Gene therapy is a technique that uses genes to treat or prevent disease. It involves the delivery of nucleic acids into a patient’s cells and tissues to treat a disease. Gene therapies are designed to introduce a normal gene into cells to replace a disease-causing gene mutation or introduce genes to help regulate the production of certain proteins. Gene therapy holds promise for treating a wide range of both rare and common diseases, like cancer, cystic fibrosis, heart disease, diabetes, hemophilia, and AIDS. It is a potential alternative to drugs and aggressive surgeries which can cause side effects and complications.
The global Gene Therapy for Rare Disease Market is estimated to be valued at US$ 193.56 Bn in 2024 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 key trends:
Increase in prevalence rates of rare diseases is expected to boost the growth of the Global gene therapy for rare disease market size over the forecast period. For instance, according to the National Organization for Rare Disorders (NORD), it is estimated that over 25 million Americans live with over 7,000 rare diseases. Rare diseases are often chronic, progressive, degenerative, and life-threatening conditions which have fewer treatment options available as compared to common diseases. Therefore, the adoption of gene therapy, which provide potential cure for such diseases, is expected to grow significantly in the coming years. Technological advancements in gene therapy research and growing clinical trials for rare diseases are also expected to propel the market growth.
SWOT Analysis
Strength: Gene therapy for rare disease provides a potentially transformative treatment option for conditions with limited therapeutic options. It aims to treat the underlying genetic mutations of rare diseases.
Weakness: High costs associated with development of gene therapies. Long-term safety and efficacy data is still limited for many gene therapies.
Opportunity: Increased investments from government and private sectors to accelerate research and development of gene therapies. Growing orphan drug designations and medical technology advancements provide a favorable environment.
Threats: Complex regulatory pathways for approval of gene therapies. Manufacturing challenges associated with producing consistent gene therapy products at commercial scale.
Key Takeaways
The global gene therapy for rare disease market is expected to witness high growth. Gene therapies have the potential to transform rare disease treatment landscape by addressing the root cause of conditions. The development of various gene therapy platforms and delivery mechanisms have enabled targeting of wide range of genetic disorders. The global Gene Therapy for Rare Disease Market is estimated to be valued at US$ 193.56 Bn in 2023 and is expected to exhibit a CAGR of 5.9% over the forecast period 2023 to 2030.
Regional analysis: North America dominates the market currently due to presence of majority of key market players and favorable regulatory environment for orphan drugs in the US. The Asia Pacific region is expected to witness fastest growth during the forecast period supported by growing investments from pharmaceutical companies and governments to strengthen R&D infrastructure for rare disease therapies.
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, EyePACS, VersaSuite, Heidelberg Engineering GmbH, ScImage, Inc., IBM Corporation, and Visbion Limited. The market is competitive with presence of large gene therapy developers and technology providers focused on various orphan indications. Collaborations between pharmaceutical companies and research institutions are also common to advance the pipeline.
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it