The gene therapy market is poised for significant growth in the forecast period, driven by several key factors. These include the increasing prevalence of genetic disorders, a rise in research and development activities, and the expanding geriatric population. Concurrently, elevated healthcare expenditure, heightened health awareness among the general population, and the growing focus of medical research organizations on finding potential cures for rare, genetic, and autoimmune diseases create lucrative prospects for market growth.
Gene therapy is an innovative treatment method that utilizes genes to treat diseases. This method involves the insertion of functional genes into cells, replacing faulty genes and targeting genetic disorders. Gene therapy is employed to rectify defective genes, ultimately aimed at either curing a disease or enhancing the body's ability to combat diseases. This is achieved through a range of ways, including replacing mutated genes with functional ones, repairing genes with mutations, and rendering diseased cells more recognizable to the immune system. Typically, a genetically engineered virus serves as a vector to carry and deliver the therapeutic gene into the patient's cells. When successful, this method results in the production of a functioning protein by the newly introduced gene. In the future, gene therapy holds the promise of treating various chronic diseases by directly inserting genes into a patient's cells, potentially reducing the reliance on drugs or surgical procedures.
Gene therapy presents a hopeful alternative for addressing a range of conditions, including inherited disorders, specific types of cancer, and certain viral infections. Gene therapy is a promising treatment approach for treating a wide range of diseases, such as cancer, diabetes, AIDS, cystic fibrosis, cardiovascular ailments, and hemophilia, among others. However, it remains an area of ongoing research to ensure safety and effectiveness. Several techniques are employed in gene therapy, such as gene augmentation therapy, gene inhibition therapy, suicide gene insertion, and other methods. Currently, gene therapy is primarily tested for diseases lacking other cures. With the advancements in therapeutic techniques and gene delivery technologies, the gene therapy market is expected to experience substantial growth. However, it is still a relatively new and complex technology, and it comes with various ethical and safety concerns which the industry players are trying to mitigate.
The global gene therapy market is witnessing growth driven by the increasing prevalence of genetic and congenital diseases on a global scale. These genetic diseases, stemming from mutations in genes, disrupt the normal functioning of the body. Traditional treatments for genetic diseases, such as medication and surgery, offer only temporary relief and do not provide a cure. Gene therapy presents a promising alternative by potentially replacing or repairing faulty genes, offering a potential cure for genetic disorders. As the number of individuals affected by genetic diseases continues to rise, there is a growing demand for this therapeutic approach.
According to the Department of Health, as cited by the Government of Western Australia, approximately six out of ten people are likely to experience ailments with genetic ties. These genetic disorders can vary in severity, with an estimated 3 to 5% of newborns in Western Australia born with genetic disorders or birth abnormalities.
Gene therapy products are subjected to stringent regulatory evaluations before receiving approval. These therapies work at the genetic level, providing therapeutic effects that are notably more effective and enduring than traditional medicines. The advantage of this longevity has led to an increasing number of approvals for gene therapy products. The US FDA, for instance, has granted approval to over 10 such products between 2021 and 2024, with a substantial number of products in the clinical pipeline anticipated to secure final approval in the forecast period. Consequently, this factor significantly propels gene therapy market growth during the forecast period.
In 2023, the spinal muscular atrophy (SMA) emerged as the leading segment, capturing a substantial share of over 40%. Despite its rarity, SMA stands as one of the most prevalent and fatal inherited disorders among infants. Notably, the advent of Zolgensma (AVXS-101) has showcased its efficacy in treating SMA, significantly altering the illness's phenotype. In May 2019, the FDA granted approval for Novartis' Zolgensma, targeting the root cause of SMA. Presently, Zolgensma stands as the sole approved gene treatment in this domain. This approval signifies the increasing adoption of therapies to combat severe hereditary ailments such as SMA.
Looking ahead, the Beta-Thalassemia Major/SCD segment anticipates the fastest CAGR. Gene therapy for Sickle Cell Disease (SCD) and β-thalassemia revolves around gene-modified hematopoietic stem cell transplantation. Both clinical and preclinical studies validate the efficacy and safety of this therapeutic approach.
The current market sees a dominant position held by the viral vectors segment, owing to the safety and high efficiency exhibited by most viral vectors in combating infectious diseases and viruses. Moreover, their ease of handling and adaptability further fuels the segment's growth in gene therapy market.
In 2023, the Adeno-Associated Virus (AAV) segment held around 20% revenue share. Numerous biopharma companies are leveraging their viral vector platforms for AAV-based gene therapy products. For example, Lonza inked an exclusive deal with Massachusetts Eye and Ear in September 2016, supporting the development and commercialization of advanced gene therapies utilizing their Anc-AAV gene therapy platform. Additionally, Biogen Inc. partnered with Capsigen Inc. in May 2021, aiming to engineer innovative AAV capsids capable of delivering groundbreaking gene therapies targeting genetic causes of various neuromuscular and CNS disorders.
In July 2021, the U.S. Department of Commerce’s National Institute of Standards and Technology (NIST), National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and United States Pharmacopeia (USP) announced a collaborative effort to evaluate analytical methods and establish standards for AAV. Through interlaboratory studies, several labs will measure crucial quality attributes, fostering the development of promising gene therapies that significantly enhance people's lives.
In 2023, North America emerged as the dominant region in the global gene therapy market, a trend expected to persist from 2024 to 2030. The region held over 60% market share in 2023, driven by advanced technologies facilitating patient eligibility assessments for gene therapy and robust hospital infrastructure. North America foresees continued leadership in routine gene therapy manufacturing, marked by increased approvals and revenue generation. Substantial R&D investments by both large corporations and smaller entities to develop optimal therapy drugs are poised to further elevate market growth.
The United States, within North America, boasts a robust gene therapy research and development ecosystem. It houses leading academic institutions, research centers, and biotech firms actively contributing to advancements in the field. This environment fosters innovation, attracting investments crucial for novel gene therapy development. Moreover, the prevalence of genetic disorders and complex ailments, like cancer, has fueled demand for innovative treatments, supported by payer willingness to endorse these therapies through reimbursement programs.
Europe, renowned for rapid clinical research, evolving healthcare infrastructure, and favorable reimbursements, is forecasted as one of the fastest-growing regions in gene therapy industry from 2024 to 2030. In Asia Pacific, increased investments, accessible resources, and government support promise significant market growth for genetic therapies in the coming years.
Report Attributes | Details |
Gene Therapy Market Forecast Years | 2024 to 2030 |
Gene Therapy Market Historical Years | 2019, 2020, 2021, 2022, 2023 |
Gene Therapy Market Size 2023 | USD 9 Billion |
Gene Therapy Market CAGR | 21% (2024 to 2030) |
Gene Therapy Market Size 2030 | USD 30 Billion |
Key Segments | Indication, Vector Type, Type of Therapy, Delivery Method, Route of Administration, and Region |
Key Regions & Countries | North America (The U.S. Canada, Mexico), Europe (Germany, The U.K, France, Netherlands, Spain, Russia, Poland, Benelux, Nordic Countries, Rest of Europe Countries), Asia Pacific (China, Japan, India, South Korea, ASEAN, Australia, Rest of APAC Countries), Middle East & Africa (GCC – UAE, Saudi Arabia, Qatar, Oman, Bahrain, Kuwait), Israel, South Africa, Egypt, Rest of MEA Countries), and South America (Brazil, Argentina, Colombia, Chile, Rest of South America Countries). |
Some Key Companies | American Gene Technologies, Amgen, AnGes, Astellas Pharma, AstraZeneca, BENITEC BIOPHARMA, Biogen, BioMarin Pharmaceutical, Bluebird Bio, Bristol-Myers Squibb Company, Cellectis, CRISPR Therapeutics, CSL Behring, Epeius Biotechnologies, Gene Biotherapeutics, Gensight Biologics, Gilead Lifesciences, Novartis, Orchard Therapeutics, Oxford BioMedica, Pfizer, REGENXBIO, Roche, Sangamo Therapeutics, SANOFI, Shanghai Sunway Biotech, Shenzhen Sibiono GeneTech, Vertex Pharmaceuticals, and Others. |
Gene therapy market players bolster their position through investments in research and development activities, strategic partnerships, portfolio expansion, regulatory approvals, and company mergers and acquisitions. These proactive strategies enable them to maintain competitiveness and address the increasing demand for gene therapies.
The global gene therapy market is expected to grow at a compound annual growth rate (CAGR) of around 21% from 2024 to 2030.
The growth of the gene therapy market is driven by several key factors, including a surge in approvals from regulatory bodies, expanded genomics clinical research initiatives, a growing emphasis on advanced targeted gene therapies, and the widening scope of gene delivery technologies. Additionally, competition among key players to commercialize gene therapies, the escalating prevalence of genetic disorders, increased research and development activities, and a growing geriatric population contribute significantly. Elevated healthcare expenditure, heightened health awareness among the general populace, and medical research organizations' intensified focus on potential cures for rare, genetic, and autoimmune diseases also propel this growth. Moreover, the market also benefits from a strong and expanding gene therapy pipeline.
The global gene therapy market size in terms of revenue was estimated to be around USD 9 Billion in 2023.
In 2023, spinal muscular atrophy (SMA) emerged as the dominant segment, securing a substantial share exceeding 40%. Despite its rarity, SMA ranks among the most prevalent and fatal inherited disorders in infants. The introduction of Zolgensma (AVXS-101) has proven its effectiveness in treating SMA, notably altering the disease's phenotype. In May 2019, the FDA granted approval for Novartis' Zolgensma, targeting the root cause of SMA. Currently, Zolgensma stands as the sole approved gene treatment in this field, marking a significant step in adopting therapies for combating severe hereditary conditions such as SMA.
Some of the key players operating in the gene therapy market include American Gene Technologies, Amgen, AnGes, Astellas Pharma, AstraZeneca, BENITEC BIOPHARMA, Biogen, BioMarin Pharmaceutical, Bluebird Bio, Bristol-Myers Squibb Company, Cellectis, CRISPR Therapeutics, CSL Behring, Epeius Biotechnologies, Gene Biotherapeutics, Gensight Biologics, Gilead Lifesciences, Novartis, Orchard Therapeutics, Oxford BioMedica, Pfizer, REGENXBIO, Roche, Sangamo Therapeutics, SANOFI, Shanghai Sunway Biotech, Shenzhen Sibiono GeneTech, Vertex Pharmaceuticals, and Others.
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