(Albany, USA) Cell and Gene Therapies in Rare Disorders Market Summary
The cell and gene therapy market for rare disorders in the 7MM was valued at nearly USD 2 billion in 2023 and is projected to grow substantially by 2034. This growth is fueled by increasing investigational new drug applications, rising regulatory approvals in the US and Europe, and expanding global clinical studies. Japan leads in rapid development and approvals. While notable progress is seen, especially in ophthalmology with stem cell therapies for limbal stem cell deficiency (LSCD), many therapeutic areas remain underdeveloped. Intensifying competition among pharma, biotech, and contract manufacturing organizations may escalate production costs, prompting companies to invest in advanced manufacturing technologies or strategic acquisitions to maintain a competitive edge.
DelveInsight’s “Cell and Gene Therapies in Rare Disorders Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Cell and Gene Therapies in Rare Disorders, historical and forecasted epidemiology as well as the Cell and Gene Therapies in Rare Disorders market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.
To Know in detail about the Cell and Gene Therapies in Rare Disorders market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Cell and Gene Therapies in Rare Disorders Market Forecast
Some of the key facts of the Cell and Gene Therapies in Rare Disorders Market Report:
- The Cell and Gene Therapies in Rare Disorders market size was valued ~USD 2,000 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034)
- In December 2024, EyeDNA Therapeutics, a subsidiary of Coave Therapeutics, has received Rare Pediatric Disease (RPD) Designation from the U.S. FDA for its investigational gene therapy, HORA-PDE6b, intended to treat retinal dystrophy caused by mutations in the PDE6b gene.
- In February 2024, REGENXBIO reported topline results from its pivotal phase trial of RGX-121 for Hunter syndrome, highlighting that the study successfully achieved its primary endpoints with statistical significance.
- In 2023, the market size for cell and gene therapies targeting rare disorders in the US was around USD 800 million and is expected to grow throughout the forecast period (2024–2034).
- By 2034, Hemophilia A is projected to generate the highest revenue among all indications in the US, followed by Retinitis Pigmentosa.
- In 2023, ZOLGENSMA (onasemnogene abeparvovec-xioi) held the largest market share among therapies in the US.
- In 2023, the total prevalent cases of selected indications for Cell and Gene Therapies in Rare Disorders across the 7MM were approximately 930,200, with projections indicating an increase during the forecast period.
- In 2023, Retinitis Pigmentosa accounted for the highest number of cases among the rare indications selected for Cell and Gene Therapies, followed by Limbal Stem Cell Deficiency.
- In 2023, the total treated cases for cell and gene therapies across selected indications in the 7MM were approximately 504,000, with numbers projected to rise throughout the forecast period (2024–2034).
- Key Cell and Gene Therapies in Rare Disorders Companies: Pfizer, Sangamo Therapeutics, Orchard Therapeutics/ SR-Tiget, Novartis, TVAX Biomedical, Aivita Biomedical, RHEACELL GmbH & Co, Capricor Therapeutics, and others
- Key Cell and Gene Therapies in Rare Disorders Therapies: Fidanacogene elaparvovec, GiroctocogenE fitelparvovec, OTL-103, OAV101, TVI-Brain-1, AV-GBM-1, ABCB5+ MSCs, CAP-1002, and others
- The Cell and Gene Therapies in Rare Disorders market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Cell and Gene Therapies in Rare Disorders pipeline products will significantly revolutionize the Cell and Gene Therapies in Rare Disorders market dynamics.
Cell and Gene Therapies in Rare Disorders Overview
Cell and Gene Therapies in Rare Disorders are expected to witness significant growth by 2034, driven by increasing investigational new drug applications and rising regulatory approvals in the United States and Europe. Cell and Gene Therapies in Rare Disorders benefit from expanding clinical research, particularly as more global studies focus on novel therapeutic approaches.
Notably, Japan is emerging as a leader in this space, with accelerated development and regulatory clearances. Cell and Gene Therapies in Rare Disorders have shown early success in ophthalmology, especially in treating Limbal Stem Cell Deficiency (LSCD), though many other therapeutic areas remain underserved. Cell and Gene Therapies in Rare Disorders face growing competition among pharmaceutical, biotechnology, and contract manufacturing organizations. As more companies enter this market, demand for manufacturing capacity intensifies, potentially driving up costs.
Cell and Gene Therapies in Rare Disorders thus compel firms to invest in advanced manufacturing technologies or pursue acquisitions to secure expertise and maintain competitive advantages. Cell and Gene Therapies in Rare Disorders continue to hold immense potential, offering hope for transformative treatments in previously neglected rare disease segments.
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Cell and Gene Therapies in Rare Disorders Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Cell and Gene Therapies in Rare Disorders Epidemiology Segmentation:
The Cell and Gene Therapies in Rare Disorders market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
- Total Prevalence of Cell and Gene Therapies in Rare Disorders
- Prevalent Cases of Cell and Gene Therapies in Rare Disorders by severity
- Gender-specific Prevalence of Cell and Gene Therapies in Rare Disorders
- Diagnosed Cases of Episodic and Chronic Cell and Gene Therapies in Rare Disorders
Download the report to understand which factors are driving Cell and Gene Therapies in Rare Disorders epidemiology trends @ Cell and Gene Therapies in Rare Disorders Epidemiology Forecast
Cell and Gene Therapies in Rare Disorders Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Cell and Gene Therapies in Rare Disorders market or expected to get launched during the study period. The analysis covers Cell and Gene Therapies in Rare Disorders market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Cell and Gene Therapies in Rare Disorders Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Cell and Gene Therapies in Rare Disorders Therapies and Key Companies
- Fidanacogene elaparvovec: Pfizer
- GiroctocogenE fitelparvovec: Pfizer/ Sangamo Therapeutics
- OTL-103: Orchard Therapeutics/ SR-Tiget
- OAV101: Novartis
- TVI-Brain-1: TVAX Biomedical
- AV-GBM-1: Aivita Biomedical
- ABCB5+ MSCs: RHEACELL GmbH & Co
- CAP-1002: Capricor Therapeutics
Discover more about therapies set to grab major Cell and Gene Therapies in Rare Disorders market share @ Cell and Gene Therapies in Rare Disorders Treatment Market
Scope of the Cell and Gene Therapies in Rare Disorders Market Report
- Study Period: 2020–2034
- Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
- Key Cell and Gene Therapies in Rare Disorders Companies: Pfizer, Sangamo Therapeutics, Orchard Therapeutics/ SR-Tiget, Novartis, TVAX Biomedical, Aivita Biomedical, RHEACELL GmbH & Co, Capricor Therapeutics, and others
- Key Cell and Gene Therapies in Rare Disorders Therapies: Fidanacogene elaparvovec, GiroctocogenE fitelparvovec, OTL-103, OAV101, TVI-Brain-1, AV-GBM-1, ABCB5+ MSCs, CAP-1002, and others
- Cell and Gene Therapies in Rare Disorders Therapeutic Assessment: Cell and Gene Therapies in Rare Disorders current marketed and Cell and Gene Therapies in Rare Disorders emerging therapies
- Cell and Gene Therapies in Rare Disorders Market Dynamics: Cell and Gene Therapies in Rare Disorders market drivers and Cell and Gene Therapies in Rare Disorders market barriers
- Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
- Cell and Gene Therapies in Rare Disorders Unmet Needs, KOL’s views, Analyst’s views, Cell and Gene Therapies in Rare Disorders Market Access and Reimbursement
To know more about Cell and Gene Therapies in Rare Disorders companies working in the treatment market, visit @ Cell and Gene Therapies in Rare Disorders Clinical Trials and Therapeutic Assessment
Table of Contents
1. Cell and Gene Therapies in Rare Disorders Market Report Introduction
2. Executive Summary for Cell and Gene Therapies in Rare Disorders
3. SWOT analysis of Cell and Gene Therapies in Rare Disorders
4. Cell and Gene Therapies in Rare Disorders Patient Share (%) Overview at a Glance
5. Cell and Gene Therapies in Rare Disorders Market Overview at a Glance
6. Cell and Gene Therapies in Rare Disorders Disease Background and Overview
7. Cell and Gene Therapies in Rare Disorders Epidemiology and Patient Population
8. Country-Specific Patient Population of Cell and Gene Therapies in Rare Disorders
9. Cell and Gene Therapies in Rare Disorders Current Treatment and Medical Practices
10. Cell and Gene Therapies in Rare Disorders Unmet Needs
11. Cell and Gene Therapies in Rare Disorders Emerging Therapies
12. Cell and Gene Therapies in Rare Disorders Market Outlook
13. Country-Wise Cell and Gene Therapies in Rare Disorders Market Analysis (2020–2034)
14. Cell and Gene Therapies in Rare Disorders Market Access and Reimbursement of Therapies
15. Cell and Gene Therapies in Rare Disorders Market Drivers
16. Cell and Gene Therapies in Rare Disorders Market Barriers
17. Cell and Gene Therapies in Rare Disorders Appendix
18. Cell and Gene Therapies in Rare Disorders Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
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