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Gene Therapy Market Driven by Increase in Occurrence of Inherited and Neurological Disorders

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Brahmayya Sunkesula
Gene Therapy Market Driven by Increase in Occurrence of Inherited and Neurological Disorders

 Growing prevalence of chronic such as cancer, cardiovascular diseases (CVD), and central nervous system (CNS) disorders is making a promising impact on the demand for applications of gene therapy across the globe. Existing cancer therapies such as chemo and radiation therapies and the prescribed drugs have resulted in many side effects that can include internal damage to the organs. Additionally, the higher costs of cancer diagnosis procedures, increase in awareness towards gene therapy products for the treatment of various diseases, and government support to promote gene therapy-related research are the major influencing factors that are anticipated to boost the market growth. Cancer is a major cause of death around the world, accounted for about 9.6 million deaths in 2018, as per the World Health Organization. Growing occurrence of inherited and neurological disorders and an increasing urgency to treat these diseases in less time are the key factors fuelling the growth of the gene therapy market. The global gene therapy market size is evaluated at $710 million as of 2018 and is anticipated to grow with a CAGR of 31.5% throughout the forecast period 2019-2025. The report covers Gene Therapy Market size by development phase and applications, Gene Therapy Market share by top 5 companies and also the market share by start-ups during the forecast period.

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According to the WHO, approximately 47.5 million people are suffering from dementia cases every year, globally. Alzheimer’s disease is a common form of dementia, which constitutes about 60-70 % of total cases. Neurological disorders, such as motor neuron disease, Parkinson’s, and Alzheimer’s diseases, can be treated by advanced gene therapy solutions efficiently. For instance, ProSavin, lentiviral vector-based gene therapy for Parkinson’s disease, which is caused by dopamine deficiency in the brain. ProSavin delivers into the brain and stimulates the target cells to begin dopamine production.

Diseases Potentially Treated by Gene Therapy

Inherited Diseases
For people with inherited diseases, gene therapy could replace a defective or missing gene or express a missing biological factor, enzyme or protein. Hemophilia, cystic fibrosis, and inherited retinal degeneration are some of the inherited diseases being treated by gene therapy technique. Replacement of a defective or dysfunctional gene altogether with the therapeutic gene is the exceptional benefit that is promoting the progress of the gene therapy marketplace.

Hemophilia
A bleeding disorder caused by the deficiency of the coagulation factor due to mutations in the genes. According to NCBI, in the United States, approximately 60% of the adults report adherence to prophylaxis due to its inherent complexity, resulting in unacceptable rates of bleeding disorders. Conventional therapy requires regular intravenous infusions of the missing coagulation proteins into the patients’ body.

Cystic Fibrosis
A genetic disorder that causes severe damage to the intestinal system and lungs. It majorly affects mucus, digestive juices, and sweat-producing cells. When there is an alteration or mutation in the genetic instructions, the production of the cystic fibrosis transmembrane conductance regulator (CFTR) gene may be affected. The CFTR protein has been cloned, and researchers are now developing gene therapies to enable the expression of CFTR.

Inherited Retinal Degeneration
Ocular gene therapies could offer a treatment for people with inherited retinal degeneration. Defects in the genes expressed in the retinal pigment epithelial cells may result in retinal degeneration. For instance, Luxturna is the first gene therapy molecule for an inherited genetic disease ever. It was developed by Spark Therapeutics and approved by the FDA in December 2017, for the United States. Luxturna is used for patients with an inherited form of retinal dystrophy, and it is injected directly into the retina of the patients so it can infect the retinal cells.

Infectious Diseases
Gene therapy vaccines are being developed and are under clinical trials for tackling infectious diseases, including malaria, tuberculosis, HIV, and influenza. Tuberculosis vaccines are being studied that use genetically modified vaccine comprising a recombinant fowlpox virus and a modified vaccine virus Ankara (MVA), each containing a tuberculosis antigen. Malaria vaccines are undergoing clinical trials utilize Chimpanzee adenovirus containing malaria antigen.

Latest Gene Therapy Products on Market
China is the first country that has approved and introduced commercial gene therapy molecules for the treatment of several chronic disorders into the market. The first product in this pipeline is Gendicine for the treatment of head and neck squamous cell carcinoma, and in the western world, the first approved gene therapy product was Glybera for the treatment of pancreatitis. In February 2019, scientists that working with Sangamo Therapeutics, an American biotechnology company has announced the first-ever in body human gene editing therapy to permanently alter DNA in a patient with Hunter Syndrome which causes severe abnormalities in many organs such as respiratory system, heart, and skeleton. Furthermore, in August 2017, the USFDA approved Novartis AG’s gene therapy molecule, Kymriah, for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL) in children and adults up to the age of 25 years. In May 2016, GlaxoSmithKline, a British pharmaceutical company introduced a stem cell gene therapy product, Strimvelis, for the treatment of patients with severe combined immunodeficiency, due to adenosine deaminase deficiency (ADA-SCID).

North America Holding Major Share of the Gene Therapy Market
North America generated approximately 40% of the global revenue in the gene therapy market in 2018. Most of the companies in the U.S. are seeking approvals from the concerned authorities for the next phase of clinical trials, and gene therapy molecules are arranged for commercial usage. FDA has approved 3 gene therapy molecules to treat cancer and inherited blindness diseases in 2018 alone. This region is anticipated to witness significant growth in the upcoming years owing to the high occurrence of cancer cases. For instance, according to a report published in the American Cancer Society’s journal, 6,09,640 cancer deaths were reported in the U.S. in 2018. Supportive initiatives by local governments and companies to promote R&D activities in the healthcare sector can stimulate regional market expansion. For instance, in 2018, the U.S government approved US$ 3 billion for the National Institutes of Health (NIH) research funding. Rising cases of diffuse large B-cell lymphoma (DLBCL) in the United States are likely to impel the demand for chimeric antigen receptor (CAR) T-cell therapy. Yescarta is the first CAR T-cell therapy approved by the U.S. food and drug administration (FDA) for the treatment of large B-cell lymphoma.

The application segment that will be creating the most lucrative opportunities for the gene therapy market is oncology. This application segment is projected to grow at a CAGR of 18.5% through to 2025. According to the National Center for Biotechnology Information, the progression in oncology study and treatment of tumor expenses is forecast to rise 7-10% annually throughout 2020, with a total cost exceeding $150 billion. Increased application of gene therapy products in the treatment of other forms of cancers is poised to drive the oncology segment during the forecast period. Viral Oncological disorders are the largest contributor toward the market growth owing to the high prevalence of cancer worldwide and the availability of regulatory approved gene therapy for cancer.

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The Major Players in the Gene Therapy Market:
The prominent players in the gene therapy market include Adaptimmune, BioCancell, Bluebird bio, Inc., Celgene, GlaxoSmithKline, Merck, OncoGenex Pharmaceuticals, Shanghai Sunway Biotech, Shenzhen SiBiono GeneTech, and SynerGene Therapeutics.

In January 2019, Bristol-Myers Squibb Company announced that it would acquire Celgene Corporation. This agreement would result in the formation of a specialty-focused biopharmaceutical company that can address the patient needs through high-value innovative medicines.

Related Reports:

A. Regenerative Medicine Market
https://www.industryarc.com/Report/16223/regenerative-medicine-market.html

B. Genomics Market
https://www.industryarc.com/Report/1275/genomics-market-analysis-and-forecast-report.html

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