Gene Therapy Market Size, Share & Trend Analysis Report By Indication (Acute Lymphoblastic Leukemia, Large B-cell Lymphoma), By Vector Type (Lentivirus), By Route of Administration, By Region,- Global Industry Analysis, Share, Growth, Regional Outlook and Forecasts, 2024-2033

The global gene therapy market size was exhibited at USD 8.75 billion in 2023 and is projected to hit around USD 52.40 billion by 2033, growing at a CAGR of 19.6% during the forecast period 2024 to 2033.

Key Takeaways:

• North America dominated the market in 2023 with the largest revenue share of 65.12% in 2023.
• Europe is estimated to be the fastest-growing regional segment from 2024 to 2033.
• The AAV segment shows a significant revenue contribution of 22.9% in 2023.
• The spinal muscular atrophy (SMA) segment dominated the market in 2023.
• The Beta-Thalassemia Major/SCD segment is anticipated to register the fastest CAGR over the forecast period. 
• The intravenous segment dominated the global gene therapy market in 2023.

U.S. Gene Therapy Market Size in U.S. 2024 to 2033

The U.S. gene therapy market size was estimated at USD 1.59 billion in 2023 and is projected to surpass around USD 9.44 billion by 2033 at a CAGR of 19.50% from 2024 to 2033.

North America dominated the market in 2023 with the largest revenue share of 65.12% in 2023. This region is expected to become the largest routine manufacturer of gene therapy in terms of the number of approvals and revenue generated during the forecast period. Increasing investments in R&D from large and small companies in the development of ideal therapy drugs are anticipated to further boost the market.

Furthermore, the increasing number of investments by the governments and the growing prevalence of targeted diseases are the factors fueling the market. According to the Spinal Muscular Atrophy Foundation, in 2020, around 10,000 to 25,000 children and adults in the U.S. were affected by spinal muscular atrophy, making it a fairly common disease among rare diseases.

Europe is estimated to be the fastest-growing regional segment from 2024 to 2030. This is attributed to its large population with unmet medical needs and increasing demand for novel technologies in the treatment of rare but increasingly prevalent diseases. Asia Pacific market for commercial application of genetic therapies is anticipated to witness significant growth in the forecast period, which can be attributed to the easy availability of resources, local presence of major companies, and increased investment, by the governments.

UK Gene Therapy Market

The UK gene therapy market is anticipated to witness accelerated growth over the forecast period, due to increased investments by various big companies and governments, including the NHS & research laboratories. For instance, in March 2022, the UK government invested USD 326.45 million to accelerate healthcare research and manufacturing. Under this investment, additional $80 million of the fund will help companies at the forefront of invention with their commercial-scale manufacturing investments in areas like gene and cell therapies, as well as improved diagnostic technologies, among others. Various mergers & partnerships between manufacturers, universities, and other government bodies are expected to boost the market over the forecast period.

Gene Therapy Market Report Scope

The growth of the market is attributed to many factors such as expanding area of advanced therapies along with gene delivery technologies and progressive competition among key players focused on commercialization of their therapies. The biotechnology companies are investing in acquisitions, mergers/collaborations, and deals as a key strategy to increase in-house expertise and strengthen the product pipelines.

The COVID-19 outbreak has negatively impacted the market growth. This sector has experienced severe disruption due to COVID-19, which has historically presented significant challenges in the supply of materials, manufacturing, and logistics operations. For instance, companies had lengthy delivery times for specific components and later discovered that it was short on clinical trial supplies when a partner contract manufacturing company was compelled to shut down.

The robust pipeline is expected to boost the market growth over the forecast period. Researchers are working to make gene therapy available at clinics. Various universities and institutes exhibit a broad portfolio of products in the pipeline which is expected to boost revenue generation over the forecast period. Clinical trials for gene therapy increased significantly from 2017 to 2018, after the FDA approved first gene therapy. According to the American Society of Gene & Cell Therapy (ASGCT), around 1,986 products, including CAR T-cell therapies and other genetically modified cell therapies, are currently under development.

Moreover, improving regulatory support creates growth opportunities for the market over the forecast period. Several positive changes have been made by many international regulatory organizations to promote therapies. Support for CAR-T technology from the FDA is one of the examples. In phase II and III studies, in particular, regulators are allowing flexibility in the usual hierarchy of how clinical trials are conducted. Moreover, FDA expects 10 to 20 new therapies to be approved annually by 2025.

Furthermore, an increase in funding and investments in this sector is expected to provide lucrative growth opportunities to market players. Several biopharma companies are investing in this sector for novel product launches. For instance, in January 2022, Ori Biotech raised more than USD 100.0 million in Series B funding to introduce a novel cell & gene therapy developing platform. This funding allowed for a rapid transition from pre-commercialization to market launch.

Market Concentration & Characteristics

Market growth for gene therapy market is exponential and market growth is accelerating. The increasing number of approvals of gene therapies within North America and Europe are the major reasons for market growth. Furthermore, strong pipeline of numerous companies is anticipated to bring new therapies in the market.

Some of the major strategies opted by the companies within gene therapy market include increased spending on R&D and new product launches. For instance, in December 2023, the U.S. FDA approved a gene therapy for treating sickle cell disease. This is the first gene therapy that is based on CRISPR gene editing. Such new launches is expected to fuel the market growth over the forecast period.

The gene therapy market has very high impact of regulations. Regional regulatory bodies such as the U.S. Food and Drug Administration, European Medicines Agency, and more are responsible for all the approvals of therapies. Lengthy approval processes and clinical trials limit the entry of new players within the market.

Currently, North America and Europe are the regions which have the most approved gene therapies as of January 2024. However, in countries such as India, South Korea, Japan, large number of clinical trials are being undertaken for various conditions. For instance, in July 2023, a team of researchers at the Narayana Nethralaya announced that they will be starting a clinical trial for a gene therapy for ocular diseases.

Vector Insights

The AAV segment shows a significant revenue contribution of 22.9% in 2023. Several biopharma companies are offering their viral vector platform for the development of AAV-based gene therapy product. For instance, in September 2016, Lonza signed an exclusive agreement with Massachusetts Eye and Ear to support its novel Anc-AAV gene therapy platform for development and commercialization of next-generation gene therapies based on their AAV platform. Similarly, RegenxBio had made an agreement with companies AveXis & Biogen in March 2014 and May 2016, respectively, which would allow both companies to use RegenxBio’s AAV vector platform for development of gene therapy molecules. Furthermore, in May 2021, Biogen Inc. and Capsigen Inc. entered into a strategic research partnership to engineer novel AAV capsids that have the possibility to deliver transformative gene therapies, which can address the fundamental genetic causes of numerous 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 collaboration to evaluate analytical methods and develop standards for AAV. As part of this partnership, NIST and USP will be conducting an interlaboratory study in which several laboratories will measure these serious quality attributes, and their results will be linked and examined. This collaboration will support the development of new promising gene therapies that will significantly advance people’s lives.

Indication Insights

The spinal muscular atrophy (SMA) segment dominated the market in 2023. Although SMA is a rare disorder, it is one of the most common fatal inherited diseases of infancy. The development of Zolgensma (AVXS-101), has proven its effectiveness in treating SMA and altering the phenotype of the illness. The FDA approved Novartis' Zolgensma approval in May 2019, which is aimed at treating the underlying cause of SMA. As of now, Zolgensma is the only gene treatment in this field to have been approved. The approval of this gene therapy is evidence of the growing use of therapies to treat serious hereditary illnesses like SMA.

The Beta-Thalassemia Major/SCD segment is anticipated to register the fastest CAGR over the forecast period. Gene therapy for SCD and β-thalassemia is based on transplantation of gene-modified hematopoietic stem cells. Clinical and preclinical studies have shown the efficacy and safety of this therapeutic modality. However, several other factors, such as suboptimal gene expression levels & gene transfer efficiency, limited stem-cell dose and quality, and toxicity of myeloablative regimens are still hampering its efficacy. Despite these challenges, in June 2019, bluebird Bio’s Zynteglo (formerly LentiGlobin) received conditional approval in Europe for the treatment of β-thalassemia and is expected to receive U.S. FDA approval in August 2022. Moreover, the product has already received Orphan Drug status by the U.S. FDA for treatment of patients with sickle cell disease (SCD). Furthermore, in April 2021, Vertex Pharmaceuticals and CRISPR Therapeutics amended partnership for the development, production, and commercialization of CTX001 in sickle beta thalassemia and cell disease. These achievements in this segment are anticipated to significantly boost the adoption of the product in this segment.

Route of Administration Insights

The intravenous segment dominated the global gene therapy market in 2023. Large number of approved products along with strong pipeline for IV candidates is the major reason for the segment dominance. The segment is also expected to emerge as the most lucrative over the forecast period.

Recent Developments

• In January 2024, Biogen and Ginkgo Bioworks announced that they have completed the gene therapy collaboration involving AAV based vectors. This is expected to fuel the demand for gene therapies in coming years.
• In December 2023, Swiss Agency for Therapeutic Products granted approval to Libmeldy for treatment of early-onset metachromatic leukodystrophy.
• In May 2023, Krystal Biotech was granted approval for VYJUVEK gene therapy for Dystrophic Epidermolysis Bullosa treatment
• In June 2023, the U.S. FDA granted approval to Sarepta for ELEVIDYS gene therapy to treat DMD in children of age 4-5 years
• In January 2023, Voyager Therapeutics and Neurocrine Biosciences entered into a strategic collaboration for commercialization & development of Voyager’s GBA1 program and other next-generation gene therapies for neurological diseases
• In January 2023, Spark Therapeutics and Neurochase established a strategic collaboration to develop Neurochase’s unique delivery technology for use with selected gene treatments for rare disorders in the CNS. In this agreement, Neurochase will contribute its extensive knowledge in direct drug delivery technology to Spark’s premier AAV platform.
• In January 2022, 64x Bio, a U.S.-based biotech company, raised USD 55.0 million in funding to advance its gene therapy manufacturing platform. This initiative was expected to expand the company’s VectorSelect platform.

Some of the prominent players in the Gene therapy market include:

• REGENXBIO, Inc.
• Oxford BioMedica plc
• Dimension Therapeutics, Inc.
• Bristol-Myers Squibb Company
• SANOFI
• Applied Genetic Technologies Corporation
• F. Hoffmann-La Roche Ltd
• bluebird Bio, Inc.
• Novartis AG
• Taxus Cardium Pharmaceuticals Group, Inc. (Gene Biotherapeutics)
• UniQure N.V.
• Shire Plc
• Cellectis S.A.
• Sangamo Therapeutics, Inc
• Orchard Therapeutics
• Gilead Lifesciences, Inc.
• BENITEC BIOPHARMA
• Sibiono GeneTech Co., Ltd
• Shanghai Sunway Biotech Co., Ltd.
• Gensight Biologics S.A.
• Transgene
• Calimmune, Inc.
• Epeius Biotechnologies Corp.
• Astellas Pharma Inc.
• American Gene Technologies
• BioMarin Pharmaceuticals, Inc.

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Nova one advisor, Inc. has segmented the global gene therapy market.

Indication

• Acute Lymphoblastic Leukemia (ALL)
• Inherited Retinal Disease
• Large B-cell Lymphoma
• ADA-SCID
• Melanoma (lesions)
• Beta-Thalassemia Major/SCD
• Head & Neck Squamous Cell Carcinoma
• Peripheral arterial disease
• Spinal Muscular Atrophy (SMA) 
• Others

Vector Type

• Lentivirus
• AAV
• RetroVirus & gamma RetroVirus
• Modified Herpes Simplex Virus
• Adenovirus
• Non-viral Plasmid Vector
• Others

Route of Administration

• Intravenous
• Others

By Region

• North America
• Europe
• Asia-Pacific
• Latin America
• Middle East & Africa (MEA)