The gene therapy for rare disease market involves the therapeutic delivery of nucleic acids into patient's cells and tissues to treat a variety of medical conditions. Gene therapy or gene transfer aims at manipulation of an individual's genes through altering, adding or knocking out a gene to treat a disease. For rare diseases, gene therapy involves administering functional genes to diseased cells in order to replace or augment their function. Over 8,000 rare diseases affect over 300 million people worldwide. The rising prevalence of rare diseases globally is fueling the demand for effective treatment options like gene therapy. Gene therapies are emerging as potential life-changing therapies for rare diseases with high unmet medical needs.

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 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.

Growing demand for effective treatment of rare diseases is propelling the gene therapy for rare disease market. Conventional treatment options for rare diseases are often associated with severe side effects. Gene therapies are emerging as promising targeted treatment approaches with fewer side effects. Advancements in gene therapy technologies and development of novel gene therapy products for rare diseases is further fueling market growth.

Increasing adoption of gene therapies globally is anticipated to drive market expansion during the forecast period. Large rare disease patient pools in the US and EU5 countries along with favorable regulatory frameworks and reimbursement policies are encouraging greater uptake of high priced gene therapies in these regions. However, emerging Asian markets also present lucrative opportunities for gene therapy providers given rising disease awareness and improving access to innovative treatments.

Market drivers

One of the major drivers for this Gene Therapy For Rare Disease Market Share is the rising approval and commercialization of novel gene therapy products for rare diseases. For instance, Zolgensma developed by AveXis, a Novartis company received FDA approval in 2019 for the treatment of spinal muscular atrophy (SMA). Additionally, several late-stage gene therapy candidates are under development that are expected to be commercialized during the forecast period, thus driving market revenues. Another key growth factor is the increasing research funding and government support for the development of gene therapies globally which is encouraging more drug makers to enter this evolving market.

The current geopolitical situation is impacting the growth of the Gene Therapy for Rare Disease Market in various ways. With rising geopolitical tensions between major economies, cross-border collaborations on developing gene therapies have become challenging. Several clinical trials initiated by global consortiums are facing delays or obstacles in patient recruitments across regions due to restrictions. At the same time, focus on indigenous innovations has increased among nations to reduce dependencies. This has led to rise in nationally funded programs for rare disease therapy development. However, limited resources and expertise within boundaries pose constraints. To sustain growth, collaborations among smaller consortiums need to be encouraged while addressing data privacy and exchange barriers. Additionally, supportive regulations and guidelines harmonized across wider geographies can help advance multi-country trials. With geopolitical uncertainties expected to continue, the market will need comprehensive long-term strategies ensuring availability of new therapies over coming years through collaborative as well as independent efforts.

In terms of geographical concentration, North America currently holds a major share of the Gene Therapy for Rare Disease Market value, led by the US. This can be attributed to the presence of several leading biotech and pharmaceutical companies involved in gene therapy development. The region also has a relatively advanced healthcare infrastructure and patient affordability for high-cost therapies. Europe is the second largest regional market with major contributors being Germany, France, and the UK. However, Asia Pacific is emerging as the fastest growing geographical region for this market. This growth can be credited to rising healthcare investments, universal healthcare programs, and increasing focus on rare disease management in major APAC economies like China and Japan.

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