![]() |
市場調查報告書
商品編碼
1519482
2024-2032 年按基因類型、載體類型、遞送方法、應用和地區分類的基因治療市場報告Gene Therapy Market Report by Gene Type, Vector Type, Delivery Method, Application, and Region 2024-2032 |
2023年全球基因治療市場規模達51IMARC Group美元。由於病毒載體技術的不斷改進和創新,遺傳性疾病和罕見疾病(如肌肉營養不良症、囊性纖維化和各種遺傳性代謝性疾病)發病率的上升,以及研發投入和資金的增加,市場正在穩步擴大。
市場成長與規模:在遺傳異常和罕見疾病盛行率上升的推動下,該產業正在迅速擴張。此外,製藥企業擴大投資於研發(R&D)活動,這對市場產生了積極影響。
技術進步:病毒載體技術的發展正在提高基因治療的有效性和安全性。而且,CRISPR-Cas9技術提升了基因編輯能力,進一步加速了基因治療市場統計。
產業應用:基因療法用於治療多種疾病,包括癌症、遺傳問題和自體免疫疾病。離體基因療法,即在體外操縱細胞,在治療特定遺傳疾病方面越來越受歡迎。
地理趨勢:根據 IMARC 的基因療法市場統計數據,北美憑藉其強大的醫療基礎設施和發達的生物技術產業,在市場上佔據主導地位。然而,在龐大患者群體的推動下,亞太地區正成為一個快速擴張的市場。
競爭格局:領先企業正在進行全面的臨床研究,以開發針對各種疾病(包括罕見遺傳異常和癌症)的新型基因療法並獲得監管許可。市場上一些主要的參與者包括 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。
挑戰與機會:雖然該行業面臨監管限制和高研究成本等限制,但它也預計有可能增加罕見疾病以外的用途並研究新的給藥方式。
未來展望:隨著對遺傳異常和罕見疾病的新療法的需求不斷成長,基因治療市場前景預計將是積極的。此外,針對獨特患者特徵的個人化基因治療預計將推動市場擴張。
病毒載體技術的進步
病毒載體技術的不斷進步和創新是推動市場成長的重要因素。病毒載體用作遞送載體,將治療基因引入患者細胞中。這項技術的最新進展大大提高了基因治療的安全性和有效性。這些進展包括創建更具特異性和針對性的病毒載體,例如腺相關病毒 (AAV) 和慢病毒,AAV 因其能夠以最小的免疫反應和損傷運輸遺傳物質而受到歡迎。此外,根據基因治療市場概況,研究人員正專注於開發可能攜帶更大遺傳負載的病毒載體,從而擴大可治癒疾病的範圍。這些技術創新不僅提高了基因療法的功效,還最大限度地減少了不良反應的可能性,使患者更安全。因此,病毒載體技術的進步正在加速基因療法的接受和功效,以解決更廣泛的醫療問題。例如,MERCK KGaA 的 VirusExpress 293 腺相關病毒 (AAV) 生產平台提供完整的病毒載體製造,包括 AAV 和慢病毒載體。此外,Catalent Inc. 推出了 UpTempo Virtuoso 平台流程,用於開發和製造腺相關病毒 (AAV) 載體。
研究與開發的投資和資助
基因治療研究和開發(R&D)工作的投資和資金不斷增加正在推動市場擴張。製藥公司、生技公司和政府機構正在投入大量資源來開發基因治療技術和療法。基因治療在解決未滿足的醫療需求和提供長期效益方面具有巨大潛力,目前正在吸引大量資金。主要製藥公司要么收購基因治療新創公司,要么建立自己的基因治療部門以加快研究速度,這對基因治療市場前景產生了積極影響。此外,政府補助和激勵措施激勵學術機構和研究公司進行基因治療實驗。美國的《孤兒藥法案》以及其他國家的類似立法為罕見疾病藥物的開發提供了誘因。例如,囊性纖維化基金會報告投資 SalioGen Therapeutics,以支持該公司對囊性纖維化新型基因療法的臨床前研究。薩洛寧的基因編碼方法旨在開啟、關閉或修改基因組中任何基因的功能。
遺傳性疾病和罕見疾病的盛行率不斷上升
遺傳異常和罕見疾病盛行率的上升正在推動市場成長。其中許多疾病幾乎沒有或根本沒有有效的治療選擇,這使得基因療法成為有趣的替代方案。隨著我們對遺傳學認知的進步,越來越多的人被診斷出來,對新藥的需求也在增加。此外,基因療法能夠透過修復或替換有缺陷的基因來治療許多疾病的根本原因。這包括肌肉營養不良症、囊性纖維化和其他遺傳性代謝失調等疾病。製藥和生物技術企業正在投資基因療法研究,以滿足不斷成長的醫療需求,從而導致該領域的市場擴張和改善。例如,根據美國疾病管制與預防中心關於脊髓性肌肉萎縮症 (SMA) 的文章,SMA 是一種遺傳性疾病,每 10,000 人中就有 1 人受到影響。因此,它是最常見的罕見疾病之一。
支持性監管環境
多個國家的監管組織,特別是美國食品和藥物管理局 (FDA) 和歐洲藥品管理局 (EMA),認知到基因療法在滿足基本醫療需求方面的潛力。因此,他們創建了結構並加速了基因治療的研究和批准。此外,快速通道指定、突破性治療指定和孤兒藥狀態也是基因治療候選者可用的監管措施之一。這些指定簡化了臨床試驗程序,縮短了開發時間表,並鼓勵企業參與基因治療研究。例如,美國 FDA 批准了 Zynteglo (betibeglogene autotemcel),這是第一個用於治療成人和兒童 BETA 地中海貧血患者的細胞基因療法。由於基因療法開發的興起,此類批准預計將推動市場成長。
IMARC Group提供了對每個細分市場的主要趨勢的分析,以及 2024-2032 年全球、區域和國家層面的預測。我們的報告根據基因類型、載體類型、遞送方法和應用對市場進行了分類。
抗原
細胞因子
腫瘤抑制劑
自殺基因
不足
生長因子
受體
其他
該報告根據基因類型對市場進行了詳細的細分和分析。這包括抗原、細胞激素、腫瘤抑制因子、自殺基因、缺陷、生長因子、受體等。
抗原基因是基因治療中的關鍵部分,特別是在癌症免疫治療的背景下。這些基因編碼腫瘤特異性抗原或與傳染原相關的抗原。透過將抗原基因引入患者的細胞中,可以激活免疫系統來識別和靶向癌細胞或病原體。它們還可以實現個人化的癌症治療,其中可以針對患者特異性抗原,以產生更有效、更有針對性的免疫反應。例如,Imugene 報告了與美國新澤西州凱尼爾沃思默克公司的商品名MSD 達成的一項新的臨床試驗合作和供應協議,以評估Imugene 的HER-Vaxx(一種B 細胞活化藥物)的安全性和有效性。
病毒載體
腺病毒
慢病毒
逆轉錄病毒
腺相關病毒
單純皰疹病毒
痘病毒
痘苗病毒
其他
非病毒技術
裸載體和質粒載體
基因槍
電穿孔
脂轉染
其他
病毒載體在產業中佔有最大佔有率。
報告還提供了基於載體類型的詳細市場細分和分析。這包括病毒載體(腺病毒、慢病毒、逆轉錄病毒、腺相關病毒、單純皰疹病毒、痘病毒、牛痘病毒等)和非病毒技術(裸載體和質粒載體、基因槍、電穿孔、脂轉染等)。報告稱,病毒載體佔最大的市場。
病毒載體,例如腺相關病毒(AAV)和慢病毒,由於能夠有效地將治療基因傳遞到目標細胞中而受到關注。 AAV 因其安全性和將遺傳物質整合到宿主基因組中的能力而受到青睞。它們廣泛用於各種基因治療應用,包括治療遺傳性疾病、罕見疾病和某些類型的癌症。病毒載體技術的持續研究和進步正在推動這一領域的成長,利用病毒載體進行了大量的臨床試驗和批准的治療。例如,美國 FDA 加速批准了 SKYSONA——一種慢病毒載體基因療法,可減緩 4-17 歲男孩神經退化性功能障礙或活動性腦腎上腺腦白質營養不良的進展。
體內基因治療
離體基因治療
該報告根據交付方式對市場進行了詳細的細分和分析。這包括體內基因治療和離體基因治療。
體內基因治療涉及將治療基因或遺傳物質直接遞送到患者體內。此方法旨在改變或糾正患者自然生理環境中組織或器官內的遺傳缺陷。體內基因治療方法通常利用病毒載體,例如腺相關病毒(AAV)或慢病毒,來運輸治療基因。基因治療的這一部分通常用於需要全身性治療的疾病,例如某些類型的遺傳性疾病或體內目標組織很容易到達的病症。例如,基因補充療法的最新進展正在擴大神經系統疾病治療的選擇。在可用的遞送載體中,腺相關病毒(AAV)通常是最受歡迎的載體。
腫瘤疾病
罕見疾病
心血管疾病
神經系統疾病
傳染病
其他
腫瘤疾病佔據了大部分市場。
該報告根據應用程式提供了詳細的市場細分和分析。這包括腫瘤疾病、罕見疾病、心血管疾病、神經系統疾病、傳染病等。根據該報告,腫瘤疾病佔最大部分。
基因療法可有效治療多種癌症,包括實體腫瘤和血液惡性腫瘤。方法範圍從引入腫瘤抑制基因到激活針對癌細胞的免疫系統。 CAR-T 細胞療法是一種基因療法,因其在治療某些類型的白血病和淋巴瘤方面的成功而受到關注。正在進行的腫瘤疾病基因療法的研發(R&D)活動正在推動市場成長。例如,美國 FDA 批准了 Abecma(idecabtagene vicleucel),這是一種基於細胞的基因療法,用於治療對多發性骨髓瘤成年患者沒有反應或在至少四線(不同類型)治療後疾病復發的患者。 Abecma 是 FDA 批准的第一個用於治療多發性骨髓瘤的細胞基因療法。
北美洲
美國
加拿大
亞太
中國
日本
印度
韓國
澳洲
印尼
其他
歐洲
德國
法國
英國
義大利
西班牙
俄羅斯
其他
拉丁美洲
巴西
墨西哥
其他
中東和非洲
北美市場處於領先地位,佔據最大的基因治療市場。
市場研究報告還對所有主要區域市場進行了全面分析,其中包括北美(美國和加拿大);亞太地區(中國、日本、印度、韓國、澳洲、印尼等);歐洲(德國、法國、英國、義大利、西班牙、俄羅斯等);拉丁美洲(巴西、墨西哥等);以及中東和非洲。報告稱,北美佔據最大的市場佔有率。
北美擁有強大的醫療基礎設施、完善的生物技術產業以及對基因治療研發 (R&D) 的大量投資。有利的監管政策,包括快速通道指定和孤兒藥地位,正在加速基因療法的批准和商業化。基因治療市場的主要參與者總部位於北美,為該地區在基因治療創新方面的領導地位做出了貢獻。例如,根據美國國立衛生研究院(NIH) 的最新消息,NIH、美國食品藥物管理局、十家製藥公司和五個非營利組織已合作,加速開發基因療法,幫助3000 萬患有基因突變的美國人罕見疾病。預計此類措施將增加對基因療法的需求,從而促進市場成長。
主要產業競爭對手目前參與了許多策略舉措。這些公司透過研究、收購和合作不斷擴大其基因治療產品組合。他們正在進行全面的臨床研究,以開發新型基因療法並獲得監管許可,這些療法將治療各種疾病,包括罕見的基因異常和癌症。此外,他們還在投資生產設施,以增加產量並確保更多患者群體能夠獲得基因治療。此外,這些行業巨頭正在尋求與學術機構和小型生物技術企業合作,以促進創新和改進基因治療技術。
Abeona 治療公司
適應免疫療法公司
Adverum 生技公司
安進公司
安斯泰來製藥公司
百健公司
藍鳥生物公司
野馬生物公司(Fortress Biotech Inc.)
諾華公司
果園治療公司
優尼庫爾公司
航海家治療公司
(請注意,這只是關鍵參與者的部分列表,報告中提供了完整列表。)
2023 年 1 月:Spark Therapeutics 和 Neurochase 建立了策略合作,開發 Neurochase 獨特的遞送技術,用於中樞神經系統罕見疾病的選定基因治療。根據該協議,Neurochase 將向 Spark 的首屈一指的 AAV 平台貢獻其在直接藥物傳輸技術方面的豐富知識。
2023 年 1 月:Voyager Therapeutics 和 Neurocrine Biosciences 就 Voyager GBA1 計畫和其他神經系統疾病下一代基因療法的商業化和開發達成策略合作。
2023年6月:美國FDA核准Sarepta用於ELEVIDYS基因療法,用於治療4-5歲兒童的DMD。
2023 年 5 月:Krystal Biotech 的 VYJUVEK 基因療法獲得批准,用於治療營養不良性大皰性表皮鬆解症。
2023 年 12 月:瑞士治療產品管理局核准 Libmeldy 用於治療早發性異染性腦白質營養不良症。
2024 年 1 月:Biogen 和 Ginkgo Bioworks 宣布,他們已經完成了涉及基於 AAV 的載體的基因治療合作。預計這將推動未來幾年對基因療法的需求。
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.