Biotechnology

Gene Therapy For Rare Disease: Gene Therapy Fueling Growth In Rare Disease Treatment

Leena Shedmake

Gene Therapy For Rare Disease: Gene Therapy Fueling Growth In Rare Disease Treatment

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 11.% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.

Market Overview:

Gene therapy products aim to correct defective genes that cause rare diseases. These products help replace faulty or missing genes in order to cure rare genetic disorders. Rising research for effective treatment is fueling the growth of this market.

Market key trends:

One of the major trends in the gene therapy for rare disease market is the increasing focus on adeno-associated virus gene therapy. Adeno associated viruses are attracting interest as they can carry therapeutic genes into cells and produce long-term expression of missing or faulty proteins without integrating into the cell's chromosome or causing diseases. They can be administered systematically and target a wide range of tissues. Several clinical trials are ongoing using these viruses as vectors to treat rare diseases like hemophilia and retinal dystrophies. This will likely create greater opportunities in the market.

Segment Analysis

The global gene therapy for rare disease market has been segmented based on vector, application, disease type, and end user. The vector segment is further divided into viral vector and non-viral vector. The viral vector segment currently dominates the market due to its higher transduction efficiency and ability to deliver larger genes compared to non-viral vectors.

Key Takeaways

The Global Gene Therapy For Rare Disease Market Size is expected to witness high growth, exhibiting CAGR of 11.% over the forecast period, due to increasing investment in R&D by key players for developing novel and effective gene therapy.

Regional analysis:

North America accounts for the largest share in the global gene therapy for rare disease market. This can be attributed to growing awareness regarding rare diseases and availability of advanced healthcare facilities. However, Asia Pacific is expected to register the fastest growth over the forecast period owing to rising healthcare expenditures, increasing discretionary incomes, and growing clinical trials of gene therapies in countries such as China and India.

Key players:

Key players operating in the gene therapy for rare disease market are Amgen Inc., Biomarin Pharmaceuticals, and Bayer AG. Amgen Inc. dominates the market with its extensive product portfolio and robust geographic presence across North America and Europe. Biomarin Pharmaceuticals focuses on developing gene therapies for rare Inherited Metabolic Diseases.

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Leena Shedmake

Gene Therapy For Rare Disease Market Growth Accelerated By Increasing Number Of Clinical Trials

Leena Shedmake 2024-01-18

The growing focus on developing new therapies for rare diseases has led to increasing number of clinical trials evaluating gene therapy. The increasing clinical research reflects the promise of gene therapy for rare diseases with unmet medical needs. The global Gene Therapy for Rare Disease Market is estimated to be valued at US$ 193. Segment AnalysisThe global gene therapy for rare disease market is dominated by the gene therapy segment. Key Takeaways The Global Gene Therapy For Rare Disease Market Demand is expected to witness high growth over the forecast period. The global Gene Therapy for Rare Disease Market is estimated to be valued at US$ 193.

Gene Therapy For Rare Disease Market Poised To Grow At Highest Pace Owing To Increasing Healthcare Expenditure

Pooja Khodke 2024-02-09

The gene therapy for rare disease market involves the development and application of treatments that modify or manipulate a person's genes to treat rare diseases. The Global Gene Therapy For Rare Disease Market is estimated to be valued at Us$ 222. Key TakeawaysKey players operating in the Gene Therapy For Rare Disease are Topcon Corporation, Agfa-Gevaert N. Impact of COVID-19 on Gene Therapy for Rare Disease Market GrowthThe COVID-19 pandemic impacted the growth of the gene therapy market for rare diseases. Geographical Concentration of Gene Therapy for Rare Disease MarketNorth America accounts for the largest share of the global gene therapy market for rare diseases in terms of value, followed by Europe.

Gene Therapy for Rare Disease Market is Estimated To Witness High Growth Owing To Increasing Investments in Research and Development

Anjali CMI 2023-09-13

The global Gene Therapy for Rare Disease Market is estimated to be valued at US$ 16. Economic: The economic factors affecting the gene therapy for rare disease market focus on the affordability and reimbursement policies for gene therapy treatments. The growing understanding of the potential benefits of gene therapy and the willingness of patients to explore new treatment options drive the demand for gene therapy products. Technological: The technological factors affecting the gene therapy for rare disease market include advancements in gene editing techniques and delivery systems. Regionally, North America is anticipated to be the fastest-growing and dominating region in the gene therapy for rare disease market.

Growing Demand For Gene Therapy For Rare Diseases To Drive Market Growth

Ashish Thapa 2023-10-27

The increasing incidence of rare diseases worldwide, coupled with advancements in gene therapy technologies, is driving the demand for gene therapy for rare diseases. Overall, the gene therapy for rare disease market is witnessing significant growth and is expected to continue expanding at a rapid pace. The increasing prevalence of rare diseases and the potential of gene therapy to offer innovative treatment options are driving market growth. Segment Analysis:The gene therapy for rare disease market can be segmented based on therapy type and disease type. The Gene Therapy For Rare Disease Market Demand is estimated to be valued at US$ 16.

Rare Disease Gene Therapy Market Size will Observe Substantial Growth by 2030

Devanand Patil 2021-07-23

Rare Disease Gene Therapy Market: SnapshotThe global rare disease gene therapy is expected to witness a significant growth in the forecast period, 2020-2030 on account of the increasing cases of genetic diseases worldwide.

These kids are expected to experience complex clinical issues in the wake of getting live-sparing bone marrow transfers to treat the condition.Request Brochure – https://www.transparencymarketresearch.com/sample/sample.php?flag=B_id=80404The report offers insights into the market emphasizing on factors boosting, repelling, challenging, and creating opportunities for the market in the forecast period.

In terms of indication, the market is grouped into inflammatory diseases, neurological diseases, infectious diseases, hematology diseases, oncology diseases, genetic diseases, and others.

What's more, clinical-stage improvement organizations are allotting critical assets towards the advancement of the uncommon illness quality treatment market.For example, in August 2020, the U.S. based Ovid Therapeutics teamed up with Italian firm Angelini Pharma to create, make and popularize drug gaboxadol for treatment of Angelman disorder (uncommon hereditary sickness) in European market.Some of the players of the global rare disease gene therapy market include Amgen Inc., AbbVie Inc., F.Hoffmann-La Roche Ltd., Merck & Co., Johnson & Johnson, Baxter, Novo Nordisk A/S, Alexion, Pfizer Inc., Bristol-Myers Squibb Company, Novartis AG, Sanofi S.A., Teva Pharmaceutical Industries Ltd., Bayer AG, Allergen plc., Vertex Pharmaceuticals, and others.Request Customization on Rare Disease Gene Therapy Market Report – https://www.transparencymarketresearch.com/sample/sample.php?flag=CR_id=80404Rare Disease Gene Therapy Market: Interesting InsightsThere has been an intense competition among biopharmaceutical players to create Covid treatment that is postponing on account of innovative work exercises in uncommon infection quality treatment market.

Stop of assembling exercises and flexibly tie disturbance because of exchanging across outskirts will have sway on uncommon illness quality treatment market development rate.

Also, clinical-stage advancement organizations are allotting critical assets towards the improvement of the uncommon infection quality treatment market.Pre Book Rare Disease Gene Therapy Market Report –https://www.transparencymarketresearch.com/checkout.php?rep_id=80404=S Rare Disease Gene Therapy Market: Regional CategorizationNorth America will keep on overwhelming the uncommon illness quality treatment market in the coming decade with a high pace of endorsements and commercialization of genomic medication.

Rare Disease Overview: Challenges and Research Innovations

Zoe Green 2023-07-24

A rare disease, also known as an orphan disease, is a medical condition that affects a small number of people. Hutchinson-Gilford progeria syndrome: This rare genetic disorder causes rapid aging in children, resulting in a variety of health issues. The development of gene therapy for rare diseases helps scientists identify disease-causing mutations and develop targeted therapies, leading to potential breakthroughs for several rare diseases. Applying precision medicine principles to rare disease research can lead to more effective and customized treatments, potentially resulting in substantial breakthroughs. Collaborative efforts, supported by technological platforms and data-sharing initiatives, can help unite researchers worldwide and provide access to larger datasets, accelerating rare disease research and facilitating breakthroughs.

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