Source: NextGen Intelligence Stats and Consulting LLP

Market share by Vector

Based on the vector type the market is segmented into Lentivirus, RetroVirus & gamma RetroVirus, AAV, Modified Herpes Simplex Virus, Adenovirus, and Others. The AAV segment shows a significant revenue contribution of over 22% in 2024. This is attributed to its superior safety profile, high efficiency in gene delivery, and long-lasting therapeutic effects. The segment is also experiencing increased adoption and approval in both oncology and rare genetic disease therapies.  Moreover, AAV-based therapies have received multiple FDA and EMA approvals, including therapies like Zolgensma (SMA), Luxturna (Leber’s congenital amaurosis), and Hemgenix (hemophilia B), which have helped cement its position as a market leader.Lentivirus segment is expected to show significant growth in the market. Lentivirus vectors are the preferred choice for CAR-T cell therapies (Chimeric Antigen Receptor T-cell therapies), a fast-growing area of gene therapy for cancer treatment. Lentivirus is used in ex vivo gene modification, where T cells are harvested from a patient, genetically modified using lentiviral vectors, and then re-infused into the patient. This technique has shown impressive results in blood cancers such as leukemia and lymphoma.

Market share by Indication

Based on the vector type the market is segmented into Large B-Cell Lymphoma, Multiple Myeloma, Spinal Muscular Atrophy (SMA), Acute Lymphoblastic Leukemia (ALL), Melanoma (lesions), Inherited Retinal Disease, Beta-Thalassemia Major/SCD, Others. Spinal Muscular Atrophy (SMA) is expected to dominate the gene therapy market. SMA treatments, particularly Zolgensma, have seen high sales and revenue generation, due to their one-time treatment for a lifelong condition. Zolgensma has been priced as one of the most expensive drugs in the world, highlighting the commercial potential within this segment. SMA gene therapies have received regulatory approvals not only from the FDA but also from other global regulatory bodies, ensuring their acceptance in major markets.

The Beta-Thalassemia Major/Sickle Cell Disease (SCD) segment is expected to experience the highest compound annual growth rate (CAGR) during the forecast period. Gene therapy has shown tremendous promise in treating SCD and beta-thalassemia major by addressing the underlying genetic defect in the hemoglobin gene. The development of CRISPR-Cas9 gene-editing technologies and other lentiviral vector-based gene therapies has paved the way for curative therapies that could potentially eliminate the need for lifelong blood transfusions and other symptomatic treatments.

Market share by Region

North America dominated the market in 2024 with the largest revenue share of over 20% in 2024. North America, particularly the U.S., has a highly developed healthcare system, with state-of-the-art hospitals, clinics, and research institutions. This advanced infrastructure supports the research and development (R&D) of gene therapies, from preclinical studies to clinical trials and commercialization. As of March 18, 2024, the U.S. Food and Drug Administration (FDA) has approved 36 gene therapies. The FDA is also expecting to approve 10–20 new gene therapies each year by 2025. The Asia Pacific (APAC) gene therapy market is projected to experience a significant compound annual growth rate (CAGR) of 18.40% over the forecast period. The Asia Pacific region is home to a large population, and genetic diseases like Sickle Cell Disease (SCD), Beta-Thalassemia, inherited retinal diseases, and muscular dystrophy are becoming increasingly prevalent. As these diseases require advanced treatment options, the demand for gene therapies offering curative solutions is growing.