Gene therapy is an experimental technique that uses genes to treat or prevent disease. In rare diseases, gene therapy aims to supplement a missing gene or inactivate a faulty gene that is causing disease. Diseases like hemophilia, cystic fibrosis, sickle cell anemia, and several types of blindness and cancer are potential targets for gene therapy. The global rare disease treatment market is largely driven by rising investment in research and development for orphan drugs and gene therapies.
The Global Gene Therapy for Rare Disease Market is estimated to be valued at US$ 222.99 Mn in 2024 and is expected to exhibit a CAGR of 5.9% over the forecast period 2024 to 2030.
Key Takeaways
Key players operating in the Gene Therapy for Rare Disease market are Topcon Corporation, Agfa-Gevaert N.V., Sonomed Escalon, Carl Zeiss Meditec AG, EyePACS, VersaSuite, Heidelberg Engineering GmbH, ScImage, Inc., IBM Corporation, and Visbion Limited. These players are extensively conducting R&D activities and launching new gene therapy products to tap growth opportunities.
Some key opportunities in the market include developing tailored gene therapies for specific rare diseases and increasing awareness about approved gene therapies. Companies are also expanding their reach globally by collaborating with regional players and setting up new manufacturing and research facilities across different countries.
The growing orphan drug pipeline and increasing approval of gene therapies are expected to drive global market expansion during the forecast period. Companies are actively conducting clinical trials across various regions to cater to the unmet needs of patients worldwide.
Market drivers
The increasing prevalence of rare genetic diseases is a key growth driver for the market. According to the National Organization for Rare Disorders (NORD), over 7,000 rare diseases affect 25-30 million Americans. Gene therapy provides novel treatment options for many such conditions with no approved therapies.
Market restraints
However, high development costs of gene therapies pose a major challenge. Developing a single gene therapy typically requires an investment of $100 million to $1 billion. Additionally, uncertainties associated with clinical success and regulatory approval also restrain the market growth.
Segment Analysis
Gene Therapy For Rare Disease Market Share has two dominating segments - replacing a disease-causing gene with a healthy copy and inactivating a disease-causing gene that is not functioning properly. The replacing a disease-causing gene with a healthy copy segment has highest market share as it provides a permanent treatment for genetic disorders by addressing the underlying cause. This sub segment is dominating as it treats the root cause of genetic disorders and provides long lasting benefits as compared to other treatment options.
Global Analysis
North America region dominates the global gene therapy for rare disease market and accounts for largest revenue share. Presence of developed healthcare infrastructure, high disposable income, rising incidence of rare diseases in the region are some factors fueling the market growth. Asia Pacific region is fastest growing region owing to increasing healthcare spending, rising awareness about gene therapy, and improving healthcare facilities in countries like China and India. Developing markets in Asia offers high growth potential for market players over the forecast period.
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