Leading Players

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

Report Coverage:

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

Key Benefits of Buying this Report

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

Leading Market Companies

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

TABLE OF CONTENTS

1. PREFACE

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

2. RESEARCH METHODOLOGY

• 2.1. Chapter Overview
• 2.2. Research Assumptions
  • 2.2.1. Market Landscape and Market Trends
  • 2.2.2. Market Forecast and Opportunity Analysis
  • 2.2.3. Comparative Analysis
• 2.3. Database Building
  • 2.3.1. Data Collection
  • 2.3.2. Data Validation
  • 2.3.3. Data Analysis
• 2.4. Project Methodology
  • 2.4.1. Secondary Research
    • 2.4.1.1. Annual Reports
    • 2.4.1.2. Academic Research Papers
    • 2.4.1.3. Company Websites
    • 2.4.1.4. Investor Presentations
    • 2.4.1.5. Regulatory Filings
    • 2.4.1.6. White Papers
    • 2.4.1.7. Industry Publications
    • 2.4.1.8. Conferences and Seminars
    • 2.4.1.9. Government Portals
    • 2.4.1.10. Media and Press Releases
    • 2.4.1.11. Newsletters
    • 2.4.1.12. Industry Databases
    • 2.4.1.13. Roots Proprietary Databases
    • 2.4.1.14. Paid Databases and Sources
    • 2.4.1.15. Social Media Portals
    • 2.4.1.16. Other Secondary Sources
  • 2.4.2. Primary Research
    • 2.4.2.1. Types of Primary Research
      • 2.4.2.1.1. Qualitative Research
      • 2.4.2.1.2. Quantitative Research
      • 2.4.2.1.3. Hybrid Approach
    • 2.4.2.2. Advantages of Primary Research
    • 2.4.2.3. Techniques for Primary
      • 2.4.2.3.1. Interviews
      • 2.4.2.3.2. Surveys
      • 2.4.2.3.3. Focus Groups
      • 2.4.2.3.4. Observational Research
      • 2.4.2.3.5. Social Media Interactions
    • 2.4.2.4. Key Opinion Leaders Considered in Primary Research
      • 2.4.2.4.1. Company Executives (CXOs)
      • 2.4.2.4.2. Board of Directors
      • 2.4.2.4.3. Company Presidents and Vice Presidents
      • 2.4.2.4.4. Research and Development Heads
      • 2.4.2.4.5. Technical Experts
      • 2.4.2.4.6. Subject Matter Experts
      • 2.4.2.4.7. Scientists
      • 2.4.2.4.8. Doctors and Other Healthcare Providers
    • 2.4.2.5. Ethics and Integrity
      • 2.4.2.5.1. Research Ethics
      • 2.4.2.5.2. Data Integrity
  • 2.4.3. Analytical Tools and Databases

3. MARKET DYNAMICS

• 3.1. Chapter Overview
• 3.2. Forecast Methodology
  • 3.2.1. Top-down Approach
  • 3.2.2. Bottom-up Approach
  • 3.2.3. Hybrid Approach
• 3.3. Market Assessment Framework
  • 3.3.1. Total Addressable Market (TAM)
  • 3.3.2. Serviceable Addressable Market (SAM)
  • 3.3.3. Serviceable Obtainable Market (SOM)
  • 3.3.4. Currently Acquired Market (CAM)
• 3.4. Forecasting Tools and Techniques
  • 3.4.1. Qualitative Forecasting
  • 3.4.2. Correlation
  • 3.4.3. Regression
  • 3.4.4. Extrapolation
  • 3.4.5. Convergence
  • 3.4.6. Sensitivity Analysis
  • 3.4.7. Scenario Planning
  • 3.4.8. Data Visualization
  • 3.4.9. Time Series Analysis
  • 3.4.10. Forecast Error Analysis
• 3.5. Key Considerations
  • 3.5.1. Demographics
  • 3.5.2. Government Regulations
  • 3.5.3. Reimbursement Scenarios
  • 3.5.4. Market Access
  • 3.5.5. Supply Chain
  • 3.5.6. Industry Consolidation
  • 3.5.7. Pandemic / Unforeseen Disruptions Impact
• 3.6. Key Market Segmentation
• 3.7. Robust Quality Control
• 3.8. Limitations

4. ECONOMIC CONSIDERATONS

• 4.1. Chapter Overview
• 4.2. Market Dynamics
  • 4.2.1. Time Period
    • 4.2.1.1. Historical Trends
    • 4.2.1.2. Current and Forecasted Estimates
  • 4.2.2. Currency Coverage
    • 4.2.2.1. Overview of Major Currencies Affecting the Market
    • 4.2.2.2. Impact of Currency Fluctuations on the Industry
  • 4.2.3. Foreign Exchange Impact
    • 4.2.3.1. Evaluation of Foreign Exchange Rates and Their Impact on Market
    • 4.2.3.2. Strategies for Mitigating Foreign Exchange Risk
  • 4.2.4. Recession
    • 4.2.4.1. Historical Analysis of Past Recessions and Lessons Learnt
    • 4.2.4.2. Assessment of Current Economic Conditions and Potential Impact on the Market
  • 4.2.5. Inflation
    • 4.2.5.1. Measurement and Analysis of Inflationary Pressures in the Economy
    • 4.2.5.2. Potential Impact of Inflation on the Market Evolution

5. EXECUTIVE SUMMARY

6. INTRODUCTION

• 6.1. Chapter Overview
• 6.2. Evolution of Gene Therapies
• 6.3. Classification of Gene Therapies
  • 6.3.1. Based on Source of Origin
  • 6.3.2. Based on Method of Delivery
    • 6.3.2.1. Ex vivo Gene Therapies
    • 6.3.2.2. In vivo Gene Therapies
• 6.4. Routes of Administration
• 6.5. Mechanism of Action of Gene Therapies
• 6.6. Advantages and Disadvantages of Gene Therapies
• 6.7. Challenges associated with Gene Therapies
  • 6.7.1. Ethical and Social Challenges
  • 6.7.2. Therapy Development Challenges
  • 6.7.3. Manufacturing Challenges
  • 6.7.4. Commercial Viability Challenges
• 6.8. Introduction to Genome Editing
  • 6.8.1. Evolution of Genome Editing
  • 6.8.2. Applications of Genome Editing
  • 6.8.3. Types of Genome Editing Techniques
• 6.9. Concluding Remarks

7. GENE DELIVERY VECTORS

• 7.1. Chapter Overview
• 7.2. Methods of Gene Transfer
• 7.3. Viral Vectors for Genetically Modified Therapies
  • 7.3.1. Types of Viral Vectors
    • 7.3.1.1. Adeno-associated Viral Vectors
    • 7.3.1.2. Adenoviral Vectors
    • 7.3.1.3. Lentiviral Vectors
    • 7.3.1.4. Retroviral Vectors
    • 7.3.1.5. Other Viral Vectors
• 7.4. Non-Viral Vectors for Genetically Modified Therapies
  • 7.4.1. Types of Non-Viral Vectors
    • 7.4.1.1. Plasmid DNA
    • 7.4.1.2. Liposomes, Lipoplexes and Polyplexes
    • 7.4.1.3. Oligonucleotides
    • 7.4.1.4. Nanoparticles
    • 7.4.1.5. Hybrid System Vectors
    • 7.4.1.6. Other Non-Viral Vectors
  • 7.4.2. Gene Delivery using Non-Viral Vectors
    • 7.4.2.1. Biolistic Methods
    • 7.4.2.2. Electroporation
    • 7.4.2.3. Receptor Mediated Gene Delivery
    • 7.4.2.4. Gene Activated matrix (GAM)

8. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIOS

• 8.1. Chapter Overview
• 8.2. Regulatory Guidelines in North America
  • 8.2.1. The US Scenario
  • 8.2.2. Canadian Scenario
• 8.3. Regulatory Guidelines in Europe
  • 8.3.1. Quality Documentation for Gene Therapy Products
  • 8.3.2. Non-Clinical Development
  • 8.3.3. Clinical Development
• 8.4. Regulatory Guidelines in Asia-Pacific
  • 8.4.1. Chinese Scenario
    • 8.4.1.1. Construction of DNA Expression Cassette and Gene Delivery Systems
    • 8.4.1.2. Generation and Characterization of Cell Banks and Engineered Bacteria Banks
      • 8.4.1.2.1. Cell Bank
      • 8.4.1.2.2. Bacterial Cell Bank
    • 8.4.1.3. Manufacturing of Gene Therapy Products
    • 8.4.1.4. Quality Control
    • 8.4.1.5. Evaluation of Efficacy of Gene Therapy Products
    • 8.4.1.6. Evaluation of Safety of Gene Therapy Products
    • 8.4.1.7. Clinical Trial of Gene Therapy Products
    • 8.4.1.8. Ethics Study
  • 8.4.2. Japanese Scenario
  • 8.4.3. South Korean Scenario
  • 8.4.4. Australian Scenario
  • 8.4.5. Hong Kong Scenario
• 8.5. Reimbursement Scenario
  • 8.5.1. Challenges Related to Reimbursement
• 8.6. Concluding Remarks and Future Outlook

9. MARKET LANDSCAPE

• 9.1. Chapter Overview
• 9.2. Marketed and Clinical Stage Gene Therapies: Market Landscape
  • 9.2.1. Analysis by Stage of Development
  • 9.2.2. Analysis by Drug Designation
  • 9.2.3. Analysis by Therapeutic Area
  • 9.2.4. Analysis by Target Gene
  • 9.2.5. Analysis by Type of Vector Used
  • 9.2.6. Analysis by Type of Therapy
  • 9.2.7. Analysis by Type of Gene Delivery Method
  • 9.2.8. Analysis by Route of Administration
  • 9.2.9. Analysis by Dosing Frequency
• 9.3. Preclinical and Discovery Stage Gene Therapies: Market Landscape
  • 9.3.1. Analysis by Stage of Development
  • 9.3.2. Analysis by Therapeutic Area
  • 9.3.3. Analysis by Target Gene
  • 9.3.4. Analysis by Type of Vector Used
  • 9.3.5. Analysis by Type of Therapy
  • 9.3.6. Analysis by Type of Gene Delivery Method

10. DEVELOPER LANDSCAPE

• 10.1. Chapter Overview
• 10.2. Gene Therapy: Competitive Landscape
  • 10.2.1. Analysis by Year of Establishment
  • 10.2.2. Analysis by Company Size
  • 10.2.3. Analysis by Location of Headquarters
  • 10.2.4. Most Active Players: Analysis by Number of Gene Therapies Developed

11. COMPANY PROFILES

• 11.1. Chapter Overview
• 11.2. Gene Therapy Developers in North America
  • 11.2.1. Amgen
    • 11.2.1.1. Company Overview
    • 11.2.1.2. Gene Therapy Portfolio
    • 11.2.1.3. Recent Developments and Future Outlook
  • 11.2.2. BioMarin Pharmaceutical
    • 11.2.2.1. Company Overview
    • 11.2.2.2. Gene Therapy Portfolio
    • 11.2.2.3. Recent Developments and Future Outlook
  • 11.2.3. bluebird bio
    • 11.2.3.1. Company Overview
    • 11.2.3.2. Gene Therapy Portfolio
    • 11.2.3.3. Recent Developments and Future Outlook
  • 11.2.4. CRISPR Therapeutics
    • 11.2.4.1. Company Overview
    • 11.2.4.2. Gene Therapy Portfolio
    • 11.2.4.3. Recent Developments and Future Outlook
  • 11.2.5. Kolon TissueGene
    • 11.2.5.1. Company Overview
    • 11.2.5.2. Gene Therapy Portfolio
    • 11.2.5.3. Recent Developments and Future Outlook
  • 11.2.6. Krystal Biotech
    • 11.2.6.1. Company Overview
    • 11.2.6.2. Gene Therapy Portfolio
    • 11.2.6.3. Recent Developments and Future Outlook
  • 11.2.7. Pfizer
    • 11.2.7.1. Company Overview
    • 11.2.7.2. Gene Therapy Portfolio
    • 11.2.7.3. Recent Developments and Future Outlook
  • 11.2.8. PTC Therapeutics
    • 11.2.8.1. Company Overview
    • 11.2.8.2. Gene Therapy Portfolio
    • 11.2.8.3. Recent Developments and Future Outlook
  • 11.2.9. Sarepta Therapeutics
    • 11.2.9.1. Company Overview
    • 11.2.9.2. Gene Therapy Portfolio
    • 11.2.9.3. Recent Developments and Future Outlook
  • 11.2.10. Spark Therapeutics
    • 11.2.10.1. Company Overview
    • 11.2.10.2. Gene Therapy Portfolio
    • 11.2.10.3. Recent Developments and Future Outlook
• 11.3. Gene Therapy Developers in Europe
  • 11.3.1. Artgen Biotech
    • 11.3.1.1. Company Overview
    • 11.3.1.2. Gene Therapy Portfolio
    • 11.3.1.3. Recent Developments and Future Outlook
  • 11.3.2. Ferring Pharmaceuticals
    • 11.3.2.1. Company Overview
    • 11.3.2.2. Gene Therapy Portfolio
    • 11.3.2.3. Recent Developments and Future Outlook
  • 11.3.3. Novartis
    • 11.3.3.1. Company Overview
    • 11.3.3.2. Gene Therapy Portfolio
    • 11.3.3.3. Recent Developments and Future Outlook
  • 11.3.4. Orchard Therapeutics
    • 11.3.4.1. Company Overview
    • 11.3.4.2. Gene Therapy Portfolio
    • 11.3.4.3. Recent Developments and Future Outlook
• 11.4. Gene Therapy Developers in Asia-Pacific and Rest of the World
  • 11.4.1. CSL Behring
    • 11.4.1.1. Company Overview
    • 11.4.1.2. Gene Therapy Portfolio
    • 11.4.1.3. Recent Developments and Future Outlook
  • 11.4.2. Shanghai Sunway Biotech
    • 11.4.2.1. Company Overview
    • 11.4.2.2. Gene Therapy Portfolio
    • 11.4.2.3. Recent Developments and Future Outlook
  • 11.4.3. Sibiono GeneTech
    • 11.4.3.1. Company Overview
    • 11.4.3.2. Gene Therapy Portfolio
    • 11.4.3.3. Recent Developments and Future Outlook

12. MARKETED GENE THERAPIES

• 12.1. Chapter Overview
• 12.2. Gendicine(R) (Shenzhen Sibiono GeneTech)
  • 12.2.1. Company Overview
  • 12.2.2. Development Timeline
  • 12.2.3. Mechanism of Action and Vector Used
  • 12.2.4. Target Indication
  • 12.2.5. Current Stage of Development
  • 12.2.6. Manufacturing, Dosage and Sales
• 12.3. Oncorine(R) (Shanghai Sunway Biotech)
  • 12.3.1. Company Overview
  • 12.3.2. Development Timeline
  • 12.3.3. Mechanism of Action and Vector Used
  • 12.3.4. Target Indication
  • 12.3.5. Current Stage of Development
  • 12.3.6. Manufacturing, Dosage and Sales
• 12.4. Rexin-G(R) (Epeius Biotechnologies)
  • 12.4.1. Company Overview
  • 12.4.2. Development Timeline
  • 12.4.3. Mechanism of Action and Vector Used
  • 12.4.4. Target Indication
  • 12.4.5. Current Stage of Development
  • 12.4.6. Manufacturing, Dosage and Sales
• 12.5. Neovasculgen(R) (Human Stem Cells Institute)
  • 12.5.1. Company Overview
  • 12.5.2. Development Timeline
  • 12.5.3. Mechanism of Action and Vector Used
  • 12.5.4. Target Indication
  • 12.5.5. Current Stage of Development
  • 12.5.6. Manufacturing, Dosage and Sales
• 12.6. Imlygic(R) (Amgen)
  • 12.6.1. Company Overview
  • 12.6.2. Development Timeline
  • 12.6.3. Mechanism of Action and Vector Used
  • 12.6.4. Target Indication
  • 12.6.5. Current Stage of Development
  • 12.6.6. Manufacturing, Dosage and Sales
• 12.7. Strimvelis(R) (Orchard Therapeutics)
  • 12.7.1. Company Overview
  • 12.7.2. Development Timeline
  • 12.7.3. Mechanism of Action and Vector Used
  • 12.7.4. Target Indication
  • 12.7.5. Current Stage of Development
  • 12.7.6. Manufacturing, Dosage and Sales
• 12.8. Luxturna(TM) (Spark Therapeutics)
  • 12.8.1. Company Overview
  • 12.8.2. Development Timeline
  • 12.8.3. Mechanism of Action and Vector Used
  • 12.8.4. Target Indication
  • 12.8.5. Current Stage of Development
  • 12.8.6. Manufacturing, Dosage and Sales
• 12.9. Zolgensa(TM) (Novartis)
  • 12.9.1. Company Overview
  • 12.9.2. Development Timeline
  • 12.9.3. Mechanism of Action and Vector Used
  • 12.9.4. Target Indication
  • 12.9.5. Current Stage of Development
  • 12.9.6. Manufacturing, Dosage and Sales
• 12.10. Collategene(R) (AnGes)
  • 12.10.1. Company Overview
  • 12.10.2. Development Timeline
  • 12.10.3. Mechanism of Action and Vector Used
  • 12.10.4. Target Indication
  • 12.10.5. Current Stage of Development
  • 12.10.6. Manufacturing, Dosage and Sales
• 12.11. Zyntelgo(TM) (bluebird bio)
  • 12.11.1. Company Overview
  • 12.11.2. Development Timeline
  • 12.11.3. Mechanism of Action and Vector Used
  • 12.11.4. Target Indication
  • 12.11.5. Current Stage of Development
  • 12.11.6. Manufacturing, Dosage and Sales
• 12.12. Libmeldy(TM) (Orchard Therapeutics)
  • 12.12.1. Company Overview
  • 12.12.2. Development Timeline
  • 12.12.3. Mechanism of Action and Vector Used
  • 12.12.4. Target Indication
  • 12.12.5. Current Stage of Development
  • 12.12.6. Manufacturing, Dosage and Sales

13. KEY COMMERCIALIZATION STRATEGIES

• 13.1. Chapter Overview
• 13.2. Successful Drug Launch Strategy: ROOTS Framework
• 13.3. Successful Drug Launch Strategy: Product Differentiation
• 13.4. Commonly Adopted Commercialization Strategies based on Phase of Development
• 13.5. List of Approved Gene Therapies
• 13.6. Key Commercialization Strategies Adopted by Gene Therapy Developers
  • 13.6.1. Strategies Adopted before Therapy Approval
    • 13.6.1.1. Participation in Global Events
    • 13.6.1.2. Collaborations with Stakeholders and Pharmaceutical Firms
    • 13.6.1.3. Indication Expansion
  • 13.6.2. Strategies adopted During / Post Therapy Approval
    • 13.6.2.1. Geographical Expansion
    • 13.6.2.2. Participation in Global Events
    • 13.6.2.3. Patient Assistance Programs
    • 13.6.2.4. Awareness through Product Websites
    • 13.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms
• 13.7. Concluding Remarks

14. LATE-STAGE GENE THERAPIES

• 14.1. Chapter Overview
• 14.2. LUMEVOQ (GS010)
• 14.3. OTL-103
• 14.4. PTC-AADC
• 14.5. BMN 270
• 14.6. rAd-IFN/Syn3
• 14.7. beti-cel
• 14.8. eli-cel
• 14.9. lovo-cel
• 14.10. SRP-9001
• 14.11. EB-101
• 14.12. ProstAtak
• 14.13. D-Fi
• 14.14. CG0070
• 14.15. Vigil-EWS
• 14.16. Engensis
• 14.17. VGX-3100
• 14.18. INVOSSA (TG-C)
• 14.19. VYJUVEKT
• 14.20. PF-06939926
• 14.21. PF06838435
• 14.22. PF-07055480
• 14.23. SPK-8011
• 14.24. AMT-061
• 14.25. VB-111
• 14.26. Generx
• 14.27. ADXS-HPV
• 14.28. AGTC 501
• 14.29. LYS-SAF302
• 14.30. NFS-01
• 14.31. AG0302-COVID19
• 14.32. RGX-314
• 14.33. Hologene 5

15. PATENT ANALYSIS

• 15.1. Chapter Overview
• 15.2. Gene Therapy: Patent Analysis
  • 15.2.1. Scope and Methodology
    • 15.2.1.1. Analysis by Publication Year
    • 15.2.1.2. Analysis by Publication Year and Type of Patent
    • 15.2.1.3. Analysis by Geography
      • 15.2.1.3.1. Analysis by Geography (North America)
      • 15.2.1.3.2. Analysis by Geography (Europe)
      • 15.2.1.3.3. Analysis by Geography (Asia-Pacific)
    • 15.2.1.4. Analysis by CPC Symbols
    • 15.2.1.5. Analysis by Emerging Focus Areas
    • 15.2.1.6. Leading Players: Analysis by Number of Patents
    • 15.2.1.7. Patent Benchmarking Analysis
      • 15.2.1.7.1. Analysis by Patent Characteristics
        • 15.2.1.7.1.1. Sanofi and Genethon
        • 15.2.1.7.1.2. Other Leading Patent Assignees
    • 15.2.1.8. Patent Valuation Analysis
• 15.3. Gene Editing Market: Patent Analysis
  • 15.3.1. Scope and Methodology
    • 15.3.1.1. Analysis by Publication Year
    • 15.3.1.2. Analysis by Publication Year and Type of Patent
    • 15.3.1.3. Analysis by Geography
      • 15.3.1.3.1. Analysis by Geography (North America)
      • 15.3.1.3.2. Analysis by Geography (Europe)
      • 15.3.1.3.3. Analysis by Geography (Asia-Pacific)
    • 15.3.1.4. Analysis by CPC Symbols
    • 15.3.1.5. Analysis by Emerging Focus Areas
    • 15.3.1.6. Leading Players: Analysis by Number of Patents
    • 15.3.1.7. Patent Benchmarking Analysis
      • 15.3.1.7.1. Analysis by Patent Characteristics
        • 15.3.1.7.1.1. Intellia Therapeutics and CRISPR Therapeutics
        • 15.3.1.7.1.2. Other Leading Patent Assignees
    • 15.3.1.8. Patent Valuation Analysis
• 15.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization

16. MERGERS AND ACQUISITIONS

• 16.1. Chapter Overview
• 16.2. Merger and Acquisition Models
• 16.3. Gene Therapy: Mergers and Acquisitions
  • 16.3.1. Analysis by Year of Merger / Acquisition
  • 16.3.2. Analysis by Type of Agreement
  • 16.3.3. Analysis by Geography
    • 16.3.3.1. Intercontinental and Intracontinental Deals
    • 16.3.3.2. Local and International Deals
  • 16.3.4. Analysis by Key Value Drivers
    • 16.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
  • 16.3.5. Analysis by Stage of Development of the Acquired Company's Product
  • 16.3.6. Analysis by Therapeutic Area
• 16.4. Analysis by Deal Multiples

17. FUNDING AND INVESTMENTS

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

18. CLINICAL TRIAL ANALYSIS

• 18.1. Chapter Overview
• 18.2. Scope and Methodology
• 18.3. Gene Therapy: Clinical Trial Analysis
  • 18.3.1. Analysis by Trial Registration Year
  • 18.3.2. Analysis by Trial Status
  • 18.3.3. Analysis by Trial Phase
  • 18.3.4. Analysis by Therapeutic Area
  • 18.3.5. Analysis by Geography
  • 18.3.6. Analysis by Trial Registration Year and Geography
  • 18.3.7. Analysis by Trial Status and Geography
  • 18.3.8. Analysis by Trial Status, Trial Phase and Geography
  • 18.3.9. Analysis by Therapeutic Area and Geography
• 18.4. Analysis by Type of Sponsor / Collaborator
• 18.5. Analysis by Prominent Treatment Sites
• 18.6. Gene Therapy: Analysis of Enrolled Patient Population
  • 18.6.1. Analysis by Trial Registration Year
  • 18.6.2. Analysis by Trial Status
  • 18.6.3. Analysis by Trial Phase
  • 18.6.4. Analysis by Therapeutic Area
  • 18.6.5. Analysis by Location of Trial Site
  • 18.6.6. Analysis by Trial Status and Location of Trial Site
  • 18.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
  • 18.6.8. Analysis by Therapeutic Area and Location of Trial Site
• 18.7. Concluding Remarks

19. COST PRICE ANALYSIS

• 19.1. Chapter Overview
• 19.2. Factors Contributing to the High Price of Gene Therapies
• 19.3. Gene Therapy Market: Price Models
  • 19.3.1. Based on Associated Product / Component Costs
  • 19.3.2. Based on Competition
  • 19.3.3. Based on Patient Segment
  • 19.3.4. Based Opinion of Industry Experts

20. START-UP VALUATION

• 20.1. Chapter Overview
• 20.2. Valuation by Years of Experience of Start-ups
  • 20.2.1. Methodology
  • 20.2.2. Results and Interpretation

21. BIG PHARMA INITIATIVES

• 21.1. Chapter Overview
• 21.2. Gene Therapy: List of Most Prominent Big Pharmaceutical Players
  • 21.2.1. Analysis by Therapeutic Area
  • 21.2.2. Analysis by Type of Vector Used
  • 21.2.3. Analysis by Type of Therapy
  • 21.2.4. Analysis by Type of Gene Delivery Method Used
• 21.3. Benchmarking Analysis of Key Parameters
  • 21.3.1. Spider Web Analysis: Pipeline Strength
  • 21.3.2. Spider Web Analysis: Mergers and Acquisitions
  • 21.3.3. Spider Web Analysis: Funding and Investments
  • 21.3.4. Spider Web Analysis: Clinical Trials
  • 21.3.5. Spider Web Analysis: Technologies
  • 21.3.6. Spider Web Analysis: Patents
• 21.4. Benchmarking Analysis of Big Pharmaceutical Players
  • 21.4.1. Spider Web Analysis: Novartis
  • 21.4.2. Spider Web Analysis: Takeda Pharmaceutical
  • 21.4.3. Spider Web Analysis: Roche
  • 21.4.4. Spider Web Analysis: Merck
  • 21.4.5. Spider Web Analysis: Pfizer
  • 21.4.6. Spider Web Analysis: Johnson & Johnson
  • 21.4.7. Spider Web Analysis: AbbVie
  • 21.4.8. Spider Web Analysis: Bristol Myers Squibb
  • 21.4.9. Spider Web Analysis: AstraZeneca
  • 21.4.10. Spider Web Analysis: Sanofi

22. DEMAND ANALYSIS

• 22.1 Chapter Overview
• 22.2. Assumptions and Methodology
• 22.3. Gene Therapy Market: Global Demand Analysis
  • 22.3.1. Analysis by Stage of Development
  • 22.3.2. Analysis by Therapeutic Area
  • 22.3.3. Analysis by Type of Therapy
  • 22.3.4. Analysis by Type of Gene Delivery Method
  • 22.3.5. Analysis by Geography

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

• 23.1. Chapter Overview
• 23.2. Market Drivers
• 23.3. Market Restraints
• 23.4. Market Opportunities
• 23.5. Market Challenges
• 23.6. Conclusion

24. GLOBAL GENE THERAPY MARKET

• 24.1. Chapter Overview
• 24.2. Key Assumptions and Methodology
• 24.3. Global Gene Therapy Market, till 2035
  • 24.3.1. Scenario Analysis
    • 24.3.1.1. Conservative Scenario
    • 24.3.1.2. Optimistic Scenario
• 24.4. Key Market Segmentations

25. GENE THERAPIES MARKET, BY THERAPEUTIC AREA

• 25.1. Chapter Overview
• 25.2. Key Assumptions and Methodology
• 25.3. Gene Therapy Market: Distribution by Therapeutic Area, 2024, 2029 and 2035
  • 25.3.1. Gene Therapy Market for Muscle Disorders, till 2035
  • 25.3.2. Gene Therapy Market for Hematological Disorders, till 2035
  • 25.3.3. Gene Therapy Market for Dermatological Disorders, till 2035
  • 25.3.4. Gene Therapy Market for Oncological Disorders, till 2035
  • 25.3.5. Gene Therapy Market for Genetic Disorders, till 2035
  • 25.3.6. Gene Therapy Market for Ophthalmic Disorders, till 2035
  • 25.3.7. Gene Therapy Market for Metabolic Disorders, till 2035
  • 25.3.8. Gene Therapy Market for Cardiovascular Disorders, till 2035
  • 25.3.9. Gene Therapy Market for Other Disorders, till 2035
• 25.4. Data Triangulation and Validation

26. GENE THERAPIES MARKET, BY TYPE OF VECTOR

• 26.1. Chapter Overview
• 26.2. Key Assumptions and Methodology
• 26.3. Gene Therapy Market: Distribution by Type of Vector, 2024, 2029 and 2035
  • 26.3.1. Gene Therapy Market for Adeno-associated Virus Vectors, till 2035
  • 26.3.2. Gene Therapy Market for Herpes Simplex Virus Vectors, till 2035
  • 26.3.3. Gene Therapy Market for Lentivirus Vectors, till 2035
  • 26.3.4. Gene Therapy Market for Adenovirus Vectors, till 2035
  • 26.3.5. Gene Therapy Market for Non-viral Vectors, till 2035
  • 26.3.6. Gene Therapy Market for Retrovirus Vectors, till 2035
  • 26.3.7. Gene Therapy Market for Other Viral Vectors, till 2035
• 26.4. Data Triangulation and Validation

27. GENE THERAPIES MARKET, BY TYPE OF THERAPY

• 27.1. Chapter Overview
• 27.2. Key Assumptions and Methodology
• 27.3. Gene Therapy Market: Distribution by Type of Therapy, 2024, 2029 and 2035
  • 27.3.1. Gene Therapy Market for Gene Augmentation, till 2035
  • 27.3.2. Gene Therapy Market for Oncolytic Immunotherapies, till 2035
  • 27.3.3. Gene Therapy Market for Gene Editing, till 2035
  • 27.3.4. Gene Therapy Market for Gene Regulation, till 2035
  • 27.3.5. Gene Therapy Market for Other Therapies, till 2035
• 27.4. Data Triangulation and Validation

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

• 28.1. Chapter Overview
• 28.2. Key Assumptions and Methodology
• 28.3. Gene Therapy Market: Distribution by Type of Gene Delivery Method, 2024, 2029 and 2035
  • 28.3.1. Gene Therapy Market for In vivo Gene Delivery, till 2035
  • 28.3.2. Gene Therapy Market for Ex vivo Gene Delivery, till 2035
• 28.4. Data Triangulation and Validation

29. GENE THERAPIES MARKET, BY ROUTE OF ADMINISTRATION

• 29.1. Chapter Overview
• 29.2. Key Assumptions and Methodology
• 29.3. Gene Therapy Market: Distribution by Route of Administration, 2024, 2029 and 2035
  • 29.3.1. Gene Therapy Market for Intravenous Route, till 2035
  • 29.3.2. Gene Therapy Market for Subretinal Route, till 2035
  • 29.3.3. Gene Therapy Market for Intratumoral Route, till 2035
  • 29.3.4. Gene Therapy Market for Intramuscular Route, till 2035
  • 29.3.5. Gene Therapy Market for Other Routes of Administration, till 2035
• 29.4. Data Triangulation and Validation

30. GENE THERAPIES MARKET, BY GEOGRAPHICAL REGIONS

• 30.1. Chapter Overview
• 30.2. Key Assumptions and Methodology
• 30.3. Gene Therapy Market: Distribution by Geographical Regions, 2024, 2029 and 2035
  • 30.3.1. Gene Therapy Market in North America, till 2035
  • 30.3.2. Gene Therapy Market in Europe, till 2035
  • 30.3.3. Gene Therapy Market in Asia-Pacific, till 2035
  • 30.3.4. Gene Therapy Market in Latin America, till 2035
  • 30.3.5. Gene Therapy Market in Rest of the World, till 2035
• 30.4. Data Triangulation and Validation

31. GENE THERAPIES MARKET, BY LEADING PLAYERS

• 31.1. Chapter Overview
• 31.2. Gene Therapy Market: Distribution by Leading Gene Therapy Developers
• 31.3. Data Triangulation and Validation

32. GENE THERAPIES MARKET, SALES FORECAST OF THERAPIES

• 32.1. Chapter Overview
• 32.2. Key Assumptions and Methodology
• 32.3. Commercialized Gene Therapies Market: Sales Forecast
  • 32.3.1. Adstiladrin(R)
    • 32.3.1.1. Sales Forecast
    • 32.3.1.2. Net Present Value
    • 32.3.1.3. Value Creation Analysis
  • 32.3.2. BEQVEZ(TM)
    • 32.3.2.1. Sales Forecast
    • 32.3.2.2. Net Present Value
    • 32.3.2.3. Value Creation Analysis
  • 32.3.3. Casgevy(TM)
    • 32.3.3.1. Sales Forecast
    • 32.3.3.2. Net Present Value
    • 32.3.3.3. Value Creation Analysis
  • 32.3.4. Elevidys(R)
    • 32.3.4.1. Sales Forecast
    • 32.3.4.2. Net Present Value
    • 32.3.4.3. Value Creation Analysis
  • 32.3.5. Gendicine(R)
    • 32.3.5.1. Sales Forecast
    • 32.3.5.2. Net Present Value
    • 32.3.5.3. Value Creation Analysis
  • 32.3.6. Hemgenix(R)
    • 32.3.6.1. Sales Forecast
    • 32.3.6.2. Net Present Value
    • 32.3.6.3. Value Creation Analysis
  • 32.3.7. IMLYGIC(R)
    • 32.3.7.1. Sales Forecast
    • 32.3.7.2. Net Present Value
    • 32.3.7.3. Value Creation Analysis
  • 32.3.8. Libmeldy(R)
    • 32.3.8.1. Sales Forecast
    • 32.3.8.2. Net Present Value
    • 32.3.8.3. Value Creation Analysis
  • 32.3.9. Luxturna(R)
    • 32.3.9.1. Sales Forecast
    • 32.3.9.2. Net Present Value
    • 32.3.9.3. Value Creation Analysis
  • 32.3.10. LYFGENIA(TM)
    • 32.3.10.1. Sales Forecast
    • 32.3.10.2. Net Present Value
    • 32.3.10.3. Value Creation Analysis
  • 32.3.11. Neovasculgen(R)
    • 32.3.11.1. Sales Forecast
    • 32.3.11.2. Net Present Value
    • 32.3.11.3. Value Creation Analysis
  • 32.3.12. Oncorine(R)
    • 32.3.12.1. Sales Forecast
    • 32.3.12.2. Net Present Value
    • 32.3.12.3. Value Creation Analysis
  • 32.3.13. Roctavian(TM)
    • 32.3.13.1. Sales Forecast
    • 32.3.13.2. Net Present Value
    • 32.3.13.3. Value Creation Analysis
  • 32.3.14. SKYSONA(R)
    • 32.3.14.1. Sales Forecast
    • 32.3.14.2. Net Present Value
    • 32.3.14.3. Value Creation Analysis
  • 32.3.15. Strimvelis(TM)
    • 32.3.15.1. Sales Forecast
    • 32.3.15.2. Net Present Value
    • 32.3.15.3. Value Creation Analysis
  • 32.3.16. Upstaza(TM)
    • 32.3.16.1. Sales Forecast
    • 32.3.16.2. Net Present Value
    • 32.3.16.3. Value Creation Analysis
  • 32.3.17. VYJUVEKT(R)
    • 32.3.17.1. Sales Forecast
    • 32.3.17.2. Net Present Value
    • 32.3.17.3. Value Creation Analysis
  • 32.3.18. Zolgensma(R)
    • 32.3.18.1. Sales Forecast
    • 32.3.18.2. Net Present Value
    • 32.3.18.3. Value Creation Analysis
  • 32.3.19. Zynteglo(TM)
    • 32.3.19.1. Sales Forecast
    • 32.3.19.2. Net Present Value
    • 32.3.19.3. Value Creation Analysis
• 32.4. Phase III Gene Therapies Market: Sales Forecast
  • 32.4.1. AAV-RPE65
    • 32.4.1.1. Sales Forecast
    • 32.4.1.2. Net Present Value
    • 32.4.1.3. Value Creation Analysis
  • 32.4.2. ABO-102 (UX111)
    • 32.4.2.1. Sales Forecast
    • 32.4.2.2. Net Present Value
    • 32.4.2.3. Value Creation Analysis
  • 32.4.3. AGTC 501 (rAAV2tYF-GRK1-RPGR)
    • 32.4.3.1. Sales Forecast
    • 32.4.3.2. Net Present Value
    • 32.4.3.3. Value Creation Analysis
  • 32.4.4. Cretostimogene Grenadenorepvec (CG0070)
    • 32.4.4.1. Sales Forecast
    • 32.4.4.2. Net Present Value
    • 32.4.4.3. Value Creation Analysis
  • 32.4.5. Dirloctocogene samoparvovec (SPK-8011)
    • 32.4.5.1. Sales Forecast
    • 32.4.5.2. Net Present Value
    • 32.4.5.3. Value Creation Analysis
  • 32.4.6. DTx-301
    • 32.4.6.1. Sales Forecast
    • 32.4.6.2. Net Present Value
    • 32.4.6.3. Value Creation Analysis
  • 32.4.7. DTx-401
    • 32.4.7.1. Sales Forecast
    • 32.4.7.2. Net Present Value
    • 32.4.7.3. Value Creation Analysis
  • 32.4.8. Generx(R) (Ad5FGF-4)
    • 32.4.8.1. Sales Forecast
    • 32.4.8.2. Net Present Value
    • 32.4.8.3. Value Creation Analysis
  • 32.4.9. Giroctocogene fitelparvovec (SB-525 / PF-07055480)
    • 32.4.9.1. Sales Forecast
    • 32.4.9.2. Net Present Value
    • 32.4.9.3. Value Creation Analysis
  • 32.4.10. Hologene 5
    • 32.4.10.1. Sales Forecast
    • 32.4.10.2. Net Present Value
    • 32.4.10.3. Value Creation Analysis
  • 32.4.11. INVOSSA(TM) (TG-C)
    • 32.4.11.1. Sales Forecast
    • 32.4.11.2. Net Present Value
    • 32.4.11.3. Value Creation Analysis
  • 32.4.12. LUMEVOQ(R)
    • 32.4.12.1. Sales Forecast
    • 32.4.12.2. Net Present Value
    • 32.4.12.3. Value Creation Analysis
  • 32.4.13. NFS-01 (rAAV2-ND4)
    • 32.4.13.1. Sales Forecast
    • 32.4.13.2. Net Present Value
    • 32.4.13.3. Value Creation Analysis
  • 32.4.14. NTLA-2001
    • 32.4.14.1. Sales Forecast
    • 32.4.14.2. Net Present Value
    • 32.4.14.3. Value Creation Analysis
  • 32.4.15. Olvi-Vec (Olvimulogene nanivacirepvec)
    • 32.4.15.1. Sales Forecast
    • 32.4.15.2. Net Present Value
    • 32.4.15.3. Value Creation Analysis
  • 32.4.16. ProstAtak(R)
    • 32.4.16.1. Sales Forecast
    • 32.4.16.2. Net Present Value
    • 32.4.16.3. Value Creation Analysis
  • 32.4.17. pz-cel (prademagene zamikeracel / EB-101)
    • 32.4.17.1. Sales Forecast
    • 32.4.17.2. Net Present Value
    • 32.4.17.3. Value Creation Analysis
  • 32.4.18. RGX-121
    • 32.4.18.1. Sales Forecast
    • 32.4.18.2. Net Present Value
    • 32.4.18.3. Value Creation Analysis
  • 32.4.19. RGX-314
    • 32.4.19.1. Sales Forecast
    • 32.4.19.2. Net Present Value
    • 32.4.19.3. Value Creation Analysis
  • 32.4.20. VGX-3100
    • 32.4.20.1. Sales Forecast
    • 32.4.20.2. Net Present Value
    • 32.4.20.3. Value Creation Analysis

33. GENE THERAPY MARKET: EMERGING TECHNOLOGIES

• 33.1. Chapter Overview
• 33.2. Gene Editing Technologies
  • 33.2.1. Applications of Gene Editing
• 33.3. Emerging Gene Editing Platforms
  • 33.3.1. CRISPR / Cas9 System
    • 33.3.1.1. Technology Providers
  • 33.3.2. Zinc Finger Nuclease
    • 33.3.2.1. Technology Providers
  • 33.3.3. TALENs
  • 33.3.4. megaTAL
    • 33.3.4.1. Technology Providers
• 33.4. Gene Therapy Regulation Technologies (Gene Switch Technology)
  • 33.4.1. Technology Providers
• 33.5. Other Emerging Technologies
• 33.6. Technology Platforms for Developing / Delivering Gene Therapies
• 33.7. Concluding Remarks

34. VECTOR MANUFACTURING FOR GENE THERAPIES

• 34.1. Chapter Overview
• 34.2. Viral Vector Manufacturing Process
  • 34.2.1. Mode of Vector Production
  • 34.2.2. Adherent and Suspension Cultures
  • 34.2.3. Unit Processes and Multiple Parallel Processes
  • 34.2.4. Cell Culture Systems for Production of Viral Vectors
    • 34.2.4.1. Small-Scale / Laboratory Scale Cell Culture Systems
    • 34.2.4.2. Large-Scale Cell Culture Systems
      • 34.2.4.2.1. Stirred Tank Reactor Systems
      • 34.2.4.2.2. Fixed Bed Reactor / Packed Bed Reactor
      • 34.2.4.2.3. WAVE Bioreactor System
  • 34.2.5. Culture Media Specifications
• 34.3. Bioprocessing of Viral Vectors
  • 34.3.1. AAV Vector Production
  • 34.3.2. Adenoviral Vector Production
  • 34.3.3. Lentiviral Vector Production
  • 34.3.4. Y - Retroviral Vector Production
• 34.4. Challenges Associated with Vector Manufacturing
• 34.5. Contract Manufacturing Organizations Involved in Viral Vector Production

35. CASE STUDY: GENE THERAPY SUPPLY CHAIN

• 35.1. Chapter Overview
• 35.2. Overview of Gene Therapy Supply Chain
• 35.3. Implementation of Supply Chain Models
• 35.4. Logistics in Gene Therapy
  • 35.4.1. Logistic Processes for Autologous and Allogenic Therapies
• 35.5. Regulatory Supply Chain Across the Globe
• 35.6. Challenges Associated with Gene Therapy Supply Chain
• 35.7. Software Applications for Gene Therapy Supply Chain Management
  • 35.7.1. Enterprise Manufacturing System
  • 35.7.2. Laboratory Information Management System
  • 35.7.3. Inventory Management System
  • 35.7.4. Quality Management System
  • 35.7.5. Logistics Management System
  • 35.7.6. Patient Management System
  • 35.7.7. Electronic Clinical Outcome Assessments System
  • 35.7.8. Supply Chain Orchestration Platform
• 35.8. Recent Developments and Upcoming Trends

36. EXECUTIVE INSIGHTS

• 36.1. Chapter Overview
• 36.2. Orchard Therapeutics
  • 36.2.1. Company Snapshot
  • 36.2.2. Interview Transcript: Molly Cameron, Former Corporate Communications Manager
• 36.3. Vivet Therapeutics
  • 36.3.1. Company Snapshot
  • 36.3.2. Interview Transcript: Jean-Phillipe Combal, Co-Founder and Chief Executive Officer
• 36.4. Kubota Pharmaceutical
  • 36.4.1. Company Snapshot
  • 36.4.2. Interview Transcript: Ryo Kubota, Chief Executive Officer, Chairman and President
• 36.5. Eyevensys
  • 36.5.1. Company Snapshot
  • 36.5.2. Interview Transcript: Patricia Zilliox, Former Chief Executive Officer and President
• 36.6. AGTC
  • 36.6.1. Company Snapshot
  • 36.6.2. Interview Transcript: Sue Washer, Former Chief Executive Officer and President
• 36.7. LogicBio Therapeutics
  • 36.7.1. Company Snapshot
  • 36.7.2. Interview Transcript: Tom Wilton, Former Chief Business Officer
• 36.8. AAVogen
  • 36.8.1. Company Snapshot
  • 36.8.2. Interview Transcript: Buel Dan Rodgers, Founder and Chief Executive Officer
• 36.9. Hemera Biosciences
  • 36.9.1. Company Snapshot
  • 36.9.2. Interview Transcript: Adam Rogers, Chief Executive Officer
• 36.10. Myonexus Therapeutics
  • 36.10.1. Company Snapshot
  • 36.10.2. Interview Transcript: Michael Tripletti, Ex Co-Founder, Chief Executive Officer and President
• 36.11. Arthrogen
  • 36.11.1. Company Snapshot
  • 36.11.2. Interview Transcript: Robert Jan Lamers, Former Chief Executive Officer
• 36.12. PlasmidFactory
  • 36.12.1. Company Snapshot
  • 36.12.2. Interview Transcript: Marco Schmeer, Project Manager and Tatjana Buchholz, Former Marketing Manager
• 36.13. Delphi Genetics
  • 36.13.1. Company Snapshot
  • 36.13.2. Interview Transcript: Cedric Szpirer, Former Executive and Scientific Director
• 36.14. Vigene Biosciences
  • 36.14.1. Company Snapshot
  • 36.14.2. Interview Transcript: Jeffrey Hung, Former Chief Commercial Officer
• 36.15. Gene Biotherapeutics
  • 36.15.1. Company Snapshot
  • 36.15.2. Interview Transcript: Christopher Reinhard, Chief Executive Officer and Chairman
• 36.16. Milo Biotechnology
  • 36.16.1. Company Snapshot
  • 36.16.2. Interview Transcript: AL Hawkins, Chief Executive Officer

37. CONCLUSION

38. APPENDIX I: TABULATED DATA

39. APPENDIX II: LIST OF COMPANIES AND ORGANIZATIONS

List of Tables

• Table 6.1 Comparison of Ex vivo and In vivo Techniques
• Table 6.2 Routes of Administration of Gene Therapies: Advantages and Disadvantages
• Table 6.3 Advantages and Disadvantages of Gene Therapies
• Table 7.1 Key Features of Viral Vectors
• Table 8.1 Payment Options Available for Gene Therapies
• Table 8.2 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
• Table 9.1 Marketed and Clinical Stage Gene Therapies: Information on Developer, Stage of Development and Drug Designation
• Table 9.2 Marketed and Clinical Stage Gene Therapies: Information on Developer, Therapeutic Area and Biological Target
• Table 9.3 Marketed and Clinical Stage Gene Therapies: Information on Developer and Type of Vector Used
• Table 9.4 Marketed and Clinical Stage Gene Therapies: Information on Developer, Type of Therapy and Type of Gene Delivery Method
• Table 9.5 Marketed and Clinical Stage Gene Therapies: Information on Developer, Route of Administration and Dosage
• Table 9.6 Preclinical and Discovery Stage Gene Therapies: Information on Developer, Stage of Development, Therapeutic Area and Biological Target
• Table 9.7 Preclinical and Discovery Stage Gene Therapies: Information on Developer and Type of Vector Used
• Table 9.8 Preclinical and Discovery Stage Gene Therapies: Information on Developer, Type of Therapy and Type of Gene Delivery Method
• Table 10.1 Gene Therapy Developers: Information on Year of Establishment, Company Size and Location of Headquarters
• Table 11.1 Gene Therapy: List of Companies Profiled
• Table 11.2 Amgen: Company Overview
• Table 11.3 Amgen: Marketed Gene Therapy Portfolio
• Table 11.4 Amgen: Clinical Stage Gene Therapy Portfolio
• Table 11.5 bluebird bio: Company Overview
• Table 11.6 bluebird bio: Marketed Gene Therapy Portfolio
• Table 11.7 bluebird bio: Preclinical and Discovery Stage Gene Therapy Portfolio
• Table 11.8 bluebird bio: Recent Developments and Future Outlook
• Table 11.9 BioMarin Pharmaceutical: Company Overview
• Table 11.10 BioMarin Pharmaceutical: Marketed Gene Therapy Portfolio
• Table 11.11 BioMarin Pharmaceutical: Preclinical Stage Gene Therapy Portfolio
• Table 11.12 BioMarin Pharmaceutical: Recent Developments and Future Outlook
• Table 11.13 CRISPR Therapeutics: Company Overview
• Table 11.14 CRISPR Therapeutics: Marketed Gene Therapy Portfolio
• Table 11.15 CRISPR Therapeutics: Preclinical and Discovery Stage Gene Therapy Portfolio
• Table 11.16 CRISPR Therapeutics: Recent Developments and Future Outlook
• Table 11.17 Kolon TissueGene: Company Overview
• Table 11.18 Kolon TissueGene: Marketed Gene Therapy Portfolio
• Table 11.19 Kolon TissueGene: Clinical Stage Gene Therapy Portfolio
• Table 11.20 Kolon TissueGene: Preclinical Stage Gene Therapy Portfolio
• Table 11.21 Kolon TissueGene: Recent Developments and Future Outlook
• Table 11.22 Krystal Biotech: Company Overview
• Table 11.23 Krystal Biotech: Marketed Gene Therapy Portfolio
• Table 11.24 Krystal Biotech: Clinical Stage Gene Therapy Portfolio
• Table 11.25 Krystal Biotech: Preclinical and Discovery Stage Gene Therapy Portfolio
• Table 11.26 Krystal Biotech: Recent Developments and Future Outlook
• Table 11.27 Pfizer: Company Overview
• Table 11.28 Pfizer: Marketed Gene Therapy Portfolio
• Table 11.29 Pfizer: Clinical Stage Gene Therapy Portfolio
• Table 11.30 Pfizer: Preclinical and Discovery Stage Gene Therapy Portfolio
• Table 11.31 Pfizer: Recent Developments and Future Outlook
• Table 11.32 PTC Therapeutics: Company Overview
• Table 11.33 PTC Therapeutics: Marketed Gene Therapy Portfolio
• Table 11.34 PTC Therapeutics: Preclinical Stage Gene Therapy Portfolio
• Table 11.35 PTC Therapeutics: Recent Developments and Future Outlook
• Table 11.36 Sarepta Therapeutics: Company Overview
• Table 11.37 Sarepta Therapeutics: Marketed Gene Therapy Portfolio
• Table 11.38 Sarepta Therapeutics: Clinical Stage Gene Therapy Portfolio
• Table 11.39 Sarepta Therapeutics: Preclinical and Discovery Gene Therapy Portfolio
• Table 11.40 Sarepta Therapeutics: Recent Developments and Future Outlook
• Table 11.41 Spark Therapeutics: Company Overview
• Table 11.42 Spark Therapeutics: Marketed Gene Therapy Portfolio
• Table 11.43 Spark Therapeutics: Clinical Stage Gene Therapy Portfolio
• Table 11.44 Spark Therapeutics: Preclinical and Discovery Stage Gene Therapy Portfolio
• Table 11.45 Spark Therapeutics: Recent Developments and Future Outlook
• Table 11.46 Artgen Biotech: Company Overview
• Table 11.47 Artgen Biotech: Marketed Gene Therapy Portfolio
• Table 11.48 Artgen Biotech: Preclinical and Discovery Stage Gene Therapy Portfolio
• Table 11.49 Artgen Biotech: Recent Developments and Future Outlook
• Table 11.50 Ferring Pharmaceuticals: Company Overview
• Table 11.51 Ferring Pharmaceuticals: Marketed Gene Therapy Portfolio
• Table 11.52 Ferring Pharmaceuticals: Clinical Stage Gene Therapy Portfolio
• Table 11.53 Ferring Pharmaceuticals: Recent Developments and Future Outlook
• Table 11.54 Novartis: Company Overview
• Table 11.55 Novartis: Marketed Gene Therapy Portfolio
• Table 11.56 Novartis: Clinical Stage Gene Therapy Portfolio
• Table 11.57 Novartis: Preclinical and Discovery Stage Gene Therapy Portfolio
• Table 11.58 Novartis: Recent Developments and Future Outlook
• Table 11.59 Orchard Therapeutics: Company Overview
• Table 11.60 Orchard Therapeutics: Marketed Gene Therapy Portfolio
• Table 11.61 Orchard Therapeutics: Clinical Stage Gene Therapy Portfolio
• Table 11.62 Orchard Therapeutics: Preclinical Stage Gene Therapy Portfolio
• Table 11.63 Orchard Therapeutics: Recent Developments and Future Outlook
• Table 11.64 CSL Behring: Company Overview
• Table 11.65 CSL Behring: Marketed Gene Therapy Portfolio
• Table 11.66 CSL Behring: Discovery Stage Gene Therapy Portfolio
• Table 11.67 CSL Behring: Recent Developments and Future Outlook
• Table 11.68 Shanghai Sunway Biotech: Company Overview
• Table 11.69 Shanghai Sunway Biotech: Marketed Gene Therapy Portfolio
• Table 11.70 Sibiono GeneTech: Company Overview
• Table 11.71 Sibiono GeneTech: Marketed Gene Therapy Portfolio
• Table 12.1 Gene Therapies: Marketed and Approved Products
• Table 12.2 Company Overview: Sibiono GeneTech
• Table 12.3 Gendicine: Patent Portfolio
• Table 12.4 Gendicine: Stage of Development
• Table 12.5 Company Overview: Shanghai Sunway Biotech
• Table 12.6 Oncorine: Stage of Development
• Table 12.7 Company Overview: Human Stem Cell Institute
• Table 12.8 Neovasculgen: Stage of Development
• Table 12.9 Company Overview: Amgen
• Table 12.10 Imlygic: Stage of Development
• Table 12.11 Imlygic: Recommended Dose and Schedule
• Table 12.12 Imlygic: Determination of Injection Volume based upon Lesion Size
• Table 12.13 Company Overview: Orchard Therapeutics
• Table 12.14 Strimvelis: Stage of Development
• Table 12.15 Company Overview: Spark Therapeutics
• Table 12.16 Luxturna: Stage of Development
• Table 12.17 Company Overview: Novartis
• Table 12.18 Zolgensma: Stage of Development
• Table 12.19 Company Overview: bluebird bio
• Table 12.20 Zynteglo: Stage of Development
• Table 12.21 Libmeldy: Stage of Development
• Table 13.1 Libmeldy: Presentations in Conferences Before Product Approval
• Table 13.2 Luxturna: Presentations in Conferences Before Product Approval
• Table 13.3 Zolgensma: Presentations in Conferences Before Product Approval
• Table 13.4 Zynteglo: Presentations in Conferences Before Product Approval
• Table 13.5 Imlygic: Presentations in Conferences After Product Approval
• Table 13.6 Libmeldy: Presentations in Conferences After Product Approval
• Table 13.7 Luxturna: Presentations in Conferences After Product Approval
• Table 13.8 Strimvelis: Presentations in Conferences After Product Approval
• Table 13.9 Zolgensma: Presentations in Conferences After Product Approval
• Table 13.10 Zynteglo: Presentations in Conferences After Product Approval
• Table 13.11 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
• Table 14.1 Gene Therapies: Phase II / III and Above Drugs
• Table 14.2 Drug Profile: LUMEVOQ (GS010)
• Table 14.3 Drug Profile: OTL-103
• Table 14.4 Drug Profile: PTC-AADC
• Table 14.5 Drug Profile: BMN 270
• Table 14.6 Drug Profile: nadofaragene firadenovec (rAd-IFN/Syn3)
• Table 14.7 Drug Profile: beti-cel
• Table 14.8 Drug Profile: eli-cel
• Table 14.9 Drug Profile: lovo-cel
• Table 14.10 Drug Profile: SRP-9001
• Table 14.11 Drug Profile: EB-101
• Table 14.12 Drug Profile: ProstAtak
• Table 14.13 Drug Profile: D-Fi
• Table 14.14 Drug Profile: CG0070
• Table 14.15 Drug Profile: Vigil-EWS
• Table 14.16 Drug Profile: Engensis
• Table 14.17 Drug Profile: VGX-3100
• Table 14.18 Drug Profile: INVOSSA (TG-C)
• Table 14.19 Drug Profile: VYJUVEKT
• Table 14.20 Drug Profile: PF-06939926
• Table 14.21 Drug Profile: PF-06838435
• Table 14.22 Drug Profile: PF-07055480
• Table 14.23 Drug Profile: SPK-8011
• Table 14.24 Drug Profile: AMT-061
• Table 14.25 Drug Profile: VB-111
• Table 14.26 Drug Profile: Generx
• Table 14.27 Drug Profile: ADXS-HPV
• Table 14.28 Drug Profile: AGTC 501
• Table 14.29 Drug Profile: LYS-SAF302
• Table 14.30 Drug Profile: NFS-01
• Table 14.31 Drug Profile: AG0302-COVID19
• Table 14.32 Drug Profile: RGX-314
• Table 14.33 Drug Profile: Hologene 5
• Table 15.1 Gene Therapy Patent Portfolio: List of Top CPC Symbols
• Table 15.2 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
• Table 15.3 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
• Table 15.4 Gene Therapy Patent Portfolio: List of Leading Patents (In terms of Highest Relative Valuation)
• Table 15.5 Gene Editing Patent Portfolio: List of Top CPC Classifications
• Table 15.6 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
• Table 15.7 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
• Table 15.8 Gene Editing Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
• Table 16.1 Gene Therapy: List of Mergers and Acquisitions, 2015-2022
• Table 16.2 Acquisitions: Key Value Drivers
• Table 16.3 Mergers and Acquisitions: Information on Phase of Development and Therapeutic Area of the Acquired Company's Gene Therapy Product
• Table 16.4 Mergers and Acquisitions: Information on Deal Multiple based on Acquisition Amount
• Table 17.1 Gene Therapy Market: Funding and Investments, 2015-2022
• Table 17.2 Funding and Investments: Summary of Investments
• Table 17.3 Funding and Investments: Summary of Venture Capital Funding
• Table 18.1 Clinical Trial Analysis: Emerging Molecules to Watch
• Table 18.2 Clinical Trial Analysis: Most Important Trials to Watch
• Table 19.1 Pricing Model: Price of Marketed Gene / Cell Therapies
• Table 19.2 Pricing Model: Price of Marketed Targeted Drugs (Antibody Drug Conjugates and Monoclonal Antibodies)
• Table 19.3 Pricing Model: Opinions of Experts / Other Analysts
• Table 20.1 Start-up Valuation: Estimates based on Overall Experience
• Table 22.1 Global Clinical Demand for Gene Therapies: Information on Trial Phase, Distribution of Patient Enrolment and Average Number of Patients Enrolled
• Table 24.1 Gene Therapy Market: List of Forecasted Therapies along with Information on Developer, Stage of Development and First Launch Year / Likely Approval Year
• Table 32.1 Adstiladrin(R): Net Present Value (USD Million)
• Table 32.2 Adstilarin(R): Value Creation Analysis (USD Million)
• Table 32.3 BEQVEZ(TM): Net Present Value (USD Million)
• Table 32.4 BEQVEZ(TM): Value Creation Analysis (USD Million)
• Table 32.5 Casgevy(TM): Net Present Value (USD Million)
• Table 32.6 Casgevy(TM): Value Creation Analysis (USD Million)
• Table 32.7 Elevidys(R): Net Present Value (USD Million)
• Table 32.8 Elevidys(R): Value Creation Analysis (USD Million)
• Table 32.9 Gendicine(R): Net Present Value (USD Million)
• Table 32.10 Gendicine(R): Value Creation Analysis (USD Million)
• Table 32.11 Hemgenix(R): Net Present Value (USD Million)
• Table 32.12 Hemgenix(R): Value Creation Analysis (USD Million)
• Table 32.13 IMLYGIC(R): Net Present Value (USD Million)
• Table 32.14 IMLYGIC(R): Value Creation Analysis (USD Million)
• Table 32.15 Libmeldy(R): Net Present Value (USD Million)
• Table 32.16 Libmeldy(R): Value Creation Analysis (USD Million)
• Table 32.17 Luxturna(R): Net Present Value (USD Million)
• Table 32.18 Luxturna(R): Value Creation Analysis (USD Million)
• Table 32.19 LYFGENIA(TM): Net Present Value (USD Million)
• Table 32.20 LYFGENIA(TM): Value Creation Analysis (USD Million)
• Table 32.21 Neovasculgen(R): Net Present Value (USD Million)
• Table 32.22 Neovasculgen(R): Value Creation Analysis (USD Million)
• Table 32.23 Oncorine(R): Net Present Value (USD Million)
• Table 32.24 Oncorine(R): Value Creation Analysis (USD Million)
• Table 32.25 Roctavian(TM): Net Present Value (USD Million)
• Table 32.26 Roctavian(TM): Value Creation Analysis (USD Million)
• Table 32.27 SKYSONA(R): Net Present Value (USD Million)
• Table 32.28 SKYSONA(R): Value Creation Analysis (USD Million)
• Table 32.29 Strimvelis(TM): Net Present Value (USD Million)
• Table 32.30 Strimvelis(TM): Value Creation Analysis (USD Million)
• Table 32.31 Upstaza(TM): Net Present Value (USD Million)
• Table 32.32 Upstaza(TM): Value Creation Analysis (USD Million)
• Table 32.33 VYJUVEKT(R): Net Present Value (USD Million)
• Table 32.34 VYJUVEKT(R): Value Creation Analysis (USD Million)
• Table 32.35 Zolgensma(R): Net Present Value (USD Million)
• Table 32.36 Zolgensma(R): Value Creation Analysis (USD Million)
• Table 32.37 Zynteglo(TM): Net Present Value (USD Million)
• Table 32.38 Zynteglo(TM): Value Creation Analysis (USD Million)
• Table 32.39 AAV-RPE65: Net Present Value (USD Million)
• Table 32.40 AAV-RPE65: Value Creation Analysis (USD Million)
• Table 32.41 ABO-102 (UX111): Net Present Value (USD Million)
• Table 32.42 ABO-102 (UX111): Value Creation Analysis (USD Million)
• Table 32.43 AGTC 501 (rAAV2tYF-GRK1-RPGR): Net Present Value (USD Million)
• Table 32.44 AGTC 501 (rAAV2tYF-GRK1-RPGR): Value Creation Analysis (USD Million)
• Table 32.45 Cretostimogene Grenadenorepvec (CG0070): Net Present Value (USD Million)
• Table 32.46 Cretostimogene Grenadenorepvec (CG0070: Value Creation Analysis (USD Million)
• Table 32.47 Dirloctocogene samoparvovec (SPK-8011): Net Present Value (USD Million)
• Table 32.48 Dirloctocogene samoparvovec (SPK-8011): Value Creation Analysis (USD Million)
• Table 32.49 DTx-301: Net Present Value (USD Million)
• Table 32.50 DTx-301: Value Creation Analysis (USD Million)
• Table 32.51 DTx-401: Net Present Value (USD Million)
• Table 32.52 DTx-401: Value Creation Analysis (USD Million)
• Table 32.53 Generx(R) (Ad5FGF-4): Net Present Value (USD Million)
• Table 32.54 Generx(R) (Ad5FGF-4): Value Creation Analysis (USD Million)
• Table 32.55 Giroctocogene fitelparvovec (SB-525 / PF-07055480): Net Present Value (USD Million)
• Table 32.56 Giroctocogene fitelparvovec (SB-525 / PF-07055480): Value Creation Analysis (USD Million)
• Table 32.57 Hologene 5: Net Present Value (USD Million)
• Table 32.58 Hologene 5: Value Creation Analysis (USD Million)
• Table 32.59 INVOSSA(TM) (TG-C): Net Present Value (USD Million)
• Table 32.60 INVOSSA(TM) (TG-C): Value Creation Analysis (USD Million)
• Table 32.61 LUMEVOQ(R): Net Present Value (USD Million)
• Table 32.62 LUMEVOQ(R): Value Creation Analysis (USD Million)
• Table 32.63 NFS-01 (rAAV2-ND4): Net Present Value (USD Million)
• Table 32.64 NFS-01 (rAAV2-ND4): Value Creation Analysis (USD Million)
• Table 32.65 NTLA-2001: Net Present Value (USD Million)
• Table 32.66 NTLA-2001: Value Creation Analysis (USD Million)
• Table 32.67 Olvi-Vec (Olvimulogene nanivacirepvec): Net Present Value (USD Million)
• Table 32.68 Olvi-Vec (Olvimulogene nanivacirepvec): Value Creation Analysis (USD Million)
• Table 32.69 ProstAtak(R): Net Present Value (USD Million)
• Table 32.70 ProstAtak(R): Value Creation Analysis (USD Million)
• Table 32.71 pz-cel (prademagene zamikeracel / EB-101): Net Present Value (USD Million)
• Table 32.72 pz-cel (prademagene zamikeracel / EB-101): Value Creation Analysis (USD Million)
• Table 32.73 RGX-121: Net Present Value (USD Million)
• Table 32.74 RGX-121: Value Creation Analysis (USD Million)
• Table 32.75 RGX-314: Net Present Value (USD Million)
• Table 32.76 RGX-314: Value Creation Analysis (USD Million)
• Table 32.77 VGX-3100: Net Present Value (USD Million)
• Table 32.78 VGX-3100: Value Creation Analysis (USD Million)
• Table 32.79 Summary of Variations in Forecasted Sales Revenue of Gene Therapies (Compared to 5th Edition)
• Table 33.1 Gene Editing Technologies: Information on Developer and Highest Phase of Drug Development Supported
• Table 33.2 Key Features of Gene Editing Platforms
• Table 33.3 ASC Therapeutics: Collaborations
• Table 33.4 CRISPR Therapeutics: Funding Instances
• Table 33.5 CRISPR Therapeutics: Collaborations
• Table 33.6 Editas Medicine: Funding Instances
• Table 33.7 Editas Medicine: Collaborations
• Table 33.8 Emendo Biotherapeutics: Collaborations
• Table 33.9 Excision Biotherapeutics: Funding Instances
• Table 33.10 Intellia Therapeutics: Funding Instances
• Table 33.11 Intellia Therapeutics: Collaborations
• Table 33.12 Sarepta Therapeutics: Funding Instances
• Table 33.13 Sarepta Therapeutics: Collaborations
• Table 33.14 ToolGen: Collaborations
• Table 33.15 Sangamo Therapeutics: Funding Instances
• Table 33.16 Sangamo Therapeutics: Collaborations
• Table 33.17 bluebird bio: Funding Instances
• Table 34.18 bluebird bio: Collaborations
• Table 34.19 Gene Switch Technology Platforms
• Table 33.20 Precigen: Funding Instances
• Table 33.21 MeiraGTx: Funding Instances
• Table 33.22 MeiraGTx: Collaborations
• Table 33.23 Gene Therapy Developers: Information on Headquarters, Technology / Platform and Type of Gene Delivery Method
• Table 34.1 Key Features of Small-scale Cell Culture Systems
• Table 34.2 List of Viral Vector Contract Manufacturing Organizations: Information on Type of Viral Vector Manufactured
• Table 36.1 Orchard Therapeutics: Company Snapshot
• Table 36.2 Vivet Therapeutics: Company Snapshot
• Table 36.3 Kubota Pharmaceutical: Company Snapshot
• Table 36.4 Eyevensys: Company Snapshot
• Table 36.5 AGTC: Company Snapshot
• Table 36.6 LogicBio Therapeutics: Company Snapshot
• Table 36.7 AAVogen: Company Snapshot
• Table 36.8 Hemera Biosciences: Company Snapshot
• Table 36.9 Myonexus Therapeutics: Company Snapshot
• Table 36.10 Arthrogen: Company Snapshot
• Table 36.11 PlasmidFactory: Company Snapshot
• Table 36.12 Delphi Genetics: Company Snapshot
• Table 36.13 Vigene Biosciences: Company Snapshot
• Table 36.14 Gene Biotherapeutics: Company Snapshot
• Table 36.15 Milo Biotechnology: Company Snapshot
• Table 38.1 Marketed and Clinical Stage Gene Therapies: Distribution by Stage of Development
• Table 38.2 Marketed and Clinical Stage Gene Therapies: Distribution by Drug Designation
• Table 38.3 Marketed and Clinical Stage Gene Therapies: Distribution by Therapeutic Area
• Table 38.4 Marketed and Clinical Stage Gene Therapies: Distribution by Biological Target
• Table 38.5 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Vector Used
• Table 38.6 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Therapy
• Table 38.7 Marketed and Clinical Stage Gene Therapies: Distribution by Type of Gene Delivery Method
• Table 38.8 Marketed and Clinical Stage Gene Therapies: Distribution by Route of Administration
• Table 38.9 Marketed and Clinical Stage Gene Therapies: Distribution by Dosage
• Table 38.10 Preclinical and Discovery Stage Gene Therapies: Distribution by Stage of Development
• Table 38.11 Preclinical and Discovery Stage Gene Therapies: Distribution by Therapeutic Area
• Table 38.12 Preclinical and Discovery Stage Gene Therapies: Distribution by Biological Target
• Table 38.13 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Vector Used
• Table 38.14 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Therapy
• Table 38.15 Preclinical and Discovery Stage Gene Therapies: Distribution by Type of Gene Delivery Method Used
• Table 38.16 Gene Therapy Developers: Distribution by Year of Establishment
• Table 38.17 Gene Therapy Developers: Distribution by Company Size
• Table 38.18 Gene Therapy Developers: Distribution by Location of Headquarters
• Table 38.19 Most Active Players: Distribution by Number of Gene Therapies
• Table 38.20 Gene Therapy Patent Portfolio: Distribution by Type of Patent
• Table 38.21 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
• Table 38.22 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
• Table 38.23 Gene Therapy Patent Portfolio: Distribution by Geography
• Table 38.24 Gene Therapy Patent Portfolio: North America Scenario
• Table 38.25 Gene Therapy Patent Portfolio: Europe Scenario
• Table 38.26 Gene Therapy Patent Portfolio: Asia-Pacific Scenario
• Table 38.27 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
• Table 38.28 Gene Therapy Patent Portfolio: Leading Industry Players: Distribution by Number of Players
• Table 38.29 Gene Therapy Patent Portfolio (Sanofi and Genethon): Benchmarking by Patent Characteristics
• Table 38.30 Gene Editing Patent Portfolio: Distribution by Type of Patent
• Table 38.31 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2017-2022
• Table 38.32 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
• Table 38.33 Gene Editing Patent Portfolio: Distribution by Geography
• Table 38.34 Gene Editing Patent Portfolio: North America Scenario
• Table 38.35 Gene Editing Patent Portfolio: Europe Scenario
• Table 38.36 Gene Editing Patent Portfolio: Asia-Pacific Scenario
• Table 38.37 Gene Editing Patent Portfolio: Distribution by CPC Symbols
• Table 38.38 Leading Industry Players (Gene Editing): Distribution by Number of Patents
• Table 38.39 Gene Editing Patent Portfolio (Intellia Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
• Table 38.40 Mergers and Acquisitions: Cumulative Year-Wise Trend, 2015-2022
• Table 38.42 Mergers and Acquisitions: Distribution by Year and Type of Agreement
• Table 38.43 Mergers and Acquisitions: Intercontinental and Intracontinental Deals
• Table 38.44 Mergers and Acquisitions: Local and International Deals
• Table 38.45 Acquisitions: Distribution by Key Value Drivers
• Table 38.46 Acquisitions: Distribution by Year of Acquisition and Key Value
• Table 38.47 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product
• Table 38.48 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company's Product
• Table 38.49 Mergers and Acquisitions: Distribution by Therapeutic Area
• Table 38.50 Funding and Investments: Cumulative Distribution of Instances by Year, 2015-2022
• Table 38.51 Funding and Investments: Cumulative Distribution of Amount Invested by Year, 2015-2022 (USD Million)
• Table 38.52 Funding and Investments: Distribution of Instances by Type of Funding, 2015-2022
• Table 38.53 Funding and Investments: Distribution of Total Amount Invested by Type of Funding (USD Million)
• Table 38.54 Funding and Investments: Summary of Investments, 2015-2022 (USD Million)
• Table 38.55 Funding and Investments: Distribution of Instances and Amount Invested by Type of Therapy
• Table 38.56 Funding and Investments: Distribution of Instances and Amount Invested by Geography
• Table 38.57 Funding and Investments: Regional Distribution of Funding Instances
• Table 38.58 Funding and Investments: Distribution of Instances and Amount Invested by Highest Phase of Development of the Company's Product
• Table 38.59 Funding and Investments: Distribution of Instances by Therapeutic Area
• Table 38.60 Most Active Players: Distribution by Number of Funding Instances
• Table 38.61 Most Active Players: Distribution by Amount Invested (USD Million)
• Table 38.62 Funding and Investments: Distribution of Instances by Type of Investors
• Table 38.63 Leading Investors: Distribution by Number of Instances
• Table 38.64 Funding and Investment Summary, 2015-2022 (USD Million)
• Table 38.65 Clinical Trial Analysis: Distribution by Trial Registration Year, 2017-2022
• Table 38.66 Clinical Trial Analysis: Distribution by Trial Status
• Table 38.67 Clinical Trial Analysis: Distribution by Trial Phase
• Table 38.68 Clinical Trial Analysis: Distribution by Therapeutic Area
• Table 38.69 Clinical Trial Analysis: Distribution by Geography
• Table 38.70 Clinical Trial Analysis: Distribution by Trial Registration Year and Geography
• Table 38.71 Clinical Trial Analysis: Distribution by Trial Status and Geography
• Table 38.72 Clinical Trial Analysis: Distribution by Type of Sponsor
• Table 38.73 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
• Table 38.74 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year, 2017-2022
• Table 38.75 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
• Table 38.76 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
• Table 38.77 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
• Table 38.78 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
• Table 38.79 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
• Table 38.80 Demand Analysis: Global Demand for Gene Therapies
• Table 38.81 Demand Analysis: Distribution by Stage of Development
• Table 38.82 Demand Analysis: Distribution by Therapeutic Area
• Table 38.83 Demand Analysis: Distribution by Type of Therapy
• Table 38.84 Demand Analysis: Distribution by Type of Gene Delivery Method
• Table 38.85 Demand Analysis: Distribution by Geography
• Table 38.86 Global Gene Therapy Market, till 2035, Base Scenario (USD Billion)
• Table 38.87 Global Gene Therapy Market, till 2035, Conservative Scenario (USD Billion)
• Table 38.88 Global Gene Therapy Market, till 2035, Optimistic Scenario (USD Billion)
• Table 38.89 Gene Therapy Market: Distribution by Therapeutic Area, 2024, 2029 and 2035
• Table 38.90 Gene Therapy Market for Muscle Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.91 Gene Therapy Market for Hematologic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.92 Gene Therapy Market for Dermatological Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.93 Gene Therapy Market for Oncological Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.94 Gene Therapy Market for Genetic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.95 Gene Therapy Market for Ophthalmic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.96 Gene Therapy Market for Metabolic Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.97 Gene Therapy Market for Cardiovascular Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.98 Gene Therapy Market for Other Disorders, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.99 Gene Therapy Market: Distribution by Type of Vector Used, 2024, 2029 and 2035
• Table 38.100 Gene Therapy Market for Adeno-associated Viral Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.101 Gene Therapy Market for Herpes Simplex Virus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.102 Gene Therapy Market for Lentivirus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.103 Gene Therapy Market for Adenovirus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.104 Gene Therapy Market for Non-Viral Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.105 Gene Therapy Market for Retrovirus Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.106 Gene Therapy Market for Other-Viral Vector, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.107 Gene Therapy Market: Distribution by Type of Therapy, 2024, 2029 and 2035
• Table 38.108 Gene Therapy Market for Gene Augmentation, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.109 Gene Therapy Market for Oncolytic Immunotherapies, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.110 Gene Therapy Market for Gene Editing, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.111 Gene Therapy Market for Gene Regulation, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.112 Gene Therapy Market for Other Therapies, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.113 Gene Therapy Market: Distribution by Type of Gene Delivery Method Used, 2024, 2029 and 2035
• Table 38.114 Gene Therapy Market for In vivo Gene Delivery, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.115 Gene Therapy Market for Ex vivo Gene Delivery, Forecasted till 2035, Conservative, Base and Optimistic Scenarios (USD
• Table 38.116 Gene Therapy Market: Distribution by Route of Administration, 2024, 2029 and 2035
• Table 38.117 Gene Therapy Market for Intravenous till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.118 Gene Therapy Market for Subretinal till 2024-2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.119 Gene Therapy Market for Intratumoral till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.120 Gene Therapy Market for Intramuscular till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.121 Gene Therapy Market for Other till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.122 Gene Therapy Market: Distribution by Geography, 2024, 2029 and 2035
• Table 38.123 Gene Therapy Market for North America, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.124 Gene Therapy Market for Europe, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.125 Gene Therapy Market for Asia-Pacific, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.126 Gene Therapy Market for Latin America, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.127 Gene Therapy Market for Rest of the World, till 2035, Conservative, Base and Optimistic Scenarios (USD Billion)
• Table 38.128 Adstiladrin(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.129 BEQVEZ(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.130 Casgevy(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.131 Elevidys(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.132 Gendicine(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.133 Hemgenix(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.134 IMLYGIC(R) (AMT-061) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.135 Libmeldy(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.136 Luxturna(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.137 LYFGENIA(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.138 Neovasculgen(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.139 Oncorine(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.140 Roctavian(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.141 SKYSONA(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.142 Strimvelis(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.143 Upstaza(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.144 VYJUVEKT(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.145 Zolgensma(R) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.146 Zynteglo(TM) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.147 AAV-RPE65 Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.148 ABO-102 (UX111) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.149 AGTC 501 (rAAV2tYF-GRK1-RPGR) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.150 Cretostimogene Grenadenorepvec (CG0070) Sales Forecast, till 2035, Conservative, Base and Optimistic Scenarios (USD Million)
• Table 38.151 Dirloctocogene samoparvovec (SPK-8011) Sales Forecast, till

List of Figures

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