Biotechnology

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

Leena Shedmake

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

Gene therapy for rare diseases incorporate the development and delivery of therapeutic genes or genetically modified cells for the treatment of rare genetic disorders. Gene therapy holds immense potential for the treatment of various rare diseases by replacing, manipulating or supplementing disease-causing genes. The growing focus on developing new therapies for rare diseases has led to increasing number of clinical trials evaluating gene therapy. According to ClinicalTrials.gov, there were more than 2,700 active gene therapy clinical trials registered as of September 2022, out of which over 150 clinical trials were for various rare genetic disorders. 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$ 193.56 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 key trends:

The increasing number of clinical trials is one of the major trends in the gene therapy for rare disease market. Gene therapies offer considerable potential in the treatment of rare genetic diseases by replacing, manipulating or supplementing defective genes. This has led to significant research efforts from biotechnology and pharmaceutical companies towards developing new gene therapies. For instance, Freeline Therapeutics is conducting a phase 1/2 study to evaluate B-L001 for the treatment of Fabry disease. Another example is Lysogene, which is evaluating LYS-SAF302, an investigational gene therapy for MPS IIIA. The positive clinical data emerging from ongoing trials is also expected to boost the adoption of approved gene therapies for rare diseases in the coming years. This increasing clinical research demonstrates the promise of gene therapy as a viable therapeutic option for several rare genetic disorders currently lacking treatment options.

Segment Analysis

The global gene therapy for rare disease market is dominated by the gene therapy segment. Gene therapy aims at manipulating the genetic material of cells and tissues to treat diseases. It involves insertion of a gene into a patient's cells and tissues to either compensate for a mutated gene or to introduce a new function. The gene therapy segment holds the major share due to the increasing demand for effective treatment options for rare diseases and less number of approved treatment available for rare diseases.

Key Takeaways

The Global Gene Therapy For Rare Disease Market Demand is expected to witness high growth over the forecast period. Rising research activities for developing advanced treatment options and growing demand for effective therapies are fueling market growth. 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.

Regional analysis

North America holds the major share in the global gene therapy for rare disease market owing to early adoption of advanced technologies, strong government support for research activities, and presence of key market players in the region. Asia Pacific is projected to grow at the fastest rate during the forecast period due to rising patient pool, increasing healthcare spending, growing awareness regarding availability of treatment options.

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. Topcon Corporation and Agfa-Gevaert N.V. hold the major market share due to their strong geographic presence and robust product portfolio for rare disease treatment.

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Leena Shedmake

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 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.

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.

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

Leena Shedmake 2023-12-22

The global Gene Therapy for Rare Disease Market is estimated to be valued at US$ 193. Several clinical trials are ongoing using these viruses as vectors to treat rare diseases like hemophilia and retinal dystrophies. Segment Analysis The global gene therapy for rare disease market has been segmented based on vector, application, disease type, and end user. 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.

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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