Gene Therapy Market, by Application (Oncology, HIV, Severe combined immuno-deficiencies, Hemophilia, and others) by Cell-type (Somatic cell and Germline), by Vector (Viral Vector and Non-Viral Vector) – Global Industry Insights, Trends and Forecast (2017-2025)

Created On: Aug-2018
Report ID: IR152
Format: PDF

Gene therapy is a method of insertion of normal genetic materials or genes into a cell in order to replace defective genes or to fix the abnormality of genes. The therapeutic delivery of genes can cure rare diseases, genetic disorders and viral infections. Gene therapy has been identified as a crucial method of treatment which can replace traditional method of treatments such as surgery or drugs that can have severe side-effects on human health. The increasing concern over occurrence of various types of cancer and global increase in death ration due to cancer has escalated the demand for gene therapy. It has been estimated that in the year 2016, around 8.9 million people died due to cancer.

Gene Therapy Market Dynamics

The market for gene therapy is expanding globally and it can be partially contributed to the continuous rise in development of number of genetic molecules, that are crucial to alter the defective genes positively or to reverse mutate selective genes. The global expansion of small biotechnology and pharmaceutical industry in last two decades has escalated the study of important molecules. It can be believed by the fact that more than 900 molecules that has potential to be used in gene therapy was identified to be in their developmental and trial phase in the year 2017. Around 83% of small biotech companies have various types of gene therapies in pipeline. The rising concern over occurrence of multiple types of cancer, cardiovascular diseases, Alzheimer’s disease and severe viral infections are also the major driving factors for gene therapy market.

Gene Therapy Market Restraint

The risks associated with gene therapy such as irregular immune response and short lived nature of the therapy (patients in some cases need to undergo multiple gene therapy rounds) can negatively impact human health which is a major hindrance for global market growth of the product. High cost of gene therapy treatment and risks of introduction of toxicity or induction of inflammatory responses by viral vectors are also a drawback of gene therapy. To ensure the safety of human health and patient under trial, there are several regulations, laws and guidelines made by the government. The U.S Food and Drug Administration (FDA) and National Institutes of Health (NIH) regulate all the research and products of gene therapy.  

The global onset of various forms of cancer has led to rise in the number of patients suffering from this life threatening disease. Gene therapy stands to be one of the most promising and least time consuming treatment method for oncology when combined with immune CPIs. Around 22% (largest portion) gene therapies are in pipeline for the treatment of oncology followed by nervous, genetic, ophthalmology and others.

Gene Therapy Market Latest Developments- Clinical Trials and Drugs

Global companies like BMS and Merck & Co. are already in the process of launching effective gene therapies to target various segments of oncology with their in-house clinical trials going on for different phases of the disease. Gene therapy in the treatment of HIV is also a major success for healthcare industry. Lentivirus gene therapy, which is a vector – based gene therapy, has shown positive results when tested on patients under trial and hence holds major hopes in the treatment of HIV. Similarly, a treatment called CERE 110 developed by Ceregene Inc to treat Alzheimer’s disease is a major success in that area till date. The significant innovations in genetic engineering tools such as CRISPR and TALEN and developed understanding of complex genetic disorders such as Autism, Gaucher disease, Progeria, Down syndrome, etc is anticipated to drive the market of gene therapy.

Gene Therapy Segment Analysis

Types of gene therapy which includes somatic cell and germline gene therapy involves the insertion of therapeutic genes into somatic cells and modification of germ cells by inserting functional genes respectively. The use of somatic cells to engineer gene therapy approaches and to be used in clinical trials is more practical than the germline therapy because it is non-hereditary in nature and hence doesn’t pose the risk to pass on to the patient’s children. There are two broad classifications of somatic cell gene therapy called ex-vivo (cell modification and gene introduction in cell is carried outside the body) and in-vivo (gene transfer is done in the cells present inside the body).

Viral vectors and non-viral vectors, which are significant carriers of genetic materials, are of great importance in gene therapy. However, viral vectors such as adenovirus and retrovirus being more efficient in delivering genetic material into the targeted cell and being used in most of the clinical trials for commercialization as compared to non-viral vectors that are still in their developmental phase. Adenovirus being the most suitable viral vector in gene therapy had been used in more than 402 gene therapy clinical trials out of 1700 gene therapies conducted till the year 2011. Ad5 and Ad2 viruses of Adenovirus species C are most eligible to create viral vectors to use in gene therapy.

North America holds the largest share in the gene therapy market as most of the clinical trials are going in U.S. The demand for better medical facilities, availability of advanced technologies and resources has established North America as a leader in the global market. The market of gene therapy in Europe and Asia Pacific is also witnessing a steep rise due to approval of drugs such as Gendicine, Oncorine, Neovasculogen, etc in China and Russia.

Gene Therapy Products in Pipeline

To treat haemophilia, which has become a common health concern globally, companies like Bayer and Freeline therapeutics are developing gene therapy products. Nightstar (U.K.) is in pipeline to develop and commercialize NSR-REP1 and NSR-RPGR gene therapies to treat Choroideremia (CHM) and X-linked retinitis pigmentosa (XLRP), which cause vision loss and night blindness. Precision Biosciences which specializes in genome editing is also a promising company which is focused to apply ARCUS (upcoming genome editing space derived from homing endonuclease, a natural genome editing enzyme)  genome editing technology to treat genetic diseases.

Major Players

Some of the key players are GlaxoSmithKline, Human Stem Cell Institute, Shire Plc, Bristol Myer’s Squibb, UniQure N.V, Spark Therapeutics LLC, Voyager Therapeutics, Celgene Corporation, Bluebird Bio, Sangamo Biosciences, Dimension Therapeutics, Chiesi Farmaceutici S.p.A., and Juno Therapeutics.

Our Research Approach Includes
  •  Market Outlining
  •  Framing discussion guide
  •  Data Validation
  • Data Analysis
  •  Re-Validation and Finalization of Data
  •  Report Insights and Publishing
Our Research Approach Includes
  •  Market Outlining
  •  Framing discussion guide
  •  Data Validation
  • Data Analysis
  •  Re-Validation and Finalization of Data
  •  Report Insights and Publishing
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