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Market Overview
Global Gene Therapy Market is expected to witness a CAGR of 18.7% during the forecast period. Certain factors that are driving the market growth includes rising investments in the R&D, technological advancements, and growing prevalence of target diseases.
Gene therapy is no longer considered a futuristic dream and instead, it has become a treatment for the present. Gene therapies are gaining momentum as promising primary successes in clinical studies accumulate and examples of regulatory approval for licensing rise in number. Somatic cells can now be treated with gene fragments or gene replacements to express required proteins. While gene therapy was originally targeted for inherited disease, it is now capable of treating a variety of acquired problems including cancer and HIV. Initially delivered through viruses, now there are safer direct or biochemical vectors, while in the future nano-particles might deliver the drug of choice directly to the nucleus of affected cells.
Scope of the Report
Gene therapy refers to an experimental technique which uses genes to treat or prevent disease. The various techniques include replacing a mutated gene which causes disease with a healthy copy of the gene, or inactivating, or “knocking out,” a mutated gene which is functioning improperly, or/and introducing a new gene into the body to help combat a disease.
The gene therapy products which include both viral vectors and non-viral vectors are included in the scope of this report.
Key Market Trends
Cancer is Expected to Hold Significant Market Share in the Indication Segment
The ever-rising prevalence of cancer all across the globe is majorly driving the growth of this segment. Getting genes into cancer cells has been one of the most difficult aspects of present day gene therapy. Researchers have been working on finding new as well as better ways of doing this. Usually, the gene is taken into the cancer cell by a carrier known as a vector. The most common types of carrier that are used in gene therapy are viruses owing to the fact that they can enter cells and eventually deliver genetic material. The viruses are generally changed so that they cannot result in any serious disease. However, they may still cause mild, flu-like symptoms. Some viruses have been modified in the laboratory so that they can target cancer cells only and not the healthy cells. Therefore, they only carry the gene into cancer cells.
North America Dominates the Market and Expected to do Same in the Forecasted Period
For gene therapy, in the United States, the DHHS (Department of Health and Human Services) has been appointed to oversee the clinical trials. Two other organizations within DHHS, the OHRP (Office for Human Research Protections) and the U.S. FDA (Food and Drug Administration), have specific authority that is described in the CFR (Code of Federal Regulations). All investigators, must obey these regulations while conducting clinical gene therapy trials. The increasing approval of various gene therapies by the afore-mentioned organizations are driving the market growth in the region. For instance, in May 2019, AveXis, a Novartis company, announced that the US FDA had approved Zolgensma (onasemnogene abeparvovec-xioi) for treating pediatric patients of less than 2 years of age having spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Competitive Landscape
The global Gene Therapy market is highly competitive and consists of a few major players. Companies like Amgen Inc., Bluebird Bio, Gilead Sciences, Inc., Novartis AG, Orchard Therapeutics, Sibiono GeneTech Co. Ltd., Spark Therapeutics LLC, and UniQure N.V., among others, hold the substantial market share in the Gene Therapy market.
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1 INTRODUCTION
1.1 Study Deliverables
1.2 Study Assumptions
1.3 Scope of the Study
2 RESEARCH METHODOLOGY
3 EXECUTIVE SUMMARY
4 MARKET DYNAMICS
4.1 Market Overview
4.2 Market Drivers
4.2.1 Rising Investments in the R&D
4.2.2 Technological Advancements
4.2.3 Growing Prevalence of Target Diseases
4.3 Market Restraints
4.3.1 Lack of Standard Regulations
4.3.2 High Price of Products
4.4 Porter’s Five Force Analysis
4.4.1 Threat of New Entrants
4.4.2 Bargaining Power of Buyers/Consumers
4.4.3 Bargaining Power of Suppliers
4.4.4 Threat of Substitute Products
4.4.5 Intensity of Competitive Rivalry
5 MARKET SEGMENTATION
5.1 By Indication
5.1.1 Cancer
5.1.2 Metabolic Disorders
5.1.3 Eye Disorders
5.1.4 Spinal Muscular Atrophy
5.1.5 Other Indications
5.2 By Technology
5.2.1 Adeno Virus Vector
5.2.2 Adeno-associated Virus Vector
5.2.3 Lentiviral Vector
5.2.4 Retroviral Vector
5.2.5 Herpes Virus Vector
5.2.6 Others
5.3 Geography
5.3.1 North America
5.3.1.1 US
5.3.1.2 Canada
5.3.1.3 Mexico
5.3.2 Europe
5.3.2.1 Germany
5.3.2.2 UK
5.3.2.3 France
5.3.2.4 Italy
5.3.2.5 Spain
5.3.2.6 Rest of Europe
5.3.3 Asia Pacific
5.3.3.1 China
5.3.3.2 Japan
5.3.3.3 India
5.3.3.4 Australia
5.3.3.5 South Korea
5.3.3.6 Rest of Asia-Pacific
5.3.4 Middle East and Africa
5.3.4.1 GCC
5.3.4.2 South Africa
5.3.4.3 Rest of Middle East and Africa
5.3.5 South America
5.3.5.1 Brazil
5.3.5.2 Argentina
5.3.5.3 Rest of South America
6 COMPETITIVE LANDSCAPE
6.1 Company Profiles
6.1.1 Amgen Inc.
6.1.2 Biogen Inc.
6.1.3 Gilead Sciences, Inc.
6.1.4 Bluebird Bio
6.1.5 Novartis AG
6.1.6 Orchard Therapeutics
6.1.7 Sibiono GeneTech Co. Ltd.
6.1.8 Spark Therapeutics LLC
6.1.9 UniQure N.V.
7 MARKET OPPORTUNITIES AND FUTURE TRENDS