The Gene Therapy for Rare Disease Market is estimated to be valued at US$ 16.3 million in 2017 and is expected to exhibit a CAGR of 35.0% over the forecast period 2018-2026, as highlighted in a new report published by Coherent Market Insights.
Gene therapy involves the introduction of normal genes into cells that have abnormal or missing genes, thus correcting genetic disorders at the molecular level. The gene therapy products for rare diseases are designed to treat a variety of genetic disorders such as inherited retinal disorders, blood disorders, neurodegenerative disorders, and muscular disorders, among others. These products offer a potential cure for patients suffering from rare diseases, which often have limited treatment options.
The gene therapy for rare disease market is primarily driven by the increasing prevalence of rare diseases globally. According to the National Institutes of Health (NIH), there are around 7,000 rare diseases identified, affecting approximately 25-30 million Americans. Additionally, advancements in gene therapy techniques, such as viral vectors, gene editing tools (such as CRISPR-Cas9), and genome sequencing technologies have significantly contributed to the growth of this market. These advancements have improved the efficiency and safety of gene therapy products, thereby increasing their adoption. However, high treatment costs and lack of reimbursement policies in some regions may hinder market growth to some extent.
The gene therapy for rare disease market can be segmented based on the type of therapy, delivery method, and end-user. Among these segments, the therapy type segment dominates the market, accounting for a significant share. This is primarily due to the increasing prevalence of rare diseases and the growing demand for effective treatment options. Gene therapy offers a promising approach for the treatment of these diseases by targeting the underlying genetic cause. Furthermore, advancements in gene editing technologies and improved understanding of genetic disorders have led to the development of more targeted and personalized therapies, further driving the dominance of this segment.
Political: The political environment plays a crucial role in the gene therapy for rare disease market. Government regulations and policies regarding gene editing technologies and gene therapies can have a significant impact on the market’s growth. For instance, stringent regulations for clinical trials and approvals can delay the commercialization of new therapies.
Economic: The economic factors influencing the market include healthcare expenditure, funding for research and development, and healthcare infrastructure. The growing investments in gene therapy research and development, along with increasing healthcare spending, are expected to positively impact market growth.
Social: The social factors shaping the gene therapy for rare disease market involve the societal acceptance of gene editing technologies and public awareness of these therapies’ potential benefits. Increased awareness and acceptance among patients, healthcare professionals, and the general public can drive the adoption of gene therapy for rare disease treatment.
Technological: Technological advancements in gene editing techniques, such as CRISPR-Cas9, have revolutionized the field of gene therapy. These advancements have improved the precision and efficiency of gene editing, opening up new possibilities for the development of effective therapies. Additionally, advancements in viral vector technologies and gene delivery systems have contributed to the growth of the gene therapy for rare disease market.
The global Gene Therapy for Rare Disease Market Size is expected to witness high growth, exhibiting a compound annual growth rate (CAGR) of 35.0% during the forecast period of 2018-2026. This growth can be attributed to increasing investments in research and development, advancements in gene editing technologies, and the rising prevalence of rare diseases.
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 major pharmaceutical and biotechnology companies in the region, favorable regulatory environment, and advanced healthcare infrastructure.
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 involved in research and development activities and strategic collaborations to strengthen their market position.