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Genes are composed of deoxyribonucleic acid (DNA), which contains essential information for producing proteins that are crucial for the optimal functioning of the body. Genetic disorder is the result of gene mutations in which proteins are made incorrectly. Gene therapy is intended to introduce healthy gene into damaged cells to counteract for abnormal genes or to make a protein of interest. Mutated gene causes missing of the important protein, with the help of gene therapy normal copy of that missing or faulty protein can be introduced to restore the function of the protein. All over the world, gene therapy is being extensively studied by the researchers for the treatment of various diseases such as HIV, Cancer, Parkinson’s disease, hemophilia and even immune deficiencies through various approaches.
For instance, According to 2015, article published in Journal Gene Technology and Resistance to Viruses has shown the future prospect of the bone marrow gene therapy for HIV treatment. Three primary approaches being practiced in the gene therapy are substituting a mutated gene with the healthier one, disabling a mutated gene that is functionally inappropriate and introducing a novel gene in the body to fight a disease. In the gene therapy treatment, vectors are responsible for insertion of functional gene into the genome of an individual tissues and cells, one of the most common vectors are viruses.
The gene therapy treatment which was once considered as unfeasible, has now become a trend among small and big companies. Result of this has been an increased the in the number of various successful startups, backed by financiers in line with giant companies these have boosted the growth of the market.
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Demand for gene therapy is significantly increasing due to increasing number of patients suffering from hereditary disorders, cancer, HIV, and various chronic diseases. For instance, 2018 American Cancer Society report showed cancer was responsible for 609,640 deaths in the U.S. Moreover, in 2017, WHO reported 1.0 million deaths were caused due to HIV-related illnesses worldwide. Increasing technological developments, innovations, and clinical and pre-clinical trials. For instance, According to published article in 2014 Regenerative Medicine reports 4749 registries of clinical trials related to stem cell treatment.
Several diseases are likely to propel growth of the gene therapy market size during the forecast period. Moreover, increasing research and development funding by government agencies and commercial market players for the development of innovative, efficient, and safer gene therapy practices is boosted the market growth. For instance, 2017 National Institutes of Health was funded US$ 1,644 million for stem cell research. Furthermore, growing awareness and community acceptance of gene therapy for various disease treatments is propelling growth of the market.
However, unwanted immune responses related to treatment, concerns regarding unethical use of gene therapy and high cost associated with the treatment can hamper the market growth.
Table of Contents
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Top players in the market
Key players operating in the global gene therapy market include GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc. These companies are conducting various clinical and pre-clinical developmental stages of gene therapies.
Key players are engaged in developing new technologies and are focusing on collaborating with leading companies in order to retain their foothold in the global market. For instance, in 2017, the U.S. Food and Drug Administration (FDA) approved a gene therapy called Luxturna, developed by Spark Therapeuticsa Biotech Company based in Philadelphia.
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