Global Gene Therapy Market Size, Demand Grows Tremendously at a CAGR of 18.60%

Introduction

Gene therapy prevents the progression of an illness by altering the expression of mutated genes or the biological characteristics of living cells for therapeutic purposes. It includes viral vectors, bacterial vectors, plasmid deoxyribonucleic acid (DNA), human gene editing technology, and patient-derived cellular gene therapy. It entails inserting a healthy gene into an individual's genome to correct a mutation causing a genetic disorder.

Numerous gene therapy treatments exist, including ex vivo, in vivo, and in situ gene therapy. Gene therapy functions by inhibiting, amplifying, and removing cells that cause disease. It can eliminate disease symptoms permanently and cure diseases for which there is no preventative medication.

Market Dynamics

Growing Prevalence of Target Diseases Like Cancer Drives the Global Market

In recent years, the prevalence of chronic diseases, such as cancer, cardiovascular disease, and others, has surged dramatically, resulting in a significant increase in the healthcare expenditures of individuals. Gene therapy is more effective than conventional medicine; as a result, it is gaining popularity among cancer patients and those with other diseases, driving market expansion.

In 2019, according to information published by the International Agency for Research on Cancer, one in five people will be diagnosed with cancer during their lifespan, and one in eight men and one in eleven women will perish due to the disease. Consequently, the growing burden of cancer increases the burden of diagnostic facilities, which will significantly contribute to the market's growth.

Technological Advancements Creates Tremendous Opportunities

Rapid advances in biomedical sciences may lead to the creation of innovative and sophisticated gene therapy techniques. Gene therapy is advancing towards perfection by utilizing a diverse range of state-of-the-art technology. It is currently positioned to capitalize on promising biotechnology revolutions. According to the March 2021 Frontier in Genome Edition article titled "CRISPR/Cas: Advances, Limitations, and Applications for Cancer Precision Research," CRISPR/Cas is a technology capable of modifying the genomes of eukaryotic cells in living organisms, making it one of the most significant scientific advances of the twenty-first century. Technological advances have made applying this method to basic and clinical research feasible in recent years. Therefore, technological advancements in CRISPR gene therapy will aid in diagnosing numerous chronic diseases, likely creating opportunities in the gene therapy market.

Regional Analysis

North America is the most significant global gene therapy market 1share and is estimated to exhibit a CAGR of 24.81% during the forecast period. The market for gene therapy in North America is driven by the robust regulatory framework for cellular therapy development and the presence of numerous biopharmaceutical companies. These organizations are increasingly endorsing gene therapy technologies, which is expected to stimulate market growth. In May 2019, AveXis, a subsidiary of Novartis, announced that the US FDA had approved the use of Zolgensma (onasemnogene abeparvovec-xioi) to treat pediatric patients with spinal muscular atrophy (SMA) who are younger than two years old and have bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Additionally, according to the Canadian Agency for Drugs and Technologies in Health (CADTH), Canadian researchers are currently or have recently participated in a large number of gene therapy trials, including two of the three trials for the drug alipogene tiparvovec for monogenic lipoprotein lipase deficiency, which has a high prevalence in Quebec due to the founder effect (151–153). Consequently, the factors above will likely contribute to future market expansion.

Key Highlights

• The global gene therapy market size was valued at USD 6.30 billion in 2024 and is projected to reach from USD 7.47 billion in 2025 to USD 29.24 billion by 2033, growing at a CAGR of 18.60% during the forecast period (2025-2033).
• Based on indication, the global gene therapy market is bifurcated into cancer, metabolic disorders, eye disorders, spinal muscular atrophy, and other indications.The cancer segment dominates the global market and is projected to exhibit a CAGR of 25.25% over the forecast period.
• Based on technology, the global gene therapy market is segmented into adenovirus vectors, adeno-associated virus vector, lentiviral vector, retroviral vector, herpes virus vector, and other technologies. The adeno-associated virus vector segment owns the highest market share and is estimated to exhibit a CAGR of 26.36% during the forecast period.
• North America is the most significant global gene therapy market by market share and is estimated to exhibit a CAGR of 24.81% during the forecast period.

Competitive Players

1. REGENXBIO, Inc.
2. Oxford BioMedica plc
3. Dimension Therapeutics, Inc.
4. Bristol-Myers Squibb Company
5. SANOFI
6. Applied Genetic Technologies Corporation
7. F. Hoffmann-La Roche Ltd
8. Bluebird Bio, Inc.
9. Novartis AG
10. Taxus Cardium Pharmaceuticals Group, Inc. (Gene Biotherapeutics)
11. UniQure N.V.
12. Shire Plc
13. Cellectis S.A.
14. Sangamo Therapeutics, Inc
15. Orchard Therapeutics
16. Gilead Lifesciences, Inc.
17. BENITEC BIOPHARMA
18. Sibiono GeneTech Co., Ltd
19. Shanghai Sunway Biotech Co., Ltd.
20. Gensight Biologics S.A.
21. Transgene
22. Calimmune, Inc.
23. Epeius Biotechnologies Corp.
24. Astellas Pharma Inc.
25. American Gene Technologies
26. BioMarin Pharmaceuticals, Inc.

Recent Developments

• June 2023: Laurus Labs Ltd. announced that it signed a MoA with IIT Kanpur (IITK) for in-licensing and commercializing novel gene therapy assets using adeno-associated virus (AAV) vectors.
• June 2023: Sarepta Therapeutics, Inc. announced that the FDA accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory pediatric patients aged 4-5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.

Segmentation

1. By Type
   1. Gene silencing
   2. Cell replacement
   3. Gene augmentation
   4. Other therapies
2. By Vector
   1. Viral vectors
   2. Non-Viral vectors
3. By Therapeutic area
   1. Neurology
   2. Oncology
   3. Hepatology
   4. Other therapeutic areas
4. By Delivery method
   1. In Vivo
   2. Ex Vivo
5. By Route of administration
   1. Intravenous
   2. Other routes of administration
6. By Indication
   1. Cancer
   2. Metabolic Disorders
   3. Eye Disorders
   4. Spinal Muscular Atrophy
   5. Other Indications
7. By Technology
   1. Adeno Virus Vector
   2. Adeno-associated Virus Vector
   3. Lentiviral Vector
   4. Retroviral Vector
   5. Herpes Virus Vector
   6. Other Technologies