Gene therapy is a pioneering treatment method that uses genes to treat diseases. Gene therapy offers a promising approach to treat and cure diseases by introducing genetic material into a patient’s cells to replace or correct abnormal genes. Gene therapy can be classified into in-vivo and ex-vivo gene therapy. This innovative treatment method has gained significant attention and investment in recent years, leading to advancements in gene therapy techniques and technologies. In addition, the development of advanced gene editing technologies, such as CRISPR-Cas9, has revolutionized the field of gene therapy. These technologies allow for precise and efficient modification of genes, making gene therapy more feasible and effective in treating genetic diseases. The increasing burden of genetic diseases and the rising number of FDA approvals of gene therapies are noteworthy factors contributing to the growing gene therapy market size. However, the high cost of gene therapy products hinders the market growth. Moreover, advancements in gene therapy technologies are expected to bring new gene therapy market trends in the coming years.
Increasing Burden of Genetic Diseases Drives Gene Therapy Market Growth
The prevalence of genetic diseases is rising worldwide and affecting the health of people at a severe level. Genetic diseases have numerous symptoms that are uncommon and are mostly noncurable. Most genetic diseases are rare and are developed due to mutations in the genetic makeup of individuals. According to the University of Sheffield research, ~300 million people worldwide have genetic diseases. According to the World Health Organization (WHO) 2021 data, 10 out of every 1,000 people are affected by single-gene diseases, signifying that 70 million and 80 million people worldwide live with any one type of single-gene disease. As per Global Genes, ~7,000 known rare diseases and disorders have been identified worldwide, and more are being discovered yearly.
In 2023, a report by MJH Life Sciences (US) estimated that 300,000 newborns worldwide are born with sickle cell disease (SCD) yearly, accounting for ∼5% of the global population. According to a study on sickle cell disease published in the National Library of Medicine, 400,000 newborns are expected to have sickle cell disease by 2050.
As per the study titled “Sickle Cell Disease,” published in August 2023, 1 in 500 African Americans is affected by SCD. In June 2020, as per Novartis AG (Switzerland) report, ∼15,000 individuals were suffering from SCD in the UK, and 270 newborns are diagnosed with the condition every year. In addition, the disease is most prevalent in emerging nations such as India. Among India’s tribal communities, ∼18 million people are affected by sickle cell trait, and 1.4 million people are suffering from SCD.
Per the Bristol Myers Squibb article, ~80–90 million people (1.5% of the global population) are carriers of beta-thalassemia. ~300 million people worldwide have the thalassemia trait, which puts them at risk of having children with some form of thalassemia. In addition, according to the Muscular Dystrophy Association, in North America and Europe, the prevalence of Duchenne muscular dystrophy is ~6 per 100,000 individuals.
Gene therapy offers a potential solution to treat and cure these diseases by targeting the underlying genetic cause of the disease, providing hope for patients and their families. This innovative treatment method has gained significant attention recently, leading to advancements in gene therapy techniques and technologies. Thus, as the burden of genetic diseases continues to rise, there is a growing need for gene therapy, which bolsters the gene therapy market growth.
Gene Therapy Market: Segmental Overview
The gene therapy market is segmented into vectors, indication, and delivery mode. Based on vectors, the market is bifurcated into non-viral vectors and viral vectors. The viral vectors segment held a significant gene therapy market share in 2023 and is projected to register a higher CAGR during 2023–2031. According to the analysis from clinicaltrials.gov, ~58% of gene therapies under development are using viral vectors. The most commonly used viral vector for gene therapy is adeno-associated virus, owing to its ability to deliver genes to non-dividing cells where the gene has a long-term therapeutic impact. Different varieties of adeno-associated viruses target different cells, making them a suitable transporter of genetic material to desired locations inside the body, providing a customized treatment. In addition, viral vectors display low immunogenicity, safety, and long-term transient expression.
Gene Therapy Market: Competitive Landscape and Key Developments
Novartis AG, Astellas Pharma Inc., Bristol-Myers Squibb Company, Bluebird Bio Inc., CSL Behring, Sanofi, F. Hoffmann-La Roche Ltd, Daiichi Sankyo, Biogen, and Oxford Biomedica are a few key companies operating in the market. These companies focus on product innovation strategies to meet evolving customer demands, along with maintaining their brand name in the gene therapy market.
A few recent developments initiated in the global gene therapy market report are mentioned below:
- In January 2024, Biogen and Ginkgo Bioworks successfully completed the gene therapy collaboration to boost the productivity of the gene therapy manufacturing platform. Together, the companies seek to redefine the industry standard for producing recombinant adeno-associated virus-based vectors.
- In May 2023, Krystal Biotech Inc. received US Food and Drug Administration (FDA) approval for VYJUVEK gene therapy for the treatment of dystrophic epidermolysis bullosa patients aged six months or older. VYJUVEK is the first-ever redosable gene therapy and the only medicine approved by the FDA for the dystrophic epidermolysis bullosa treatment.
- In January 2023, Spark Therapeutics entered into a strategic collaboration with Neurochase to develop Neurochase’s unique delivery technology for use with selected gene therapy treatments for rare disorders in the central nervous system. Through the agreement, Neurochase will contribute its extensive knowledge in direct drug delivery technology to Spark’s leading adeno-associated virus platform.
- In January 2023, Voyager Therapeutics Inc. and Neurocrine Biosciences Inc. entered into a strategic collaboration to advance the development and commercialization of Voyager’s GBA1 program and other next-generation gene therapies for the treatment of neurological diseases. In addition, Neurocrine Biosciences and Voyager have agreed to work together on three new gene therapy programs directed to rare central nervous system targets.