Gene Therapy - Market Insights, Competitive Landscape, and Market Forecast - 2030

The global gene therapy market is estimated to grow at a CAGR of 19.28% during the forecast period from 2024 to 2030. The demand for gene therapy is primarily being boosted by the rising prevalence of serious genetic disorders such as spinal muscular atrophy, hemophilia, retinitis pigmentosa, and others, increasing number of pharmaceutical trials for gene therapy, growing advances in gene editing tools, and others are further contributing in the overall growth of the gene therapy market during the forecast period from 2024-2030.

Gene Therapy Market Dynamics:

The increasing number of individuals with genetic disorders and the growing therapeutic importance of gene therapy in their cure and management is the major factor driving the growth of the gene therapy market. As per the report published by the World Federation of Hemophilia 2022, globally about 347,026 people were reported to be suffering from bleeding disorders in 2020. As per the same source in 2020, nearly around 209,614 people had hemophilia which includes 165,379 cases of hemophilia A, 33,076 cases of hemophilia B, and 11,159 cases of hemophilia type unknown or type not reported.

The above-mentioned source further stated that 84,197 were reported to have Von Willebrand disease and 53,215 people were suffering from other bleeding disorders across the world in 2020.

Hemgenix (etranacogene dezaparvovec) is CE marked and US Food and Drug Administration-approved gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency).

Retinal dystrophies (RD) associated with progressive visual dysfunction and complete blindness in some patients are a group of degenerative disorders of the retina with clinical and genetic heterogeneity. Gene therapy can be used for the effective treatment and management of the patient suffering from this condition. For example, LUXTURNA by Spark Therapeutics, Inc. under F. Hoffmann-La Roche Ltd. is US FDA approved prescription gene therapy product indicated for the treatment of patients with inherited retinal disease due to mutations in both copies of the RPE65 gene.

The rapid expansion of the gene therapy pipeline is expected to escalate the market for the same. For instance, in December 2022 Pfizer Inc., announced positive top-line results from the Phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B.

However, ethical issues associated with gene therapy diagnostics and the high treatment cost of gene therapy may restrict the growth of the overall gene therapy market.

The gene therapy market was largely impacted during the period of COVID-19 pandemic owing to lockdown impositions as a measure to prevent the spread of COVID-19 infection which led to the suspension of research and development activities based on gene therapy owing to the lack of availability of raw materials and required workforce. Nevertheless, the gene therapy market is in a recovery phase owing to the introduction of vaccines against COVID-19 and the resumption of activities across all sectors including healthcare and pharmaceutical, thereby providing ample opportunities for the gene therapy market to grow at significant revenue during the forecast period.

Gene Therapy Market Segment Analysis:

Gene Therapy Market by Vector Type (Viral and Non-Viral), Delivery Type (Ex Vivo and In Vivo), Indication (Neurological, Oncological, Hematological, Ophthalmological, and Others), and Geography (North America, Europe, Asia-Pacific, and Rest of the World)

In the vector type segment of the gene therapy market, the viral vector category is expected to amass a significant revenue share in the year 2023.

The ability of viruses to naturally infect cells and to efficiently transport gene materials of interest into host cells makes them the suitable vehicle for gene therapy. Viruses can deliver nucleic acid content to specific cell types while avoiding immunosurveillance by an infected host.

A plethora of viral vectors including adenovirus (Ad), adeno-associated virus (AAV), and herpes simplex virus (HSV) has been employed for the safe and efficient delivery of gene therapies.

Adeno-associated virus (AAV) is the most commonly used viral vector for in vivo gene therapy applications. It has several essential characteristics including the ability to transduce both diving and non-dividing cells and enable long-term, stable gene expression which makes them ideal for gene therapy. AAV do not integrate DNA into the host genome and also have low immunogenicity.

Adenoviruses facilitate a high level of gene expression by delivering multiple copies of the genome into one host cell. They can transduce both dividing and non-dividing cells.

The viral vectors can be designed and developed for both short-term and permanent long-term gene expression.

For example, Zolgensma intended for the treatment of children suffering from spinal muscular atrophy (SMA) is an adeno-associated virus (AAV) vector-based gene therapy. The vector delivers a fully functional copy of the human SMN gene into the target motor neuron cells. Zolgensma results in the expression of the SMN protein in a child’s motor neurons, which improves muscle movement and function, and the survival of a child with SMA.

Therefore, considering the advantages associated with a viral vector and their growing usage, this category is expected to generate considerable revenue thereby pushing the overall growth of the global gene therapy market during the forecast period.

North America is expected to dominate the overall Gene Therapy Market:

Among all the regions, North America is estimated to account for the largest share of the Gene Therapy Market in the year 2023. Owing to the significance of key growth factors such as the increasing patient population suffering from genetic disorders, the rising incidence of cancer, sophisticated healthcare infrastructure, the presence of key players, and fast regulatory approval for new products, among others, the market for gene therapy is expected to register significant growth in the region.

According to National Center on Birth Defects and Developmental Disabilities under the Centers for Disease Control and Prevention (CDC) 2022, sickle cell anemia affects about 100,000 Americans. As per the same source, Hemophilia A affects 1 in 5,000 male births and each year about 400 babies are born with hemophilia A in the United States.

The data published by CDC 2022 says that every year 57,000 men and 18,000 women get bladder cancer in the United States. As per Global Cancer Observatory 2020, about 80.6 thousand cases of bladder cancer were reported in the US in 2020 and by 2040 this number is expected to reach 122 thousand.

In January 2019, US Food and Drug Administration released a statement based on an assessment of the gene therapy pipeline and the clinical success rates of these products, that by 2025, FDA will be approving 10 to 20 gene therapy products a year. Also, it was estimated by FDA that by 2020 they will be receiving 200 investigational new drug (INDs) applications per year. Thus, an increasing number of product development activities in the region is going to accelerate the growth of the gene therapy market.

Hence, the above-mentioned factors are going to increase the growth of the gene therapy market in the region.

Gene Therapy Market Key Players:

Some of the key market players operating in the gene therapy market include Novartis AG, F. Hoffmann-La Roche Ltd., Ferring B.V., bluebird bio, Inc., Ultragenyx Pharmaceutical Inc., PTC Therapeutics, BioMarin, Biogen, Prevail Therapeutics, CSL, and others.

Recent Developmental Activities in the Gene Therapy Market:

• In February 2023, CSL received the European Commission approval for Hemgenix which is the first gene therapy to gain EU approval for hemophilia B.
• In December 2022, Ferring Pharmaceuticals announced that it has received the US Food and Drug Administration (FDA) approval for Adstiladrin® (nadofaragene firadenovec-vncg), a novel adenovirus vector-based gene therapy, for the treatment of adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.
• In August 2022, PTC Therapeutics, Inc. received marketing authorization for Upstaza (eladocagene exuparvovec), the first approved gene therapy treatment developed for aromatic L-amino acid decarboxylase (AADC) deficiency from European Commission.

Key Takeaways from the Gene Therapy Market Report Study

• Market size analysis for current gene therapy market size (2023), and market forecast for 6 years (2024-2030)
• The effect of the COVID-19 pandemic on this market is significant. To capture and analyze suitable indicators, our experts are closely watching the gene therapy market.
• Top key product/services/technology developments, mergers, acquisitions, partnerships, and joint ventures happened for the last 3 years
• Key companies dominating the global gene therapy market.
• Various opportunities available for the other competitor in the gene therapy market space.
• What are the top-performing segments in 2023? How these segments will perform in 2030?
• Which are the top-performing regions and countries in the current gene therapy market scenario?
• Which are the regions and countries where companies should have concentrated on opportunities for gene therapy market growth in the coming future?

Target Audience who can be benefited from this Gene Therapy Market Report Study

• Gene Therapy products providers
• Research organizations and consulting companies
• Gene Therapy- related organizations, associations, forums, and other alliances
• Government and corporate offices
• Start-up companies, venture capitalists, and private equity firms
• Distributors and Traders dealing in gene therapy
• Various End-users who want to know more about the gene therapy market and the latest innovations and developments in the gene therapy market.

Frequently Asked Questions for the Gene Therapy Market:

1. What is gene therapy?

Gene therapy is a technique that involves modification or manipulation of a person’s genes to treat or cure disease. It can be done by several mechanisms including the replacement of a disease-causing gene with a healthy copy of a gene, the inactivation of a disease-causing gene, and the introduction of a new or modified gene into the body to help treat a disease.

2. What is the market for global gene therapy?

The global gene therapy market is estimated to grow at a CAGR of 19.28% during the forecast period from 2024 to 2030.

3. What are the drivers for the global gene therapy market?

The growth of the gene therapy market is primarily being boosted by the increasing therapeutic importance of gene therapy, the rapid expansion of the gene therapy pipeline, advances in gene editing technology, and rising research and development activities related to gene therapy are contributing to the overall growth of the gene therapy market during the forecast period from 2024-2030.

4. Who are the key players operating in the global gene therapy market?

Some of the key market players operating in the gene therapy market include Novartis AG, F. Hoffmann-La Roche Ltd., Ferring B.V., bluebird bio, Inc., Ultragenyx Pharmaceutical Inc., PTC Therapeutics, BioMarin, Biogen, Prevail Therapeutics, CSL, and others.

5. Which region has the highest share in the gene therapy market?

North America is expected to dominate the overall gene therapy market during the forecast period from 2024-2030. Factors such as the increasing patient population suffering from genetic disorders, the rising incidence of cancer, sophisticated healthcare infrastructure, the presence of key players, and fast regulatory approval for new products, among others, are driving the growth of the gene therapy market in the region.


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