According to Precedence Research, the global gene therapy market size is evaluated at USD 11.07 billion in 2025 and is projected to exceed around USD 55.43 billion by 2034, growing at a CAGR of 19.60% from 2025 to 2034.
In terms of revenue, the global gene therapy market was valued at USD 9.26 billion in 2024, grew to USD 11.07 billion in 2025 and is expected to reach over USD 55.43 billion by 2034. The global cell and gene therapy market was calculated at USD 21.28 billion in 2024 and is anticipated to reach around USD 117.46 billion by 2034.
In terms of CAGR, the global gene therapy market is poised to grow at a 19.60% of CAGR between 2024 and 2034, On the other hand the global cell and gene therapy market is expanding at a CAGR of 18.6% from 2024 to 2034.
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The rising advancement in the healthcare infrastructure, ongoing clinical trials, increasing demand for innovative treatments, increasing number of approvals of gene therapies, increasing prevalence of genetic diseases, and are expected to boost the growth of the gene therapy market during the forecast period.
Gene Therapy Market Highlights:
🔹 North America dominated the global market by holding 54% of market share in 2024.
🔹Asia Pacific is expected to show the fastest growth during the projected period.
🔹The gene silencing segment led the market in 2024.
🔹The viral vector segment dominated the market in 2024.
🔹The oncology segment contributed the highest market share in 2024.
🔹The in vivo segment captured the largest market share in 2024.
🔹The intravenous segment contributed the biggest market share in 2024.
Market Overview and Industry Potential
In recent years, gene therapy has been experiencing a breakthrough in targeting specific cell groups across numerous therapeutic areas. Gene therapy is a widely used technique that uses genes to prevent, treat, and cure a disease. The innovative approach of gene therapy involves adding new copies of a broken gene or replacing a defective or missing gene in a patient's cells with a healthy version. Both inherited genetic diseases such as hemophilia and sickle cell disease and acquired disorders such as leukemia can be treated with gene therapy effectively. In recent years, the pipeline of gene therapies that utilize advanced genetic engineering tools has significantly increased.
By leveraging the power of genetic engineering, gene therapies intend to correct or replace faulty genes within an individual's cells, offering the potential of one-dose cures for several chronic and genetic disorders. Several biotechnology companies are increasingly investing in acquisitions, mergers or collaborations, and deals as a prominent strategy to enhance in-house expertise and strengthen product pipelines. The increasing demand for personalized treatments, rising investment by the market players in gene therapies, supportive government framework, rising R&D funding by government and private organizations, and advancements in gene editing technologies, boost the market expansion in the coming years.
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How does AI integration impact on gene therapies?
With the continuous advancement of technology, the integration of Artificial Intelligence with genome editing can transform genetics, biomedicine, and healthcare. This could represent new opportunities for rapid advancements in human health through enhanced data analysis and tailored treatments. AI is widely adopted to streamline several aspects of gene therapy to enhance treatment development, optimization, and effective implementation. AI integration holds the promising potential to offer transformative advancements in several pre-clinical and clinical gene therapy development stages.
The major applications of AI in gene therapy development include biomarker prediction, viral vector and biomanufacturing optimization, personalized medicine, target identification and validation, clinical predictions, optimizing viral vector for manufacturing, viral vector design optimization, and drug discovery and development. The combined power of AI and gene therapy enhances patient selection, improves monitoring of therapeutic outcomes, and supports adaptive treatment protocols.
🔹In February 2025, ExpressionEdits announced a strategic partnership and licensing deal with Boehringer Ingelheim, under which the companies will work on developing two novel gene therapies. As part of the collaboration, Boehringer will harness ExpressionEdits' AI-powered Genetic Syntax Engine to enhance therapeutic gene expression by restoring introns.
Major Trends in the Gene Therapy Market
Rising Research Funding
The increasing research funding in gene therapy is projected to create immense growth opportunities for the gene therapy market during the forecast period. The increasing incidence of chronic illnesses, including cancer and rare genetic disorders is propelling demand for innovative treatments, which has increased investments in genomics and molecular biology research for groundbreaking discoveries and applications. In addition, clinical trials for gene therapy increased significantly over the years.
Clinical tests play a crucial role in medical research, and they assist the biopharmaceutical and pharmaceutical industries to develop and bring innovative cell and gene treatments to the market. The rising need for innovative medications to cater to unmet medical requirements has substantially increased the number of clinical trials that have been conducted during the past several years.
For instance, In February 2025, Ocugen, Inc., a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, announced that dosing is complete, ahead of schedule in the Phase 2 portion of the Phase 1/2 ArMaDa clinical trial for OCU410—a novel multifunctional modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). Age-related macular degeneration (AMD) affects 1 in 8 people 60 years and older. The global prevalence of dAMD is 266 million worldwide and by 2050 more than 5 million Americans may suffer from this incurable condition. Today, GA – the later stage of dAMD – affects approximately 2-3 million people in the United States (U.S.) and Europe.
According to the report from the IQVIA Institute reflects on the current state of the end-to-end environment for cell and gene therapies on March 2024:
🔹As of the end of 2023, 76 cell and gene therapies have been launched globally, more than double the number of therapies that had been launched by 2013.
🔹Spending on cell and gene therapies is increasing rapidly and reached $5.9Bn in 2023, up 38% from 2022 although spending accounted for only 0.4% of the $1.6Tn spent on medicines globally in 2023.
Rising partnership and acquisition among organizations
The increasing partnership and acquisition among organizations is expected to accelerate the growth of the gene therapy market during the forecast period. Several prominent organizations are actively engaged in forming strategic partnerships to develop advanced gene therapy products for detecting genetic disorders and unlocking new possibilities in gene therapy. The collaboration seeks to accelerate the development of gene therapy, contributing to improved personalized treatment, enhanced manufacturing efficiency, and cost reduction in the production of gene therapy-based therapeutics. These partnerships or collaborations create opportunities for mutual growth and gain a competitive edge in the market.
For instance, In October 2024, Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to enable in vivo gene delivery, announced its research collaboration with Roche to develop next-generation adeno-associated virus (AAV) vectors for gene therapies targeting neurological diseases.
Gene Therapy Market Report Scope
|
Report Attribute |
Key Statistics |
|
Market Size in 2024 |
USD 9.26 Billion |
|
Market Size by 2034 |
USD 55.43 Billion |
|
CAGR 2025 to 2034 |
19.60% |
|
Dominating Region |
North America |
|
Fastest Growing Region |
Asia Pacific |
|
Base Year |
2024 |
|
Historic Years |
2020 to 2023 |
|
Forecast Years |
2024 to 2034 |
|
Segments Covered |
Type, Vector, Therapeutic Area, Delivery Method, Route of Administration and Regions |
|
Regions Covered |
North America, Europe, Asia-Pacific, Latin America and Middle East & Africa |
Gene Therapy Market Regional Outlook: U.S. Gene Therapy Market Size to Achieve USD 22.08 Bn by 2034 The
U.S. gene therapy market size was estimated at USD 3.63 billion in 2024 and is
predicted to achieve around USD 22.08 billion by 2034, expanding at a CAGR of
19.79% from 2025 to 2034. North America held the dominant share
of the gene
therapy market
in 2024. The region is observed to witness notable growth during the forecast
period. The
growth of the region is attributed to the presence of sophisticated healthcare
infrastructure, the surge in research activities, rising healthcare
expenditure, and the increasing prevalence of chronic and genetic diseases. This region is the home to numerous
pharmaceutical and biotech companies involved in the process of developing and
commercializing cell and gene therapies.
The region has witnessed an early adopter of several advanced medical
therapies, including gene therapies. Prominent universities, research
institutions, and medical centers in the region are aggressively working to be
at the forefront of gene therapy research and discoveries. The presence of a
strong pipeline of numerous companies to bring innovative therapies to the
market along with the rising number of approvals of gene therapies within North
America is some of the of the major contributors to the gene therapy market
growth. Additionally, the rapid growth of the market is attributed to the surge
in the number of gene therapy clinical trials conducted across the U.S.
The
approval pipeline for cell and gene therapy (CGT) continues to grow. Per Evaluate, CGT
made up 10% of all U.S. FDA novel approvals in 2023, up from 7% and 6% in 2022
and 2021, respectively. With the rise in approvals, such as the first
CRISPR-based therapeutic approved for marketing in the U.S., In December 2023,
The U.S. Food and Drug Administration approved two milestone treatments,
Casgevy and Lyfgenia, representing the first cell-based gene therapies for the
treatment of sickle cell disease (SCD) in patients 12 years and older.
Additionally, one of these therapies, Casgevy, is the first FDA-approved
treatment to utilize a type of novel genome editing technology, signaling an
innovative advancement in the field of gene therapy. Sickle cell disease is a
group of inherited blood disorders affecting approximately 100,000 people in
the U.S.
The gene therapy development pipeline is expanding rapidly, with numerous new
therapies being developed for a broad range of diseases. In 2023, the Food and
Drug Administration (FDA) approved several major gene therapies, including
treatments for sickle cell disease, Duchenne muscular dystrophy, and type 1
diabetes. As of March 18, 2024, there are now 36 gene therapies approved
by the FDA, with an additional 500 in the pipeline and the expectation that
10–20 will be approved annually by 2025.
On the other hand, Europe is observed
to expand rapidly during the forecast period owing to the presence of advanced
healthcare infrastructure, rising clinical trials, an increasing number of
approvals of gene therapies by gene therapy is regulated by the European
Medicines Agency (EMA), rapid technological improvements, and increasing demand
for innovative gene therapies for treating genetic disorders such as cystic
fibrosis, hemophilia, sickle cell disease, Leber congenital amaurosis and
increasingly prevalent diseases including cancer, heart disease, diabetes,
AIDS, and others. The market is also expected to witness significant growth during the
forecast period, owing to the increased investment by several prominent industry players and governments, including
the NHS & research laboratories.
🔹In January 2025, Interius
BioTherapeutics, a clinical-stage company engineering targeted, programmable
vectors for the precision delivery of genetic medicines, announced that the
German regulatory agency, the Paul Ehrlich Institute (PEI), has approved the expansion
of the INVISE first-in-human Phase 1 clinical trial evaluating INT2104 for the
treatment of B-cell malignancies to Europe. INVISE is the first clinical trial
to announce clearance to evaluate in vivo CAR gene therapy in Europe. Gene Therapy Market Segmentation: Type Insights The
gene silencing segment dominated the market with the largest share in 2024.
Gene silencing involves the transfer of genetic material that prevents the
activity of an existing gene in a cell. This technique reduces the amount of a
specific protein the cell is making by targeting messenger RNA (mRNA). Gene
silencing is an advanced technique utilized in controlling the disease,
studying genes, and developing innovative drugs. This targeted approach is
widely adopted to treat a broad range of diseases, including genetic disorders,
cancers, and viral infections. Vector Insights: The viral vector segment held the largest segment of the gene therapy
market in 2024, the segment is expected to sustain the position throughout
the forecast period. Viral vectors are the most effective means of gene transfer.
Viral vectors are widely being used for a broad spectrum of gene therapy for
both chronic and acute diseases. Viral vectors have shown various
promising results for treating chronic diseases including severe combined
immunodeficiency (SCID), hemophilia, muscular dystrophy, β-thalassemia, and
sickle cell disease (SCD). In the context of cancer, viral vectors are used to
carry genes that can change cancer cells back to normal cells or to replace a
gene that is not working effectively with a working copy of the gene. In cancer
vaccines, viral vectors deliver specific cancer-related antigens to the body.
Viral vectors have also been used in vaccines to help prevent specific viral
infections such as adenoviruses, adeno-associated viruses, retroviruses,
lentiviruses, and herpes simplex viruses. Thus, driving the growth of the
segment in the coming years. On the other hand, the
non-viral vector segment is expected to grow significantly during the forecast
period
owing to the rising availability of non-viral vector gene treatments. Recently, researchers
have increasingly focused on non-viral vectors such as polymers, inorganic
particles, lipids, or combinations of these different types. In comparison with
a viral vector, non-viral vectors have lower cytotoxicity, mutagenesis, and
immunogenicity, making them an attractive option for researchers to explore
promising delivery system advancements in the gene therapy field. Thereby,
bolstering the segment’s expansion. Therapeutic Area Insights: The oncology segment accounted for the
dominating segment in the year 2024 owing to the rising incidence of cancer
around the world. In recent years, Cancer Gene Therapy has played an integral
role in advancing the next generation of cancer treatment. Gene therapy is an
innovative and potentially curative approach to treating cancer patients. Gene
therapy aims to control the altered genes or genetic
mutations associated with cancer to inhibit its growth. Gene therapy
involves the process of replacing a defective gene with a functional, healthy
copy of that gene. This approach may offer a potentially beneficial treatment
for cancer, particularly when compared to chemotherapy, which often lacks
selectivity and can lead to non-specific toxicity. The
neurology segment accounted for the considerable growth in the global
gene therapy market over the forecast period.
The growth of the segment is majorly driven by the
rising prevalence of neurological diseases, including Alzheimer’s and
Parkinson’s. There are various appealing characteristics of gene therapy
approaches that make them ideal for treating neurological diseases. The
FDA-approved several gene therapies for neurological disorders such as
ZOLGENSMA for spinal muscular atrophy, SKYSONA for cerebral
adrenoleukodystrophy, ELEVIDYS for Duchenne muscular dystrophy, and LENMELDY
for Metachromatic Leukodystrophy (MLD). The in vivo segment is expected to hold the dominating
share of the market during the forecast period. With the rising demand for
targeted therapies, in vivo gene therapy has gained significant traction. In
vivo gene therapy, corrected genes are given directly into the patient’s body.
This approach is less invasive and eliminates the necessity for complicated
procedures. This can occur through an IV or local delivery to a specific organ,
like the eye. On the other hand, the ex vivo segment for considerable growth in
the global cell line development market over the forecast period. In ex vivo gene therapy, the cells of
the patients are extracted from the body and new genes are introduced to those
cells in a laboratory setting using gene therapy techniques. The modified cells
are then transplanted back to the patient body to combat disease or repair
nonfunctioning genes. Route of Administration Insights
The intravenous segment held the largest share of the market in 2023. The
growth of the segment is attributed to its ability of intravenous (IV)
administration to deliver treatments directly into the bloodstream of the
patient. This method is commonly used to treat several medical conditions. The
rapid improvements in vector design and delivery mechanisms have made
intravenous administration the most preferred option for many gene therapies,
propelling the segment’s growth during the forecast period. Browse Related Reports: 🔸Viral Vectors &
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Therapy Processing System Market: https://www.precedenceresearch.com/automated-and-closed-cell-therapy-processing-system-market 🔹Dimension Therapeutics Inc. 🔹Taxus
Cardium Pharmaceuticals Group Inc. 🔹Epeius Biotechnologies Corp. 🔹Shanghai Sunway
Biotech Co. Ltd 🔹Applied Genetic Technologies
Corporation 🔹Bristol-Myers Squibb Company 🔹American Gene Technologies 🔹BioMarin Pharmaceuticals Inc. 🔹Gensight Biologics S.A. 🔹Sibinono GeneTech Co. Ltd What is Going Around the Globe? 🔹In January 2025, NHS is to offer
'groundbreaking' sickle cell gene therapy. A gene-editing therapy for sickle
cell disease, with a price tag of £1.65m, is to be offered to patients on the
NHS in England. According to the experts, nearly 50 people a year with an
inherited blood disorder is likely to receive it. Campaigners have referred to
the treatment as "groundbreaking," and its availability on the NHS as
a "milestone." 🔹In February 2025, GenSight Biologics,
a biopharma company focused on developing and commercializing innovative gene
therapies for retinal neurodegenerative diseases and central nervous system
disorders, reported final efficacy and safety results after the REFLECT Phase
III clinical trial with LUMEVOQ. The results show that five years after a
one-time administration of the gene therapy, the visual acuity improvement
among patients with LHON (Leber Hereditary Optic Neuropathy) was sustained
while maintaining a favorable safety profile. 🔹In October 2024, Princess Royal
opened a pioneering Gene Therapy Innovation and Manufacturing Centre (GTIMC) at
the University of Sheffield. The state-of-the-art facility marks a significant
milestone in accelerating the development of life-changing treatments for
patients with rare and inherited diseases. The new GTIMC facility will
accelerate the development of new gene therapies and clinical trials and build
career pathways for people in the region and develop Sheffield and Yorkshire as
a hub for gene therapy in the future. The University of Sheffield is already
one of the leading players in gene therapy research and innovation and the new
facility will build on existing research to find cutting-edge gene therapy
treatments for more than 7,000 rare and inherited diseases that are currently
without a cure. 🔹In October 2024, Governor Hochul
launched the next phase of Long Island's nation-leading cell and gene therapy
innovation hub. With the $430 million New York BioGenesis Park on Long Island
and the $98 million expansion at Roswell Park Comprehensive Cancer Center in
Buffalo, these projects represent a combined total investment of over half a
billion dollars in Cell and Gene Therapy innovation across New York. The research report categorizes the gene therapy market into the
following segments and subsegments: By Type 🔹Gene Silencing 🔹Cell Replacement 🔹Gene Augmentation 🔹Other Therapies By Vector 🔹Viral vectors 🔹Non-Viral vectors By Therapeutic Area 🔹Neurology 🔹Oncology 🔹Hepatology 🔹Other therapeutic areas By Delivery Method 🔹In Vivo 🔹Ex Vivo By Route of Administration 🔹Intravenous 🔹Other routes of administration By Geography 🔹North America 🔹Europe 🔹Asia Pacific 🔹Middle East & Africa 🔹Latin America Immediate Delivery Available | Buy This Premium Research Report@ https://www.precedenceresearch.com/checkout/1462 You can place an order or ask any
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Gene
Therapy Market Top Companies
