Gene therapy is a technique in which genetic material is used to prevent or cure diseases such as genetically inherited disorders, neurodegenerative diseases, viral infections, and even cancer. CNS gene therapy can offer permanent treatment for life-threatening diseases such as Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, Batten disease and many more. In CNS gene therapy DNA is introduced in the host body through a vector that is designed to modify the expression of an abnormal gene and treat the disease. According to WHO, the burden of CNS is increasing each year and is expected to reach 103 million disability-adjusted life years (DALYs) in 2030. CNS gene therapy aims to augment the gene expression or inhibit it. Desirable gene expression is thus achieved by undergoing CNS gene therapy, however, only a select number of genes are modifiable to cure diseases till date.
The exponentially increasing demand for treatment of incurable CNS disease is luring manufacturers to invest in CNS gene therapy market. Many leading players in biotechnology and pharmaceutical sectors such as Pfizer, Sanofi, Novartis, Abbott Laboratories, etc. are entering the CNS gene therapy market and the global gene therapy finances increased by 35% in 2018 as compared to 2017. The growing number of clinical trials and research papers supporting the pieces of evidence of successful CNS gene therapy is expected to drive the CNS gene therapy market over the forecast period. CNS gene therapy is considered to be very expensive, as it therefore often requires an individual, case-by-case approach. This factor may hamper mass adoption of CNS gene therapy in the market over the forecast period. Although the future of CNS gene therapy beholds huge potential for growth, it is limited to developed countries with high healthcare and research and development expenditure over the forecast period. Stringent regulations and complications related to interference of foreign genetic material are expected to be a restraint in the expansion of CNS gene therapy market.
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Based on the therapeutic platform, the global CNS gene therapy market is segmented into the following:
- Viral Vectors
- Herpes Simplex
- Adeno-Associated Virus (AAV)
- Non-Viral Vectors (Nanospheres and Nanoparticles)
- Genetically Modified Cell-Based Immunotherapies
- Gene Editing
Based on the indication, the global CNS gene therapy market is segmented into the following:
- Alzheimer’s disease (AD)
- Huntington’s disease (HD)
- Parkinson’s disease (PD)
- Amyotrophic Lateral Sclerosis (ALS) and Motor Neuron diseases
- AtaxiasMultiple sclerosis (MS)
- Adrenoleukodystrophy and Metachromatic Leukodystrophy
- Batten disease (BD)
The global CNS gene therapy market is pegged to grow at a high CAGR over the forecast period owing to the increasing investments by manufacturers and government organizations for developing a treatment for neurological diseases. In 2018, 28 products have received the designation for CNS gene therapy. Increasing regulatory approvals are expected to favor the growth of CNS gene therapy in the market. Genetically modified cell-based immunotherapies segment in the therapeutic platform is expected to grow with high CAGR owing to the increasing number of genetically modified cell-based immunotherapies undergoing preclinical and clinical trials. On the basis of indication, Parkinson’s disease (PD) segment is expected to contribute maximum revenue share in CNS gene therapy market over the forecast period.
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On the basis of geography, the global CNS gene therapy market is split into seven key regions viz. North America, Latin America, Europe, East Asia, South Asia, Oceania, and the Middle East & Africa. North America CNS gene therapy market is anticipated to be the most dominant because of higher healthcare expenditure in the region. There are 476 gene and cell therapies therapeutic developers, 206 gene therapy & gene-based medicine companies and 18 gene therapy & gene-based medicine companies active in neurodegenerative indications in North America responsible for the generation of maximum market share in terms of revenue over the forecast period. East Asia CNS gene therapy market is projected to grow with the highest CAGR over the forecast period owing to the increasing research and development expenditure. Europe is expected to contribute second largest share in the global CNS gene therapy market owing to higher spending on healthcare and faster adoption of biotechnology products in the region. MEA and Latin America are expected to represent the least incremental opportunity in the global CNS gene therapy market over the forecast period due to higher cost associated with the product and stringent approvals for CNS gene therapy.
Some of the players operating in the CNS gene therapy market are Abbott Laboratories, Pfizer, Inc., Abeona Therapeutics, Sanofi, Amicus Therapeutics, PTC Therapeutics, Orchard Therapeutics, Novartis AG, GenSight Biologics, Spark Therapeutics, Prevail Therapeutics, and many others.