Gene Therapy Industry Overview

The global gene therapy market size was estimated at USD 5.54 billion in 2023 and is projected to grow at a CAGR of 18.88% from 2024 to 2030. The market growth is attributed to many factors such as the 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 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 the 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.

Gather more insights about the market drivers, restrains and growth of the Gene Therapy Market

Detailed Segmentation:

Vector Insights

The AAV segment shows a significant revenue contribution of 22% in 2023. Several biopharma companies are offering their viral vector platform for the development of AAV-based gene therapy products. 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 the 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 the 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 can 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.

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 the 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.

Route of Administration Insights

The intravenous segment dominated the global gene therapy market in 2023, primarily due to its effectiveness in delivering treatments directly into the bloodstream, ensuring widespread distribution and rapid uptake by target cells. This method's ability to treat a wide range of conditions, coupled with advancements in vector design and delivery mechanisms, has made intravenous administration the preferred choice for many gene therapies, contributing significantly to its market leadership. The segment is also expected to emerge as the most lucrative over the forecast period.

Regional Insights

North America dominated the market in 2023 with the largest revenue share of 18.95% 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.

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Gene Therapy Market Segmentation

Grand View Research has segmented the global gene therapy market report based on indication, vector type, route of administration, and region:

Gene Therapy Indication Outlook (Revenue, USD Million, 2018 - 2030)

• Large B-Cell Lymphoma
• Multiple Myeloma
• Spinal Muscular Atrophy (SMA)
• Acute Lymphoblastic Leukemia (ALL)
• Melanoma (lesions)
• Inherited Retinal Disease
• Beta-Thalassemia Major/SCD
• Others

Gene Therapy Vector Type Outlook (Revenue, USD Million, 2018 - 2030)

• Lentivirus
• RetroVirus & gamma RetroVirus
• AAV
• Modified Herpes Simplex Virus
• Adenovirus
• Others

Gene Therapy Route of Administration Outlook (Revenue, USD Million, 2018 - 2030)

• Intravenous
• Others

Gene Therapy Regional Outlook (Revenue, USD Million, 2018 - 2030)

• North America
  • S.
  • Canada
  • Mexico
• Europe
  • UK
  • Germany
  • Switzerland
• Asia Pacific
  • Japan
  • China
  • South Korea
  • Australia

Rest of the world 

Key Gene Therapy Companies:

The following are the leading companies in the gene therapy market. These companies collectively hold the largest market share and dictate industry trends.

• Amgen Inc.
• Novartis AG
• F. Hoffmann-La Roche
• Gilead Sciences, Inc.
• bluebird bio, Inc.
• Bristol-Myers Squibb Company
• Legend Biotech.
• BioMarin.
• uniQure N.V.
• Merck & Co.
• Sarepta Therapeutics, Inc.
• Krystal Biotech, Inc.
• CRISPR Therapeutics.

Order a free sample PDF of the Gene Therapy Market Intelligence Study, published by Grand View Research.

Recent Developments

• In January 2024, Biogen and Ginkgo Bioworks announced the completion of their gene therapy collaboration involving AAV based vectors. This is expected to fuel the demand for gene therapies in the coming years.
• In December 2023, the Swiss Agency for Therapeutic Products granted approval to Libmeldy for the 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 the 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.