Biotechnology

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

Ashish Thapa

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

Market Overview:

Gene therapy for rare diseases involves the introduction of genetic material into a patient's cells to treat or prevent a rare genetic disorder. It offers great potential in addressing diseases that have no cure or limited treatment options. This therapy holds immense advantages, including the potential to provide long-term solutions, target specific genetic mutations, and improve patient outcomes and quality of life. The increasing incidence of rare diseases worldwide, coupled with advancements in gene therapy technologies, is driving the demand for gene therapy for rare diseases.

Market Key Trends:

One key trend in the gene therapy for rare disease market is the increasing investment in research and development activities. Many pharmaceutical companies are investing significantly in developing gene therapies for various rare diseases, aiming to bring novel treatments to market. This trend is fueled by the growing understanding of the genetic basis of rare diseases and the potential of gene therapy to provide targeted and personalized treatment options. Moreover, favorable regulatory policies and initiatives to accelerate the development and commercialization of gene therapies are further facilitating market growth.

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. Investments in research and development activities and supportive regulatory frameworks are further contributing to the expansion of this market.

Segment Analysis:

The gene therapy for rare disease market can be segmented based on therapy type and disease type. Based on therapy type, the dominating sub-segment is ex vivo gene therapy. Ex vivo gene therapy involves the extraction of cells from the patient's body, manipulation of the genetic material to introduce therapeutic genes, and then reintroduction of the modified cells back into the patient. This therapy type is dominating due to its higher success rates and broader applicability across various rare diseases. It allows for precise genetic modification and can target specific cells, resulting in better outcomes for patients.

The Gene Therapy For Rare Disease Market Demand is estimated to be valued at US$ 16.3 Million in 2023 and is expected to exhibit a CAGR of 35.0% over the forecast period 2023–2030, as highlighted in a new report by Coherent Market Insights.

Key Takeaways:

This growth can be attributed to various factors, including increasing government initiatives to support rare disease treatment, advancements in genetic research and technology, and a growing number of clinical trials for gene therapy. For example, the US Food and Drug Administration (FDA) has designated orphan drug status and accelerated review pathways for gene therapies, facilitating their development and approval.

In terms of regional analysis, North America is expected to be the fastest-growing and dominating region in the gene therapy for rare disease market. This can be attributed to factors such as high healthcare expenditure, robust infrastructure for gene therapy research and development, and favorable reimbursement policies. The region also has a large patient population affected by rare diseases, which creates a strong demand for gene therapies.

Key players operating in the gene therapy for rare disease market include Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc. These key players are actively involved in research and development activities, strategic collaborations, and mergers and acquisitions to gain a competitive edge in the market. For example, Kite Pharma, Inc. was acquired by Gilead Sciences, Inc. to strengthen its gene therapy portfolio and expand its market presence.

Ashish Thapa

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.

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.

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.

Gene Therapy Market 2022: New Trends, Drivers and Restraints

Biswadeep 2022-02-15

TBRC’s market report includes a chapter on the COVID-19 impact on the gene therapy industry, which gives valuable insights on supply chain disruptions, logistical challenges, and other economic implications of the virus on the market and how companies can strategize to bounce back from it. With updated market numbers according to the effects of the coronavirus, the report provides the most accurate expected gene therapy market growth numbers from 2022-2031. id=3395&type=smp The global gene therapy market is expected to grow from $5. The gene therapy market is expected to reach $21. Key Mergers And Acquisitions In The Gene Therapy Market30.

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