Gene Therapy

Gene Therapy

The gene therapy market is experiencing significant growth on account of the surging prevalence of cancer and rare diseases. According to WHO, in the United States, over 400 products have been approved as therapy for more than 200 rare disease indications, and in the EU, over 70 products for about 45 indications. Furthermore, in the field of molecular and cellular biology, rapid and important developments, led by technological advances in genomics and gene editing techniques, have contributed to a growing number of approved gene therapies and an expanding pipeline. According to the Alliance for Regenerative Medicine (ARM), by the end of Q2 2019, there were more than 700 ongoing clinical trials globally. The American Medical Association reports that around 4,000 diseases have been linked to gene disorders (including cancers, AIDS, cystic fibrosis, and Parkinson's and Alzheimer's diseases). The successful treatment with gene therapy could positively affect millions of lives.

The gene therapy market is expected to grow at a steady rate of around 30.1% owing to the surge in cancer indications or increasing funding for gene therapy research. Investment from biopharma companies and traction towards the research and development of gene therapy are acting as a growth catalyst to the growth of the market. Numerous companies and laboratories are researching gene therapy, therefore, getting funding from seed funders and angel investors. For instance, Ori Biotech Ltd, a London and New Jersey-based innovator in the cell and gene therapy manufacturing sphere, announced the USD 30 million series A funding in 2020. So, the total funding in the company reached USD 41 million.

• Based on vector, the market is segmented into viral vector (retroviral and adeno-associated) and non-viral vector (oligonucleotides). The non-viral vector category to witness higher CAGR during the forecast period owing to the non-viral methods present certain advantages over viral methods, with simple large-scale production and low host immunogenicity being just two. Previously, low levels of transfection and expression of the gene held non-viral methods at a disadvantage; however, recent advances in vector technology have yielded molecules and techniques with transfection efficiencies related to those of viruses.
• On the basis of gene type the market is categorized into antigen, cytokine, receptor, and others. Among these, the cytokine held a significant growth during the forecast period. This is mainly due to the growing demand for gene therapy in some rare genetic diseases such as cancers, infectious diseases, and autoimmune disorders as it seeks to modulate immune function in order to resolve disease. These are used as an adjuvant in DNA vaccines, to restore homeostasis and reduce inflammation in autoimmune disorders or stimulate host inflammatory responses and immunity to cancers. Decades of preclinical research have culminated in clinical studies investigating the electro-transfer of cytokine genes for the treatment of cancers and infectious diseases.
• Based on indication, the gene therapy market has been classified into oncology, rare genetic diseases, cardiovascular, neurology, and others. The rare genetic diseases category is to witness higher adoption of gene therapies during the forecast period. This is mainly due to rise in the incidences of rare diseases. There are over 300 million people living with one or more of over 6,000 identified rare diseases around the world1, each supported by family, friends and a team of careers that make up the rare disease community. Each rare disease may only affect a handful of people, scattered around the world, but taken together the number of people directly affected is equivalent to the population of the world’s third largest country. Rare diseases currently affect 3.5% - 5.9% of the worldwide population. 72% of rare diseases are genetic whilst others are the result of infections (bacterial or viral), allergies and environmental causes, or are degenerative and proliferative while 70% of those genetic rare diseases start in childhood.
• Based on the delivery method, the market is bifurcated into in vivo and ex vivo. In vivo held a dominant share of the market in 2022. In vivo, gene therapy benefits from its simplicity and the reduced volume of material introduced into the brain. These properties make it an ideal method when the target is a specific, small area of the brain. In vivo gene therapy could also be used to obtain high levels of CNTF expression that would be appropriate for a significant physiological effect (both on neuronal survival and clinical symptoms). Viral vectors - such as adeno-associated viruses, lentiviruses, or adenoviruses can transduce high numbers of cells and lead to stable transgene expression.
• For a better understanding of the market adoption of the gene therapy industry, the market is analyzed based on its worldwide presence in countries such as North America (U.S., Canada, Rest of North America), Europe (Germany, U.K., France, Spain, Italy, Rest of Europe), Asia-Pacific (China, Japan, India, Rest of Asia-Pacific), Rest of World. North America is anticipated to grow at a substantial CAGR during the forecast period. While hundreds of potential cell and gene therapies are being developed, a few of these cutting-edge medicines have already been approved by the US Food and Drug Administration (FDA) and are currently helping patients. As of February 2020, nine cell or gene therapy drugs for the treatment of cancer, eye defects, and rare genetic diseases have been approved in the United States. Currently, more than 100 diseases are being explored for potential treatment with cell and gene therapies. Cellular and gene therapy-related research and development in the United States continue to grow at a fast rate, with several products advancing in clinical development
• Some of the major players operating in the market include Biogen; BioMarin; bluebird bio, Inc.; Bristol-Myers Squibb Company; Gilead Sciences, Inc.; Novartis AG; Orchard Therapeutics plc; REGENXBIO INC.; Spark Therapeutics, Inc.; and uniQure NV.