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Global Gene Therapy Market Clinical Trials Research Insight 2028

12-23-2021 11:01 AM CET | Health & Medicine

Press release from: Kuick Resarch

Global Gene Therapy Market Clinical Trials Research Insight

Global Gene Therapy Market & Clinical Trials Intelligence Report Highlights

• Global Market Gene Therapy Landscape USD 24 Billion Opportunity
• Comprehensive Commercial & Clinical insight on 16 Approved Gene Therapies
• Gene Therapy Products Dosage & Price Analysis
• Comprehensive Clinical Insight On 1700 Gene Therapies In Clinical Trials
• Information On 45 Orphan Designated Gene Therapies By Indication, Country, Company
• Global Gene Therapy Clinical Trials Insight By Phase, Country, Formulations, Company, Indication, Target
• Global & Regional Market Insight

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Genes are small fragments of DNA which encode proteins that are used by cell to carry out vital activities. However, the disruptions in DNA sequence of gene can lead to loss or change in cell’s ability to carry out role in the body. This often leads to widespread changes that manifests as disease, including genetic disorders and cancer. The novel gene therapy aims to treat these disorders by replacing, inactivating, or introducing genes in cells which can compensate the activities of abnormal gene.

Several gene therapies have entered the market which has shown to improve survival outcomes in several rare genetic diseases, cancers, and other disorders. Although Spinal Muscular Atrophy (SMA) is a rare disease, it has constituted for the highest share in the market. The development of gene therapy, Zolgensma (AVXS-101), has significantly proven its efficiency in the treatment of SMA, along with changing the disease phenotype. In May 2019, Novartis received approval from the U.S. FDA for Zolgensma, which is designed to address the root cause of SMA. Zolgensma is the first and only approved gene therapy in this segment. Apart from this, the emergence of CAR T cell therapy in the management of cancer has also shown encouraging response in the market.

Currently the field of gene therapy is highly unexplored and researchers are conducting research and development to drive the sector. Much of the innovation and development in the space is being driven by smaller biotech companies or research universities, sometimes in partnership with big pharmaceutical or an entity specialized in the targeted therapy. For instance, in December 2021, BioMarin Pharmaceutical and Skyline Therapeutics announced a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated Virus (AAV) gene therapies to treat genetic cardiovascular diseases. In addition, Decibel Therapeutics collaborated with Regeneron to discover and develop gene therapies for hearing loss. These rising trends in the market help to increase the investment, which further boosts the growth of market.

At present, more than 1500 clinical trials are ongoing which are evaluating the role of gene therapies in wide range of therapeutic conditions including neurological diseases, ocular disorders, Alzheimer’s disease, HIV/AIDs, Parkinson Disease, and cancers. Chimeric antigen receptor (CAR) T-cell therapy accounts for more than 50% of the clinical pipeline. Several researches are going to expand their use in solid tumors and to overcome the limitations of previously approved CAR T-cell products. Recently, researchers have developed bispecific CAR T cells to enhance the efficacy and specificity, which are present in initial stages of development.

The promising future of gene therapies is driven by improvement and advancement of the underpinning technologies and cellular mechanisms, such as improvements in the safety of gene editing tools, fine-tuning of regulating mechanisms designed into cells, or the use of cells to deliver drugs in a controlled and targeted way. Further, recent scientific and technological advances have also aid in reducing the development time for personalized treatment making it more available and accessible. The drug development industry is moving rapidly towards precision medicine and leading pharmaceutical companies are expecting to increase investment in forthcoming years.

The global gene therapy market is anticipated to grow with high rates during the forecast period. The growing prevalence of chronic diseases, such as cancer and various rare diseases is one of the major factors driving the progress of the market. The pipeline for cancer gene therapy is quite strong and recently several industry players have been focused on developing several effective gene therapies and its vectors, which will further boost the growth of market. As per our analysis, the global gene therapy market is expected to surpass US$ 20 Billion by 2028.


Neeraj Chawla
Research Head
Kuick research


Kuick Research is a market research and analytics company that provides targeted information for critical decisions at business, product and service levels. We are quick, predictive and known by the recommendations we have made in the past. Our result-oriented research methodology offers understanding of multiple issues in a short period of time and gives us the capability to keep you full with loads of practical ideas. By translating research answers into strategic insight and direction, we not only rate the success potential of your products and/or services, but also help you identify the opportunities for growth in new demographies and find ways to beat competition.

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