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基因治療學和RNAi 基因療法

市場調查報告書

商品編碼

1574086

美國基因治療市場：依類型、載體、基因傳遞方法、應用、給藥途徑、最終用戶、地區、機會、預測，2017-2031

United States Gene Therapy Market Assessment, By Type, By Vector, By Gene Delivery Method, By Application, By Route of Administration, By End-user, By Region, Opportunities and Forecast, 2017-2031F

出版日期: 2024年10月22日 | 出版商:

Market Xcel - Markets and Data | 英文 131 Pages | 商品交期: 3-5個工作天內

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簡介目錄圖表

預計2024-2031年期間，美國基因治療市場規模將以21.53%的年複合成長率擴大，從2023年的31.0126億美元成長到2031年的142.8731億美元。市場成長的推動因素包括研究活動的增加、監管機構批准的增加、慢性病和遺傳疾病病例數量的增加以及主要市場參與者的投資增加。

基於 CRISPR 的基因組編輯技術的最新進展預計也將對市場擴張產生積極影響。此外，病例數的增加引起了人們對心血管研究的關注。美國心臟協會估計，2023年心血管疾病的年齡調整死亡率將比2022年增加4%。新興的Cas 核酸酶加速藥物和生命科學的發展，其擴展的應用能夠擴展基於 CRISPR 的基因組編輯工具箱。基因組編輯技術透過增強心血管疾病基因改造模型的生成，徹底改變心血管研究。

醫療補助和醫療保險等政府政策以及平價醫療法案（ACA）等法律也會影響美國的償付和基因治療的取得。透過生物醫學高級研究與進步管理局（BARDA）和美國國立衛生研究院（NIH）推進生物技術研究的聯邦資金也推動基因治療的創新。

本報告調查了美國基因治療市場，提供了市場概述，依類型、載體、基因傳遞方法、應用、給藥途徑、最終用戶、地區趨勢和進入市場的公司概況。

前5名市場領導者的競爭矩陣
前5名進入公司的SWOT 分析
市場前10位主要參與者的情況
- Gilead Sciences Inc. (Kite Pharma)
- Bristol-Myers Squibb Company
- Amgen, Inc.
- Sarepta Therapeutics, Inc.
- F. Hoffmann-la Roche ltd. (Spark Therapeutics, Inc.)
- Bluebird Bio, Inc.
- Novartis AG
- CSL Behring LLC (Manufactured by uniQure Inc.)
- Johnson & Johnson Services, Inc. (Janssen Research & Development, LLC)
- CRISPR Therapeutics AG

第17章策略建議

第18章查詢及免責聲明

簡介目錄圖表

Product Code: MX12124

United States gene therapy market is projected to witness a CAGR of 21.53% during the forecast period 2024-2031, growing from USD 3,101.26 million in 2023 to USD 14,287.31 million in 2031. The growth of the market is bolstered by rising research activities, increasing approvals by regulatory bodies, growing cases of chronic and genetic diseases, and increasing investments by key market players.

Recent advancements in CRISPR-based genome editing technology are also expected to positively influence the market's expansion. Additionally, increasing emphasis is being placed on cardiovascular research due to the rising cases. According to the estimates of the American Heart Association, the age-adjusted death rate from cardiovascular diseases in 2023 increased by 4% from 2022. The emerging Cas nucleases are promoting the development of medicines and life sciences with the help of their extended applications that are allowing the expansion of the CRISPR-based genome editing toolbox. The genome editing technology is revolutionizing cardiovascular research by bolstering the generation of genetically modified models of cardiovascular diseases.

The government policies such as Medicaid and Medicare and laws including Affordable Care Act (ACA) have also impacted reimbursement and access to gene therapies in the United States. Federal funding for advancing biotechnology research from the Biomedical Advanced Research and Progress Authority (BARDA) and National Institutes of Health (NIH) are also fostering innovations in gene therapy.

Increasing Research Activities Boost Market Growth

The recent advancements in innovative technologies have propelled the development of novel therapeutic solutions and bolstered awareness about the potential of gene therapy for treating genetic disorders and rare diseases. Thus, this will bolster the investments towards research activities in gene therapy approaches across the country, which are ushering in a wide range of new therapeutic solutions to combat different diseases. In January 2024, a team of researchers from the Harvard Medical School and Massachusetts Eye and Ear Infirmary developed a novel gene therapy approach that allowed children with congenital deafness to hear for the first time. The project was the first to use gene therapy to treat the condition and has opened doors to support the development of other treatment solutions to reduce the burden of different types of genetic deafness. Such collaborations between the leading research organizations are expected to boost the market in the coming years.

Rising Approvals by FDA Support Market Expansion

The increasing efforts of the key players in the market to bolster the development and availability of novel therapeutic solutions in the country are propelling the United States gene therapy market growth. In April 2024, the United States Food and Drug Administration (US FDA) approved fidanacogene elaparvovec (Beqvez), the second gene therapy for hemophilia B. The patients eligible for a one-time infusion include those with historical or current life-threatening hemorrhages, spontaneous and serious bleeding episodes, and those on clotting factor IX prophylaxis therapy. The first therapy to be approved by the FDA for the condition was etranacogene dezaparvovec (Hemgenix), in November 2022. The growing approvals for gene therapy for chronic diseases can be attributed to the efficacy and safety demonstrated by them in different trial phases. Such approvals are expected to provide lucrative growth opportunities to the market and aid the patient population in combating chronic diseases.

Rising Cases of Genetic and Chronic Diseases to Increase Market Demand

The increasing prevalence of chronic and genetic diseases is propelling the demand for gene therapy in the United States. Genetic disorders, with often no effective conventional treatments, have become a main target for interventions involving gene therapy. Some of such disorders include cystic fibrosis, sickle cell anemia, muscular dystrophy, and hemophilia. Additionally, the expansion of the aging population is also one of the major reasons contributing to rising cases of chronic diseases and thus increasing the demand for gene therapies in the United States. Age-related disorders, including cardiovascular diseases, diabetes, and neurodegenerative disorders such as Parkinson's and Alzheimer's disease, require novel treatment modalities. Gene therapies offer symptom alleviation and modification of the disease process, thus providing effective long-term solutions. Meanwhile, the rise in cancer cases in the United States has drawn attention to gene therapy in oncology in the country. As per the estimates of the National Cancer Institute, approximately 2,001,140 new cases of cancer are expected to be diagnosed in the United States in 2024.

Oncological Diseases Hold Major Market Share

The rising cases of cancer in the country and the presence of a strong healthcare sector that is committed towards advancing the next generation of cancer treatment are propelling the growth of the United States gene therapy market. The gene therapy approach is deployed to either weaken the cancer cells or strengthen the immune system.

The increasing efforts towards the development of novel gene therapies to battle different types of cancer are also supporting the market's expansion. In July 2024, UC San Diego Health became the first health system in San Diego County to provide new gene therapy for treating localized bladder cancer. Such developments are expected to aid healthcare organizations in meeting the evolving requirements of patients with the help of gene therapy and provide lucrative growth opportunities to the market.

Future Market Scenario (2024-2031F)

As per the United States gene therapy market analysis, the market is expected to witness significant growth in the coming years owing to the growing awareness about the potential of gene therapy in reducing the burden of different diseases, increasing investments by the key market players, and rising research activities in the country. Various research studies and clinical trials are underway to evaluate the safety and effectiveness of gene therapy. For instance, the Methodist Hospital Research Institute in Texas is conducting an interventional study to evaluate the efficacy and safety of chemotherapy, radiotherapy, valacyclovir, and gene therapy in recurrent glioblastoma multiforme. The study aims to assess the toxicity and efficacy of the combination therapy in anaplastic astrocytoma or glioblastoma multiforme and comprises of patients who have previously failed standard-of-care treatment. The study is expected to conclude in December 2025.

Additionally, the growing efforts of the leading market players to bolster the production of different therapeutic solutions are also expected to boost the market's expansion. For instance, Gilead Sciences Inc. is actively working towards quadrupling the production of their cell therapy cancer treatments by 2026 by ensuring improvements in their manufacturing processes.

Key Players Landscape and Outlook

Mergers and acquisitions aid the key players of the market to ensure the availability and development of innovative therapies, bolstering the market. In March 2024, Gilead Sciences Inc. announced that they had acquired CymaBay Therapeutics, Inc. for approximately USD 4.3 billion. The acquisition aligns with the former's long-term commitment to ensuring the availability of transformational medicines for patients by adding CymaBay's investigational candidate, seladelpar, to their existing liver portfolio. Such investments are expected to transform the treatment landscape for primary biliary cholangitis and allow companies to meet the unmet requirements of the patients.

Additionally, the rising investments by the key players towards the construction of manufacturing facilities in the country is also supporting the market's expansion. In February 2024, Amgen, Inc. announced the opening of a new manufacturing facility in Central Ohio. The site is expected to play a crucial role in aiding the company in combatting the threat of chronic diseases and ensuring the availability of innovative therapeutic solutions.

1. Project Scope and Definitions

2. Research Methodology

3. Executive Summary

4. United States Gene Therapy Market Outlook, 2017-2031F

4.1. Market Size Analysis & Forecast
- 4.1.1. By Value
- 4.1.2. By Volume
4.2. Market Share Analysis & Forecast
- 4.2.1. By Type
  - 4.2.1.1. Gene Augmentation
  - 4.2.1.2. Gene Silencing
  - 4.2.1.3. Others
- 4.2.2. By Vector
  - 4.2.2.1. Viral Vectors
  - 4.2.2.2. Non-Viral Vectors
- 4.2.3. By Gene Delivery Method
  - 4.2.3.1. In vivo
  - 4.2.3.2. Ex vivo
- 4.2.4. By Application
  - 4.2.4.1. Oncological Diseases
  - 4.2.4.2. Genetic Diseases
  - 4.2.4.3. Cardiovascular Diseases
  - 4.2.4.4. Neuromuscular Diseases
  - 4.2.4.5. Hematological Diseases
  - 4.2.4.6. Infectious Diseases
  - 4.2.4.7. Others
- 4.2.5. By Route of Administration
  - 4.2.5.1. Intravenous
  - 4.2.5.2. Others
- 4.2.6. By End-user
  - 4.2.6.1. Hospitals
  - 4.2.6.2. Cancer and Research Institutes
  - 4.2.6.3. Clinics
  - 4.2.6.4. Others
- 4.2.7. By Region
  - 4.2.7.1. Northeast
  - 4.2.7.2. Midwest
  - 4.2.7.3. West
  - 4.2.7.4. South
- 4.2.8. By Company Market Share Analysis (Top 5 Companies and Others - By Value, 2023)
4.3. Market Map Analysis, 2023
- 4.3.1. By Type
- 4.3.2. By Vector
- 4.3.3. By Gene Delivery Method
- 4.3.4. By Application
- 4.3.5. By Route of Administration
- 4.3.6. By End-user
- 4.3.7. By Region

5. Demand Supply Analysis

6. Import and Export Analysis

7. Value Chain Analysis

8. Porter's Five Forces Analysis

9. PESTLE Analysis

10. Pricing Analysis

11. Market Dynamics

11.1. Market Drivers
11.2. Market Challenges

12. Market Trends and Developments

13. Regulatory Framework and Innovation

13.1. Clinical Trials
13.2. Regulatory Approvals

14. Patent Landscape

15. Case Studies

16. Competitive Landscape

16.1. Competition Matrix of Top 5 Market Leaders
16.2. SWOT Analysis for Top 5 Players
16.3. Key Players Landscape for Top 10 Market Players
- 16.3.1. Gilead Sciences Inc. (Kite Pharma)
  - 16.3.1.1. Company Details
  - 16.3.1.2. Key Management Personnel
  - 16.3.1.3. Products and Services
  - 16.3.1.4. Financials (As Reported)
  - 16.3.1.5. Key Market Focus and Geographical Presence
  - 16.3.1.6. Recent Developments/Collaborations/Partnerships/Mergers and Acquisition
- 16.3.2. Bristol-Myers Squibb Company
- 16.3.3. Amgen, Inc.
- 16.3.4. Sarepta Therapeutics, Inc.
- 16.3.5. F. Hoffmann-la Roche ltd. (Spark Therapeutics, Inc.)
- 16.3.6. Bluebird Bio, Inc.
- 16.3.7. Novartis AG
- 16.3.8. CSL Behring LLC (Manufactured by uniQure Inc.)
- 16.3.9. Johnson & Johnson Services, Inc. (Janssen Research & Development, LLC)
- 16.3.10. CRISPR Therapeutics AG

Companies mentioned above DO NOT hold any order as per market share and can be changed as per information available during research work.

17. Strategic Recommendations

18. About Us and Disclaimer

簡介目錄圖表

List of Tables

Table 1. Pricing Analysis of Products from Key Players
Table 2. Competition Matrix of Top 5 Market Leaders
Table 3. Mergers & Acquisitions/ Joint Ventures (If Applicable)
Table 4. About Us - Regions and Countries Where We Have Executed Client Projects

List of Figures

Figure 1. United States Gene Therapy Market, By Value, In USD Million, 2017-2031F
Figure 2. United States Gene Therapy Market, By Volume, In Thousand Therapies, 2017-2031F
Figure 3. United States Gene Therapy Market Share (%), By Type, 2017-2031F
Figure 4. United States Gene Therapy Market Share (%), By Vector, 2017-2031F
Figure 5. United States Gene Therapy Market Share (%), By Gene Delivery Method, 2017-2031F
Figure 6. United States Gene Therapy Market Share (%), By Application, 2017-2031F
Figure 7. United States Gene Therapy Market Share (%), By Route of Administration, 2017-2031F
Figure 8. United States Gene Therapy Market Share (%), By End-user, 2017-2031F
Figure 9. United States Gene Therapy Market Share (%), By Region, 2017-2031F
Figure 10. By Type Map-Market Size (USD Million) & Growth Rate (%), 2023
Figure 11. By Vector Map-Market Size (USD Million) & Growth Rate (%), 2023
Figure 12. By Gene Delivery Method Map-Market Size (USD Million) & Growth Rate (%), 2023
Figure 13. By Application Size Map-Market Size (USD Million) & Growth Rate (%), 2023
Figure 14. By Route of Administration Map-Market Size (USD Million) & Growth Rate (%), 2023
Figure 15. By End-user Map-Market Size (USD Million) & Growth Rate (%), 2023
Figure 16. By Region Map-Market Size (USD Million) & Growth Rate (%), 2023