Gene Therapy Market Size, Trends, and Growth Report by 2031
市场洞察和分析师观点：
基因治疗是一种通过灭活致病物质来治疗疾病的过程基因，用该基因的健康副本替换致病基因，或者将新的或修改的基因引入体内以帮助治疗和预防疾病。基因治疗可分为体内基因治疗和离体基因治疗。基因疗法的目的是用正常基因替换或纠正有缺陷的基因，使身体产生正常功能所需的正确蛋白质或酶，从而有可能治愈疾病的根本原因。全球范围内遗传疾病和癌症的患病率不断上升，以及 FDA 批准基因疗法的数量不断增加，有利于基因疗法市场的增长。此外，基因治疗市场趋势包括基因治疗技术的进步，这将有利于未来市场的增长。
增长动力：
生物技术的进步带动了发展针对多种适应症的治疗方法。基因疗法被用于治疗各种疾病，例如癌症、神经系统疾病和遗传性疾病。由于美国食品和药物管理局 (FDA) 批准的产品的可用性，基因疗法在全球范围内得到广泛采用。以下是 FDA 近年来批准的基因治疗产品的一些示例：
2023 年 12 月，FDA 批准了两种基于细胞的镰状细胞病基因疗法。 Bluebird Bio 的 Lyfgenia (lovotibeglogene autotemcel) 已被批准用于 12 岁及以上有血管闭塞事件史的镰状细胞病患者。它与 Vertex Pharmaceuticals 和 CRISPR Therapeutics 的 Casgevy (exagamglogene autotemcel) 一起获得批准。 2023 年 6 月，FDA 批准了 Roctavian，一种基于腺相关病毒载体的基因疗法，用于治疗患有严重 A 型血友病的成人，且无需预先存在抗体腺相关病毒血清型 5。遗传性 A 型血友病是一种严重的出血性疾病，由基因突变引起，该基因突变负责产生因子 VIII (FVIII)，这是一种能够促进血液凝固的蛋白质。 Roctavian 是一种一次性基因治疗产品，含有携带凝血因子 VIII 基因的病毒载体。2023 年 6 月，FDA 批准了 Elevidys，这是第一个治疗 4 至 5 岁儿童杜氏肌营养不良症的基因疗法。已确认杜氏肌营养不良症基因突变，并且没有妨碍该疗法治疗的既往病史。2022 年 11 月，FDA 批准了由 CSL Behring LLC 生产的 HEMGENIX，一种重组腺相关病毒 5 型基因治疗患有某些类型的 B 型血友病的成年患者。因此，这些基因疗法的越来越多的批准正在推动基因疗法市场的增长。
报告细分和范围：
基因治疗市场分析是通过考虑以下部分进行的：载体、适应症、递送模式和地理位置。根据载体，市场分为非病毒载体和病毒载体。从适应症来看，市场分为神经系统疾病、癌症、杜氏肌营养不良症、肝病等适应症。就递送模式而言，市场分为体内和体外。基因治疗市场报告的范围涵盖北美（美国、加拿大和墨西哥）、欧洲（法国、德国、英国、西班牙、意大利和欧洲其他地区）、亚太地区（中国、日本、印度、澳大利亚、韩国以及亚太其他地区）、中东和非洲（沙特阿拉伯、南非、阿联酋以及中东和非洲其他地区）以及南美洲和中美洲（巴西、阿根廷和南美洲和中美洲其他地区）。
细分分析：
基因治疗市场按载体分为非病毒载体和病毒载体。病毒载体细分市场在 2023 年占据了重要的市场份额。预计 2023 年至 2030 年市场复合年增长率将更高。
基因治疗市场，按 Vector - 2023 年和 2031 年
根据适应症，市场分为神经系统疾病、癌症、杜氏肌营养不良症、肝病和其他适应症。癌症细分市场在 2023 年占有重要的基因治疗市场份额，预计 2023-2030 年复合年增长率最高。
根据递送模式，市场分为体内和体外。体内细分市场在 2023 年占据了重要的基因治疗市场份额，预计 2023 年至 2030 年期间复合年增长率将更高。体内基因治疗可以实现系统治疗，这意味着它可以到达全身的多个部位和器官。这对于影响多个区域或具有系统症状的疾病特别有帮助，从而可以采取全面的治疗方法。此外，病毒载体、纳米粒子和脂质载体等先进递送技术的发展提高了体内治疗的有效性和特异性。这些进步促进了遗传物质的靶向递送，并提高了治疗的安全性和有效性。
区域分析：
从地理上看，基因治疗市场分为北美、欧洲、亚太地区、南美和南亚地区。和中美洲、中东和非洲。 2023 年，北美占据了重要的市场份额。 2023年，美国主导了该地区的基因治疗市场。北美市场的增长归因于遗传性疾病患病率的不断上升、癌症患者数量的增加、政府资助的增加、先进基因疗法在疾病治疗中的采用不断增加以及产品批准的不断增加。
根据美国疾病控制与预防中心 (CDC) 表示，2020 年美国新诊断出约 1,603,844 例癌症病例，其中 602,347 例癌症死亡。每 10 万人中报告了 403 例新癌症病例。此外，根据国际癌症研究机构的数据，预计到 2040 年，新增癌症病例将达到 3020 万。根据美国政府问责办公室 2021 年 10 月发布的估计，约有 25 至 3000 万人患有罕见癌症。国内的疾病；近50%的罕见病患者是儿童。罕见疾病通常是基因突变的结果；据估计，80% 的罕见病是遗传性的。
根据美国国立卫生研究院 2021 年 10 月的更新，10 家制药公司和 5 家非营利组织已合作加速基因疗法的开发3000万美国人患有罕见疾病。美国 FDA 已批准 7 种细胞和基因治疗药物，新产品线达到约 1,200 种实验疗法。其中一半处于 2 期临床试验中，根据《化学与工程新闻》2023 年报告的估计，细胞疗法的年销售额增长预计为 15%，基因疗法的年销售额增长约为 30%。上述所有这些因素都促进了该地区基因治疗市场规模的不断增长。
行业发展和未来机遇：
在全球基因治疗市场运营的市场参与者采取的一些举措是如下：
“2022 年 1 月，Ori Biotech Ltd 在超额认购的 B 轮融资中获得了超过 1 亿美元的资金，以在市场上推出创新的细胞和基因治疗制造平台。” 2020 年 1 月，Astellas Pharma Inc. 收购了 Audentes Therapeutics, Inc.。此次收购使合并后的公司成为基于 AAV 的基因医学领域的全球领导者。竞争格局和主要公司：
基因治疗市场预测可以提供帮助利益相关者规划他们的增长战略。诺华公司、安斯泰来制药公司、百时美施贵宝公司、蓝鸟生物公司、CSL Behring、赛诺菲、罗氏公司、第一三共、百健和牛津生物医学公司都是基因治疗领域的知名参与者市场报告。这些公司专注于推出新的高科技产品、现有产品的进步以及地域扩张，以满足全球不断增长的消费者需求。
Report Coverage
Revenue forecast, Company Analysis, Industry landscape, Growth factors, and Trends
Segment Covered
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Regional Scope
North America, Europe, Asia Pacific, Middle East & Africa, South & Central America
Country Scope
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Frequently Asked Questions
The gene therapy market majorly consists of the players, including Novartis AG, Astellas Pharma Inc., Bristol-Myers Squibb Company, Bluebird Bio Inc., CSL Behring, Sanofi, F. Hoffmann-La Roche Ltd, Daiichi Sankyo, Biogen, and Oxford Biomedica.
The gene therapy market was valued at US$ 5.11 billion in 2023.
The factors driving the growth of the gene therapy market include the increasing burden of genetic diseases and the rising number of FDA approvals of gene therapies. However, the high cost of gene therapy products hampers the growth of the gene therapy market.
The gene therapy market is expected to be valued at US$ 25.69 billion in 2031.
The global gene therapy market is segmented based on vectors, indication, and delivery mode. Based on indication, the gene therapy market is categorized into neurological diseases, cancer, Duchenne muscular dystrophy, hepatological diseases, and other indications. The cancer segment held a significant market share in 2023 and is anticipated to record the highest CAGR in the market during 2023–2031.
Gene therapy is a process for the treatment of diseases by inactivating a disease-causing gene, replacing a disease-causing gene with a healthy copy of the gene, or introducing a new or modified gene into the body to help treat and prevent the disease.
The List of Companies - Gene Therapy Market
- Novartis AG
- Astellas Pharma Inc.
- Bristol-Myers Squibb Company
- Bluebird Bio Inc.
- Sanofi
- F. Hoffmann-La Roche Ltd
- Daiichi Sankyo
- CSL Behring
- Biogen
- Oxford Biomedica.
The Insight Partners performs research in 4 major stages: Data Collection & Secondary Research, Primary Research, Data Analysis and Data Triangulation & Final Review.
- Data Collection and Secondary Research:
As a market research and consulting firm operating from a decade, we have published and advised several client across the globe. First step for any study will start with an assessment of currently available data and insights from existing reports. Further, historical and current market information is collected from Investor Presentations, Annual Reports, SEC Filings, etc., and other information related to company’s performance and market positioning are gathered from Paid Databases (Factiva, Hoovers, and Reuters) and various other publications available in public domain.
Several associations trade associates, technical forums, institutes, societies and organization are accessed to gain technical as well as market related insights through their publications such as research papers, blogs and press releases related to the studies are referred to get cues about the market. Further, white papers, journals, magazines, and other news articles published in last 3 years are scrutinized and analyzed to understand the current market trends.
- Primary Research:
The primarily interview analysis comprise of data obtained from industry participants interview and answers to survey questions gathered by in-house primary team.
For primary research, interviews are conducted with industry experts/CEOs/Marketing Managers/VPs/Subject Matter Experts from both demand and supply side to get a 360-degree view of the market. The primary team conducts several interviews based on the complexity of the markets to understand the various market trends and dynamics which makes research more credible and precise.
A typical research interview fulfils the following functions:
- Provides first-hand information on the market size, market trends, growth trends, competitive landscape, and outlook
- Validates and strengthens in-house secondary research findings
- Develops the analysis team’s expertise and market understanding
Primary research involves email interactions and telephone interviews for each market, category, segment, and sub-segment across geographies. The participants who typically take part in such a process include, but are not limited to:
- Industry participants: VPs, business development managers, market intelligence managers and national sales managers
- Outside experts: Valuation experts, research analysts and key opinion leaders specializing in the electronics and semiconductor industry.
Below is the breakup of our primary respondents by company, designation, and region:
Once we receive the confirmation from primary research sources or primary respondents, we finalize the base year market estimation and forecast the data as per the macroeconomic and microeconomic factors assessed during data collection.
- Data Analysis:
Once data is validated through both secondary as well as primary respondents, we finalize the market estimations by hypothesis formulation and factor analysis at regional and country level.
- Macro-Economic Factor Analysis:
We analyse macroeconomic indicators such the gross domestic product (GDP), increase in the demand for goods and services across industries, technological advancement, regional economic growth, governmental policies, the influence of COVID-19, PEST analysis, and other aspects. This analysis aids in setting benchmarks for various nations/regions and approximating market splits. Additionally, the general trend of the aforementioned components aid in determining the market's development possibilities.
- Country Level Data:
Various factors that are especially aligned to the country are taken into account to determine the market size for a certain area and country, including the presence of vendors, such as headquarters and offices, the country's GDP, demand patterns, and industry growth. To comprehend the market dynamics for the nation, a number of growth variables, inhibitors, application areas, and current market trends are researched. The aforementioned elements aid in determining the country's overall market's growth potential.
- Company Profile:
The “Table of Contents” is formulated by listing and analyzing more than 25 - 30 companies operating in the market ecosystem across geographies. However, we profile only 10 companies as a standard practice in our syndicate reports. These 10 companies comprise leading, emerging, and regional players. Nonetheless, our analysis is not restricted to the 10 listed companies, we also analyze other companies present in the market to develop a holistic view and understand the prevailing trends. The “Company Profiles” section in the report covers key facts, business description, products & services, financial information, SWOT analysis, and key developments. The financial information presented is extracted from the annual reports and official documents of the publicly listed companies. Upon collecting the information for the sections of respective companies, we verify them via various primary sources and then compile the data in respective company profiles. The company level information helps us in deriving the base number as well as in forecasting the market size.
- Developing Base Number:
Aggregation of sales statistics (2020-2022) and macro-economic factor, and other secondary and primary research insights are utilized to arrive at base number and related market shares for 2022. The data gaps are identified in this step and relevant market data is analyzed, collected from paid primary interviews or databases. On finalizing the base year market size, forecasts are developed on the basis of macro-economic, industry and market growth factors and company level analysis.
- Data Triangulation and Final Review:
The market findings and base year market size calculations are validated from supply as well as demand side. Demand side validations are based on macro-economic factor analysis and benchmarks for respective regions and countries. In case of supply side validations, revenues of major companies are estimated (in case not available) based on industry benchmark, approximate number of employees, product portfolio, and primary interviews revenues are gathered. Further revenue from target product/service segment is assessed to avoid overshooting of market statistics. In case of heavy deviations between supply and demand side values, all thes steps are repeated to achieve synchronization.
We follow an iterative model, wherein we share our research findings with Subject Matter Experts (SME’s) and Key Opinion Leaders (KOLs) until consensus view of the market is not formulated – this model negates any drastic deviation in the opinions of experts. Only validated and universally acceptable research findings are quoted in our reports.
We have important check points that we use to validate our research findings – which we call – data triangulation, where we validate the information, we generate from secondary sources with primary interviews and then we re-validate with our internal data bases and Subject matter experts. This comprehensive model enables us to deliver high quality, reliable data in shortest possible time.
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