by Gene therapy works by treating genetic disorders by introducing corrected genes into the patient’s cells or by blocking abnormal genes that cause rare diseases. Some of the main advantages of gene therapy include long-term effects, targeted approach, reduced need for medications and potential to cure genetic conditions. With increasing research in genetic science, gene therapy holds promise for treating several rare genetic disorders like muscular dystrophy, hemophilia, cystic fibrosis, and some forms of cancer.
The Global Gene Therapy for Rare Disease Market is estimated to be valued at US$ 8.9 BN in 2024 and is expected to exhibit a CAGR of 9.0% 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. With increasing R&D investments, these players are developing novel gene therapy techniques and products to address unmet medical needs.
The growing demand for gene therapy in cancer treatments is fueling market growth. Gene therapy is emerging as a promising approach for treating cancer by removing or correcting the genetic defects leading to tumor growth. Many gene therapy clinical trials are underway for rare cancer indications.
Globally, the adoption of Gene Therapy For Rare Disease Market Demand is increasing across key markets like North America, Europe, and Asia Pacific. Government support and increased healthcare spending are encouraging translational research in gene therapy. At the same time, rising awareness about gene therapy and its potential to treat previously untreatable diseases is prompting more patients to opt for this treatment approach.
Market Key Trends
One of the key trends in the gene therapy market is the growing popularity of CAR T-cell therapy for treating hematological cancers. CAR T-cell therapy is an immunotherapy that utilizes gene therapy methods to reprogram patient’s T cells to attack cancer cells. Drugs like Kymriah and Yescarta have significantly improved survival rates in relapsed/refractory leukemia and lymphoma. With further advancements, CAR T-cell therapy offers hope to cancer patients worldwide.
Porter’s Analysis
Threat of new entrants: High costs in gene therapy research and manufacturing acts as a barrier.
Bargaining power of buyers: Patients have limited options thus less power.
Bargaining power of suppliers: Suppliers have some control as number of approved gene therapies is limited.
Threat of new substitutes: Potential for new gene therapy approaches but targets are still narrow.
Competitive rivalry: Companies compete on technology pipelines and manufacturing capacity.
Geographical Regions
North America holds the largest share of the gene therapy for rare disease market currently due to higher awareness and adoption of advanced therapies. The availability of robust healthcare infrastructure and reimbursement for new treatment options also contributes to its large market size.
Asia Pacific is poised to grow at the fastest rate during the forecast period. This is attributed to rising healthcare expenditures, growing middle-class population with greater access to treatment, and increasing collaborations with global gene therapy companies. Countries like China and India are aggressively funding R&D to develop indigenous research capabilities as the need for therapies in genetic and rare disorders grows.
*Note:
1.Source: Coherent Market Insights, Public sources, Desk research
2.We have leveraged AI tools to mine information and compile it
