by Gene therapy for rare diseases aims to modify the genetic cause of disorders by substituting the defective gene or supplementing a missing gene. Over 7,000 rare diseases affect an estimated 350 million people worldwide and 80% have a known genetic cause. Newer gene delivery technologies like viral and non-viral vectors are revolutionizing treatment options for rare genetic disorders like hemophilia, retinal dystrophies and cystic fibrosis.
Gene therapy products replace, inactivate or supplement defective genes to correct the underlying cause of a rare disease. Growing investment in gene therapy research and support from regulatory agencies have fostered development of new treatments. Recent approvals and significant pipeline demonstrate promise of gene therapy to treat previously untreatable orphan conditions.
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 2023-2030, as highlighted in a new report published by Coherent Market Insights.
Market key trends:
The gene therapy landscape is accelerating with advancement of vector technologies. Significant investments in viral vector manufacturing is enhancing vector engineering and delivery to target tissues. Non-viral systems mimicking viruses are also gaining focus due to their potential for repeat administration. Regulatory agencies are providing accelerated assessments and designations to expedite therapies for rare diseases. With continued innovation in vector design and characterization, gene therapies hold promise to transform treatment of many currently incurable rare conditions.
Porter’s Analysis
Threat of new entrants: The biotechnology market for gene therapy of rare diseases has moderate barriers for new entrants due to the need for extensive R&D efforts and approval from regulatory authorities.
Bargaining power of buyers: The bargaining power of buyers is moderate as patients cannot forego treatment for rare life-threatening diseases even if prices are high.
Bargaining power of suppliers: Suppliers such as gene therapy manufacturers have high bargaining power given their specialized expertise and resources required to develop new therapies.
Threat of new substitutes: The threat of new substitutes is low as alternative treatment options for most rare diseases are limited.
Competitive rivalry: The competitive rivalry in the market is intense as established players are investing heavily in R&D to develop novel gene therapies.
Key Takeaways
The Global Gene Therapy For Rare Disease Market Demand is expected to witness high growth. 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 2023-2030.
The North America region currently dominates the global gene therapy for rare diseases market due to presence of established healthcare infrastructure, large patient population, increasing focus of key players, and high awareness levels regarding rare diseases. Asia Pacific region is poised to witness fastest growth owing to growing healthcare expenditure, increasing prevalence of rare disorders, and growing initiatives by government and private organizations to spread awareness.
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
*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
