Gene Therapy for Rare Disease Market Valued at US$16.3 Million in 2017, Expected to Reach US$35.0 Million by 2026

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Gene Therapy for Rare Disease Market
Gene Therapy for Rare Disease Market

A) Market Overview:

The Gene Therapy for Rare Disease Market is estimated to be valued at US$ 16.3 million in 2017 and is expected to reach US$ 35.0 million by 2026. With a compound annual growth rate (CAGR) of 9.8% over the forecast period. Gene therapy involves the use of genetic material to treat or prevent rare diseases caused by genetic mutations. These therapies show immense potential in curing rare diseases that have limited treatment options. Advancements in genetic engineering techniques, such as the use of viral vectors and gene editing technologies, have revolutionized the field of gene therapy. The gene therapy market offers promising solutions to patients suffering from rare diseases, including inherited disorders, genetic disorders, and certain types of cancer.

B) Market Key Trends:

One of the key trends in the gene therapy for rare disease market is the increasing investment in research and development activities. Pharmaceutical companies are actively investing in the development of gene therapies to tap into the potential of this emerging market. This trend is driven by the growing understanding of genetic mutations and advancements in technology that enable precise targeting and delivery of therapeutic genes. Companies like Kite Pharma, Novartis, and Bluebird Bio are among the key players investing heavily in gene therapy research. These investments are expected to accelerate the development of new therapies and drive market growth. Additionally, collaborations and partnerships between pharmaceutical companies and research institutions are further fueling the advancement of gene therapy for rare diseases.
Segment Analysis:

The Gene Therapy for Rare Disease market can be segmented based on the type of rare diseases it caters to. One dominant sub-segment in this market is gene therapy for genetic disorders. Genetic disorders are inherited conditions caused by abnormalities in an individual's DNA. They can affect various body systems and lead to chronic health issues. Gene therapy offers a promising solution by correcting the genetic defects and restoring normal gene function.

Gene therapy for genetic disorders dominates the market due to the high prevalence of these conditions and the significant unmet medical needs. According to the World Health Organization, genetic disorders affect around 1 in 25 individuals worldwide. The increasing awareness about the potential of gene therapy in treating genetic disorders has led to extensive research and development efforts in this sub-segment. Several gene therapy products targeting specific genetic disorders, such as hemophilia, spinal muscular atrophy, and Duchenne muscular dystrophy, have gained regulatory approvals and are available in the market.

Gene therapy for genetic disorders also receives substantial support from regulatory authorities and government organizations. In many countries, specialized regulations and funding programs exist to facilitate the development and commercialization of gene therapy products for genetic disorders. This favorable regulatory environment further strengthens the dominance of this sub-segment in the gene therapy for rare disease market.

Key Takeaways:

The global Gene Therapy for Rare Disease Market Size is expected to witness high growth, exhibiting a CAGR of 35.0% over the forecast period of 2018-2026. This growth can be attributed to several factors, including the increasing prevalence of rare genetic disorders, advancements in gene editing technologies, and growing investments in gene therapy research.

In terms of regional analysis, North America is anticipated to be the fastest-growing and dominating region in the gene therapy for rare disease market. The region has a well-established healthcare infrastructure, a large patient population, and a high prevalence of rare genetic disorders. Additionally, North America has a favorable regulatory environment for the development and commercialization of gene therapy products.

Key players operating in the gene therapy for rare disease market include Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc. These companies are actively engaged in research and development activities to advance gene therapy technologies and develop innovative treatments for rare genetic disorders.

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