DelveInsight’s Gene Therapy Market report delivers an in-depth understanding of the 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), covering historical and forecasted market trends through 2032.
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Some of the key facts of the Gene Therapy Market Report:
- The global gene therapy market was valued at USD 8,570.28 million in 2024 and is growing at a CAGR of 19.25% during the forecast period from 2025 to 2032, expected 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, supported by the rapid expansion of the gene therapy pipeline and numerous clinical trials targeting conditions previously considered untreatable.
- 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 gene therapy market globally.
- According to 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 an estimated 830,895 individuals worldwide living with haemophilia, including about 282,266 severe cases.
- Therapies such as 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.
- Von Willebrand disease (VWD) was diagnosed in over 2,500 people in Australia as of 2024 per the Haemophilia Foundation Australia, though many cases were believed to be undiagnosed, driving demand for gene-editing approaches such as CRISPR-based therapies.
- In July 2024, Pfizer Inc. announced positive topline results from the Phase 3 AFFINE study evaluating giroctocogene fitelparvovec, an investigational gene therapy for adults with moderately severe to severe hemophilia A.
- In the vector-type segment, the viral category is expected to hold a significant share in 2024, with vectors such as Adeno-Associated Viruses (AAV), Lentiviruses, and Adenoviruses used extensively due to their ability to deliver therapeutic genes into target cells with precision and efficiency.
- In July 2022, PTC Therapeutics announced that the European Commission granted marketing authorization for Upstaza™ (eladocagene exuparvovec), the first approved disease-modifying treatment for AADC deficiency and the first gene therapy directly infused into the brain to reach the market.
- North America is expected to account for the highest proportion of the gene therapy market in 2024, out of all regions, driven by advanced healthcare infrastructure, strong R&D capabilities, and substantial financial investment.
- Per the Centers for Disease Control and Prevention (2024), about 1 in every 5,000 males aged 5-9 years in the US had Duchenne or Becker muscular dystrophy (DBMD), and approximately 33,000 males were estimated to be living with hemophilia in the US.
- In April 2024, Pfizer Inc. announced FDA approval of BEQVEZ™ (fidanacogene elaparvovec-dzkt), a one-time gene therapy for adults with hemophilia B.
- In November 2024, PTC Therapeutics announced FDA approval of KEBILIDI™ (eladocagene exuparvovec-tneq) for AADC deficiency in children and adults.
- In June 2023, Sarepta Therapeutics announced that the US FDA granted accelerated approval for ELEVIDYS (delandistrogene moxeparvovec-rokl) for ambulatory pediatric patients aged 4 to 5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.
- Key Gene Therapy Companies: 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, Kriya Therapeutics, Affinia Therapeutics, Caribou Biosciences, Pfizer Inc., and others.
Gene Therapy Overview
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. Gene therapy offers a promising alternative to conventional treatment by targeting the root cause of disease through delivery of functional genes — for example, delivering clotting factor genes (Factor VIII for haemophilia A and Factor IX for haemophilia B) or micro-dystrophin variants for Duchenne/Becker muscular dystrophy using viral vectors such as adeno-associated viruses (AAVs).
Viral vectors are used extensively in gene therapy because of their innate ability to enter cells and introduce genetic material, achieving high levels of therapeutic gene expression in targeted tissues with minimal damage to surrounding cells. Viral gene therapies are being applied across a wide range of diseases, including hemophilia, inherited blindness, and oncology. AAV vectors are used to deliver clotting factor genes to treat hemophilia A and B — for instance, Valoctocogene Roxaparvovec — while AAV-based therapies like Luxturna are used to treat genetic forms of retinal disorders, including Leber congenital amaurosis.
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Gene Therapy Market Dynamics
The high global prevalence of haemophilia, Von Willebrand disease, and other genetic and rare diseases continues to boost the overall gene therapy market, supported by increasing focus on personalized medicine and advancements in gene-editing technologies such as CRISPR. The rapid expansion of the gene therapy pipeline — with several promising therapies advancing to Phase 3 clinical trials, regulatory fast-track designations, and innovative delivery mechanisms — further accelerates market evolution.
However, ethical and regulatory issues, especially concerning germline modifications, along with the high cost of gene therapies, may pose slight obstacles to the growth of the gene therapy market during the forecast period.
Gene Therapy Market Segment Analysis
The Gene Therapy market is segmented 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, the viral category is expected to hold a significant share in 2024, driven by its effectiveness in delivering therapeutic genes into target cells with precision and efficiency across diseases such as rare genetic disorders, hemophilia, inherited blindness, and certain cancers. Increasing strategic activity among key market players, including regulatory approvals such as Upstaza™, is further boosting the segment.
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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, attributed to its advanced healthcare infrastructure, strong research and development capabilities, and substantial financial investments in innovative biopharmaceuticals. The increasing prevalence of rare and genetic disorders, the presence of key market players such as Biogen, Novartis, and PTC Therapeutics, government initiatives, and favorable reimbursement policies further strengthen the region’s market position, alongside a well-established clinical trial pipeline in the United States.
Gene Therapy Market Strengths
- A rapidly expanding pipeline with multiple therapies advancing to Phase 3 trials, alongside regulatory fast-track designations, provides strong near-term commercial momentum for the market.
- The dominance of viral vector platforms (AAV, Lentivirus, Adenovirus) offers proven, precise delivery mechanisms already validated across hemophilia, inherited blindness, and AADC deficiency indications.
Gene Therapy Market Opportunities
- The high global prevalence of haemophilia (an estimated 830,895 individuals worldwide) and under-diagnosed conditions such as Von Willebrand disease represent significant unaddressed patient populations for gene-based interventions.
- Advancements in gene-editing technologies such as CRISPR and the growing focus on personalized medicine open opportunities for next-generation therapies targeting bleeding disorders, muscular dystrophy, and other genetic conditions beyond current approved indications.
Scope of the Gene Therapy Market Report
- Study Period: 2020-2028
- Forecast Period: 2023-2028
- Coverage: Global
- Key Gene Therapy Companies: 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, Kriya Therapeutics, Affinia Therapeutics, Caribou Biosciences, Pfizer Inc., and others
- Segmentation: By Vector Type (Viral and Non-Viral), Delivery Type (Ex Vivo and In Vivo), Indication (Neurological, Oncological, Hematological, Ophthalmological, and Others), and Geography
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