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Gene Therapy Market

The market for Gene Therapy was estimated at $8.8 billion in 2025; it is anticipated to increase to $20.6 billion by 2030, with projections indicating growth to around $48.2 billion by 2035.

Report ID:DS1804018
Author:Debadatta Patel - Senior Consultant
Published Date:
Datatree
Life Science
Biotechnology
Gene Therapy
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Report Summary
Market Data
Methodology
Table of Contents

Global Gene Therapy Market Outlook

Revenue, 2025

$8.8B

Forecast, 2035

$48.2B

CAGR, 2026 - 2035

18.5%

The Gene Therapy industry revenue is expected to be around $8.8 billion in 2026 and expected to showcase growth with 18.5% CAGR between 2026 and 2035. With the field of gene therapy now becoming a cornerstone of advanced medicine it is moving from a speculative concept to an established field of study, due to the rise in the number of clinical trials, plus favourable regulatory environment and a steady flow of investment from pharmaceutical companies and investors. Therapies designed to treat blood diseases and cancer now make up 57.3% of demand for genetic platforms due to the applications of these technologies in the treatment of certain cancers and inherited blood conditions. The continued relevance of gene therapy is underpinned by the fact that the range of diseases it can treat is being broadened, through developments in production and logistics, and through longer term data on how well the treatment works and how safe it is in real world use. The leading source of revenue in this sector is Gene Augmentation Therapy, generating $3.28 billion in sales in 2025. This is because it has a greater body of clinical evidence, clearer legal pathways to follow and is used in the treatment of many different genetic conditions.

Among its various applications, gene therapy offers the prospect of treatments that permanently correct faulty genes through the modification of the genome. This process provides an alternative to the management of chronic symptoms of a condition. A one time treatment could be possible. The characteristics of these therapies include the targeting of specific mechanisms and pathways, the ability to treat the root cause of mutations and the possibility of eradicating disease in the case of conditions including cancer, blood disorders, rare hereditary conditions, eye diseases, neurological conditions and muscular dystrophy. Research in this field is currently focused on two major region's therapies applied outside or inside the body. These treatments target hematological malignancies, other forms of cancer, blood diseases like sickle cell, and disorders caused by a single faulty gene. With gene therapy development gathering pace, there is growing demand for more advanced delivery systems and the genetic engineering techniques necessary for the most complex treatments. The trend is moving away from standardised treatments and more towards personalised therapy that will select patients based on their individual biomarkers. Another factor driving the industry is increased co operation between researchers in academia and the pharmaceutical industry. Finally the cost of these treatments is now being covered by the patients insurance.

Gene Therapy market outlook with forecast trends, drivers, opportunities, supply chain, and competition 2025-2035
Gene Therapy Market Outlook

Market Key Insights

  • The Gene Therapy market is projected to grow from $8.8 billion in 2025 to $48.2 billion in 2035. This represents a CAGR of 18.5%, reflecting rising demand across Cancer Treatment, Monogenic Disorders Management, and Chronic Diseases Intervention.

  • Leading the market are BioMarin Pharmaceutical, Sarepta Therapeutics and Novartis, with these companies defining the competitive market landscape.

  • Gene therapy is expected to show its highest growth rate in the US and China. Between 2025 and 2030, it is predicted that the Compound Annual Growth Rate in these countries will be 17.8% and 25.9% respectively.

  • Brazil, India and South Africa are expected to exhibit the highest growth rates of 13.9% to 19.2% per annum.

  • The expanding gene therapy applications will contribute $3 billion to the market size by 2030.

  • Gene therapy is expected to experience a significant expansion, with predicted revenue growth of $39.4 billion by the year 2035. The product sector that will gain the most market share is that supplying research institutes and specialist clinics.

  • The gene therapy sector is now growing within oncology and this is being backed by the regulatory bodies with the fast track approval of products. It is anticipated that the gene therapy market will grow by 446% between 2035 and 2025.

gene therapy market size with pie charts of major and emerging country share, CAGR, trends for 2025 and 2032
Gene Therapy - Country Share Analysis

Opportunities in the Gene Therapy

In Europe there is also an emerging market for gene silencing technologies for diagnosing rare blood diseases. By 2030, the market for gene silencing globally is predicted to grow to $4.26 billion from $1.92 billion in the year 2025. The compound annual growth rate here is 17.3%. There is a fast increasing number of RNAi and CRISPR based cell and gene therapies in Europe's landscape of rare disease. This is due to hospital consortiums, biopharmaceutical partnerships and clinical trial pipelines that utilise patient registries and regulatory incentives. They are driving the commercialisation of this technology.

Growth Opportunities in North America and Europe

The Gene Therapy industry within North America is driven by the availability of considerable funding for research and development, swift regulatory approvals, along with the widespread adoption of advanced biologic treatments. This has made oncology the most profitable Gene Therapy application, with neurology being a Gene Therapy region that is growing rapidly. Key region's of growth include the expansion of gene therapies that are used for cancer and blood disorders, the development of gene therapies that are used outside the body and gene therapies used in the body for rare diseases, and the application of gene editing technology. With the market becoming increasingly competitive biotech firms and the large pharmaceutical companies are scrambling for locations to conduct clinical trials, vector manufacturing sites and payer contracts. In response, companies are pursuing long term supply agreements and partnerships with CDMOs that are strategic. The region is characterised by the presence of a well equipped clinical infrastructure, favourable reimbursement schemes for high cost treatments, and strong patient lobbying groups. This creates a market environment that is conducive to value based contracts, rapid adoption of new cell and gene therapies and premium pricing.
Across the region of Europe the gene therapy market is essentially supported by a comprehensive system for the licensing of gene based drugs. Consequently it is seen that haematology makes up the greatest portion of the gene therapy drug market, by its size. Ophthalmology is its closest rival due to the large number of scientific trials which have already been completed in the fields of both inherited retinal diseases and disorders which cause patients to bleed. Key region's of opportunity include long duration haematology gene therapies which can demonstrate long term factor expression, expansion of ophthalmology services via the establishment of centres of excellence and the introduction of region wide patient outcome registries to support the cost benefits of one off treatments. The biotechnology industry in the region is driven by a cluster of major academic hospitals, small and medium sized biotechnology companies and research institutes. This has led to intense competition within the sector with the companies involved focusing on developing manufacturing scalability, the optimisation of adeno associated virus vectors and safety data following the release of any new treatments. Centralised assessment processes along with government backing for novel treatments and uniform quality standards are core drivers for the region. These factors mean Gene Therapy platforms which deliver consistent manufacture and control processes, transparent long term risk management and cost offset cases are favoured.

Market Dynamics and Supply Chain

01

Driver: Rising Demand for Personalized Medicine and Advanced Cancer Therapeutics Driving Gene Therapy Market

The gene therapy market is also strongly propelled by the increasing adoption of personalized medicine and the rising demand for advanced cancer treatments. Personalized medicine leverages patient-specific genetic information to tailor therapies, particularly CAR-T cell therapies and viral vector-based interventions, improving efficacy and reducing adverse effects. Concurrently, oncology applications, including oncolytic viruses and gene-modified immune cells, address unmet clinical needs for hematologic and solid tumors. Companies such as Novartis, Gilead Sciences, and bluebird bio are also developing innovative gene therapy solutions, enhancing treatment precision. These growth factors collectively drive research, regulatory approvals, and commercialization efforts, supporting the expansion of gene therapy pipelines, accelerating patient access, and enabling better clinical outcomes across hospitals, research institutes, and biotech centers globally.
The development of safer, more efficient viral vectors such as AAVs and lentiviruses has also accelerated gene therapy adoption across multiple indications. Innovations in gene editing, vector design, and delivery methods enhance transduction efficiency, reduce immunogenicity, and improve tissue targeting. Companies like Sarepta Therapeutics, Spark Therapeutics, and UniQure are also leveraging these technologies to expand applications in monogenic disorders and chronic diseases. Improved delivery systems enable scalable production and reproducible therapeutic effects, strengthening clinical adoption and fostering market growth.
02

Restraint: High Cost of Gene Therapy

In times there has been a notable advancement in gene therapy as a groundbreaking medical innovation; nonetheless; one of the major obstacles hindering its widespread adoption is the exorbitant cost involved in the treatment process which can soar up to millions of dollars, per patient rendering it out of reach for a significant portion of the populace This high cost reduces the potential customer base thus impacting market demand adversely Moreover insurance firms find it challenging to cope with these steep expenses which in turn adds complexity to the healthcare landscape and hinders market growth.
03

Opportunity: Emerging demand for cell replacement gene therapy in Asia-Pacific diabetes and metabolic disease populations and Rapid expansion of gene augmentation therapies for inherited retinal diseases in North American ophthalmology markets

In the Asia Pacific region, there is a substantial market for gene therapy due to the high incidence of diabetes and metabolic syndrome. A prediction is made that cell replacement therapies will generate revenue of $2.51 billion in 2025, and by 2030 this should rise to $6.06 billion. This rate of growth would be 19.3% per annum. In this region the ex vivo engineered cell and gene therapies which allow for long term insulin production coupled with the presence of manufacturing hubs and also reimbursement pilots, are contributing to cell replacement therapy being the gene therapy which is advancing fastest in this region for treating metabolic diseases.
Gene therapy is set to become adopted in ophthalmology in North America. It comes as inherited retinal diseases treatments move from clinical trials towards market. Worldwide revenue from gene therapies where the genome is augmented is predicted to grow from $3.28 billion in 2025 to a figure of $8 billion by the year 2030; this represents the quickest growing gene therapy in the world with a CAGR of 19.5%. This trend is driven by the growth of precision medicine, along with advances in viral vector delivery technologies and regenerative medicine, in which cell and gene therapies are delivered in the body.
04

Challenge: Regulatory Hurdles

The process of gene therapy is intricate as it involves modifying genes at a level and is subjected to strict regulatory oversight and close examination by authorities worldwide. Approval criteria are stringent due to the newness of the technology and the potential risks associated with modification techniques. Any delays or refusals, in obtaining approvals can affect the speed at which gene therapies enter the market and how quickly they are adopted by people.

Supply Chain Landscape

1

Viral Vector Supply

Thermo Fisher ScientificLonza GroupCatalent
2

Gene Therapy Development

NovartisSarepta TherapeuticsCRISPR Therapeutics
3

Gene Therapy Manufacturing

BioMarin PharmaceuticalBluebird bioOxford Biomedica
4

Clinical Applications

hospitalsspecialty clinicsacademic medical centers
Gene Therapy - Supply Chain

Use Cases of Gene Therapy in Cancer Treatment & Monogenic Disorders Management

Cancer Treatment : Gene therapy is increasingly applied in cancer treatment to modify or replace defective genes, enhance immune response, and directly target tumor cells. CAR-T cell therapy and oncolytic viral vectors are the most common approaches, primarily used by oncology centers, research hospitals, and biotech firms. Companies like Novartis, Gilead Sciences (Kite Pharma), and bluebird bio lead the market with FDA-approved therapies such as Kymriah and Zynteglo, offering highly personalized treatments that improve patient outcomes and reduce relapse rates in hematologic cancers.
Monogenic Disorders Management : Gene therapy has transformed the management of monogenic disorders, which are caused by mutations in a single gene. Adeno-associated viral vectors and lentiviral vectors are widely employed to deliver functional genes to affected tissues. This therapy is primarily utilized by specialized clinical centers and hospitals treating rare genetic diseases. Key players such as Sarepta Therapeutics, Spark Therapeutics, and UniQure provide cutting-edge solutions for disorders like Duchenne muscular dystrophy, hemophilia, and spinal muscular atrophy, offering durable and potentially curative effects.
Chronic Diseases Intervention : Gene therapy is emerging as a promising approach for chronic diseases, including cardiovascular, metabolic, and neurodegenerative disorders. Ex vivo and in vivo viral vector systems are commonly used to deliver therapeutic genes to targeted tissues, enabling long-term disease modulation. End-users include specialty clinics, research institutes, and hospitals. Companies such as Voyager Therapeutics, REGENXBIO, and Editas Medicine are advancing gene therapy pipelines for chronic conditions, aiming to reduce disease progression, enhance quality of life, and minimize lifelong dependence on conventional medications.

Recent Developments

The gene therapy market is advancing rapidly as CRISPR and viral vector innovations improve safety, targeting, and efficiency for inherited diseases and oncology. Recent developments emphasize scalable manufacturing, regulatory approvals of next‑generation AAV and lentiviral products, and broader clinical adoption of CAR‑T and gene‑editing therapies. A key trend is integration of precision medicine, enabling bespoke genetic treatments that enhance therapeutic outcomes and reduce side effects, driving investment from biotech and big pharma in R&D and commercialization pipelines.

November 2025 : The U.S. Food and Drug Administration approved Itvisma for the treatment of spinal muscular atrophy in patients aged 2 and older, expanding gene therapy use beyond infants. This expansion is confirmed by both Novartis and the FDA.
June 2024 : The U.S. FDA expanded the use of Sarepta’s Elevidys for Duchenne muscular dystrophy to a broader patient population, though later safety issues impacted distribution and labeling.

Impact of Industry Transitions on the Gene Therapy Market

As a core segment of the Biotechnology industry, the Gene Therapy market develops in line with broader industry shifts. Over recent years, transitions such as Expanding Applications and Regulatory Shifts have redefined priorities across the Biotechnology sector, influencing how the Gene Therapy market evolves in terms of demand, applications and competitive dynamics. These transitions highlight the structural changes shaping long-term growth opportunities.
01

Expanding Applications

The expansion of gene therapy from being experimental to widespread use is having a marked impact on the treatment of various rare genetic disorders, chronic conditions, cancer, and infectious diseases. Gene therapy is expected to increase the gene therapy market by $3 billion by the year 2030. The trend is due to gene therapies that are increasingly being used by clinicians to modify disease pathways, not only just treat the symptoms, making them more attractive than traditional medicines. Developers of biopharmaceuticals are benefiting from a growing potential patient market and less risk to their product development programmes as a result of the progress made in the technology and a maturing legal framework. These factors are altering the position of gene therapy, so that it becomes a cornerstone of the field of personalised medicine. This is now having an impact on competitive strategies, investments and research priorities globally across the pharmaceutical industry.
02

Regulatory Shifts

Significant changes have occurred in regulations over the last few years concerning the development and investigation of gene therapy. Regulatory authorities such as the FDA have approved gene therapies including Luxturna and Kymriah. This approval demonstrates a growing acceptance of gene therapies by the governing bodies. The new rules will speed up clinical trials and the subsequent granting of licenses to market new biotech products. The movement in regulatory policies enables the swift progression of gene therapy products through the development phases and on to the market. This results in patients having quicker access to new treatments.