Gene therapy is an experimental medical treatment that uses specific
types of genes to treat or prevent several types of diseases. Gene
therapy is also used to treat several hereditary diseases, wherein
the mutated defective gene is replaced with the functional gene. Gene
therapy is being widely studied by researchers all over the world for
the treatment of several diseases such as immune deficiencies,
hemophilia, Parkinson’s disease, cancer, and even HIV, through
different approaches. Three primary approaches that are being studied
and practiced in the gene therapy are replacement of the mutated
disease-causing gene with the healthy gene, inactivation of the
mutated gene, and introduction of the new gene to fight against the
disease. In the gene therapy treatment, a functional gene is inserted
into the genome of an individual’s cells and tissues by using a
carrier known as ‘vector’. Viruses is the most common type of
vectors used in gene therapy, which is genetically altered to carry
the normal human DNA. Over the last few years, gene therapy has
emerged as a promising treatment option for several diseases
including inherited disorders and certain types of cancers and viral
infections. However, the treatment is still reviewed for its
effectiveness and safety. Currently, the gene therapy is being tested
only for those diseases for which no other treatment is available.
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The demand for gene therapy is greatly influenced by the increasing
number of patients with cancer, hereditary disorders, and viral
infections. The International Agency for Research on Cancer has
estimated that more than 21.7 million cases of cancer are likely to
be diagnosed worldwide and more than 13 million deaths would occur
due to cancer across the world by the end of 2030. Moreover,
according to Joint United Nations Programme on HIV and AIDS () in
2015, there were approximately 36.7 million people living with
HIV/AIDS across the globe. Technological advancements and several
pre-clinical and clinical trials for the treatment of different
disease are likely to boost the gene therapy market during the
forecast period. Also, successful clinical trials of gene therapy for
the treatment of central nervous system disorders would open new
avenues for the global gene therapy market in the near future.
Moreover, increase in R&D budgets by commercial players and
governments for the development of safe and effective gene therapy
for various diseases would propel the global gene therapy market in
the near future. However, high cost of gene therapy treatment and
unwanted immune response in some cases are some of the restraining
factors for the market.
The global gene therapy market can be broadly segmented based on type
of gene therapy, type of vector used for gene transfer, disease
indication, and geography. Basically, there exist two types of gene
therapy: germline gene therapy (which involves the modification of
genes inside the germ cells i.e. sperms and ova) and somatic gene
therapy (where the therapeutic gene is inserted into the body cells
or tissues and not in germ cells or gametes). The somatic gene
therapy is widely studied and practiced for the treatment of cancer
and infectious diseases. Use of the germline gene therapy for the
treatment of hereditary disorders is prohibited in countries such as
Germany, Switzerland, and Australia, due to and long-term effects on
the future generation. Based on type of vector used, the market has
been segmented into viral vectors and non-viral vectors such as
bacteria and plasmid DNA. Viral vectors are most commonly used and
studied. The viral vectors segment accounts for a major share of the
global gene therapy market. Based on indication, the global gene
therapy market has been segmented into infectious diseases, genetic
diseases, cancer, and other diseases.
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Geographically, the global gene therapy market has been segmented
into four regions: North America, Europe, Asia Pacific, and Rest of
World. North America is projected to dominate the global gene therapy
market by 2025. The major share held by North America is attributable
to well-established health care facilities, high per capita health
care expenditure, favorable medical reimbursement policies, and
extensive R&D activities for the gene therapy in the region.
Additionally, increasing number of patients with cancer and genetic
disorders in the region is projected to propel the demand for gene
therapy in North America during the forecast period. The market for
gene therapy in North America is anticipated to expand at the maximum
CAGR from 2017 to 2025. Europe is anticipated to follow North America
in the market for gene therapy. The market in Asia Pacific is likely
to expand at a moderate growth rate during the forecast period.
Key players operating in the global gene therapy market are Bluebird
Bio, Inc., CELGENE CORPORATION, Adaptimmune Therapeutics plc,
GlaxoSmithKline plc., Merck KGaA, OncoGenex Pharmaceuticals, Inc.,
Shanghai Sunway Biotech Co. Ltd., and Transgene SA. Most of these
companies are witnessing different pre-clinical and clinical stages
of development of gene therapies.
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