Gene Therapy Market

SKU: DMBT1341 | Last Updated On: Nov 30 2022 | Available Formats

> Gene Therapy Market Expected to reach a high CAGR of 33.4% By 2029

Gene Therapy Market is segmented By Therapy Type (Somatic Gene Therapy, Germline Therapy), By Application Type (Cardio, Neuro, Oncology, Infectious Diseases, Inherited Diseases, Others), and By Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2022-2029.

[150 Pages Report] Gene therapy market is expected to grow at a CAGR of 33.4% during the forecast period (2022-2029).

Gene therapy is introduction of DNA into a patient to treat a genetic disease. The new DNA usually contains a functioning gene to correct the effects of a disease-causing mutation. Researchers are studying gene therapy for a number of diseases such as severe combined immune deficiencies, hemophilia, Parkinson's disease, cancer, and even HIV through a number of approaches.

Market Dynamics

  • Advancements in treatments to cure diseases such as cancer, neuro, and cardio diseases are driving the global gene therapy market in recent years and anticipated to remain the same over the forecast period. For instance, in 2017, in France, researchers from Bluebird Bio announced that a teenage boy in France had been cured of sickle-cell disease after receiving experimental gene therapy. Scientists removed stem cells from the boy’s bone marrow and modified them in the lab by introducing copies of a gene to prevent his red blood cells from becoming sickled. 
  • Rising prevalence of chronic diseases such as cancer, asthma, diabetes, and COPD are boosting the requirement of gene therapy over the forecast period. For instance, according to the National Cancer Institute in 2018, in the United States, an estimated 1,735,350 new cases of cancer will be diagnosed and 609,640 people will die from the disease.
  • An increasing number of gene therapy start-ups in 2019 is expected to be one of the major factors driving the growth of the market during the forecast period (2019-2026). For instance, in January 2019, Apic Bio launched with $40 million to treat ALS and liver diseases. Also, Neurogene launched with $68.5 million to tackle several neurological and lysosomal storage disorder diseases.
  • However, the high cost of gene therapies and stringent regulatory framework are hampering the global gene therapy market over the forecast period. The cost of these therapies can be extremely expensive and present a large burden to the healthcare system, in the range of $400,000 to $850,000 at the high end, currently, for the approved gene therapies, and could be amplified depending on the size of the patient population. According to estimates from the Institute for Clinical and Economic Review, if gene therapies were used just by one in 10 patients who had a genetic condition, the cumulative budget impact of therapies priced at $1m–$2m (which some may consider an overestimate now) could reach $3tn in the US alone. 
  • As the pipeline advances, the demand grows which leads gene therapy developers to turn towards contract manufacturing organizations (CMOs) or contract development and manufacturing organizations (CDMOs) for vector supply services. The demand for manufacturing will provide lucrative opportunities for CMOs and CDMOs in the specialized market of cell and gene therapy, but that demand will likely exceed the supply. 

Market Segmentation

  • Based on application, the global gene therapy market is segmented into oncology, neurological diseases, inherited diseases, cardiac diseases, and infectious diseases. Oncology had the highest market share in 2018 and is expected to remain dominant same over the forecast period.  One-third of the gene therapy pipeline consists of candidates in development for cancer. About 43% of the rare disease gene therapy pipeline is considered rare oncologic diseases. Cancers for which gene therapy products are being developed include glioblastoma, metastatic melanoma, head and neck cancer, prostate cancer, renal cell cancer, and colorectal cancer. For instance, in 2017, FDA approved gene therapy treatment for cancer known as CAR-T therapy. The therapy is designed to treat an often-lethal type of blood and bone marrow cancer that affects children and young adults and will be sold by Novartis for $475,000. 
  • Gene therapy applications in cardio diseases have the second-largest share in recent years due to increasing number of heart-related diseases. According to the World Health Organization in 2017, 17.9 million people died from CVDs in 2016 globally, representing 31% of all global deaths. Researchers and scientists are have been working effectively on gene therapy in cardiology areas to provide solutions.
  • Based on therapy type, the global gene therapy market is categorized into somatic gene therapy and germline gene therapy. In somatic gene therapy, transfer of a section of DNA to any cell of the body that doesn’t produce sperm or eggs whereas in germline gene therapy, transfer of a section of DNA to cells that produce eggs or sperm.

Gene Therapy Market - Strategic Insights



Market CAGR


Segments Covered

By Therapy Type, By Application Type, and By Region

Report Insights Covered

Competitive Landscape Analysis, Company Profile Analysis, Market Size, Share, Growth, Demand, Recent Developments, Mergers and acquisitions, New Product Launches, Growth Strategies, Revenue Analysis, and Other key insights.

Fastest Growing Region

Asia Pacific

Largest Market Share 

North America

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Geographical Share

  • North America holds the dominant market share for plasma fractionation in 2018, and it is expected to maintain the growth over the forecast period, owing to the rapid advancements in gene therapy market. For instance, in 2018, Spark Therapeutics developed Luxturna that was approved by the Food and Drug Administration (FDA) for treatment of RPE65-mediated inherited retinal dystrophy. Luxturna is the first targeted gene therapy approved in the US, representing a huge milestone in medical care. The American Society of Clinical Oncology’s (ASCO), Clinical Cancer Advances Report 2018 recently named adoptive immunotherapy with chimeric antigen receptor T cells (CART T as the most clinical advance of the year.)
  • Europe has the second largest market share in global gene therapy market due to increasing number of chronic diseases such as cancer and heart attack. According to the WHO, with more than 3.7 million new cases and 1.9 million deaths each year, cancer represents the second most important cause of death and morbidity in Europe. Hence, with rising research and development for the treatment of chronic diseases, the market for gene therapy is growing in this region.
  • Hemophilia is a challenging market by biotechs across Europe and North America competing to launch the first gene therapy. Gene therapy emerges as a potential new treatment for patients with hemophilia. In July 2019, the International Society on Thrombosis and Haemostasis (ISTH) launched Gene Therapy in Hemophilia: An ISTH Education Initiative. The France Foundation collaborated with the ISTH to educate clinicians around the world about the science and potential role of gene therapy for patients with hemophilia.

Competitive Analysis

  • Key pharmaceutical companies are investing significant money in the clinical research and development of gene therapy products. Competition is immense in global gene therapy market and companies see a huge opportunity in gene therapy market.
  • The key players in gene therapy market are Novartis AG, UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Kite Pharmaceuticals, Gilead Sciences, Inc, Human Stem Cell Institute, Shire Plc, Cellectis, and GlaxoSmithKline plc.
  • Partnerships provide an important vehicle for both the licenser and licensee to grow. Over the 2012–18 time period (through September 2018), 351 gene therapy alliances were signed, with a total deal value of $41bn. The players are adopting various key strategies such as product launches, investments, mergers & acquisitions which are leading to expanding their business globally, hence contributing to the growth of the gene therapy market. For instance,
  • In May 2019, Novartis, a Swiss pharmaceutical company received the US regulatory approval for a gene therapy that treats a rare childhood disorder and has a price tag of US$2.1 million, making it the most expensive drug in history.
  • In May 2019, ElevateBio raised $150 million to launch cell and gene therapy incubator.
  • On February 15, 2019, Gene therapy pioneer James Wilson launches startup Passage Bio with $115.5 million. It has raised $115.5 million in series financing to develop gene therapies for rare genetic diseases of the central nervous system.
  • In 2019, Novartis has a deal with Spark to market Luxturna in Europe, and is therefore at the forefront of not only the groundbreaking R&D behind cell and gene therapy, but is also looking to lead the revolution in pricing and reimbursement required to make them a commercial success.

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Q1.  What is gene therapy?
A.  In the broadest sense, gene therapy is the use of genetic material in the treatment or prevention of disease. 

Q2 Which is the largest regional market for Gene Therapy?

A. North America is the largest regional market for Gene Therapy

Q3 Which are the top companies to hold the market share in Gene Therapy?

A. Novartis AG, Gilead Sciences., Jazz Pharmaceuticals, Orchard Therapeutics., Bristol-Myers Squibb., Amgen Inc., Shenzhen SiBiono Genentech, Shanghai Sunway Biotech Co., Ltd., Adaptimmune Therapeutics plc are the top companies to hold the market share in Gene Therapy.

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