Gene therapy is an experimental technique that uses genes to treat or prevent disease. In gene therapy, a normal gene replaces a mutated gene or is introduced to compensate for the abnormal gene.

The global gene therapy market is estimated to be valued at US$ 10.37 billion in 2024 and is expected to exhibit a CAGR of 4.7% over the forecast period of 2024 to 2030.

Gene therapy has shown promise in clinical trials for treating a number of inherited and acquired diseases, with fewer side effects than drugs or other medical procedures. The technique aims to supplement, manipulate, or silence genes for therapeutic benefit.

Key Takeaways

Key players operating in the gene therapy market are Novartis AG (Switzerland), Biogen Inc. (US), Gilead Sciences, Inc. (US), Bristol-Myers Squibb (US), Alnylam Pharmaceuticals, Inc. (US), and Sarepta Therapeutics, Inc. (US) among others. These key players are focusing on development of gene therapies for genetic as well neurological disorders. For instance, Novartis received approval for Zolgensma for pediatric patients with spinal muscular atrophy in 2019.

There is high potential for growth in gene therapy as it provides long term benefits over traditional drugs and offers potential cures. Areas such as cancer, cardiovascular diseases, infectious diseases and neurodegenerative disorders are creating opportunities for development of gene therapies.

Major players are expanding globally to capture market opportunities. For example, Biogen expanded manufacturing facilities in North Carolina to support production of gene therapies. Gilead Sciences partnered with Sangamo Therapeutics to develop gene regulation therapies for genetic disorders.

Market Drivers

Rising prevalence of genetic disorders is a key factor driving growth of the gene therapy market. The technology offers promise for conditions with no effective treatment options. Additionally, increasing R&D investments from government as well as private organizations is supporting development of novel gene therapies. For instance, National Institutes of Health is investing heavily in gene therapy research.

Market Restraints

High costs associated with development and manufacturing are a major challenge for the gene therapy market. The regulatory landscape is also stringent which increases complexity and time required to commercialize therapies. Safety concerns over long term effects and payload delivery methods can also limit widespread adoption of gene therapies.

Segment Analysis

The Gene Therapy Market Growth can be segmented according to vector type, application, and region. Viral vectors dominate the market currently, accounting for over 70% share owing to their high transfection efficiency. Retroviruses and adenoviruses are commonly used viral vectors for gene therapy. However, adenoviruses face challenges regarding immune responses. Among non-viral vectors, naked/plasmid DNA holds significant potential due to its safety and large transgene capacity but faces low transfection efficiency issues. In terms of application, oncology is the leading segment and is projected to witness high growth during the forecast period. This is due to the rising cancer incidence and the approval of multiple gene therapy products for cancer treatment. Neurological disorders are also expected to provide lucrative opportunities owing to the rising R&D in this application area.

Global Analysis

North America dominates the global gene therapy market currently due to supportive regulatory environment, strong research base and presence of key market players. Within North America, the US gene therapy market witnesses high growth and is expected to maintain its lead during the forecast period. Europe is also a major regional market driven by ongoing clinical research from countries including the UK, Germany, Spain and France. The Asia Pacific region is anticipated to emerge as the fastest growing market during the forecast period with China and Japan being the major countries. This growth can be attributed to increasing healthcare expenditure, rising incidence of chronic diseases and growing focus of Western companies on emerging Asia-Pacific countries.