Gene therapy involves modifying genes or introducing new ones into patient’s cells and tissues to treat diseases. It aims at replacing, manipulating, or supplementing non-functional genes to cure rare inherited diseases, some types of cancers, and other medical conditions. With the rising prevalence of rare diseases due to genetic disorders and mutations in chromosomes, the need for effective therapies has grown exponentially. Government initiatives aimed at creating funding and reimbursement policies are also strengthening the research and development activities in gene therapy.
Some key applications of gene therapy include treatment of cancer, genetic disorders like cystic fibrosis, hemophilia, sickle cell disease, and HIV/AIDS. Manufacturers are extensively conducting research for developing highly advanced and innovative gene therapies such as CAR T-cell therapy, gene editing, and gene therapy vaccines.
The global gene therapy for rare disease market is estimated to be valued at US$ 193.56 billion in 2023 and is expected to exhibit a CAGR of 5.9% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.
Market Dynamics
The increasing awareness among patients and physicians about gene therapy and the availability of various clinical trials are driving the market growth. The heading had highlighted that gene therapy for rare diseases is expected to be flourished by increasing awareness and government support. Government bodies are providing reimbursement for gene therapy drugs and are investing heavily in researcher. This government support positively impacts the market by encouraging research and facilitating commercialization.
Additionally, continuous improvements in genetic engineering technologies including gene editing tools such as CRISPR-Cas9 are propelling the gene therapy market as they enable accurate modification of genes. CRISPR-Cas9 has emerged as a promising gene therapy approach and many companies have initiated clinical trials for rare diseases using CRISPR-Cas9.
Segment Analysis
The gene therapy for rare disease market is dominated by the segment Disease Type. Within disease type, neurological disorders hold the dominating share of the segment owing to the increasing prevalence of neurological rare diseases such as Mucopolysaccharidosis disease, Pompe disease etc. Also, there are promising candidates in pipeline and approved products available for the treatment of these diseases using gene therapy.
PEST Analysis
Political: Government regulations and initiatives related to orphan drug designation and tax benefits are supporting research and development of gene therapies for rare diseases. However, high costs related to gene therapy development and manufacturing require supportive healthcare policies.
Economic: Rising healthcare spending and increasing demand for effective treatment of rare diseases is driving the gene therapy market growth. However, high costs of these therapies pose challenges.
Social: Growing support from patient advocacy groups and awareness about rare diseases is contributing to the market growth.
Technological: Advancements in vector engineering, targeted delivery and manufacturing technologies are expanding applications of gene therapies and reducing costs. CRISPR/Cas9 technology also provides opportunities.
Key Takeaways
The Global Gene Therapy For Rare Disease Market Growth is expected to witness high. The market size for 2023 is estimated to be US$ 193.56 Bn with a CAGR of 5.9% during the forecast period of 2023 to 2030.
Regional analysis
North America is expected to dominate the market during the forecast period owing to established healthcare system, high healthcare spending, presence of major players and early approval of gene therapies in the region. However, Asia Pacific is anticipated to witness fastest growth during the forecast period attributed to growing healthcare infrastructure, increasing disposable incomes and expanding patient population in countries such as China and India.
Key players
Key players operating in the gene therapy for rare disease market are Topcon Corporation, Agfa-Gevaert N.V., Sonomed Escalon, Carl Zeiss Meditec AG. Topcon Corporation and Carl Zeiss Meditec AG are recognized as global leaders in the ophthalmic imaging systems space with expanded product portfolios and global presence.
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1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it