Gene therapy for rare disease involves the introduction of genetic material into a patient’s cells to replace abnormal or missing genes, thereby treating or preventing the occurrence of rare diseases. This therapy has witnessed significant advancements in recent years and has the potential to offer long-term therapeutic benefits for patients suffering from rare diseases. Several gene therapies have been approved by regulatory agencies, providing hope for patients who have limited or no treatment options. The market for gene therapy for rare disease is expected to witness substantial growth as more research is conducted, and new therapies are developed.
The gene therapy for rare disease market is driven by the increasing prevalence of rare diseases globally. According to the World Health Organization, there are around 6,000 to 8,000 identified rare diseases, and around 400 million people worldwide are affected by these diseases. The rising investments in research and development activities for gene therapy also contribute to market growth. Governments, pharmaceutical companies, and research organizations are investing heavily in developing innovative gene therapies for rare diseases. Additionally, advancements in genetic engineering technologies, such as CRISPR-Cas9, are expected to further drive the market by enabling more precise and efficient gene editing. These factors are expected to propel the growth of the gene therapy for rare disease market over the forecast period.
The gene therapy for rare disease market can be segmented based on the type of disease. The dominating sub-segment in this market is genetic disorders. Genetic disorders such as cystic fibrosis, muscular dystrophy, and hemophilia have a high prevalence rate globally. These disorders are caused by mutations or abnormalities in a person’s genetic makeup, and gene therapy offers a promising solution for their treatment. The development of advanced gene editing technologies such as CRISPR-Cas9 has further enhanced the potential of gene therapy in treating genetic disorders. Due to the high demand for effective treatment options for genetic disorders, the genetic disorders segment is expected to dominate the gene therapy for rare disease market.
The Gene Therapy For Rare Disease Market Demand is estimated to be valued at US$ 16.3 Million in 2023 and is expected to exhibit a CAGR of 35.0% over the forecast period 2023–2030, as highlighted in a new report by Coherent Market Insights.
Political: Gene therapy for rare diseases is subject to extensive regulation by government authorities. The approval process for gene therapy products involves stringent regulations to ensure safety and efficacy.
Economic: The gene therapy market is driven by the increasing healthcare expenditure and reimbursement policies for gene therapy treatments. The high cost of gene therapy is a major economic factor influencing market growth.
Social: The rising prevalence of rare genetic diseases and the increasing awareness about gene therapy as a potential treatment option are driving market growth. The acceptance of gene therapy by patients and healthcare professionals is also a social factor impacting the market.
Technological: Technological advancements in gene editing techniques and delivery systems are driving the development of more efficient and targeted gene therapies. The use of viral vectors and non-viral vectors in gene therapy has revolutionized treatment options for rare diseases.
The global gene therapy for rare disease market is expected to witness high growth, exhibiting a CAGR of 35.0% over the forecast period. This growth can be attributed to the increasing prevalence of rare genetic diseases and advancements in gene editing technologies. The genetic disorders segment is expected to dominate the market, driven by the high demand for effective treatments for genetic disorders.
In terms of regional analysis, North America is the fastest-growing and dominating region in the gene therapy for rare disease market. This can be attributed to the presence of key players, high healthcare expenditure, and favorable reimbursement policies in the region.
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 key players have a strong market presence and are actively involved in research and development activities to introduce innovative gene therapy products.
- Source: Coherent Market Insights, Public sources, Desk research
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