Gene Therapy Market Size, Share & Trends Analysis Report By Indication (Acute Lymphoblastic Leukemia, Large B-cell Lymphoma), By Vector Type (Lentivirus), By Region, And Segment Forecasts, 2023 - 2030

Report Overview

The global gene therapy market was valued at USD 7.54 billion in 2022 and is expected to expand at a compound annual growth rate (CAGR) of 19.1% from 2023 to 2030. The market's growth is attributed to the factors such as expanding the area of advanced therapies along with gene delivery technologies and has developed progressive competition among key players focused on the commercialization of their therapies. The biotechnology companies are investing in acquisitions, mergers/collaborations, and expansions as key strategies to increase in-house expertise and strengthen product pipelines.

The outbreak of COVID-19 has negatively impacted market growth. This sector has experienced severe disruption as a result of COVID-19, which has historically presented significant challenges in the supply of materials, manufacturing, and logistics operations. For instance, a gene therapy company 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.

A robust gene therapy pipeline is expected to boost the gene therapy market growth in the forecast period. Researchers are working to make gene therapy available at clinics. Various universities and institutes are observed to exhibit a broad portfolio of products in the pipeline which, in turn, is expected to boost revenue generation in the near future. 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 gene therapy products, including CAR T-cell therapies and other genetically modified cell therapies are under development.

Moreover, improving regulatory support creates a growth opportunity for the gene therapy market in the forecast period. Positive changes have been made in the ways that several international regulatory organizations 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 expected 10 to 20 new cell and gene therapies to be approved each year 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 the 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.

Vector Insights

Retroviruses have shown a significant revenue growth share of more than 20.0% in 2022. Retroviruses were the most widely used viruses in gene therapy applications owing to the ease of isolation and incorporation of DNA into the virus. Retroviruses provide the ability to convert the two copies of single-stranded RNA to double-stranded DNA that can be integrated into the host cell chromosomes. A large proportion of ongoing clinical trials are being focused on using retroviruses. Furthermore, with the approval of Yescarta in October 2017, this segment witnessed significant revenue growth in 2022.

On the other hand, Adeno-Associated Viral (AAV) vectors are expected to show significant market share in the coming years. AAVs are highly in demand and their application in clinical trials is rising every year as these viruses deliver the highest efficiency in delivering the gene to the region of interest. The rise is due to the result of clinical trials relating to the progress of orthopedic and ocular gene therapy treatment showing increased efficiency and efficacy. In recent years, usage of AAV is increasing significantly across various therapeutic fields, consequently witnessing a significant boost in adoption rate during the forecast years.

Indication Insights

Spinal Muscular Atrophy (SMA) is dominating the segment in 2022 with a share of 43.44% in terms of revenue. 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 to demonstrate its effectiveness in treating SMA and altering the phenotypic of the illness. The FDA approved Novartis' Zolgensma, which aims to treat the underlying cause of SMA, approval in May 2019. 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.

Inherited retinal disease is expected to witness a significant CAGR during the forecast period. An increase in R&D activities for the development of gene therapies used for treating ocular diseases, including inherited retinal disease, has driven the segment. Luxturna is being commercialized by Spark therapeutics in the U.S. and by Novartis outside the U.S. as a treatment for a form of inherited retinal disease. Luxturna, with its approval, became the first gene therapy that can be directly administered for targeting a genetic disease caused by mutations in a single genetic factor.

Regional Insights

North America dominated the gene therapy market in 2022 and accounted for the major share of more than 65.0% of the overall revenue. 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.

Asia Pacific is estimated to be the fastest-growing regional segment from 2023 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, the local presence of major companies, and increased investment, by the governments.

Key Companies & Market Share Insights

A rise in investment and funding for the advancements in this field is anticipated to provide growth opportunities to companies. Furthermore, key players are engaged in strategic initiatives to boost their offerings in the market, which is expected to strengthen the market competition during the coming years. For instance, 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. Moreover, in April 2022, Pfizer, Inc. announced its plan to introduce its first U.S. site in the Phase 3 trials of investigational gene therapy for Patients with Duchenne Muscular Dystrophy. This initiative started after approval for restarting the global Phase 3 CIFFREO study from regulatory authorities in Canada, Taiwan, the UK, Spain, and Belgium. Some of the key players in the global 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.

Gene Therapy Market Report Scope

Global Gene Therapy Market Segmentation

This report forecasts revenue growth at global, regional, and country levels in addition to providing an analysis of the latest industry trends in each of the subsegments from 2018 to 2030. For this study, Grand View Research has segmented the global gene therapy market report based on the indication, vector type, and region:

• Indication (Revenue, USD Million; Number of Patients, 2018 - 2030)

  • 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 (Revenue, USD Million; 2018 - 2030)

  • Lentivirus

  • AAV

  • RetroVirus & gamma RetroVirus

  • Modified Herpes Simplex Virus

  • Adenovirus

  • Non-viral Plasmid Vector

  • Others

• Region (Revenue, USD Million; 2018-2030)

  • North America

    • U.S.

    • Canada

  • Europe

    • UK

    • Germany

    • France

    • Italy

    • Spain

  • Asia Pacific

    • Japan

    • China

    • India

    • Australia

  • Rest of the World