美国基因治疗市场：依类型、载体、基因传递方法、应用、给药途径、最终用户、地区、机会、预测，2017-2031

预计2024-2031年期间，美国基因治疗市场规模将以21.53%的年复合成长率扩大，从2023年的31.0126亿美元成长到2031年的142.8731亿美元。市场成长的推动因素包括研究活动的增加、监管机构批准的增加、慢性病和遗传疾病病例数量的增加以及主要市场参与者的投资增加。

基于 CRISPR 的基因组编辑技术的最新进展预计也将对市场扩张产生积极影响。此外，病例数的增加引起了人们对心血管研究的关注。美国心臟协会估计，2023年心血管疾病的年龄调整死亡率将比2022年增加4%。新兴的Cas 核酸酶加速药物和生命科学的发展，其扩展的应用能够扩展基于 CRISPR 的基因组编辑工具箱。基因组编辑技术透过增强心血管疾病基因改造模型的生成，彻底改变心血管研究。

医疗补助和医疗保险等政府政策以及平价医疗法案（ACA）等法律也会影响美国的偿付和基因治疗的取得。透过生物医学高级研究与进步管理局（BARDA）和美国国立卫生研究院（NIH）推进生物技术研究的联邦资金也推动基因治疗的创新。

本报告调查了美国基因治疗市场，提供了市场概述，依类型、载体、基因传递方法、应用、给药途径、最终用户、地区趋势和进入市场的公司概况。

目录

第1章 专案范围与定义

第2章 研究方法

第3章 执行摘要

第4章 美国基因治疗市场前景，2017-2031

• 市场规模分析与预测
• 市场占有率分析与预测
• 2023年市场地图分析
  • 依类型
  • 依媒介
  • 依基因传递法
  • 依用途
  • 依给药途径
  • 依最终用户
  • 依地区

第5章 供需分析

第6章 导入汇出分析

第7章 价值链分析

第8章 波特五力分析

第9章 PESTLE分析

第10章 价格分析

第11章 市场动态

第12章 市场趋势与发展

第13章 监理架构与创新

第14章 专利状况

第15章 个案研究

第16章 竞争态势

• 前5名市场领导者的竞争矩阵
• 前5名进入公司的SWOT 分析
• 市场前10位主要参与者的情况
  • 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 策略建议

第18章 查询及免责声明

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.

Table of Contents

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

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