The 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 11.% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.
Market Overview:
Gene therapy for rare diseases uses genetic material to treat or cure diseases caused by errors in genes or chromosomes.It involves inserting a functional gene into a person's cells to replace a gene that is nonfunctional or missing. This repairs or replaces a gene that is not working properly.Gene therapy aims to cure diseases by targeting the root cause of the disease at the genetic level rather than just alleviating symptoms. It has the potential to transform the treatment of previously untreatable diseases.
Market key trends:
Development of gene corrections therapies: There has been significant advancement in developing gene correcting therapies by addressing the root cause of disease. Therapies like in-vivo gene editing and in-vivo mRNA delivery is showing promising results in clinical trials for treating rare genetic diseases. Advancement in gene delivery vectors is also helping in improving efficacy and safety of gene therapies.
Porter's Analysis
Threat of new entrants: The threat is low as the gene therapy market requires huge investment for R&D and clinical trials.
Bargaining power of buyers: The bargaining power of buyers is high as the treatment options for rare diseases are limited.
Bargaining power of suppliers: Suppliers have moderate bargaining power due to the specialized nature of inputs required for gene therapy development.
Threat of new substitutes: Gene therapy faces minimal threat from substitutes currently as it offers a curative approach for some rare genetic disorders.
Competitive rivalry: Intense as major players are focusing on pipeline development and product approvals.
SWOT Analysis
Strengths: Gene therapy offers a curative and long-lasting treatment approach for some rare genetic disorders. Growing R&D investments and technological advancements.
Weaknesses: High costs involved, complex development process, safety and efficacy concerns. Limited treatment options for some rare diseases.
Opportunities: Increasing prevalence of rare diseases, rising healthcare expenditure, growing focus on orphan drugs. Collaborations between pharma companies and research institutes.
Threats: Stringent regulations, reimbursement challenges, IP issues, biosimilars introduction post patent expiration.
Key Takeaways
The Global Gene Therapy For Rare Disease Market Demand is expected to witness high growth, exhibiting CAGR of 11% over the forecast period, due to increasing prevalence of rare genetic disorders globally. As per estimation, over 350 million people are affected by rare diseases worldwide. Gene therapy offers a permanent cure by correcting the underlying genetic defects, driving its adoption.
Regionally, North America dominated the global market and captured over 40% share in 2023 owing to presence of advanced healthcare facilities, favorable reimbursement policies and increasing awareness. Europe was the second largest market led by growing R&D investments by key players. Asia Pacific is anticipated to be the fastest growing region during the forecast period due to rising healthcare spending, availability of low-cost manufacturing facilities and expanding target patient pool.
Key players operating in the gene therapy for rare disease market are Amgen Inc., Biomarin Pharmaceuticals, Bayer AG. Major players are focusing on strengthening their pipeline through partnerships, M&A and new product approvals to gain competitive edge. In 2023, Amgen launched Libmeldy for treatment of metachromatic leukodystrophy in Europe.
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