Gene Therapy Market

Gene Therapy Market by Vector Type (Viral and Non-Viral), Delivery Type (Ex Vivo and In Vivo), Indication (Neurological, Oncological, Hematological, Ophthalmological, and Others), and Geography (North America, Europe, Asia-Pacific, and Rest of the World) is expected to grow at a steady CAGR forecast till 2032 owing to the rising prevalence of genetic and rare diseases, rapid expansion of the gene therapy pipeline, and increasing strategic activities among the key players across the globe.

The global gene therapy market was valued at USD  8,570.28 million in 2024, growing at a CAGR of 19.25% during the forecast period from 2025 to 2032 to reach USD 34,933.71 million by 2032. The rising prevalence of genetic and rare diseases has driven demand for innovative treatments, positioning gene therapy as a promising solution. This demand is supported by the rapid expansion of the gene therapy pipeline, which includes numerous clinical trials and advanced therapies targeting conditions previously considered untreatable. Additionally, strategic activities such as mergers, acquisitions, and partnerships among key players have accelerated research, development, and commercialization efforts, collectively fostering innovation and boosting the overall market for gene therapy globally. Thus, all the factors mentioned above collectively contribute to the positive growth of the gene therapy market during the forecast period from 2025 to 2032.

Gene Therapy Market Dynamics:

According to data from the Haemophilia Foundation Australia (2024), over 7,400 individuals in Australia were diagnosed with bleeding disorders. Globally, haemophilia affected approximately 1 in 6,000–10,000 males, with haemophilia A occurring in 1 in 6,000 males and haemophilia B in 1 in 26,000 males. An estimated 830,895 individuals worldwide had haemophilia, including about 282,266 with severe cases. Gene therapy offers a promising alternative by targeting the root cause of the disease through the delivery of functional genes capable of producing the missing clotting factors (Factor VIII for haemophilia A and Factor IX for haemophilia B). For example, therapies like BioMarin's Roctavian for haemophilia A and UniQure's Hemgenix for haemophilia B have shown the potential to reduce or eliminate the need for factor replacement therapy, improve quality of life, and address underlying genetic defects. Thus, the high global prevalence of haemophilia can significantly boost the overall market of gene therapy across the globe.

Additionally, according to the recent data provided by the Haemophilia Foundation Australia (2024), Von Willebrand disease (VWD), an inherited bleeding disorder that affects individuals of all genders and racial backgrounds, was diagnosed in over 2,500 people in Australia, though many cases were believed to be undiagnosed. With VWD affecting both males and females and a significant number of cases remaining undiagnosed, the demand for innovative therapeutic solutions has risen. Gene therapy offers a promising avenue to address the root cause of such disorders by correcting or replacing faulty genes, potentially providing long-term or permanent relief. The increasing focus on personalized medicine and advancements in gene-editing technologies, such as CRISPR, has further accelerated the development of gene therapies targeting bleeding disorders like VWD.

Furthermore, the rapid expansion of the gene therapy pipeline is significantly boosting the overall market, with several promising therapies advancing to Phase 3 clinical trials. For instance, in July 2024, Pfizer Inc. announced positive topline results from the Phase 3 AFFINE study, evaluating giroctocogene fitelparvovec, an investigational gene therapy for the treatment of adults with moderately severe to severe hemophilia A. These advancements reflect the broader interest and investment in gene therapy, highlighting its potential to address rare and complex diseases, thereby driving market growth. Regulatory fast-track designations and innovative delivery mechanisms also contribute to the pipeline's rapid evolution thereby boosting the overall market of gene therapy across the globe.

Thus, the factors mentioned above are expected to boost the overall market of gene therapy across the globe during the forecast period from 2025 to 2032.

However, the ethical and regulatory issues especially concerning germline modifications and the high cost of gene therapies may result in slight obstacles to the growth of the gene therapy market.

Gene Therapy Market Segment Analysis:

Gene Therapy Market by Vector Type (Viral and Non-Viral), Delivery Type (Ex Vivo and In Vivo), Indication (Neurological, Oncological, Hematological, Ophthalmological, and Others), and Geography (North America, Europe, Asia-Pacific, and Rest of the World) 

In the vector-type segment of the gene therapy market, the viral category is expected to hold a significant share in 2024.  Viral gene therapy has become a significant driver in boosting the overall market of gene therapy due to its effectiveness in delivering therapeutic genes into target cells with precision and efficiency. The ability of viral vectors to transport therapeutic payloads to specific cells is pivotal in treating a variety of diseases, including rare genetic disorders, hemophilia, inherited blindness, and certain cancers.

Viral vectors, such as Adeno-Associated Viruses (AAV), Lentiviruses, and Adenoviruses, are used extensively in gene therapy because of their innate ability to enter cells and introduce genetic material. This delivery system can achieve high levels of therapeutic gene expression in targeted tissues with minimal damage to surrounding cells, improving the success rates of treatments. Viral gene therapies are being applied across a wide range of diseases, such as hemophilia, inherited blindness, oncology treatments, and others. AAV vectors are being used to deliver clotting factor genes to treat hemophilia A and B. For instance, therapies like Valoctocogene Roxaparvovec have been developed using AAV vectors to treat hemophilic patients. Additionally, AAV-based therapies like Luxturna are being used to treat genetic forms of retinal disorders, including Leber congenital amaurosis.

Additionally, the increase in strategic activities among the key market players is further boosting the overall market of viral gene therapy. For instance, In July 2022, PTC Therapeutics, Inc. announced that the European Commission granted marketing authorization for Upstaza™ (eladocagene exuparvovec). Upstaza became the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency and the first gene therapy directly infused into the brain to reach the market. Approved for patients aged 18 months and older, Upstaza is an AAV2 viral-based gene therapy containing the human DDC gene delivered via recombinant technology.

Thus, the factors mentioned above are expected to boost the segment thereby boosting the overall market of gene therapy across the globe.

North America is expected to dominate the overall Gene Therapy market:

North America is expected to account for the highest proportion of the gene therapy market in 2024, out of all regions. This can be ascribed to its advanced healthcare infrastructure, strong research and development capabilities, and substantial financial investments in innovative biopharmaceuticals. Additionally, the increasing prevalence of rare and genetic disorders and the presence of key market players, such as Biogen, Novartis, and PTC Therapeutics, along with government initiatives and favorable reimbursement policies, further strengthen the region's market position. Furthermore, the rapid adoption of advanced gene therapy technologies and a well-established clinical trial pipeline, particularly in the United States, are expected to fuel this growth. Moreover, North America's higher prevalence of genetic and rare diseases contributes to the rising demand for gene therapy solutions, solidifying its market leadership in the coming years.

As per the recent data provided by the Centre for Disease Control and Prevention (2024), in the United States, about 1 in every 5,000 males aged 5-9 years had Duchenne or Becker muscular dystrophy (DBMD). These conditions are caused by mutations in the dystrophin gene, leading to progressive muscle weakness and loss of function. Gene therapy offers the potential to address the root cause by delivering functional versions of the dystrophin gene or micro-dystrophin variants using viral vectors such as adeno-associated viruses (AAVs) thereby boosting the overall market of gene therapy across the region.

Additionally, according to the recent data provided by the Centre for Disease Control and Prevention (2024), in the United States, approximately, 33,000 males were estimated to be living with hemophilia. Hemophilia, particularly hemophilia A and B, is characterized by a deficiency in clotting factors, leading to prolonged bleeding episodes. Gene therapies, such as BioMarin’s Roctavian for hemophilia A and CSL Behring’s Hemgenix for hemophilia B, have emerged as transformative options, offering the potential for long-term treatment by introducing functional genes to restore clotting factor production and hence, boosting the overall market of gene therapy across the region.

Furthermore, the growing product approval of new gene therapy further escalates the market of gene therapy. For instance, in April 2024, Pfizer Inc. announced the FDA approval of BEQVEZ™ (fidanacogene elaparvovec-dzkt), a one-time gene therapy for adults with hemophilia B.

Thus, the above-mentioned factors are expected to escalate the market of gene therapy in the region.

Gene Therapy Market Key Players:

Some of the key market players operating in the gene therapy market include F. Hoffmann-La Roche Ltd., Novartis AG, Ferring B.V., bluebird bio, Inc., CSL, Ultragenyx Pharmaceutical Inc., PTC Therapeutics, BioMarin, Biogen, Prevail Therapeutics, Sarepta Therapeutics, Inc., Kriya Therapeutics, Affinia Therapeutics, Caribou Biosciences, Pfizer Inc., and others.

Recent Developmental Activities in the Gene Therapy Market:

• In November 2024, PTC Therapeutics announced FDA approval of AADC deficiency gene therapy with the brand name KEBILIDI™ (eladocagene exuparvovec-tneq), indicated for the treatment of children and adults with AADC deficiency.
• In June 2023, Sarepta Therapeutics, Inc. announced that the U.S. FDA granted accelerated approval for ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy for ambulatory pediatric patients aged 4 to 5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.

Key Takeaways From the Gene Therapy Market Report Study

• Market size analysis for current gene therapy size (2024), and market forecast for 8 years (2025 to 2032)
• Top key product/technology developments, mergers, acquisitions, partnerships, and joint ventures happened over the last 3 years.
• Key companies dominating the gene therapy market.
• Various opportunities available for the other competitors in the gene therapy market space.
• What are the top-performing segments in 2024? How these segments will perform in 2032?
• Which are the top-performing regions and countries in the current gene therapy market scenario?
• Which are the regions and countries where companies should have concentrated on opportunities for gene therapy market growth in the coming future?

Target Audience Who Can be Benefited From This Gene Therapy Market Report Study

• Gene therapy product providers
• Research organizations and consulting companies
• Gene therapy-related organizations, associations, forums, and other alliances
• Government and corporate offices
• Start-up companies, venture capitalists, and private equity firms
• Distributors and traders dealing in gene therapy
• Various end-users who want to know more about the gene therapy market and the latest technological developments in the gene therapy market. 

Frequently Asked Questions for the Gene Therapy Market:

1. What is gene therapy?

• Gene therapy is a medical technique that involves modifying or introducing genetic material into a patient’s cells to treat or prevent disease. This innovative approach can correct defective genes, replace missing genes, or introduce new genes to help the body fight or prevent illnesses, particularly genetic disorders, cancers, and some viral infections.

2. What is the market for gene therapy?

• The global gene therapy market was valued at USD  8,570.28 million in 2024, growing at a CAGR of 19.25% during the forecast period from 2025 to 2032 to reach USD 34,933.71 million by 2032.

3. What are the drivers for the global gene therapy market?

• The rising prevalence of genetic and rare diseases has driven demand for innovative treatments, positioning gene therapy as a promising solution. This demand is supported by the rapid expansion of the gene therapy pipeline, which includes numerous clinical trials and advanced therapies targeting conditions previously considered untreatable. Additionally, strategic activities such as mergers, acquisitions, and partnerships among key players have accelerated research, development, and commercialization efforts, collectively fostering innovation and boosting the overall market for gene therapy globally. Thus, all the factors mentioned above collectively contribute to the positive growth of the gene therapy market during the forecast period from 2025 to 2032.

4. Who are the key players operating in the global gene therapy market?

• Some of the key market players operating in gene therapy are F. Hoffmann-La Roche Ltd., Novartis AG, Ferring B.V., bluebird bio, Inc., CSL, Ultragenyx Pharmaceutical Inc., PTC Therapeutics, BioMarin, Biogen, Prevail Therapeutics, Sarepta Therapeutics, Inc., Kriya Therapeutics, Affinia Therapeutics, Caribou Biosciences, Pfizer Inc., and others.

5. Which region has the highest share in the global gene therapy market?

• North America is expected to account for the highest proportion of the gene therapy market in 2024, out of all regions. This can be ascribed to its advanced healthcare infrastructure, strong research and development capabilities, and substantial financial investments in innovative biopharmaceuticals. Additionally, the increasing prevalence of rare and genetic disorders and the presence of key market players, such as Biogen, Novartis, and PTC Therapeutics, along with government initiatives and favorable reimbursement policies, further strengthen the region's market position. Furthermore, the rapid adoption of advanced gene therapy technologies and a well-established clinical trial pipeline, particularly in the United States, are expected to fuel this growth. Moreover, North America's higher prevalence of genetic and rare diseases contributes to the rising demand for gene therapy solutions, solidifying its market leadership in the coming years.

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