Gene Therapy Market Research Report Information by Vector Type (Viral Vector (Lentiviral Vectors (LVVs), Retrovirus Vectors, Adenoviral Vectors (AdVs), Adeno-associated Viral Vectors (AAVs)) and Non-Viral Vector (Physical Vector, Chemical Vector)), by Gene Type (Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth Factors, Receptors, and Others), by Application (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Diseases, and Others), by Delivery Method (In Vivo Gene Therapy and Ex

Gene Therapy Market Overview

The Gene Therapy Market was valued at USD 6.11 Billion in 2024. The Gene Therapy Market industry is projected to grow from USD 7.24 Billion in 2025 to USD 46.91 Billion by 2035, exhibiting a compound annual growth rate (CAGR) of 19.04% during the forecast period (2025-2035).

The growing incidences and prevalence of cancer and rare disorders and increasing approval for gene therapy products and favourable government support for gene therapy are driving the growth of the Gene Therapy Market.

As per the Analyst at MRFR, the growing incidences of cancer and rare disorders are significantly fueling the Gene Therapy Market as the demand for innovative treatments escalates. As cancer rates rise and the prevalence of rare genetic disorders increases, traditional therapeutic options often fall short, creating a pressing need for advanced solutions. Gene therapy, with its potential to address the underlying genetic causes of these conditions, offers a promising alternative by targeting and modifying the specific genes involved. 

For instance, according to the International Agency for Research on Cancer, there were cancer cases of nearly 9,826,539 in Asia, 4,471,422 in Europe, 2,673 174 in Northern America, 1,551,060 in Latin America and the Caribbean, 1,185,216 in Africa, and 269 088 in Oceania cancer cases of both genders in 2022. Furthermore, India also reported increasing incidences of cancer, which is contributing to the growth of the global gene therapy market.

 Source: Secondary Research, Primary Research, MRFR Database, and Analyst Review

Gene Therapy Market Opportunity

GROWING INVESTMENT IN THE FIELD OF GENE THERAPY

The increasing investment in gene therapy is one of the major opportunities across the globe. The growing investment leads to an increase in the availability of technological advancements, including CRISPR-Cas9. Also, the investment assists in developing gene therapy with a focus on broad therapeutic areas. Furthermore, rising funding also assists in expanding the market, which fosters collaboration, competition, innovation, and investment. For instance,

• June 2024: Beacon Therapeutics (US) has secured $170 million in Series B funding to propel its work on ophthalmic gene therapies. This round was led by Forbion, with participation from existing investors Syncona and Oxford Science Enterprises, along with new contributors TCGX and Advent Life Sciences. The funds are earmarked to advance the clinical development of their lead asset, AGTC-501, which is targeting X-Linked Retinitis Pigmentosa (XLRP), and to generate data for their Dry Age-related Macular Degeneration (dAMD) program.


• February 2024: Vivet Therapeutics received USD 5.35 million in funding from the French government through the France Health Innovation Plan 2030, which will significantly boost the development of a gene therapy aimed at treating this rare and debilitating condition. CTX is a rare genetic disorder that impairs the body’s ability to metabolize cholesterol, leading to the accumulation of fatty deposits in various tissues, including the brain. This results in a range of serious symptoms, from chronic diarrhea in infancy to progressive neurological issues like dementia and seizures in adulthood. Currently, treatment options are limited to managing symptoms and slowing the progression of the disease.


• January 2024: Novartis AG (Switzerland) signed a deal with Voyager Therapeutics (US) in order to pay USD 100 million upfront as a part of a licensing deal to develop gene therapy candidates for genetic disorders.


• June 2023: Laurus Labs (India) invested USD 9.59 million for gene therapy firm ImmunoACT (India). ImmunoACT will be able to accelerate the scale-up of its lead candidate, HCAR-19, by expanding its multi-location cGMP facilities with the new funding.

The data show a global increase in investment and collaboration in the gene therapy sector. Companies such as Beacon Therapeutics, Vivet Therapeutics, Novartis AG, and Laurus Labs are strategically collaborating and investing in R&D and manufacturing facilities to accelerate the commercialization and scaling-up of innovative gene therapies.

Gene Therapy Market Segment Insights

Gene Therapy System by Vector Type Insights

Based on Vector Type, this segment includes Viral Vector (Lentiviral Vectors (LVVs), Retrovirus Vectors, Adenoviral Vectors (AdVs) and Adeno-associated Viral Vectors (AAVs)) and Non-Viral Vector (Physical Vector, Chemical Vector). The Viral Vector segment dominated the global market in 2024, while the Non-Viral Vector is projected to be the fastest–growing segment during the forecast period. Among viral vectors, adeno-associated viruses (AAVs) and lentiviral vectors (LVVs) are widely used for their safety profiles and long-term gene expression. 

Non-viral vectors, including physical methods (like electroporation) and chemical carriers (like lipid nanoparticles), offer lower immunogenicity and easier large-scale manufacturing. The success of lipid nanoparticle-based mRNA vaccines during the COVID-19 pandemic has accelerated interest and innovation in non-viral delivery systems. Additionally, advancements in nanotechnology and material sciences are enhancing the efficiency and targeting capability of non-viral vectors, making them an attractive option for next-generation gene therapies and expanding their potential across various therapeutic areas.

FIGURE 2: GENE THERAPY MARKET SHARE BY VECTOR TYPE 2024 AND 2035 (USD BILLION)

Source: Secondary Research, Primary Research, MRFR Database and Analyst Review

Gene Therapy System by Gene Type Insights

Based on Gene Type, this segment includes Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth Factors, Receptors, and Others. The Antigen segment dominated the global market in 2024, while it is projected to be the fastest–growing segment during the forecast period. These therapies have shown remarkable clinical success and have received regulatory approvals, boosting market confidence. Additionally, antigen-based approaches are expanding into other areas such as infectious diseases and autoimmune disorders, supported by advancements in gene delivery technologies and personalized medicine. 

The segment benefits from a robust pipeline, significant investment, and favorable regulatory designations like orphan drug and breakthrough therapy status, which accelerate development and commercialization. Overall, the antigen segment's ability to deliver targeted, effective, and personalized treatments positions it as a key driver of innovation and growth in the gene therapy landscape.

Gene Therapy System by Application Insights

Based on Application, this segment includes Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Diseases, and Others. The Oncological Disorders segment dominated the global market in 2024, while it is projected to be the fastest–growing segment during the forecast period. This is primarily due to the rising global cancer burden and the increasing adoption of gene therapies like CAR-T cell therapy and oncolytic viruses, which offer targeted and effective treatment options. 

Advances in personalized medicine and immune-based gene therapies have shown significant success in treating hematologic and solid tumors, driving demand. Additionally, strong clinical pipelines, regulatory approvals, and substantial investments in cancer gene therapy research are fueling growth. The high unmet need for more effective cancer treatments and the ability of gene therapy to address previously untreatable cancers further strengthen this segment’s market position.

Gene Therapy System by Delivery Method Insights

Based on the Delivery Method, this segment includes In Vivo Gene Therapy and Ex Vivo Gene Therapy. The In Vivo Gene Therapy segment dominated the global market in 2024, while it is projected to be the fastest–growing segment during the forecast period. This method involves directly delivering genetic material into a patient’s body, allowing for simpler, less invasive procedures compared to Ex Vivo approaches. In Vivo therapies are particularly effective for treating genetic and rare diseases, especially those affecting the liver, muscles, and central nervous system. 

The rise of adeno-associated viral (AAV) vectors and lipid nanoparticles has improved the safety and precision of in vivo delivery. Additionally, reduced complexity, lower treatment costs, and faster clinical translation are driving increased adoption. Growing research investments and successful clinical trials continue to accelerate development in this segment.

Gene Therapy System Regional Insights

Based on the Region, the global Gene Therapy is segmented into North America, Europe, Asia-Pacific and Rest of the World. Major demand factors driving the North America market are the growing incidences and prevalence of cancer and rare disorders and increasing approval for gene therapy products and favourable government support for gene therapy. Additionally, the availability of reimbursements and insurance coverage is making gene therapies more accessible to patients. Increased healthcare expenditure and investment in research and development are further fueling market expansion. 

Supportive regulatory frameworks and incentives are streamlining the approval process, while strategic partnerships and collaborations are accelerating innovation. The pipeline of gene therapy drugs includes a wide range of treatments for various genetic and acquired diseases, such as rare genetic disorders, cancer, and autoimmune diseases. The potential of these drugs to provide targeted and personalized treatments for patients with complex medical conditions has generated significant interest from biopharmaceutical companies, investors, and healthcare providers. The competitive landscape features established players who have adopted strategies such as acquisition, agreement, strategic alliance, collaboration, clinical trial, expansion, joint venture, new product development, and product approval to expand their product portfolio.

FIGURE 3: GENE THERAPY MARKET VALUE BY REGION 2024 AND 2035 (USD BILLION)

Source: Secondary Research, Primary Research, MRFR Database, and Analyst Review

Further, the countries considered in the scope of the Application Tracking System Market are the US, Canada, Germany, France, the UK, Italy, Spain, China, India, Japan, South Korea, Australia, Middle East & Africa, South America and others.

Global Gene Therapy Key Market Players & Competitive Insights

Many global, regional, and local vendors characterize the Gene Therapy Market. The market is highly competitive, with all the players competing to gain market share. Intense competition, rapid advances in technology, frequent changes in government policies, and environmental regulations are key factors that confront market growth. The vendors compete based on cost, product quality, reliability, and government regulations. Vendors must provide cost-efficient, high-quality products to survive and succeed in an intensely competitive market.

The major competitors in the market are Bristol-Myers Squibb Company, Lonza, UniQure N.V., Bluebird Bio, Inc., Orchard Therapeutics Plc, GenSight Biologics, Novartis AG, Amgen Inc., Sarepta Therapeutics Inc., Spark Therapeutics, Inc., are among others. The Gene Therapy Market is a consolidated market due to increasing competition, acquisitions, mergers and other strategic market developments and decisions to improve operational effectiveness.

Key Companies in the Gene Therapy Market include

• Bristol-Myers Squibb Company


• Lonza


• UniQure N.V.


• Bluebird Bio, Inc.


• Orchard Therapeutics Plc


• GenSight Biologics


• Novartis AG


• Amgen Inc.


• Sarepta Therapeutics Inc.


• Spark Therapeutics, Inc.,

Gene Therapy Market Industry Developments

January 2024: Novartis AG has entered into a strategic partnership with Voyager Therapeutics, Inc., which includes an exclusive license for Voyager's TRACER capsids and related intellectual property. This collaboration will focus on developing a preclinical gene therapy candidate for Huntington's Disease (HD).

October 2023: SpliceBio, a genetic medicines company specializing in Protein Splicing to advance gene therapies, has announced an exclusive collaboration and licensing agreement with Spark Therapeutics. This partnership will leverage SpliceBio's proprietary Protein Splicing platform to develop a gene therapy for an undisclosed inherited retinal disease.

December 2023: Bluebird bio, Inc. (US) received an approval from US Food Drug and Administration for Lyfgenia representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older.

September 2021: GenSight Biologics announced that the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) has designated its gene therapy LUMEVOQ as a Promising Innovative Medicine (PIM) for the treatment of vision loss caused by Leber Hereditary Optic Neuropathy (LHON), which is caused by a confirmed G11778A mutation in the ND4 mitochondrial gene.

Gene Therapy Market Segmentation

Gene Therapy by Vector Type Outlook

• Viral Vector
  
  
  
  • Lentiviral Vectors (LVVs)
  
  
  • Retrovirus Vectors
  
  
  • Adenoviral Vectors (AdVs)
  
  
  • Adeno-associated Viral Vectors (AAVs)
  
  
  
  


• Non-Viral Vector
  
  
  
  • Physical Vector
  
  
  • Chemical Vector
  
  
  
  

Gene Therapy by Gene Type Outlook

• Antigen


• Cytokine


• Tumor Suppressor


• Suicide


• Deficiency


• Growth Factors


• Receptors


• Others

Gene Therapy by Application Outlook

• Oncological Disorders


• Rare Diseases


• Cardiovascular Diseases


• Neurological Disorders


• Infectious Diseas


• Others

Gene Therapy by Delivery Method Outlook

• In Vivo Gene Therapy


• Ex Vivo Gene Therapy

Gene Therapy Regional Outlook

• North America
  
  
  
  • US
  
  
  • Canada
  
  
  
  


• Europe
  
  
  
  • Germany
  
  
  • France
  
  
  • UK
  
  
  • Italy
  
  
  • Spain
  
  
  • Rest of Europe
  
  
  
  


• Asia-Pacific
  
  
  
  • China
  
  
  • India
  
  
  • Japan
  
  
  • South Korea
  
  
  • Australia
  
  
  • Rest of Asia-Pacific
  
  
  
  


• Rest of the world
  
  
  
  • Middle East & Africa
  
  
  • South America
  
  
  
  

Report Attribute/Metric	Details
Market Size 2024	USD 6.11 Billion
Market Size 2025	USD 7.24 Billion
Market Size 2035	USD 46.91 Billion
Compound Annual Growth Rate (CAGR)	19.04 % (2025-2035)
Base Year	2024
Forecast Period	2025-2035
Historical Data	2019-2023
Forecast Units	Value (USD Billion)
Report Coverage	Revenue Forecast, Competitive Landscape, Growth Factors, and Trends
Segments Covered	By Vector Type, By Gene Type, By Application, By Delivery Method
Geographies Covered	North America, Europe, Asia Pacific, Rest of the World
Countries Covered	The US, Canada, Germany, France, the UK, Italy, Spain, China, India, Japan, South Korea, Australia, Middle East & Africa, South America
Key Companies Profiled	Bristol-Myers Squibb Company, Lonza, UniQure N.V., Bluebird Bio, Inc., Orchard Therapeutics Plc, GenSight Biologics, Novartis AG, Amgen Inc., Sarepta Therapeutics Inc., Spark Therapeutics, Inc.
Key Market Opportunities	·         Growing investment in the field of gene therapy ·         Ongoing clinical trials associated with gene therapy
Key Market Dynamics	·         Growing incidences and prevalence of cancer and rare disorders ·         Increasing approval for gene therapy products ·         Favourable government support for gene therapy