Global Gene therapy Market

Global Gene Therapy Market Size, Share, Growth Analysis, By Vector(Lentivirus, AAV), By Indication(Acute Lymphoblastic Leukemia (ALL), Inherited Retinal Disease) - Industry Forecast 2024-2031


Report ID: SQMIG35H2218 | Region: Global | Published Date: February, 2024
Pages: 157 | Tables: 73 | Figures: 75

Global Gene Therapy Market Insights

Gene Therapy Market size was valued at USD 6.39 Billion in 2021 and is poised to grow from USD 7.60 Billion in 2022 to USD 38.34 Billion by 2030, at a CAGR of 19% during the forecast period (2023-2030).

The global gene therapy market is a rapidly growing market that is poised for significant growth in the coming years. The purpose of the gene therapy market is to provide a cure for genetic disorders that are currently untreatable. Gene therapy market is focused on developing new and innovative therapies that can address the root cause of these diseases.

The key driving factors for the gene therapy market is the increasing prevalence of genetic disorders. The prevalence of genetic disorders is increasing globally, which is driving the demand for gene therapy. The main restraints for the gene therapy market are the high cost of these therapies. Gene therapies are complex and expensive to develop, which results in high costs for patients. The delivery mechanism is critical for the success of gene therapies, as it needs to deliver the healthy gene to the targeted cells in the body. Additionally, the immune system can recognize the gene therapy as a foreign invader, which can lead to an immune response that reduces the effectiveness of the therapy. The gene therapy market presents a significant opportunity for pharmaceutical companies to develop new and innovative therapies.

US Gene Therapy Market is poised to grow at a sustainable CAGR for the next forecast year.

Market snapshot - 2024-2031

Global Market Size

USD 6.39 Billion

Largest Segment

Retrovirus & gamma Retrovirus

Fastest Growth

Retrovirus & gamma Retrovirus

Growth Rate

19% CAGR

Global Gene therapy Market 2022-2030 ($ Bn)
Country Share for North America Region- 2022 (%)
Global Gene therapy Market Size By Vector, 2022-2030 ($ Bn)
Global Gene therapy Market Size By Vector, 2022-2030 (%)

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Global Gene Therapy Market Segmental Analysis

The global gene therapy market is segmented on the basis of vector, indication, and region. Based on vectors, the market is segmented into lentivirus, AAV, retrovirus & gamma retrovirus, modified herpes simplex virus, adenovirus, non-viral plasmid vector, and others. In terms of indication, the market is segmented into acute lymphoblastic leukemia (ALL), inherited retinal disease, large B-cell lymphoma, melanoma (lesions), beta-thalassemia major/SCD, head & neck squamous cell carcinoma, peripheral arterial disease, spinal muscular atrophy (SMA), and others. Regionwise, the market is segmented into North America, Europe, Asia Pacific, Middle East and Africa, and Latin America.

Gene Therapy Market Analysis by Vector

In 2022, retroviruses segment accounted for over 20% of the revenue share in the global gene therapy market due to their ease of isolation and incorporation of DNA into the virus, allowing the conversion of single-stranded RNA to double-stranded DNA for integration into host cell chromosomes. Ongoing clinical trials are predominantly using retroviruses, and the approval of Yes carta in 2017 has led to a significant revenue growth in this segment. Meanwhile, Adeno-Associated Viral (AAV) vectors are expected to show substantial market share growth in the future. AAVs have become highly sought after and their clinical trial applications are increasing annually due to their ability to deliver genes to the region of interest with the highest efficiency. The rise of AAV usage can be attributed to the success of orthopedic and ocular gene therapy treatments, which have shown increased efficacy. Therefore, AAV is expected to witness a significant adoption rate boost in various therapeutic fields during the forecast period.

Gene Therapy Market Analysis by Indication

As of 2022, spinal muscular atrophy (SMA) holds the largest market share of 43.44% in terms of revenue. Although SMA is rare, it is a fatal inherited disease that commonly affects infants. Zolgensma (AVXS-101), developed to treat SMA, has been proven to be effective and has altered the phenotypic of the illness. In May 2019, the FDA approved Novartis' Zolgensma, making it the only gene therapy for SMA to be approved. This approval demonstrates the growing use of gene therapies to treat serious hereditary illnesses like SMA. The inherited retinal disease segment is expected to have a significant compound annual growth rate (CAGR) during the forecast period. R&D activities for developing gene therapies to treat ocular diseases, including inherited retinal disease, have driven this segment. Spark Therapeutics commercializes Luxturna in the U.S. and Novartis outside the U.S. as a treatment for a form of inherited retinal disease. Luxturna is the first gene therapy that can be directly administered to target a genetic disease caused by mutations in a single genetic factor.

Global Gene therapy Market Size By Vector, 2022(%), 2023 (%)

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Global Gene Therapy Market Regional Insights

In 2022, North America emerged as the dominant region in the gene therapy market, contributing to over 65.0% of the overall revenue. It is projected to remain the largest routine manufacturer of gene therapy drugs during the forecast period, with increasing investments in R&D by companies of all sizes. The market is being driven by rising government investments and the growing prevalence of targeted diseases in the region, such as spinal muscular atrophy, affecting 10,000 to 25,000 individuals in the U.S. alone, according to the Spinal Muscular Atrophy Foundation.

Asia Pacific is expected to be the fastest-growing regional segment from 2023 to 2030, owing to its large population with unmet medical needs and the growing demand for innovative technologies to treat rare but increasingly prevalent diseases. The commercial application of genetic therapies is likely to witness significant growth in this region due to the availability of resources, the presence of major companies, and government investments.

Global Gene therapy Market Size By Geography, 2022-2030, 2024-2031
  • Largest
  • Fastest

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Global Gene Therapy Market Dynamics

Gene Therapy Market Drivers

Increasing Prevalence of Genetic Disorders

  • Genetic disorders, such as spinal muscular atrophy, cystic fibrosis, and hemophilia, are on the rise worldwide. The increasing prevalence of these disorders is driving the growth of the gene therapy market, as gene therapies have shown promising results in the treatment of these disorders.

Rising Demand for Personalized Medicine

  • Gene therapy offers personalized treatment options that can be tailored to individual patients based on their genetic makeup. As the demand for personalized medicine continues to rise, the gene therapy market is expected to grow significantly.

Gene Therapy Market Restraints

High Costs

  • The high costs associated with gene therapy, including research and development, clinical trials, and manufacturing, are a major restraint on the growth of the gene therapy market. This makes it difficult for patients to access gene therapies, particularly in low-income countries.

Regulatory Hurdles

  • The gene therapy market is highly regulated, with strict guidelines governing the development and approval of gene therapies. This can be a significant restraint on the growth of the market, as companies must spend significant time and resources obtaining regulatory approval for their products.

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Global Gene Therapy Market Competitive Landscape

The global gene therapy market is highly competitive with a large number of players operating in the industry. The key players in the market are focusing on the development of new and advanced therapies to enhance their market presence. The gene therapy market is highly competitive, with a number of players focusing on the development of novel therapies for the treatment of a range of diseases. The market is expected to continue to grow in the coming years, driven by increasing investment in R&D and growing demand for new and advanced therapies for the treatment of rare and life-threatening diseases.

Gene Therapy Market Top Player’s Company Profiles

  • Spark Therapeutics Inc. (US)
  • Sarepta Therapeutics Inc. (US)
  • bluebird bio-Inc. (US)
  • Gilead Sciences Inc. (US)
  • Amgen Inc. (US)
  • Regenxbio Inc. (US)
  • Krystal Biotech Inc. (US)
  • uniQure N.V. (Netherlands)
  • Avexis Inc. (US)
  • Orchard Therapeutics plc (UK)
  • Abeona Therapeutics Inc. (US)
  • Solid Biosciences Inc. (US)
  • Audentes Therapeutics Inc. (US)
  • Adaptimmune Therapeutics plc (UK)
  • Intellia Therapeutics Inc. (US)
  • Editas Medicine Inc. (US)
  • Precision BioSciences Inc. (US)

Gene Therapy Market Recent Developments

  • In July 2022, Novartis Pharmaceuticals UK announces the launch of the Novartis Biome UK Heart Health Catalyst 2022, in a world-first investor partnership with Medtronic ltd, RYSE Asset Management, and Chelsea and Westminster Hospital NHS Foundation Trust and its official charity CW+
  • In March 2022, the National Heart Lung and Blood Institute granted USD 12 million to an Indiana University School of Medicine researcher. The grant is for exploring major themes in a gene therapy approach to safer and potentially curative treatments for hemophilia.
  • In August 2021, Spark Therapeutics announced that it had completed its acquisition by Roche for $4.3 billion. Spark Therapeutics is known for developing the first FDA-approved gene therapy, Luxturna, for an inherited retinal disease.
  • In July 2021, the European Commission approved Novartis' gene therapy Zolgensma for the treatment of patients with SMA, making it the second approved gene therapy for SMA in Europe after Biogen's Spinraza.

Global Gene Therapy Key Market Trends

  • Increased focus on rare diseases: Gene therapy is becoming increasingly popular for the treatment of rare genetic disorders, which were previously ignored due to their low prevalence. For example, Zolgensma, a gene therapy for spinal muscular atrophy, was approved by the FDA in 2019 and has since become a popular treatment option. Similarly, Luxturna, a gene therapy for inherited retinal disease, was approved in 2017.
  • Development of innovative gene therapies: Several companies are investing heavily in R&D to develop innovative gene therapies. For example, Bluebird Bio is developing gene therapies for sickle cell disease and beta-thalassemia, while CRISPR Therapeutics is working on gene therapies for cancer and blood disorders.

Global Gene Therapy Market SkyQuest Analysis

SkyQuest’s ABIRAW (Advanced Business Intelligence, Research & Analysis Wing) is our Business Information Services team that Collects, Collates, Correlates, and Analyzes the Data collected by means of Primary Exploratory Research backed by robust Secondary Desk research.

According to our global gene therapy market analysis, the increasing investments in R&D activities, the growing prevalence of targeted diseases, and rising demand for novel technologies in the treatment of rare but increasingly prevalent diseases are the key growth drivers. The market is also driven by the increasing number of clinical trials and approvals by regulatory bodies for gene therapy products. The competitive landscape of the gene therapy market is highly fragmented, with the presence of several small and large players. Some of the key players in the market include Novartis International AG (Switzerland), Pfizer Inc. (US), Spark Therapeutics Inc. (US), bluebird bio, Inc. (US), and uniQure N.V. (Netherlands). These players are focusing on strategies such as collaborations, partnerships, and acquisitions to expand their market presence and improve their product portfolios. The global gene therapy market is expected to witness significant growth in the coming years due to the increasing demand for targeted and personalized therapies, rising investments in R&D activities, and the growing prevalence of targeted diseases.

Report Metric Details
Market size value in 2023 USD 6.39 Billion
Market size value in 2031 USD 38.34 Billion
Growth Rate 19%
Forecast period 2024-2031
Forecast Unit (Value) USD Billion
Segments covered
  • Vector
    • Lentivirus, AAV, Retrovirus & gamma Retrovirus, Modified Herpes Simplex Virus, Adenovirus, Non-viral Plasmid Vector, and Others
  • Indication
    • Acute Lymphoblastic Leukemia (ALL), Inherited Retinal Disease, Large B-cell Lymphoma, Melanoma (lesions), Beta-Thalassemia Major/SCD, Head & Neck Squamous Cell Carcinoma, Peripheral arterial disease, Spinal Muscular Atrophy (SMA), and Others
Regions covered North America (US, Canada), Europe (Germany, France, United Kingdom, Italy, Spain, Rest of Europe), Asia Pacific (China, India, Japan, Rest of Asia-Pacific), Latin America (Brazil, Rest of Latin America), Middle East & Africa (South Africa, GCC Countries, Rest of MEA)
Companies covered
  • Spark Therapeutics Inc. (US)
  • Sarepta Therapeutics Inc. (US)
  • bluebird bio-Inc. (US)
  • Gilead Sciences Inc. (US)
  • Amgen Inc. (US)
  • Regenxbio Inc. (US)
  • Krystal Biotech Inc. (US)
  • uniQure N.V. (Netherlands)
  • Avexis Inc. (US)
  • Orchard Therapeutics plc (UK)
  • Abeona Therapeutics Inc. (US)
  • Solid Biosciences Inc. (US)
  • Audentes Therapeutics Inc. (US)
  • Adaptimmune Therapeutics plc (UK)
  • Intellia Therapeutics Inc. (US)
  • Editas Medicine Inc. (US)
  • Precision BioSciences Inc. (US)
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Table Of Content

Executive Summary

Market overview

  • Exhibit: Executive Summary – Chart on Market Overview
  • Exhibit: Executive Summary – Data Table on Market Overview
  • Exhibit: Executive Summary – Chart on Global Gene Therapy Market Characteristics
  • Exhibit: Executive Summary – Chart on Market by Geography
  • Exhibit: Executive Summary – Chart on Market Segmentation
  • Exhibit: Executive Summary – Chart on Incremental Growth
  • Exhibit: Executive Summary – Data Table on Incremental Growth
  • Exhibit: Executive Summary – Chart on Vendor Market Positioning

Parent Market Analysis

Market overview

Market size

  • Market Dynamics
    • Exhibit: Impact analysis of DROC, 2021
      • Drivers
      • Opportunities
      • Restraints
      • Challenges
  • SWOT Analysis

KEY MARKET INSIGHTS

  • Technology Analysis
    • (Exhibit: Data Table: Name of technology and details)
  • Pricing Analysis
    • (Exhibit: Data Table: Name of technology and pricing details)
  • Supply Chain Analysis
    • (Exhibit: Detailed Supply Chain Presentation)
  • Value Chain Analysis
    • (Exhibit: Detailed Value Chain Presentation)
  • Ecosystem Of the Market
    • Exhibit: Parent Market Ecosystem Market Analysis
    • Exhibit: Market Characteristics of Parent Market
  • IP Analysis
    • (Exhibit: Data Table: Name of product/technology, patents filed, inventor/company name, acquiring firm)
  • Trade Analysis
    • (Exhibit: Data Table: Import and Export data details)
  • Startup Analysis
    • (Exhibit: Data Table: Emerging startups details)
  • Raw Material Analysis
    • (Exhibit: Data Table: Mapping of key raw materials)
  • Innovation Matrix
    • (Exhibit: Positioning Matrix: Mapping of new and existing technologies)
  • Pipeline product Analysis
    • (Exhibit: Data Table: Name of companies and pipeline products, regional mapping)
  • Macroeconomic Indicators

COVID IMPACT

  • Introduction
  • Impact On Economy—scenario Assessment
    • Exhibit: Data on GDP - Year-over-year growth 2016-2022 (%)
  • Revised Market Size
    • Exhibit: Data Table on Global Gene Therapy Market size and forecast 2021-2027 ($ million)
  • Impact Of COVID On Key Segments
    • Exhibit: Data Table on Segment Market size and forecast 2021-2027 ($ million)
  • COVID Strategies By Company
    • Exhibit: Analysis on key strategies adopted by companies

MARKET DYNAMICS & OUTLOOK

  • Market Dynamics
    • Exhibit: Impact analysis of DROC, 2021
      • Drivers
      • Opportunities
      • Restraints
      • Challenges
  • Regulatory Landscape
    • Exhibit: Data Table on regulation from different region
  • SWOT Analysis
  • Porters Analysis
    • Competitive rivalry
      • Exhibit: Competitive rivalry Impact of key factors, 2021
    • Threat of substitute products
      • Exhibit: Threat of Substitute Products Impact of key factors, 2021
    • Bargaining power of buyers
      • Exhibit: buyers bargaining power Impact of key factors, 2021
    • Threat of new entrants
      • Exhibit: Threat of new entrants Impact of key factors, 2021
    • Bargaining power of suppliers
      • Exhibit: Threat of suppliers bargaining power Impact of key factors, 2021
  • Skyquest special insights on future disruptions
    • Political Impact
    • Economic impact
    • Social Impact
    • Technical Impact
    • Environmental Impact
    • Legal Impact

Market Size by Region

  • Chart on Market share by geography 2021-2027 (%)
  • Data Table on Market share by geography 2021-2027(%)
  • North America
    • Chart on Market share by country 2021-2027 (%)
    • Data Table on Market share by country 2021-2027(%)
    • USA
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • Canada
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
  • Europe
    • Chart on Market share by country 2021-2027 (%)
    • Data Table on Market share by country 2021-2027(%)
    • Germany
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • Spain
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • France
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • UK
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • Rest of Europe
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
  • Asia Pacific
    • Chart on Market share by country 2021-2027 (%)
    • Data Table on Market share by country 2021-2027(%)
    • China
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • India
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • Japan
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • South Korea
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • Rest of Asia Pacific
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
  • Latin America
    • Chart on Market share by country 2021-2027 (%)
    • Data Table on Market share by country 2021-2027(%)
    • Brazil
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • Rest of South America
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
  • Middle East & Africa (MEA)
    • Chart on Market share by country 2021-2027 (%)
    • Data Table on Market share by country 2021-2027(%)
    • GCC Countries
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • South Africa
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)
    • Rest of MEA
      • Exhibit: Chart on Market share 2021-2027 (%)
      • Exhibit: Market size and forecast 2021-2027 ($ million)

KEY COMPANY PROFILES

  • Competitive Landscape
    • Total number of companies covered
      • Exhibit: companies covered in the report, 2021
    • Top companies market positioning
      • Exhibit: company positioning matrix, 2021
    • Top companies market Share
      • Exhibit: Pie chart analysis on company market share, 2021(%)

Methodology

For the Global Gene Therapy Market, our research methodology involved a mixture of primary and secondary data sources. Key steps involved in the research process are listed below:

1. Information Procurement: This stage involved the procurement of Market data or related information via primary and secondary sources. The various secondary sources used included various company websites, annual reports, trade databases, and paid databases such as Hoover's, Bloomberg Business, Factiva, and Avention. Our team did 45 primary interactions Globally which included several stakeholders such as manufacturers, customers, key opinion leaders, etc. Overall, information procurement was one of the most extensive stages in our research process.

2. Information Analysis: This step involved triangulation of data through bottom-up and top-down approaches to estimate and validate the total size and future estimate of the Global Gene Therapy Market.

3. Report Formulation: The final step entailed the placement of data points in appropriate Market spaces in an attempt to deduce viable conclusions.

4. Validation & Publishing: Validation is the most important step in the process. Validation & re-validation via an intricately designed process helped us finalize data points to be used for final calculations. The final Market estimates and forecasts were then aligned and sent to our panel of industry experts for validation of data. Once the validation was done the report was sent to our Quality Assurance team to ensure adherence to style guides, consistency & design.

Analyst Support

Customization Options

With the given market data, our dedicated team of analysts can offer you the following customization options are available for the Global Gene Therapy Market:

Product Analysis: Product matrix, which offers a detailed comparison of the product portfolio of companies.

Regional Analysis: Further analysis of the Global Gene Therapy Market for additional countries.

Competitive Analysis: Detailed analysis and profiling of additional Market players & comparative analysis of competitive products.

Go to Market Strategy: Find the high-growth channels to invest your marketing efforts and increase your customer base.

Innovation Mapping: Identify racial solutions and innovation, connected to deep ecosystems of innovators, start-ups, academics, and strategic partners.

Category Intelligence: Customized intelligence that is relevant to their supply Markets will enable them to make smarter sourcing decisions and improve their category management.

Public Company Transcript Analysis: To improve the investment performance by generating new alpha and making better-informed decisions.

Social Media Listening: To analyze the conversations and trends happening not just around your brand, but around your industry as a whole, and use those insights to make better Marketing decisions.

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FAQs

Gene Therapy Market size was valued at USD 6.39 Billion in 2021 and is poised to grow from USD 7.60 Billion in 2022 to USD 38.34 Billion by 2030, at a CAGR of 19% during the forecast period (2023-2030).

The global gene therapy market is highly competitive with a large number of players operating in the industry. The key players in the market are focusing on the development of new and advanced therapies to enhance their market presence. The gene therapy market is highly competitive, with a number of players focusing on the development of novel therapies for the treatment of a range of diseases. The market is expected to continue to grow in the coming years, driven by increasing investment in R&D and growing demand for new and advanced therapies for the treatment of rare and life-threatening diseases. 'Novartis International AG (Switzerland)', 'Pfizer Inc. (US)', 'Biogen Inc. (US)', 'Spark Therapeutics Inc. (US)', 'Sarepta Therapeutics Inc. (US)', 'bluebird bio-Inc. (US)', 'Gilead Sciences Inc. (US)', 'Amgen Inc. (US)', 'Regenxbio Inc. (US)', 'Krystal Biotech Inc. (US)', 'uniQure N.V. (Netherlands)', 'Avexis Inc. (US)', 'Orchard Therapeutics plc (UK)', 'Abeona Therapeutics Inc. (US)', 'Solid Biosciences Inc. (US)', 'Audentes Therapeutics Inc. (US)', 'Adaptimmune Therapeutics plc (UK)', 'Intellia Therapeutics Inc. (US)', 'Editas Medicine Inc. (US)', 'Precision BioSciences Inc. (US)'

Genetic disorders, such as spinal muscular atrophy, cystic fibrosis, and hemophilia, are on the rise worldwide. The increasing prevalence of these disorders is driving the growth of the gene therapy market, as gene therapies have shown promising results in the treatment of these disorders.

Increased focus on rare diseases: Gene therapy is becoming increasingly popular for the treatment of rare genetic disorders, which were previously ignored due to their low prevalence. For example, Zolgensma, a gene therapy for spinal muscular atrophy, was approved by the FDA in 2019 and has since become a popular treatment option. Similarly, Luxturna, a gene therapy for inherited retinal disease, was approved in 2017.

In 2022, North America emerged as the dominant region in the gene therapy market, contributing to over 65.0% of the overall revenue. It is projected to remain the largest routine manufacturer of gene therapy drugs during the forecast period, with increasing investments in R&D by companies of all sizes. The market is being driven by rising government investments and the growing prevalence of targeted diseases in the region, such as spinal muscular atrophy, affecting 10,000 to 25,000 individuals in the U.S. alone, according to the Spinal Muscular Atrophy Foundation.

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