Gene Therapy Market: Current Analysis and Forecast (2023-2030)


Emphasis on Vector {Viral Vector (Retroviral and Adeno-Associated) and Non-Viral Vector (Oligonucleotides)}; Gene Type (Antigen, Cytokine, Receptor, and Others); Indication (Oncology, Rare Genetic Diseases, Cardiovascular, Neurology, and Others); Delivery Method (In-Vivo and Ex-Vivo); and Region/Country.







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Research Methodology

Report Description

Gene Therapy Market
Gene Therapy Market

The Gene Therapy Market was valued at USD 5.66 Billion in the year 2022 and is expected to grow at a strong CAGR of around 30.1% during the forecast period (2023-2030) owing to the growing gene therapy pipeline. The surging gene therapy pipeline across the globe is one of the major factors which is contributing to the growth of the gene therapy market. As per a study by PhRMA there were around 289 cell and gene therapies for clinical development by biopharmaceutical companies in the United States in Jan –Feb. 2020 moreover the number of novel cell and gene therapies range from early to late stages of clinical development increased to 362 , and till March 2020. Furthermore, it was reveled that the gene therapy market across the globe has a strong pipeline with USD 12 Billion opportunities by 2030. Also, some of these innovative medicines have already been approved by the U.S. Food and Drug Administration (FDA) that are beneficial for the patients. In addition, there are nine-cell or gene therapy products approved in the U.S. – treating cancer, eye diseases, and rare hereditary diseases as of February 2020. So, gene therapy pipeline would drive the market in the coming years as well 

Some of the major players operating in the market include Biogen; BioMarin; bluebird bio, Inc.; Bristol-Myers Squibb Company; Gilead Sciences, Inc.; Novartis AG; Orchard Therapeutics plc; REGENXBIO INC.; Spark Therapeutics, Inc.; and uniQure NV. Several M&As along with partnerships have been undertaken by these players to facilitate customers with hi-tech and innovative products/technologies.

Insights Presented in the Report

“Amongst vector, the smart appliances segment held a dominating share of the market in 2022”

Based on on vector, the market is segmented into viral vector (retroviral and adeno-associated) and non-viral vector (oligonucleotides). The viral vector held dominant share of the market in 2022. Lentiviral vectors, derived from the human immunodeficiency virus, have been extensively investigated and optimized over the past two decades. Third-generation, self-inactivating lentiviral vectors have recently been used in multiple clinical trials to introduce genes into hematopoietic stem cells to correct primary immunodeficiencies and hemoglobinopathies. CAR T-cell therapies engineered using lentiviral vectors have demonstrated noteworthy clinical success in patients with B-cell malignancies leading to regulatory approval of the first genetically engineered cellular therapy using lentiviral vectors.

“Amongst gene type, the tumor supressor gene therapy technology held the majority share of the market in 2022”

By gene type the market is categorized into antigen, cytokine, receptor, and others. The tumor suppressor gene therapy held majority share of the market in 2022. Tumor-suppressor genes play pivotal roles in maintaining genome integrity and in regulating cell proliferation, differentiation, and apoptosis. Their loss-of-function mutations are related directly to tumorigenesis. Thus, use of tumor-suppressor genes as anticancer therapeutics has been investigated rigorously in both experimental and clinical researches. Clinical trials with tumor-suppressor genes, especially the p53 gene, have demonstrated that the treatment is well tolerated, and; favorable clinical responses, including a pathologically complete responses, have been observed in a subset of patients with advanced disease or with cancers resistant to conventional therapy.

“Amongst delivery method, the ex vivo segment will grow with high CAGR during the forecast period”

By delivery method, the market is bifurcated into in vivo and ex vivo. The ex vivo segment is anticipated to grow with high CAGR during the forecast period. The expression of the gene and health of the transfected cells can be verified before it is introduced to the patient via ex vivo delivery. This method can be useful for therapies where compounds should be released or expressed by specific cell types that can be selected on the culture dish before transplant. In ex vivo cells are modified outside the patient’s body and the corrected version is transplanted back into the patient. The cells are treated with either a viral or non viral gene therapy vector carrying the corrected copy of the gene.

“North America dominated the gene therapy market in 2022”

North America dominated the Gene Therapy market and is anticipated to have the maximum revenue owing to the robust regulatory agenda for promoting the development of cellular therapy and the presence of a substantial number of biopharma companies, several government agencies are investing in the region. While hundreds of potential cell and gene therapies are being developed, a few of these cutting-edge medicines have already been approved by the US Food and Drug Administration (FDA) and are currently helping patients. As of February 2020, nine cell or gene therapy drugs for the treatment of cancer, eye defects, and rare genetic diseases have been approved in the United States. Currently, more than 100 diseases are being explored for the potential treatment with cell and gene therapies. Cellular and gene therapy-related research and development in the United States continue to grow at a fast rate, with several products advancing in clinical development

Gene Therapy Market Report Coverage

Gene Therapy Market
Gene Therapy Market

Reasons to buy this report:

  • The study includes market sizing and forecasting analysis validated by authenticated key industry experts.
  • The report presents a quick review of overall industry performance at one glance.
  • The report covers an in-depth analysis of prominent industry peers with a primary focus on key business financials, product portfolios, expansion strategies, and recent developments.
  • Detailed examination of drivers, restraints, key trends, and opportunities prevailing in the industry.
  • The study comprehensively covers the market across different segments.
  • Deep dive regional level analysis of the industry.

Customization Options:

The global gene therapy market can further be customized as per the requirement or any other market segment. Besides this, UMI understands that you may have your own business needs, hence feel free to contact us to get a report that completely suits your requirements.

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1.1.Market Definitions
1.2.Main Objective
2.1.       Research Process of the Gene Therapy Market
2.2.       Research Methodology of the Gene Therapy Market
2.3.       Respondent Profile 






7.1.    Viral Vector 
7.1.1.       Retroviral
7.1.2.       Adeno-Associated
7.2.    Non-Viral Vector 
7.2.1.       Oligonucleotides


8.1.    Antigen
8.2.    Cytokine
8.3.    Receptor
8.4.    Others 


9.1.    Oncology
9.2.    Rare Genetic Diseases
9.3.    Cardiovascular
9.4.    Neurology
9.5.    Others 


10.1.  In-Vivo
10.2.  Ex-Vivo


11.1.   North America    
11.1.1.    U.S.  
11.1.2.    Canada  
11.1.3.    Rest of North America
11.2.   Europe   
11.2.1.    Germany  
11.2.2.    U.K.  
11.2.3.    France  
11.2.4.    Italy  
11.2.5.    Spain  
11.2.6.    Rest of Europe 
11.3.   Asia-Pacific   
11.3.1.    China  
11.3.2.    Japan  
11.3.3.    India  
11.3.4.    Rest of Asia-Pacific
11.4.   Rest of World   


12.1.      Market Drivers 
12.2.      Market Challenges
12.3.      Impact Analysis 






15.1.   Demand Side Analysis
15.2.   Supply Side Analysis






18.1.      LUXTURNA 
18.2.      KYMRIAH (tisagenlecleucel)
18.3.      TECARTUS (brexucabtagene autoleucel)
18.4.      YESCARTA (axicabtagene ciloleucel)




21.1.      Biogen 
21.2.      BioMarin 
21.3.      bluebird bio, Inc. 
21.4.      Bristol-Myers Squibb Company
21.5.      Gilead Sciences, Inc.
21.6.      Novartis AG 
21.7.      Orchard Therapeutics plc
21.8.      REGENXBIO INC. 
21.9.      Spark Therapeutics, Inc.
21.10.   uniQure NV 

Research Methodology

Research Methodology for the Gene Therapy Market Analysis (2022-2030)

Analyzing the historical market, estimating the current market, and forecasting the future market of the global gene therapy market were the three major steps undertaken to create and analyze the adoption of gene therapys in major regions globally. Exhaustive secondary research was conducted to collect the historical market numbers and estimate the current market size. Secondly, to validate these insights, numerous findings and assumptions were taken into consideration. Moreover, exhaustive primary interviews were also conducted, with industry experts across the value chain of the global gene therapy market. Post assumption and validation of market numbers through primary interviews, we employed a top-down/bottom-up approach to forecasting the complete market size. Thereafter, market breakdown and data triangulation methods were adopted to estimate and analyze the market size of segments and sub-segments of the industry pertains to. Detailed methodology is explained below:

Analysis of Historical Market Size

Step 1: In-Depth Study of Secondary Sources:

Detail secondary study was conducted to obtain the historical market size of the gene therapy market through company internal sources such as annual reports & financial statements, performance presentations, press releases, etc., and external sources including journals, news & articles, government publications, competitor publications, sector reports, third-party database, and other credible publications.

Step 2: Market Segmentation:

After obtaining the historical market size of the gene therapy market, we conducted a detailed secondary analysis to gather historical market insights and share for different segments & sub-segments for major regions. Major segments are included in the report as vector, gene type, delivery method, and indication. Further country-level analyses were conducted to evaluate the overall adoption of testing models in that region.

Step 3: Factor Analysis:

After acquiring the historical market size of different segments and sub-segments, we conducted a detailed factor analysis to estimate the current market size of the gene therapy market. Further, we conducted factor analysis using dependent and independent variables such as vector, gene type, delivery method, and indication of the gene therapy market. A thorough analysis was conducted for demand and supply-side scenarios considering top partnerships, mergers and acquisitions, business expansion, and product launches in the gene therapy market sector across the globe.

Current Market Size Estimate & Forecast

Current Market Sizing: Based on actionable insights from the above 3 steps, we arrived at the current market size, key players in the global gene therapy market, and market shares of the segments. All the required percentage shares split, and market breakdowns were determined using the above-mentioned secondary approach and were verified through primary interviews.

Estimation & Forecasting: For market estimation and forecast, weights were assigned to different factors including drivers & trends, restraints, and opportunities available for the stakeholders. After analyzing these factors, relevant forecasting techniques i.e., the top-down/bottom-up approach were applied to arrive at the market forecast for 2030 for different segments and sub-segments across the major markets globally. The research methodology adopted to estimate the market size encompasses:

  • The industry’s market size, in terms of revenue (USD) and the adoption rate of the gene therapy market across the major markets domestically
  • All percentage shares, splits, and breakdowns of market segments and sub-segments
  • Key players in the global gene therapy market in terms of products offered. Also, the growth strategies adopted by these players to compete in the fast-growing market

Market Size and Share Validation

Primary Research: In-depth interviews were conducted with the Key Opinion Leaders (KOLs) including Top Level Executives (CXO/VPs, Sales Head, Marketing Head, Operational Head, Regional Head, Country Head, etc.) across major regions. Primary research findings were then summarized, and statistical analysis was performed to prove the stated hypothesis. Inputs from primary research were consolidated with secondary findings, hence turning information into actionable insights.

Split of Primary Participants in Different Regions

Gene Therapy Market
Gene Therapy Market

Market Engineering

The data triangulation technique was employed to complete the overall market estimation and to arrive at precise statistical numbers for each segment and sub-segment of the global gene therapy market. data was split into several segments & sub-segments post studying various parameters and trends in the areas of the product, technology, and end-users in the global gene therapy market.

The main objective of the Global Gene Therapy Market Study

The current & future market trends of the global gene therapy market were pinpointed in the study. Investors can gain strategic insights to base their discretion for investments on the qualitative and quantitative analysis performed in the study. Current and future market trends determined the overall attractiveness of the market at a regional level, providing a platform for the industrial participant to exploit the untapped market to benefit from a first-mover advantage. Other quantitative goals of the studies include:

  • Analyze the current and forecast market size of the gene therapy market in terms of value (USD). Also, analyze the current and forecast market size of different segments and sub-segments
  • Segments in the study include areas of the vector, gene type, delivery method, and indication
  • Define and analysis of the regulatory framework for the gene therapy industry
  • Analyze the value chain involved with the presence of various intermediaries, along with analyzing customer and competitor behaviors of the industry
  • Analyze the current and forecast market size of the gene therapy market for the major region
  • Major countries of regions studied in the report include Asia Pacific, Europe, North America, and the Rest of the World
  • Company profiles of the gene therapy market and the growth strategies adopted by the market players to sustain in the fast-growing market
  • Deep dive regional level analysis of the industry

You can also purchase parts of this report. Do you want to check out a section wise
price list?