• In January 2024, Biogen and Ginkgo Bioworks announced that they have completed the gene therapy collaboration involving AAV based vectors. This is expected to fuel the demand for gene therapies in coming years.

• In December 2023, Swiss Agency for Therapeutic Products granted approval to Libmeldy for treatment of early-onset metachromatic leukodystrophy.

• In May 2023, Krystal Biotech was granted approval for VYJUVEK gene therapy for Dystrophic Epidermolysis Bullosa treatment

• In June 2023, the U.S. FDA granted approval to Sarepta for ELEVIDYS gene therapy to treat DMD in children of age 4-5 years

• In January 2023, Voyager Therapeutics and Neurocrine Biosciences entered into a strategic collaboration for commercialization & development of Voyager’s GBA1 program and other next-generation gene therapies for neurological diseases

• In January 2023, Spark Therapeutics and Neurochase established a strategic collaboration to develop Neurochase’s unique delivery technology for use with selected gene treatments for rare disorders in the CNS. In this agreement, Neurochase will contribute its extensive knowledge in direct drug delivery technology to Spark’s premier AAV platform.

• 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.

In this report, the Global Gene Therapy Market has been segmented by Indication Type, Therapeutic Area, Technology, Application, and Geography.

Global Gene Therapy Market, Segmentation by Indication Type

The Global Gene Therapy Market has been segmented by Indication Type into Cancer, Cardiovascular Indication, Infectious diseases indication, Genetic diseases indication, Neurological diseases indication and Others.

The gene therapy market for cancer represents a significant share, accounting for approximately 35% of the overall market. This dominance is due to the rising incidence of various cancers and the growing adoption of targeted gene-based treatments. Innovative therapies like CAR-T cell therapy are driving growth, offering new hope for previously untreatable cancers.

Gene therapy applications in cardiovascular diseases contribute to nearly 15% of the market. With the increasing prevalence of heart conditions globally, especially in aging populations, gene therapy is emerging as a promising intervention. It focuses on enhancing vascular regeneration and improving cardiac function through genetic modification.

The segment for infectious diseases accounts for around 10% of the gene therapy market. Innovations in viral vector technologies and the need for effective treatments against pathogens like HIV and hepatitis have propelled this segment. Gene editing tools are enabling the development of durable therapeutic responses.

Holding a robust 25% share, gene therapy for genetic disorders is one of the fastest-growing segments. Conditions such as cystic fibrosis, sickle cell anemia, and muscular dystrophy are being addressed with curative intent. Advances in CRISPR and other gene-editing technologies are transforming treatment paradigms.

Neurological diseases represent about 10% of the gene therapy market. Disorders like Parkinson’s, ALS, and Huntington’s are witnessing increased investment in gene-based treatments. The ability of gene therapy to target the central nervous system offers promising breakthroughs in treating these complex neurodegenerative disorders.

This category includes various emerging indications and contributes roughly 5% to the gene therapy market. Conditions like ophthalmologic disorders and autoimmune diseases are gradually becoming viable candidates for gene-based interventions. Continued research and clinical trials are expanding the scope of gene therapy beyond traditional applications.

Global Gene Therapy Market, Segmentation by Therapeutic Area

The Global Gene Therapy Market has been segmented by Therapeutic Area into Oncology, Genetic Disorders, Infectious Diseases, Cardiovascular Diseases, and Neurological Disorders.

Oncology dominates the global gene therapy market, accounting for nearly 40% of the total share. The growing burden of cancer and the shift towards personalized medicine are fueling demand for innovative gene-based treatments. Therapies like CAR-T cells and oncolytic viruses are gaining traction for their precision and effectiveness.

Genetic disorders hold a substantial market share of around 30%, driven by the need for curative therapies for conditions like sickle cell anemia, Duchenne muscular dystrophy, and cystic fibrosis. The progress in gene editing technologies such as CRISPR has significantly enhanced treatment outcomes for inherited diseases.

Infectious diseases contribute approximately 12% to the gene therapy market. The focus on long-term solutions for conditions like HIV and hepatitis has led to increased research in this area. Gene therapies offer the potential for immune modulation and permanent suppression of viral replication.

Cardiovascular diseases make up nearly 10% of the market, as gene therapies aim to address ischemic heart disease, heart failure, and arterial disorders. By promoting angiogenesis and improving cardiac repair, gene-based treatments are reshaping cardiovascular care.

Neurological disorders account for roughly 8% of the gene therapy market. Conditions like Parkinson’s disease, ALS, and spinal muscular atrophy are receiving increasing attention. Gene therapy enables targeted delivery to the nervous system, offering hope for diseases once deemed untreatable.

Global Gene Therapy Market, Segmentation by Technology

The Global Gene Therapy Market has been segmented by Technology into Adeno Virus Vector, Adeno-associated Virus Vector, Lentiviral Vector, Retroviral Vector, Herpes Virus Vector, and Others.

The Adeno virus vector segment accounts for approximately 22% of the gene therapy market. Known for its high gene delivery efficiency, this vector is widely used in oncology and vaccination therapies. Its ability to infect both dividing and non-dividing cells makes it a preferred option in various therapeutic applications.

Adeno-associated virus (AAV) vectors hold the largest share, contributing nearly 30% to the market. Their low immunogenicity and ability to provide long-term gene expression have made AAV vectors a standard in treatments for genetic disorders and neurological diseases.

Lentiviral vectors represent around 20% of the market and are extensively used for ex vivo gene therapy in treating hematologic disorders. Their ability to integrate into the host genome ensures stable gene expression, a critical feature for long-lasting therapies.

The retroviral vector segment comprises about 10% of the market. While similar to lentiviral vectors, retroviral vectors are mainly limited to dividing cells. They have proven effective in earlier gene therapy trials for immune deficiencies and select cancer therapies.

Herpes virus vectors contribute nearly 8% of the gene therapy market. Their large genome capacity and ability to remain latent in nerve cells make them ideal for treating neurological disorders. However, safety and complexity issues continue to limit their broader adoption.

The others category, accounting for around 10%, includes emerging vector technologies like non-viral methods and hybrid vectors. These are gaining traction due to their reduced risk of immunogenic reactions and potential for customized gene delivery solutions.

Global Gene Therapy Market, Segmentation by Application

The Global Gene Therapy Market has been segmented by Application into Research, Clinical, and Commercial.

The research segment dominates the application-based gene therapy market, holding around 45% of the total share. This is driven by significant investments in preclinical studies and basic gene function analysis. Academic institutions and biotech firms are leading efforts to explore new gene delivery mechanisms and therapeutic targets.

The clinical application segment contributes approximately 35% to the market. Numerous clinical trials are underway globally, focusing on both rare genetic disorders and common chronic diseases. The rising number of regulatory approvals for gene-based treatments is accelerating market growth in this segment.

Holding about 20% market share, the commercial segment is steadily expanding as more gene therapies receive regulatory clearance. The focus is on the development and distribution of FDA-approved gene therapies for conditions like spinal muscular atrophy and inherited blindness. This marks the transition from experimental to widely accessible treatments.

Global Gene Therapy Market, Segmentation by Geography

In this report, the Global Gene Therapy Market has been segmented by Geography into five regions; North America, Europe, Asia Pacific, Middle East and Africa and Latin America.

Global Gene Therapy Market Share (%), by Geographical Region, 2024

North America leads the global gene therapy market, capturing nearly 45% of the total share. The region benefits from robust R&D infrastructure, significant government funding, and the presence of major biopharmaceutical companies. The U.S. is a frontrunner in clinical trials and gene therapy approvals.

Europe accounts for approximately 25% of the global gene therapy market. Countries like Germany, the UK, and France are investing heavily in genomic medicine and biotech innovations. The region has a strong focus on regulatory frameworks that support advanced therapeutic development.

The Asia Pacific region contributes about 18% to the gene therapy market and is witnessing rapid growth. Rising healthcare investments, expanding biotech ecosystems, and supportive government initiatives in countries like China, Japan, and South Korea are key drivers.

Middle East and Africa hold a modest share of around 7% in the gene therapy market. Although adoption is still emerging, there is increasing interest in personalized medicine and genomic healthcare solutions across countries like UAE and South Africa.

Latin America represents nearly 5% of the market, with Brazil and Mexico being the major contributors. The region is gradually embracing gene-based research and expanding access to advanced therapeutic solutions through public-private collaborations.