Gene therapy is a new generation of medicine where a functioning gene is delivered to a targeted tissue in the body to produce a missing or non-functioning protein. It is potential one-time treatment which is aimed at targeting the underlying cause of a disease at cellular level and may deliver transformational improvement in quality of life. Till date, gene therapies have been successfully used in the management of wide range of diseases including cystic fibrosis, cancer, heart diseases, AIDS, hemophilia, and others.
The advent of gene therapy has raised hopes towards viable treatment options for rare genetic disease for which there is no treatment option available. The approval of Zolgensma (onasemnogene abeparvovec), which is an adeno-associated virus gene mediated therapy for spinal muscular atrophy is considered as breakthrough in gene therapy market. In addition to this, several gene therapy products for rare diseases have entered the market including Libmeldy for metachromatic leukodystrophy and Spinarza for spinal muscular atrophy. Apart from this, the therapeutic wave of gene therapy was also employed to treat cancer with the advent of chimeric antigen receptor (CAR) T-cell therapy. Several CAR T-cell therapy have entered the market and have shown to significantly improve survival outcomes in cancer patients.
In last few years, significant milestones have been achieved in terms of marketing authorization of gene therapy products and real benefit for a large number of patients has been established. Despite this, the global gene therapy market is still present in an immature phage, and indicates huge potential for future growth. Significant barriers to the growth of market includes efficacy and safety concerns, immune system responses, laborious approaches for developing and manufacturing, and the high cost of drugs, which needs to be overcome in forthcoming years.
The increasing investment in this sector has propelled the research and development activities in this field. At present, there are more than 3000 ongoing clinical trials related to gene therapy which will propel the growth of market. The majority of these drugs are present in initial stage of clinical development, suggesting that novel gene therapies will enter the market in next 5-7 years. For instance, in November 2021, US FDA has granted priority review he Biologics License Application (BLA) for betibeglogene autotemcel (beti-cel) for patients with β-thalassemia who require regular red blood cell transfusions. The drug is currently being evaluated in phase-I/II clinical trial.
Many key players in the market are focused on adopting strategies, such as mergers and acquisitions, to enhance their product portfolio, which in turn, is expected to propel the market growth over the forecast period. Recently in October 2021, National Institute of Health, US FDA, 10 pharmaceutical companies, and 5 non-profit organizations have partnered for the development of gene therapies for rare diseases. The increasing investment in this sector will further propel the growth of market. As per our report findings, the global gene therapy market is anticipated to grow with high growth rates during the forecast period. With the growing incidences of cancer and targeted diseases, the adoption of gene therapy will be increased. Among region, US is expected to dominate the global cell therapy market owing to large number of ongoing clinical trials, favorable reimbursement policies, and increasing awareness among population,
The global gene therapy market provides insights into the global and regional market along with the analysis of different influencing factors like drivers, restraints, and opportunities. By geography, the market has been segmented into several regions including US, Europe, China, Japan, Taiwan, South Korea, Germany, and India. By indication, the market has been segmented into cancer, CNS, and others. Apart from this, a comprehensive list of key players operating in global gene therapy market is also mentioned in the report.