目次

Table of Contents

1 INTRODUCTION
1.1 STUDY OBJECTIVES
1.2 MARKET DEFINITION
1.3 STUDY SCOPE
1.3.1 MARKETS COVERED & REGIONAL SEGMENTATION
1.3.2 INCLUSIONS & EXCLUSIONS
1.3.3 YEARS CONSIDERED
1.3.4 CURRENCY CONSIDERED
1.4 STAKEHOLDERS
1.5 SUMMARY OF CHANGES

2 RESEARCH METHODOLOGY
2.1 RESEARCH DATA
2.1.1 SECONDARY RESEARCH
2.1.1.1 Key data from secondary sources
2.1.2 PRIMARY RESEARCH
2.1.2.1 Insights of primary experts
2.2 MARKET SIZE ESTIMATION
2.2.1 GLOBAL MARKET ESTIMATION
2.2.2 SEGMENTAL MARKET SIZE ESTIMATION
2.3 MARKET GROWTH RATE PROJECTIONS
2.4 MARKET BREAKDOWN AND DATA TRIANGULATION
2.5 RESEARCH ASSUMPTIONS
2.6 RESEARCH LIMITATIONS
2.7 RISK ANALYSIS

3 EXECUTIVE SUMMARY

4 PREMIUM INSIGHTS
4.1 GENE THERAPY MARKET OVERVIEW
4.2 NORTH AMERICA: GENE THERAPY MARKET, BY THERAPY TYPE & COUNTRY (2024)
4.3 GENE THERAPY MARKET SHARE, BY END USER, 2024 VS 2032
4.4 GENE THERAPY MARKET: GEOGRAPHIC GROWTH OPPORTUNITIES

5 MARKET OVERVIEW
5.1 INTRODUCTION
5.2 MARKET DYNAMICS
5.2.1 DRIVERS
5.2.1.1 Growing demand for curative therapies for genetic disorders, rare diseases, and cancers
5.2.1.2 Increasing investments in gene therapy research
5.2.1.3 Technological advancements
5.2.1.4 Robust clinical trial pipeline for gene therapy
5.2.2 RESTRAINTS
5.2.2.1 High cost of gene therapy products
5.2.2.2 Complex regulatory frameworks and ethical concerns regarding genetic modifications
5.2.2.3 Limited manufacturing scalability
5.2.3 OPPORTUNITIES
5.2.3.1 Expansion into rare diseases and unmet needs
5.2.3.2 Increasing focus on precision medicine
5.2.3.3 Advancements in gene therapy delivery technologies
5.2.4 CHALLENGES
5.2.4.1 Complex commercial manufacturing and scaling process
5.2.4.2 Short shelf life and supply chain challenges
5.2.4.3 Lack of reimbursement
5.3 TECHNOLOGY ANALYSIS
5.3.1 KEY TECHNOLOGIES
5.3.1.1 CRISPR-Cas9
5.3.1.2 RNA interference
5.3.1.3 Viral vectors
5.3.2 COMPLEMENTARY TECHNOLOGIES
5.3.2.1 Stem cell therapy
5.3.2.2 Omics technologies
5.3.3 ADJACENT TECHNOLOGIES
5.3.3.1 Nanoparticles
5.4 VALUE CHAIN ANALYSIS
5.5 SUPPLY CHAIN ANALYSIS
5.5.1 PROMINENT COMPANIES
5.5.2 SMALL AND MEDIUM-SIZED ENTERPRISES
5.5.3 END USERS
5.5.4 INVESTORS, VENTURE CAPITALISTS, AND FUNDING BODIES
5.5.5 REGULATORY BODIES
5.6 ECOSYSTEM ANALYSIS
5.7 PATENT ANALYSIS
5.8 PIPELINE ANALYSIS
5.9 REGULATORY LANDSCAPE
5.9.1 REGULATORY BODIES, GOVERNMENT AGENCIES, AND OTHER ORGANIZATIONS, BY REGION
5.9.2 REGULATORY FRAMEWORK
5.9.3 REIMBURSEMENT SCENARIO
5.10 PRICING ANALYSIS
5.10.1 AVERAGE SELLING PRICE TREND, BY KEY PLAYER
5.10.2 AVERAGE SELLING PRICE TREND, BY REGION
5.11 TRENDS/DISRUPTIONS IMPACTING CUSTOMERS’ BUSINESSES
5.12 KEY CONFERENCES & EVENTS, 2025-2026
5.13 PORTER’S FIVE FORCES ANALYSIS
5.13.1 THREAT OF NEW ENTRANTS
5.13.2 THREAT OF SUBSTITUTES
5.13.3 BARGAINING POWER OF SUPPLIERS
5.13.4 BARGAINING POWER OF BUYERS
5.13.5 INTENSITY OF COMPETITIVE RIVALRY
5.14 KEY STAKEHOLDERS & BUYING CRITERIA
5.14.1 KEY STAKEHOLDERS IN BUYING PROCESS
5.14.2 BUYING CRITERIA
5.15 INVESTMENT & FUNDING SCENARIO
5.16 IMPACT OF AI ON GENE THERAPY MARKET

6 GENE THERAPY MARKET, BY THERAPY TYPE
6.1 INTRODUCTION
6.2 GENE SILENCING THERAPY
6.2.1 FLEXIBILITY AND PRECISION OF GENE SILENCING THERAPIES TO SUPPORT GROWTH
6.3 CELL REPLACEMENT THERAPY
6.3.1 LONG-TERM EFFECT OF THERAPY TO SUPPORT GROWTH IN ADOPTION
6.4 GENE AUGMENTATION THERAPY
6.4.1 EFFECTIVENESS AGAINST MONOGENIC DISORDERS TO DRIVE MARKET
6.5 OTHER THERAPIES

7 GENE THERAPY MARKET, BY VECTOR
7.1 INTRODUCTION
7.2 VIRAL VECTORS
7.2.1 RETROVIRAL VECTORS
7.2.1.1 Advantages of retroviral vectors to support market growth
7.2.1.2 Gamma-retroviral vectors
7.2.1.2.1 Availability of gamma-retroviral vectors to support market growth
7.2.1.3 Lentiviral vectors
7.2.1.3.1 North America to dominate lentiviral vectors market
7.2.2 AAV VECTORS
7.2.2.1 Focus of clinical pipeline on adeno-associated viral vectors to drive growth
7.2.3 OTHER VIRAL VECTORS
7.3 NON-VIRAL VECTORS
7.3.1 OLIGONUCLEOTIDE VECTORS
7.3.1.1 Oligonucleotides to account for largest share of non-viral vectors market
7.3.2 OTHER NON-VIRAL VECTORS

8 GENE THERAPY MARKET, BY GENE TYPE
8.1 INTRODUCTION
8.2 DEFICIENCY
8.2.1 TECHNOLOGICAL ADVANCEMENT IN GENE THERAPIES TO BOOST GROWTH
8.3 RECEPTOR
8.3.1 INCREASING DEMAND FOR DEVELOPING RECEPTOR-BASED GENE THERAPY TO SUPPORT MARKET GROWTH
8.4 ANTIGEN
8.4.1 INCREASING DEMAND FOR TARGETED THERAPIES TO PROPEL GROWTH
8.5 CYTOKINE
8.5.1 RISING R&D TO SUPPORT MARKET GROWTH
8.6 TUMOR SUPPRESSOR
8.6.1 RISING PREVALENCE OF CANCER TO PROPEL MARKET GROWTH
8.7 OTHER GENES

9 GENE THERAPY MARKET, BY THERAPEUTIC AREA
9.1 INTRODUCTION
9.2 NEUROLOGY
9.2.1 ENHANCED DELIVERY METHODS AND ADVANCEMENTS IN GENE EDITING TECHNOLOGIES TO BOOST MARKET GROWTH
9.3 ONCOLOGY
9.3.1 RISING BURDEN OF CANCER TO SUPPORT MARKET GROWTH
9.4 HEMATOLOGY
9.4.1 INCREASING PREVALENCE OF GENETIC BLOOD DISORDERS TO BOOST MARKET GROWTH
9.5 OTHER THERAPEUTIC AREAS

10 GENE THERAPY MARKET, BY DELIVERY METHOD
10.1 INTRODUCTION
10.2 IN VIVO DELIVERY
10.2.1 IN VIVO DELIVERY METHOD TO ACCOUNT FOR LARGEST MARKET SHARE IN 2023

10.3 EX VIVO DELIVERY
10.3.1 GROWING FOCUS ON ONCOLOGY THERAPEUTICS TO SUPPORT MARKET GROWTH

11 GENE THERAPY MARKET, BY ROUTE OF ADMINISTRATION
11.1 INTRODUCTION
11.2 INTRAVENOUS ROUTE
11.2.1 HIGH EFFECTIVENESS AND LARGE SHARE OF INTRAVENOUS PRODUCTS TO SUPPORT MARKET GROWTH
11.3 SUBCUTANEOUS ROUTE
11.3.1 DEVELOPMENT OF IMPROVED DELIVERY DEVICES TO SUPPORT MARKET GROWTH
11.4 OTHER ROUTES

12 GENE THERAPY MARKET, BY END USER
12.1 INTRODUCTION
12.2 HOSPITALS
12.2.1 GROWING DEMAND FOR ADVANCED THERAPIES TO BOOST MARKET
12.3 SPECIALTY CENTERS
12.3.1 ADVANCEMENTS IN GENE THERAPY TECHNOLOGIES TO SUPPORT MARKET GROWTH
12.4 OTHER END USERS

13 GENE THERAPY MARKET, BY REGION
13.1 INTRODUCTION
13.2 NORTH AMERICA
13.2.1 NORTH AMERICA: MACROECONOMIC ANALYSIS
13.2.2 US
13.2.2.1 US to dominate North American market during forecast period
13.2.3 CANADA
13.2.3.1 Government support for expanding manufacturing facilities to propel market growth
13.3 EUROPE
13.3.1 EUROPE: MACROECONOMIC ANALYSIS
13.3.2 GERMANY
13.3.2.1 Growing pharmaceutical R&D and manufacturing to drive market
13.3.3 UK
13.3.3.1 Strong government support to propel market growth
13.3.4 FRANCE
13.3.4.1 Government investment in pharmaceutical industry to drive growth
13.3.5 ITALY
13.3.5.1 Initiatives for gene therapy development to drive growth
13.3.6 SPAIN
13.3.6.1 Expansion of biomanufacturing facilities to support market growth
13.3.7 SWITZERLAND
13.3.7.1 Presence of key pharmaceutical and biopharmaceutical companies to support market growth
13.3.8 NETHERLANDS
13.3.8.1 Growing awareness and strong focus on pharmaceutical R&D to support market growth
13.3.9 REST OF EUROPE
13.4 ASIA PACIFIC
13.4.1 ASIA PACIFIC: MACROECONOMIC ANALYSIS
13.4.2 CHINA
13.4.2.1 China to dominate Asia Pacific market during forecast period
13.4.3 JAPAN
13.4.3.1 Increasing initiatives supporting gene therapy adoption to propel market growth
13.4.4 INDIA
13.4.4.1 Favorable scenario for foreign direct investment to favor market growth
13.4.5 AUSTRALIA
13.4.5.1 Increasing demand for innovative therapies to propel market
13.4.6 SOUTH KOREA
13.4.6.1 Increased export of drugs to support market growth
13.4.7 THAILAND
13.4.7.1 Favorable government policies to propel adoption of advanced therapies
13.4.8 REST OF ASIA PACIFIC
13.5 LATIN AMERICA
13.5.1 LATIN AMERICA: MACROECONOMIC ANALYSIS
13.5.2 BRAZIL
13.5.2.1 Brazil to dominate LATAM market during forecast period
13.5.3 MEXICO
13.5.3.1 Rising demand for chronic disease treatment to support market growth
13.5.4 REST OF LATIN AMERICA
13.6 MIDDLE EAST
13.6.1 MIDDLE EAST: MACROECONOMIC ANALYSIS
13.6.2 GCC COUNTRIES
13.6.2.1 Saudi Arabia
13.6.2.1.1 Growing healthcare expenditure to boost market growth
13.6.2.2 UAE
13.6.2.2.1 Growing focus on genetic medicine development projects to drive market
13.6.2.3 Rest of GCC Countries
13.6.3 REST OF MIDDLE EAST

13.7 AFRICA
13.7.1 HIGH GROWTH POTENTIAL OF AFRICAN MARKETS TO ATTRACT KEY PLAYERS
13.7.2 AFRICA: MACROECONOMIC ANALYSIS

14 COMPETITIVE LANDSCAPE
14.1 OVERVIEW
14.2 KEY PLAYER STRATEGIES/RIGHT TO WIN, 2022-2024
14.2.1 OVERVIEW OF STRATEGIES ADOPTED BY KEY PLAYERS IN GENE THERAPY MARKET, 2022−2024
14.3 REVENUE ANALYSIS, 2021-2023
14.4 MARKET SHARE ANALYSIS, 2023
14.5 COMPANY EVALUATION MATRIX: KEY PLAYERS, 2023
14.5.1 STARS
14.5.2 EMERGING LEADERS
14.5.3 PERVASIVE PLAYERS
14.5.4 PARTICIPANTS
14.5.5 COMPETITIVE BENCHMARKING: KEY PLAYERS, 2023
14.5.5.1 Company footprint
14.5.5.2 Region footprint
14.5.5.3 Therapy type footprint
14.5.5.4 Therapeutic area footprint
14.5.5.5 Route of administration footprint
14.6 COMPANY EVALUATION MATRIX: STARTUPS/SMES, 2023
14.6.1 PROGRESSIVE COMPANIES
14.6.2 RESPONSIVE COMPANIES
14.6.3 DYNAMIC COMPANIES
14.6.4 STARTING BLOCKS
14.6.5 COMPETITIVE BENCHMARKING: STARTUPS/SMES, 2023
14.6.5.1 Detailed list of key startups/SMEs
14.6.5.2 Competitive benchmarking of key startups/SMEs
14.7 VALUATION & FINANCIAL METRICS
14.7.1 FINANCIAL METRICS
14.7.2 COMPANY VALUATION
14.8 BRAND/PRODUCT COMPARISON
14.9 COMPETITIVE SCENARIO
14.9.1 PRODUCT APPROVALS
14.9.2 DEALS
14.9.3 EXPANSIONS

15 COMPANY PROFILES
15.1 INTRODUCTION
15.2 KEY PLAYERS
15.2.1 BIOGEN INC.
15.2.1.1 Business overview
15.2.1.2 Products offered
15.2.1.3 Recent developments
15.2.1.3.1 Deals
15.2.1.4 MnM view
15.2.1.4.1 Key strengths
15.2.1.4.2 Strategic choices
15.2.1.4.3 Weaknesses and competitive threats
15.2.2 NOVARTIS AG
15.2.2.1 Business overview
15.2.2.2 Products offered
15.2.2.3 Recent developments
15.2.2.3.1 Deals
15.2.2.3.2 Expansions
15.2.2.3.3 Other developments
15.2.2.4 MnM view
15.2.2.4.1 Key strengths
15.2.2.4.2 Strategic choices
15.2.2.4.3 Weaknesses and competitive threats
15.2.3 ALNYLAM PHARMACEUTICALS, INC.
15.2.3.1 Business overview
15.2.3.2 Products offered
15.2.3.3 Recent developments
15.2.3.3.1 Product approvals
15.2.3.3.2 Deals
15.2.3.3.3 Other developments
15.2.3.4 MnM view
15.2.3.4.1 Key strengths
15.2.3.4.2 Strategic choices
15.2.3.4.3 Weaknesses and competitive threats
15.2.4 SAREPTA THERAPEUTICS, INC.
15.2.4.1 Business overview
15.2.4.2 Products offered
15.2.4.3 Recent developments
15.2.4.3.1 Product approvals
15.2.4.3.2 Deals
15.2.4.4 MnM view
15.2.4.4.1 Key strengths
15.2.4.4.2 Strategic choices
15.2.4.4.3 Weaknesses and competitive threats
15.2.5 JAZZ PHARMACEUTICALS PLC
15.2.5.1 Business overview
15.2.5.2 Products offered
15.2.5.3 Recent developments
15.2.5.3.1 Expansions
15.2.5.4 MnM view
15.2.5.4.1 Key strengths
15.2.5.4.2 Strategic choices
15.2.5.4.3 Weaknesses and competitive threats
15.2.6 FERRING B.V.
15.2.6.1 Business overview
15.2.6.2 Products offered
15.2.6.3 Recent developments
15.2.6.3.1 Product approvals
15.2.6.3.2 Deals
15.2.6.3.3 Expansions
15.2.7 PFIZER INC.
15.2.7.1 Business overview
15.2.7.2 Products offered
15.2.7.3 Recent developments
15.2.7.3.1 Product approvals
15.2.7.3.2 Deals
15.2.8 AMGEN, INC.
15.2.8.1 Business overview
15.2.8.2 Products offered
15.2.8.3 Recent developments
15.2.8.3.1 Deals
15.2.8.3.2 Expansions
15.2.9 JOHNSON & JOHNSON SERVICES, INC.
15.2.9.1 Business overview
15.2.9.2 Products offered
15.2.9.3 Recent developments
15.2.9.3.1 Deals
15.2.9.3.2 Other developments
15.2.10 DAIICHI SANKYO COMPANY, LIMITED.
15.2.10.1 Business overview
15.2.10.2 Products offered

15.2.11 BIOMARIN PHARMACEUTICAL INC.
15.2.11.1 Business overview
15.2.11.2 Products offered
15.2.11.3 Recent developments
15.2.11.3.1 Product approvals
15.2.11.3.2 Expansions
15.2.12 KYOWA KIRIN CO., LTD.
15.2.12.1 Business overview
15.2.12.2 Products offered
15.2.12.3 Recent developments
15.2.12.3.1 Product approvals
15.2.12.3.2 Deals
15.2.13 NIPPON SHINYAKU CO., LTD.
15.2.13.1 Business overview
15.2.13.2 Products offered
15.2.13.3 Recent developments
15.2.13.3.1 Deals
15.2.14 PTC THERAPEUTICS
15.2.14.1 Business overview
15.2.14.2 Products offered
15.2.14.3 Recent developments
15.2.14.3.1 Product approvals
15.2.15 UNIQURE NV.
15.2.15.1 Business overview
15.2.15.2 Products offered
15.2.15.3 Recent developments
15.2.15.3.1 Deals
15.3 OTHER PLAYERS
15.3.1 JUVENTAS CELL THERAPY LTD.
15.3.2 CRISPR THERAPEUTICS
15.3.3 BLUEBIRD BIO, INC.
15.3.4 KRYSTAL BIOTECH, INC.
15.3.5 SHANGHAI SUNWAY BIOTECH CO., LTD.
15.3.6 JW (CAYMAN) THERAPEUTICS CO. LTD
15.3.7 IASO BIOTECHNOLOGY
15.3.8 CARSGEN THERAPEUTICS HOLDINGS LIMITED
15.3.9 ADAPTIMMUNE
15.3.10 SIBIONO GENETECH CO. LTD.
15.3.11 ULTRAGENYX PHARMACEUTICAL INC.

16 APPENDIX
16.1 DISCUSSION GUIDE
16.2 KNOWLEDGESTORE: MARKETSANDMARKETS’ SUBSCRIPTION PORTAL
16.3 CUSTOMIZATION OPTIONS
16.4 RELATED REPORTS
16.5 AUTHOR DETAILS