Gene therapy for rare diseases aims to treat a wide range of inherited and acquired diseases through the delivery of genetic material into a patient’s cells and tissues. Gene therapy products including viral and non-viral vectors are capable of delivering corrective genes to treat rare inherited disorders such as hemophilia, muscular dystrophy, cystic fibrosis, and Gaucher disease. Growing research into complex gene therapies has led to the development of CRISPR/Cas9 gene editing tools which precisely target defective genes and replace them.
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:
One of the key trends driving growth in the gene therapy for rare diseases market is the increasing support and initiatives by governments worldwide. Many governments are actively collaborating with biopharmaceutical companies and research organizations to invest in developing safer and more effective gene therapies. In countries like the US, EU, Australia and Japan, funding schemes, tax incentives and special regulatory designations are accelerating clinical research. Additionally, patient advocacy groups are raising awareness and philanthropic donations are supplementing government support. These efforts aim to address the high costs and bring down pricing barriers to make transformative gene therapies accessible to more patients suffering from rare genetic disorders.
Threat of new entrants: The threat of new entrants is moderate in the Global gene therapy for rare disease market size given the large investment required for R&D of new therapies. However, collaboration with CROs and research institutes can help lower barriers.
Bargaining power of buyers: The bargaining power of buyers is moderate as most therapies target specific rare diseases with small patient pools. However, presence of alternatives like chemotherapy and surgery increases buyers’ options.
Bargaining power of suppliers: The bargaining power of suppliers is low due to the specialized nature of inputs required in gene therapy development such as viral vectors and gene editing tools. This limits options for companies.
Threat of new substitutes: The threat of new substitutes is high given the ongoing focus on developing alternative treatment options like genetically engineered cell therapies and gene editing tools which can potentially replace gene therapies.
Competitive rivalry: The competitive rivalry is high among major players focused on commercializing gene therapies for multiple rare diseases. Frequent M&As and collaborations are common to gain access to new therapies and expand portfolios.
The global gene therapy for rare disease market is expected to witness high growth over the forecast period driven by approvals of novel therapies, rising prevalence of rare diseases, and improving reimbursement scenario. 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.
North America currently dominates the market and is expected to maintain its leading position throughout the forecast period owing to availability of advanced healthcare facilities and presence of major market players in the region.
Europe is also a significant market for gene therapy and is anticipated to exhibit a fast growth rate over the next few years supported by strong government funding for research.
Key players operating in the gene therapy for rare disease market include 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 highly competitive with these players focusing on new product launches, acquisitions, and collaborations to strengthen their product portfolios and global presence.
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it