市场调查报告书
商品编码
1519482
2024-2032 年按基因类型、载体类型、递送方法、应用和地区分類的基因治疗市场报告Gene Therapy Market Report by Gene Type, Vector Type, Delivery Method, Application, and Region 2024-2032 |
2023年全球基因治疗市场规模达51IMARC Group美元。由于病毒载体技术的不断改进和创新,遗传性疾病和罕见疾病(如肌肉营养不良症、囊性纤维化和各种遗传性代谢性疾病)发病率的上升,以及研发投入和资金的增加,市场正在稳步扩大。
市场成长与规模:在遗传异常和罕见疾病盛行率上升的推动下,该产业正在迅速扩张。此外,製药企业越来越多地投资于研发(R&D)活动,这对市场产生了积极影响。
技术进步:病毒载体技术的发展正在提高基因治疗的有效性和安全性。而且,CRISPR-Cas9技术提升了基因编辑能力,进一步加速了基因治疗市场统计。
产业应用:基因疗法用于治疗多种疾病,包括癌症、遗传问题和自体免疫疾病。离体基因疗法,即在体外操纵细胞,在治疗特定遗传疾病方面越来越受欢迎。
地理趋势:根据 IMARC 的基因疗法市场统计数据,北美凭藉其强大的医疗基础设施和发达的生物技术产业,在市场上占据主导地位。然而,在庞大患者群体的推动下,亚太地区正成为一个快速扩张的市场。
竞争格局:领先企业正在进行全面的临床研究,以开发针对各种疾病(包括罕见遗传异常和癌症)的新型基因疗法并获得监管许可。市场上一些主要的参与者包括 Abeona Therapeutics Inc.、Adaptimmune Therapeutics plc、Adverum Biotechnologies Inc.、Amgen Inc.、Astellas Pharma Inc.、Biogen Inc.、Bluebird Bio Inc.、Mustang Bio Inc. (Fortress Biotech Inc.) )、Novartis AG、Orchard Therapeutics plc、UniQure NV、Voyager Therapeutics Inc. 等。
挑战与机会:虽然该行业面临监管限制和高研究成本等限制,但它也预计有可能增加罕见疾病以外的用途并研究新的给药方式。
未来展望:随着对遗传异常和罕见疾病的新疗法的需求不断增长,基因治疗市场前景预计将是积极的。此外,针对独特患者特征的个人化基因治疗预计将推动市场扩张。
病毒载体技术的进步
病毒载体技术的不断进步和创新是推动市场成长的重要因素。病毒载体用作递送载体,将治疗基因引入患者细胞中。这项技术的最新进展大大提高了基因治疗的安全性和有效性。这些进展包括创建更具特异性和针对性的病毒载体,例如腺相关病毒 (AAV) 和慢病毒,AAV 因其能够以最小的免疫反应和损伤运输遗传物质而受到欢迎。此外,根据基因治疗市场概况,研究人员正专注于开发可能携带更大遗传负载的病毒载体,从而扩大可治癒疾病的范围。这些技术创新不仅提高了基因疗法的功效,还最大限度地减少了不良反应的可能性,使患者更安全。因此,病毒载体技术的进步正在加速基因疗法的接受和功效,以解决更广泛的医疗问题。例如,MERCK KGaA 的 VirusExpress 293 腺相关病毒 (AAV) 生产平台提供完整的病毒载体製造,包括 AAV 和慢病毒载体。此外,Catalent Inc. 推出了 UpTempo Virtuoso 平台流程,用于开发和製造腺相关病毒 (AAV) 载体。
研究与开发的投资和资助
基因治疗研究和开发(R&D)工作的投资和资金不断增加正在推动市场扩张。製药公司、生技公司和政府机构正在投入大量资源来开发基因治疗技术和疗法。基因治疗在解决未满足的医疗需求和提供长期效益方面具有巨大潜力,目前正在吸引大量资金。主要製药公司要么收购基因治疗新创公司,要么建立自己的基因治疗部门以加快研究速度,这对基因治疗市场前景产生了积极影响。此外,政府补助和激励措施激励学术机构和研究公司进行基因治疗实验。美国的《孤儿药法案》以及其他国家的类似立法为罕见疾病药物的开发提供了诱因。例如,囊性纤维化基金会报告投资 SalioGen Therapeutics,以支持该公司对囊性纤维化新型基因疗法的临床前研究。萨洛宁的基因编码方法旨在开启、关闭或修改基因组中任何基因的功能。
遗传性疾病和罕见疾病的盛行率不断上升
遗传异常和罕见疾病盛行率的上升正在推动市场成长。其中许多疾病几乎没有或根本没有有效的治疗选择,这使得基因疗法成为有趣的替代方案。随着我们对遗传学认识的进步,越来越多的人被诊断出来,对新药的需求也在增加。此外,基因疗法能够透过修復或替换有缺陷的基因来治疗许多疾病的根本原因。这包括肌肉营养不良症、囊性纤维化和其他遗传性代谢失调等疾病。製药和生物技术企业正在投资基因疗法研究,以满足不断增长的医疗需求,从而导致该领域的市场扩张和改善。例如,根据美国疾病管制与预防中心关于脊髓性肌肉萎缩症 (SMA) 的文章,SMA 是一种遗传性疾病,每 10,000 人中就有 1 人受到影响。因此,它是最常见的罕见疾病之一。
支持性监管环境
多个国家的监管组织,特别是美国食品和药物管理局 (FDA) 和欧洲药品管理局 (EMA),认识到基因疗法在满足基本医疗需求方面的潜力。因此,他们创建了结构并加速了基因治疗的研究和批准。此外,快速通道指定、突破性治疗指定和孤儿药状态也是基因治疗候选者可用的监管措施之一。这些指定简化了临床试验程序,缩短了开发时间表,并鼓励企业参与基因治疗研究。例如,美国 FDA 批准了 Zynteglo (betibeglogene autotemcel),这是第一个用于治疗成人和儿童 β 地中海贫血患者的细胞基因疗法。由于基因疗法开发的兴起,此类批准预计将推动市场成长。
IMARC Group提供了对每个细分市场的主要趋势的分析,以及 2024-2032 年全球、区域和国家层面的预测。我们的报告根据基因类型、载体类型、递送方法和应用对市场进行了分类。
抗原
细胞因子
肿瘤抑制剂
自杀基因
不足
生长因子
受体
其他的
该报告根据基因类型对市场进行了详细的细分和分析。这包括抗原、细胞激素、肿瘤抑制因子、自杀基因、缺陷、生长因子、受体等。
抗原基因是基因治疗中的关键部分,特别是在癌症免疫治疗的背景下。这些基因编码肿瘤特异性抗原或与传染原相关的抗原。透过将抗原基因引入患者的细胞中,可以激活免疫系统来识别和靶向癌细胞或病原体。它们还可以实现个人化的癌症治疗,其中可以针对患者特异性抗原,以产生更有效、更有针对性的免疫反应。例如,Imugene 报告了与美国新泽西州凯尼尔沃思默克公司的商品名MSD 达成的一项新的临床试验合作和供应协议,以评估Imugene 的HER-Vaxx(一种B 细胞活化药物)的安全性和有效性。
病毒载体
腺病毒
慢病毒
逆转录病毒
腺相关病毒
单纯疱疹病毒
痘病毒
痘苗病毒
其他的
非病毒技术
裸载体和质粒载体
基因枪
电穿孔
脂转染
其他的
病毒载体在产业中占有最大份额。
报告还提供了基于载体类型的详细市场细分和分析。这包括病毒载体(腺病毒、慢病毒、逆转录病毒、腺相关病毒、单纯疱疹病毒、痘病毒、牛痘病毒等)和非病毒技术(裸载体和质粒载体、基因枪、电穿孔、脂转染等)。报告称,病毒载体占最大的市场。
病毒载体,例如腺相关病毒(AAV)和慢病毒,由于能够有效地将治疗基因传递到目标细胞中而受到关注。 AAV 因其安全性和将遗传物质整合到宿主基因组中的能力而受到青睐。它们广泛用于各种基因治疗应用,包括治疗遗传性疾病、罕见疾病和某些类型的癌症。病毒载体技术的持续研究和进步正在推动这一领域的成长,利用病毒载体进行了大量的临床试验和批准的治疗。例如,美国 FDA 加速批准了 SKYSONA——一种慢病毒载体基因疗法,可减缓 4-17 岁男孩神经退化性功能障碍或活动性脑肾上腺脑白质营养不良的进展。
体内基因治疗
离体基因治疗
该报告根据交付方式对市场进行了详细的细分和分析。这包括体内基因治疗和离体基因治疗。
体内基因治疗涉及将治疗基因或遗传物质直接递送到患者体内。此方法旨在改变或纠正患者自然生理环境中组织或器官内的遗传缺陷。体内基因治疗方法通常利用病毒载体,例如腺相关病毒(AAV)或慢病毒,来运输治疗基因。基因治疗的这一部分通常用于需要全身性治疗的疾病,例如某些类型的遗传性疾病或体内目标组织很容易到达的病症。例如,基因补充疗法的最新进展正在扩大神经系统疾病治疗的选择。在可用的递送载体中,腺相关病毒(AAV)通常是最受欢迎的载体。
肿瘤疾病
罕见疾病
心血管疾病
神经系统疾病
传染病
其他的
肿瘤疾病占据了大部分市场。
该报告根据应用程式提供了详细的市场细分和分析。这包括肿瘤疾病、罕见疾病、心血管疾病、神经系统疾病、传染病等。根据该报告,肿瘤疾病占最大部分。
基因疗法可有效治疗多种癌症,包括实体肿瘤和血液恶性肿瘤。方法范围从引入肿瘤抑制基因到激活针对癌细胞的免疫系统。 CAR-T 细胞疗法是一种基因疗法,因其在治疗某些类型的白血病和淋巴瘤方面的成功而受到关注。正在进行的肿瘤疾病基因疗法的研发(R&D)活动正在推动市场成长。例如,美国 FDA 批准了 Abecma(idecabtagene vicleucel),这是一种基于细胞的基因疗法,用于治疗对多发性骨髓瘤成年患者没有反应或在至少四线(不同类型)治疗后疾病復发的患者。 Abecma 是 FDA 批准的第一个用于治疗多发性骨髓瘤的细胞基因疗法。
北美洲
美国
加拿大
亚太
中国
日本
印度
韩国
澳洲
印尼
其他的
欧洲
德国
法国
英国
义大利
西班牙
俄罗斯
其他的
拉丁美洲
巴西
墨西哥
其他的
中东和非洲
北美市场处于领先地位,占据最大的基因治疗市场。
市场研究报告还对所有主要区域市场进行了全面分析,其中包括北美(美国和加拿大);亚太地区(中国、日本、印度、韩国、澳洲、印尼等);欧洲(德国、法国、英国、义大利、西班牙、俄罗斯等);拉丁美洲(巴西、墨西哥等);以及中东和非洲。报告称,北美占据最大的市场份额。
北美拥有强大的医疗基础设施、完善的生物技术产业以及对基因治疗研发 (R&D) 的大量投资。有利的监管政策,包括快速通道指定和孤儿药地位,正在加速基因疗法的批准和商业化。基因治疗市场的主要参与者总部位于北美,为该地区在基因治疗创新方面的领导地位做出了贡献。例如,根据美国国立卫生研究院(NIH) 的最新消息,NIH、美国食品药物管理局、十家製药公司和五个非营利组织已合作,加速开发基因疗法,帮助3000 万患有基因突变的美国人罕见疾病。预计此类措施将增加对基因疗法的需求,从而促进市场成长。
主要产业竞争对手目前参与了许多策略倡议。这些公司透过研究、收购和合作不断扩大其基因治疗产品组合。他们正在进行全面的临床研究,以开发新型基因疗法并获得监管许可,这些疗法将治疗各种疾病,包括罕见的基因异常和癌症。此外,他们还在投资生产设施,以增加产量并确保更多患者群体能够获得基因治疗。此外,这些行业巨头正在寻求与学术机构和小型生物技术企业合作,以促进创新和改进基因治疗技术。
Abeona 治疗公司
适应免疫疗法公司
Adverum 生技公司
安进公司
安斯泰来製药公司
百健公司
蓝鸟生物公司
野马生物公司(Fortress Biotech Inc.)
诺华公司
果园治疗公司
优尼库尔公司
航海家治疗公司
(请注意,这只是关键参与者的部分列表,报告中提供了完整列表。)
2023 年 1 月:Spark Therapeutics 和 Neurochase 建立了策略合作,开发 Neurochase 独特的递送技术,用于中枢神经系统罕见疾病的选定基因治疗。根据该协议,Neurochase 将向 Spark 的首屈一指的 AAV 平台贡献其在直接药物传输技术方面的丰富知识。
2023 年 1 月:Voyager Therapeutics 和 Neurocrine Biosciences 就 Voyager GBA1 计画和其他神经系统疾病下一代基因疗法的商业化和开发达成策略合作。
2023年6月:美国FDA核准Sarepta用于ELEVIDYS基因疗法,用于治疗4-5岁儿童的DMD。
2023 年 5 月:Krystal Biotech 的 VYJUVEK 基因疗法获得批准,用于治疗营养不良性大疱性表皮鬆解症。
2023 年 12 月:瑞士治疗产品管理局核准 Libmeldy 用于治疗早发性异染性脑白质营养不良症。
2024 年 1 月:Biogen 和 Ginkgo Bioworks 宣布,他们已经完成了涉及基于 AAV 的载体的基因治疗合作。预计这将推动未来几年对基因疗法的需求。
The global gene therapy market size reached US$ 5.1 Billion in 2023. Looking forward, IMARC Group expects the market to reach US$ 16.8 Billion by 2032, exhibiting a growth rate (CAGR) of 13.9% during 2024-2032. The market is expanding steadily due to ongoing improvements and innovations in viral vector technology, rising incidences of genetic disorders and rare diseases such as muscular dystrophy, cystic fibrosis, and various inherited metabolic disorders, as well as increased investment and funding in R&D.
Market Growth and Size: The industry is expanding rapidly, fueled by the rising prevalence of genetic abnormalities and rare diseases. Additionally, pharmaceutical businesses are increasingly investing in research and development (R&D) activities, which are positively impacting the market.
Technological Advancements: Developments in viral vector technology are enhancing the efficacy and safety of gene therapy delivery. Moreover, CRISPR-Cas9 technology improves gene editing capabilities, which further accelerates gene therapy market statistics.
Industry Applications: Gene therapy is used to treat a variety of ailments, including cancer, genetic problems, and autoimmune conditions. Ex vivo gene therapy, in which cells are manipulated outside the body, is gaining popularity for treating specific genetic illnesses.
Geographical Trends: As per the gene therapy market statistics by IMARC, North America dominates the market, owing to its strong healthcare infrastructure and developed biotechnology sector. However, Asia Pacific is emerging as a rapidly expanding market, propelled by a big patient population.
Competitive Landscape: Leading players are conducting comprehensive clinical studies to develop and get regulatory clearance for novel gene treatments aimed at a variety of ailments, including rare genetic abnormalities and cancer. Some of the key players in the market include Abeona Therapeutics Inc., Adaptimmune Therapeutics plc, Adverum Biotechnologies Inc., Amgen Inc., Astellas Pharma Inc., Biogen Inc., Bluebird Bio Inc., Mustang Bio Inc. (Fortress Biotech Inc.), Novartis AG, Orchard Therapeutics plc, UniQure N.V., Voyager Therapeutics Inc., etc.
Challenges and Opportunities: While the industry confronts constraints like regulatory limitations and high research costs, it also expects possibilities for increasing uses beyond rare disorders and investigating novel delivery modalities.
Future Outlook: The gene therapy market outlook is expected to be positive, with rising demand for novel therapies for genetic abnormalities and uncommon illnesses. Furthermore, individualized gene therapy tailored to unique patient characteristics is projected to drive market expansion.
Advancements in Viral Vector Technology
Continuous progress and innovation in viral vector technology are important factors driving market growth. Viral vectors are used as delivery vehicles to introduce therapeutic genes into the cells of patients. Recent advances in this technique have greatly improved the safety and efficacy of gene therapy therapies. These developments include the creation of more specific and focused viral vectors, such as adeno-associated viruses (AAVs) and lentiviruses, with AAVs gaining popularity due to their ability to transport genetic material with minimal immune reaction and damage. Furthermore, as per the gene therapy market overview, researchers are focusing on developing viral vectors that may carry bigger genetic payloads, broadening the range of curable disorders. These technical innovations not only increase the efficacy of gene treatments but also minimize the likelihood of adverse effects, making them safer for patients. As a result, the advancement of viral vector technology is accelerating the acceptance and efficacy of gene therapy treatments for a broader range of medical problems. For instance, MERCK KGaA, the VirusExpress 293 Adeno-Associated Virus (AAV) Production Platform, offers full viral vector manufacturing, including AAV and lentiviral vectors. Additionally, Catalent Inc. launched UpTempo Virtuoso platform process for the development and manufacturing of adeno-associated viral (AAV) vectors.
Investment And Funding in Research and Development
The growing investments and funding for gene therapy research and development (R&D) efforts are driving market expansion. Pharmaceutical businesses, biotechnology firms, and government agencies are putting major resources into developing gene therapy technologies and therapeutics. Gene treatments have a strong potential for addressing unmet medical needs and providing long-term benefits, which is drawing significant funding. Major pharmaceutical corporations are either purchasing gene therapy startups or building their own gene therapy sections to speed up research, which is positively impacting the gene therapy market outlook. Furthermore, government subsidies and incentives motivate academic institutions and research firms to conduct gene therapy experiments. The Orphan Drug Act in the United States, as well as comparable legislation in other countries, gives incentives for the development of rare illness medicines. For instance, the Cystic Fibrosis Foundation reported investing in SalioGen Therapeutics to support the company's preclinical research into novel gene therapy for cystic fibrosis. Salonen's Gene Coding approach is designed to turn on, turn off, or modify the function of any gene in the genome.
Rising Prevalence of Genetic Disorders and Rare Diseases
The rising prevalence of genetic abnormalities and rare illnesses is propelling the market growth. Many of these diseases have little or no effective treatment choices, making gene therapy an intriguing alternative. As our understanding of genetics advances, more people are diagnosed, and the demand for novel medicines grows. Moreover, gene therapy has the ability to treat the underlying causes of many disorders by repairing or replacing defective genes. This comprises illnesses including muscular dystrophy, cystic fibrosis, and other hereditary metabolic disorders. Pharmaceutical and biotechnology businesses are investing in gene therapy research to fulfill this expanding medical demand, resulting in market expansion and improvements in the area. For instance, as per the United States Center for Disease Control and Prevention article on spinal muscular atrophy (SMA), SMA is a genetic disorder that affects around 1 in every 10,000 people. Therefore, it is one of the most common rare diseases.
Supportive Regulatory Environment
Regulatory organizations in several countries, especially the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), recognize the potential of gene therapy to address essential medical needs. Consequently, they have created structures and accelerated the research and approval of gene treatments. Additionally, fast-track designations, breakthrough treatment designations, and orphan drug status are among the regulatory measures available to gene therapy candidates. These designations ease the clinical trial procedure, shorten development schedules, and encourage corporations to participate in gene therapy research. For instance, the U.S. FDA approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. Such approvals are expected to drive market growth due to the rise in the development of gene therapies.
IMARC Group provides an analysis of the key trends in each segment of the market, along with forecasts at the global, regional and country levels for 2024-2032. Our report has categorized the market based on gene type, vector type, delivery method, and application.
Antigen
Cytokine
Tumor Suppressor
Suicide Gene
Deficiency
Growth Factors
Receptors
Others
The report has provided a detailed breakup and analysis of the market based on the gene type. This includes antigen, cytokine, tumor suppressor, suicide gene, deficiency, growth factors, receptors, and others.
Antigen genes are a critical segment in gene therapy, especially in the context of cancer immunotherapy. These genes code for tumor-specific antigens or antigens associated with infectious agents. By introducing antigen genes into the cells of the patients, the immune system can be activated to recognize and target cancer cells or pathogens. They also enable personalized cancer treatments, where patient-specific antigens can be targeted for a more effective and targeted immune response. For instance, Imugene reported a new clinical trial collaboration and supply agreement with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ, USA, to evaluate the safety and efficacy of Imugene's HER-Vaxx, a B-cell activating immunotherapy, in combination with MSD's anti-PD-1 therapy, pembrolizumab (KEYTRUDA), in patients with HER-2 positive gastric cancer.
Viral Vector
Adenoviruses
Lentiviruses
Retroviruses
Adeno-Associated Virus
Herpes Simplex Virus
Poxvirus
Vaccinia Virus
Others
Non-Viral Techniques
Naked and Plasmid Vectors
Gene Gun
Electroporation
Lipofection
Others
Viral vector holds the largest share in the industry.
A detailed breakup and analysis of the market based on the vector type have also been provided in the report. This includes viral vector (adenoviruses, lentiviruses, retroviruses, adeno-associated virus, herpes simplex virus, poxvirus, vaccinia virus, and others) and non-viral techniques (naked and plasmid vectors, gene gun, electroporation, lipofection, and others). According to the report, viral vector accounted for the largest market share.
Viral vectors, such as adeno-associated viruses (AAVs) and lentiviruses, are gaining traction due to their efficient delivery of therapeutic genes into target cells. AAVs are favored for their safety profile and ability to integrate genetic material into the host genome. They are widely used for a variety of gene therapy applications, including treating genetic disorders, rare diseases, and certain types of cancer. Ongoing research and advancements in viral vector technology are driving the growth of this segment, with numerous clinical trials and approved therapies utilizing viral vectors. For instance, the U.S. FDA granted approval at an accelerated pace for SKYSONA- a lentiviral vector gene therapy shown to slow the progression of neurodegenerative dysfunction, or active cerebral adrenoleukodystrophy in boys with the age range of 4-17.
In-Vivo Gene Therapy
Ex-Vivo Gene Therapy
The report has provided a detailed breakup and analysis of the market based on the delivery method. This includes in-vivo gene therapy and ex-vivo gene therapy.
In-vivo gene therapy involves the direct delivery of therapeutic genes or genetic material into the body of the patient. This approach aims to modify or correct genetic defects within the tissues or organs of patients in their natural physiological environment. In-vivo gene therapy methods typically utilize viral vectors, such as adeno-associated viruses (AAVs) or lentiviruses, to transport the therapeutic genes. This segment of gene therapy is often used for diseases that require systemic treatment, such as certain types of inherited disorders or conditions where the target tissue is easily accessible within the body. For instance, recent advancements in gene supplementation therapy are expanding the options for the treatment of neurological disorders. Among the available delivery vehicles, adeno-associated virus (AAV) is often the favored vector.
Oncological Disorders
Rare Diseases
Cardiovascular Diseases
Neurological Disorders
Infectious Disease
Others
Oncological disorders account for the majority of the market share.
The report has provided a detailed breakup and analysis of the market based on the application. This includes oncological disorders, rare diseases, cardiovascular diseases, neurological disorders, infectious diseases, and others. According to the report, oncological disorders represented the largest segment.
Gene therapy is effective in the treatment of various cancers, including solid tumors and hematological malignancies. Approaches range from introducing tumor-suppressing genes to activating the immune system against cancer cells. CAR-T cell therapy, a type of gene therapy, is gaining traction for its success in treating certain types of leukemia and lymphoma. The ongoing research and development (R&D) activities of gene therapies for oncological disorders are impelling the market growth. For instance, the U.S. FDA approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to or whose disease has returned after at least four last lines (different types) of treatment. Abecma is the first cell-based gene therapy approved by the FDA for treating multiple myeloma.
North America
United States
Canada
Asia-Pacific
China
Japan
India
South Korea
Australia
Indonesia
Others
Europe
Germany
France
United Kingdom
Italy
Spain
Russia
Others
Latin America
Brazil
Mexico
Others
Middle East and Africa
North America leads the market, accounting for the largest gene therapy market share.
The market research report has also provided a comprehensive analysis of all the major regional markets, which include North America (the United States and Canada); Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia, and others); Europe (Germany, France, the United Kingdom, Italy, Spain, Russia, and others); Latin America (Brazil, Mexico, and others); and the Middle East and Africa. According to the report, North America accounted for the largest market share.
North America has a robust healthcare infrastructure, a well-established biotechnology industry, and significant investment in gene therapy research and development (R&D). Favorable regulatory policies, including fast-track designations and orphan drug status, are accelerating the approval and commercialization of gene therapies. Key players in the gene therapy market are headquartered in North America, contributing to the leadership of the region in gene therapy innovations. For instance, as per an update by the National Institutes of Health (NIH), the NIH, USFDA, ten pharmaceutical companies, and five non-profit organizations have partnered to accelerate the development of gene therapies for the 30 million Americans who suffer from a rare disease. Such initiatives are anticipated to increase the demand for gene therapy, thereby contributing to market growth.
Key industry competitors are currently involved in a number of strategic initiatives. These firms are constantly growing their gene therapy portfolios via research, acquisitions, and collaborations. They are undertaking comprehensive clinical studies to develop and get regulatory clearance for novel gene treatments that will treat a variety of ailments, including rare genetic abnormalities and cancer. In addition, they are investing in manufacturing facilities to increase output and ensure gene treatments are available to a larger patient population. Furthermore, these industry heavyweights are pursuing collaborations with academic institutions and smaller biotech businesses to promote innovation and improve gene therapy technology.
Abeona Therapeutics Inc.
Adaptimmune Therapeutics plc
Adverum Biotechnologies Inc.
Amgen Inc.
Astellas Pharma Inc.
Biogen Inc.
Bluebird Bio Inc.
Mustang Bio Inc. (Fortress Biotech Inc.)
Novartis AG
Orchard Therapeutics plc
UniQure N.V.
Voyager Therapeutics Inc.
(Please note that this is only a partial list of the key players, and the complete list is provided in the report.)
January 2023: Spark Therapeutics and Neurochase established a strategic collaboration to develop Neurochase's unique delivery technology for use with selected gene treatments for rare disorders in the CNS. In this agreement, Neurochase will contribute its extensive knowledge in direct drug delivery technology to Spark's premier AAV platform.
January 2023: Voyager Therapeutics and Neurocrine Biosciences entered into a strategic collaboration for the commercialization & development of Voyager's GBA1 program and other next-generation gene therapies for neurological diseases.
June 2023: The U.S. FDA granted approval to Sarepta for ELEVIDYS gene therapy to treat DMD in children of age 4-5 years.
May 2023: Krystal Biotech was granted approval for VYJUVEK gene therapy for Dystrophic Epidermolysis Bullosa treatment.
December 2023: Swiss Agency for Therapeutic Products granted approval to Libmeldy for the treatment of early-onset metachromatic leukodystrophy.
January 2024: Biogen and Ginkgo Bioworks announced that they have completed the gene therapy collaboration involving AAV-based vectors. This is expected to fuel the demand for gene therapies in the coming years.