Gene therapy for rare disease involves gene transfer techniques that alter or modify the expression of a gene or its activity to cure genetic disorders. Gene therapy is emerging as a viable treatment option for various rare genetic diseases, such as hemophilia, hearing loss, retinitis pigmentosa, Gaucher's disease, and Sanfilippo syndrome, for which conventional treatment options are not available or are highly inadequate. The method is advantageous as it can potentially provide a functional cure for inherited disorders that are difficult to treat using traditional drugs. Additionally, gene therapy has the potential for one-time treatment instead of lifelong treatments needed for conventional therapies.
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, as highlighted in a new report published by Coherent Market Insights.
Market Opportunity:
The high unmet medical needs associated with rare diseases present a major market opportunity for gene therapy. Rare diseases impact a large patient population but have limited treatment options. Currently, approximately 95% of rare diseases lack approved disease-modifying therapies. Gene therapy offers the promise of providing targeted treatments for several rare diseases by supplementing or correcting defective genes. This establishes a significant market opportunity for companies developing gene therapies for rare diseases to address unmet medical needs and improve patient outcomes.
Porter's Analysis
Threat of new entrants: The threat of new entrants is low as developing gene therapies requires huge R&D investment and clinical trials. Strict regulations by regulatory authorities also act as a barrier.
Bargaining power of buyers: The bargaining power of buyers is high due to presence of various gene therapy providers. Buyers can choose from various treatment options based on individual needs.
Bargaining power of suppliers: Gene therapy developers and manufacturers have significant bargaining power over buyers as developing gene therapies require specialized expertise and facilities.
Threat of new substitutes: Threat of substitutes is low as alternative treatment options like small molecule drugs or biologics cannot effectively treat genetic diseases.
Competitive rivalry: The competition is high among existing gene therapy developers to develop safer and effective treatments for rare genetic diseases.
SWOT Analysis
Strengths: Gene therapies offer potentially curative treatments for various rare genetic diseases with no other treatment options. Growing research activities support the development of new gene therapies.
Weaknesses: High costs associated with R&D and production of gene therapies. Safety concerns remain major challenges due to complex nature of these therapies. Limited patient pool also affects commercial viability.
Opportunities: Increasing support from governments and funding agencies boost research in this sector. Growing orphan drug designation and incentives accelerate the approval process.
Threats: Risks of gene therapy resistance and immune reactions. Stringent regulations and lengthy approval process delay market access of new therapies. Manufacturing challenges at commercial scale also threaten smooth supplies.
Key Takeaways
The Global Gene Therapy For Rare Disease Market Growth is expected to witness high due to increasing pipeline promising therapies and approvals. 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.
The US and major European countries dominate the market due to high healthcare spending and presence of key market players. North America is currently the largest and fastest growing regional market for gene therapies due to supportive regulatory environment and increasing R&D activities in this sector supported by private and public funding. The US market in particular has a stable patient pool supported by reimbursement programs boosting commercial adoption of approved gene therapies. Europe is also witnessing significant growth due to initiatives promoting gene therapy research and availability of advanced healthcare facilities.
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. These players are focused on enhancing their gene therapy portfolios through acquisitions, new product launches, and expanded manufacturing facilities globally to cater to the growing demand.
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