Exagamglogene Autotemcel(Exagamglogene Autotemcel) - 药物靶点：BCL11A_在研适应症：镰状细胞血症，输血依赖性β地中海贫血，输血依赖性地中海贫血_专利_临床

概要

基本信息

药物类型

CRISPR/Cas9

别名

Autologous CRISPR-Cas9 modified CD34+ hHSPCs、CRISPR-Cas9-based-therapy、Exa-cel

+ [4]

靶点

BCL11A

作用方式

抑制剂

作用机制

BCL11A抑制剂(BCL11A蛋白抑制剂)、Gene transference(基因转移)

治疗领域

遗传病与畸形血液及淋巴系统疾病其他疾病

在研适应症

镰状细胞血症输血依赖性β地中海贫血输血依赖性地中海贫血+ [1]

非在研适应症-

原研机构

CRISPR Therapeutics AG

在研机构

Vertex Pharmaceuticals, Inc.

CRISPR Therapeutics AGVertex Pharmaceuticals (Europe) Ltd. (Ireland)

非在研机构-

权益机构

Vertex Pharmaceuticals, Inc.

Charles River Laboratories, Inc.

MaxCyte, Inc.

+ [2]

最高研发阶段批准上市

首次获批日期

英国 (2023-11-15),

镰状细胞血症输血依赖性β地中海贫血

最高研发阶段(中国)-

特殊审评优先审评 (美国)、快速通道 (美国)、孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、优先药物（PRIME） (欧盟)、再生医学先进疗法 (美国)、孤儿药 (英国)、附条件批准 (英国)、优先审评 (加拿大)

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关联

8

项与 Exagamglogene Autotemcel 相关的临床试验

NCT05951205

/ Not yet recruiting临床3期

A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype

The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).

开始日期2027-07-31

申办/合作机构

Vertex Pharmaceuticals, Inc.

ChiCTR2300073795

/ Recruiting早期临床1期IIT

An International, Multicenter, Phase I Open Label Study to Evaluate Safety and Efficacy of Administration of Autologous CD34+ Cells Gene Edited Ex Vivo Using a CRISPR/AAV6 Platform to Introduce a Codon Optimized cDNA Version of the Red-Cell Type Pyruvate Kinase Genein Adult and Pediatric Patients with Severe Pyruvate Kinase

开始日期2023-11-09

申办/合作机构

上海交通大学医学院附属上海儿童医学中心

NCT05477563

/ Recruiting临床3期

A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

开始日期2022-08-02

申办/合作机构

Vertex Pharmaceuticals, Inc.

100 项与 Exagamglogene Autotemcel 相关的临床结果

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100 项与 Exagamglogene Autotemcel 相关的转化医学

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100 项与 Exagamglogene Autotemcel 相关的专利（医药）

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37

项与 Exagamglogene Autotemcel 相关的文献（医药）

2025-08-01·VALUE IN HEALTH

Implementing the Generalized Risk-Adjusted Cost-Effectiveness Model for Sickle Cell Disease: A Case Study

Article

作者: Land, Natalie ; Hasan, Md Tahsin ; Graf, Marlon ; Mulligan, Karen ; Chou, Jacquelyn W ; Kleintjens, Joris

OBJECTIVES:

This study aims to quantify the societal value of curing sickle cell disease by implementing the generalized risk-adjusted cost-effectiveness (GRACE) method, thereby adjusting for factors such as disease severity, reduction of uncertainty in treatment outcomes, and trade-offs between quality of life (QoL) and life expectancy in a cost-effectiveness analysis (CEA).

METHODS:

Using GRACE, we recalibrated a recent health technology assessment in sickle cell disease for 2 gene therapies, lovo-cel and exa-cel. The GRACE framework modified existing CEA by adjusting willingness-to-pay thresholds based on untreated illness severity, incorporating treatment outcome uncertainty, and varying the substitution rates between life expectancy and QoL across health states.

RESULTS:

Implementing the GRACE framework resulted in a 6% reduction in both direct and societal incremental cost-effectiveness ratios for lovo-cel and exa-cel, demonstrating a decrease from $192 651 and $161 816 to $182 036 and $152 900 per quality-adjusted life year, respectively. Additionally, willingness-to-pay thresholds increased by approximately 50%, reflecting a higher valuation of treatments under GRACE. GRACE-adjusted estimates suggest that lovo-cel and exa-cel are cost-effective from both direct payer and societal perspectives.

CONCLUSIONS:

The GRACE method offers a more comprehensive and precise estimation of societal value, leading to more efficient and equitable resource allocation. This study not only highlights the limitations of traditional CEA in capturing the total societal value of treatments for severe diseases but also provides a roadmap for incorporating GRACE model elements into health technology assessments, thereby facilitating a broader acceptance of innovative therapies that significantly enhance patient QoL.

2025-07-31·STEM CELLS AND DEVELOPMENT

Advances in Sickle Cell Disease Treatment: A Comparative Review of Hematopoietic Stem Cell Transplantation and Gene Therapy (Casgevy and Lyfgenia).

Review

作者: Kawari, Mohammed ; Abdelazim, Omer Tarig Fathi ; Sharafeldin, Abu-Baker Khalid ; Hasan, Zahra Abdulla Isa Yusuf ; Toorani, Zainab Abdulmajeed

Sickle cell disease (SCD), affecting approximately 2.1% of Bahrain's population, is a prevalent inherited disorder that necessitates effective treatments and long-term management. This review highlights two innovative gene therapies (Casgevy and Lyfgenia) and compares their efficacy and safety with hematopoietic stem cell transplantation (HSCT)-the only curative option currently available for SCD. While HSCT offers a 90% success rate with suitable donors, its limitations include donor scarcity and toxicity. Gene therapies like Casgevy and Lyfgenia show promising efficacy in reducing SCD complications while bypassing such limitations. In the Kingdom of Bahrain, the Bahrain Oncology Center approved Casgevy in December 2023 and completed its first patient treatment in mid-February 2025, making Bahrain an early adopter. This milestone marks a crucial moment in the history of both SCD and gene therapies and thus warrants exploring the considerations revolving around their implementation. Although these therapies seem to offer hope for patients ineligible for HSCT, their long-term outcomes remain unassessed-further studies with extended follow-up are needed to confirm their safety and durability.

2025-07-14·Cardiovascular & hematological disorders drug targets

Updated Review of Current Therapeutic Approaches for the Management of Sickle Cell Disease

Article

作者: Bhardwaj, Harish ; Jangde, Rajendra Kumar ; Puri, Devwati ; Verma, Bhupendra

Abstract::

Sickle cell disease is a severe genetic blood disorder marked by the production of abnormal hemoglobin (HbS), leading to sickle-shaped red blood cells that obstruct blood flow and cause various problems, such as the increased risk of infection, persistent anemia, acute pain episodes, and organ damage. Roughly 100,000 Americans suffer from SCD, with approximately 40,000 of them being children. Black people have the highest frequency of the disease. There are six Food and Drug Administration (FDA)-approved drugs, hydroxyurea, L-glutamine, crizanlizumab- TMCA, voxelotor, Casgevy, and Lyfgenia, that are used for the prophylaxis and treatment of serious complications of sickle cell disease. Current treatment approaches focus on symptom management, including pain control, hydroxyurea to reduce pain crises, and transfusions for severe anemia. Based on the clinical trial results, L-glutamine and crizanlizumab-TMCA prevent cell damage and hemoglobin sickling by reducing the sickle cell crisis episodes. At the same time, voxelotor improves hemoglobin oxygen-binding capacity in patients with SCD. Novel therapies, such as gene therapy and clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR-Cas9) technology, aim to correct the genetic defect. At the same time, stem cell and bone marrow transplants offer potential cures but are limited by the availability of donors and side effects. Ongoing research seeks to enhance treatment options and develop potential cures for SCD. This review attempts to present a comprehensive overview of the current therapeutic approaches and newly developed innovative medicines to combat and potentially eradicate SCD with an emphasis on their mechanisms, efficacy, and clinical implications.

833

项与 Exagamglogene Autotemcel 相关的新闻（医药）

2025-09-10

·医药地理

中国首例！碱基编辑疗法成功治愈镰刀型细胞贫血病

近日，正序生物宣布与广西医科大学第一附属医院合作开展的针对镰刀型细胞贫血病（SCD）的碱基编辑药物CS-101注射液的研究者发起的临床研究成功治愈首位患者，患者在治疗后胎儿血红蛋白（HbF）水平显著且持续上升，镰状血红蛋白（HbS）水平显著且持续降低，治疗后6个月起HbF与HbS比例稳定在6.5:3.5，总血红蛋白浓度稳定至120 g/L以上，治疗后6个月中没有出现血管闭塞危象，已回归到正常生活中。这是中国首次通过碱基编辑疗法治愈镰刀型细胞贫血病的成功案例。被“扭曲”的红细胞镰刀型细胞贫血病，也称为镰状细胞贫血，是一种常染色体显性遗传血红蛋白病，根本病因在于β-珠蛋白肽链第6位的谷氨酸被缬氨酸替代，形成异常的血红蛋白S。在低氧条件下，氧分压下降时血红蛋白S分子之间相互作用，成为螺旋形多聚体，使红细胞扭曲成镰状细胞，这一过程称为“镰变”。镰刀型细胞贫血病临床表现为慢性溶血性贫血、再发性疼痛危象、易感染、慢性局部缺血导致器官组织损害等，严重时可危及生命。全球约有3.5%的人口携带镰刀型细胞贫血病突变基因，每年约有30万婴儿出生时患有此病，在非洲、地中海沿岸、中东和东南亚等地区发病率较高。目前，镰刀型细胞贫血病的常规治疗手段包括药物治疗和输血治疗，这两种方法均属于对症治疗，虽可缓解患者的临床症状，但无法实现根本性治愈。此外，造血干细胞移植是一种有望根治该病的策略，但传统的异体移植依赖于匹配的供体，在实际中往往难以获得。而基因编辑疗法的出现为彻底治愈带来了新希望：该技术通过编辑患者自身的造血干细胞，激活胎儿血红蛋白（HbF）的表达，不仅避免了寻找供体的等待时间，也显著缩短了治疗周期，为疾病的彻底治愈带来了曙光。 Pharma ONE药物研发大数据平台显示，目前全球仅有一款基因编辑疗法——Casgevy（通用名：exagamglogene autotemcel）获批上市。该疗法由Vertex Pharmaceuticals与CRISPR Therapeutics共同开发，是一种自体、体外CRISPR/Cas9基因编辑治疗方法，于2023年首次在英国获得批准，用于治疗地中海贫血和镰状细胞性贫血。它的成功上市标志着基因编辑技术在临床应用中取得重大突破，并为未来更多基因编辑疗法的研发与应用奠定了重要基础。镰状细胞性贫血药物全球获批情况数据来源：Pharma ONE药物研发大数据平台 CS-101展现革命性疗效 CS-101注射液是正序生物利用上海科技大学自主研发的高精准变形式碱基编辑器tBE（transformer Base Editor）开发的一款针对β-血红蛋白病的基因编辑药物，具有高效、安全的治疗效果。CS-101注射液通过采集患者自体造血干细胞，利用tBE对患者自体造血干细胞中的HBG1/2启动子区域进行精准碱基编辑，模拟健康人群中天然存在的有益碱基突变，重新激活γ-珠蛋白的表达，再将编辑后的造血干细胞回输至患者体内，使得患者胎儿血红蛋白浓度快速升高，有效抑制红细胞镰变，显著减少血管闭塞危象和溶血。由于CS-101编辑靶点的高效性和tBE技术的安全性，相较于其他基于CRISPR技术的β-血红蛋白病的编辑疗法，CS-101可以使患者迅速地完成造血重建，更快速并大幅提升胎儿血红蛋白水平从而降低镰刀状血红蛋白的占比，更高效且稳定地阻止红细胞发生镰变；同时不会引发患者基因组DNA大片段缺失、染色体突变、脱靶突变等风险，显示出了更好的有效性和安全性。官方表示，CS-101注射液已经在临床试验中成功治愈了近20例β-地贫患者和镰刀型细胞贫血病患者，首位β-地贫患者在接受CS-101治疗后已经摆脱输血依赖超过22个月。CS-101注射液有望成为β-血红蛋白病全球首创（First-in-Class）的碱基编辑治疗药物和同类最好（Best-in-Class）的基因编辑治疗药物。结语此次中国首例碱基编辑疗法成功治愈镰刀型细胞贫血病，不仅是基因编辑技术在临床应用的又一重大突破，更为众多患者点燃了根治的希望。CS-101注射液凭借其高效、安全、精准的优势，有望引领β-血红蛋白病治疗迈入“终身治愈”的新时代，也标志着中国在全球基因治疗赛道上迈出了坚实而领先的一步。 END 如需获取更多数据洞察信息或公众号内容合作，请联系医药地理小助手微信号：pharmadl001

基因疗法临床研究上市批准

2025-09-08

·PRNewswire

ToolGen Files Patent Infringement Lawsuit Against Lonza in the Netherlands

Lawsuit filed against Lonza, the Dutch manufacturer of the world's first CRISPR-based genome editing therapy, CASGEVY Second European action following UK litigation, enforcing ToolGen's rights to fundamental CRISPR-Cas9 RNP technology SEOUL, South Korea, Sept. 8, 2025 /PRNewswire/ -- ToolGen, Inc. (KOSDAQ: 199800), a global leader in genome editing technology, today announced that it has filed a patent infringement lawsuit against Lonza Netherlands B.V., a contract development and manufacturing organization (CDMO) that produces CASGEVY, the world's first approved CRISPR-based gene therapy. The case was filed with the District Court of The Hague. The lawsuit charges that Lonza, in its production of Vertex's CASGEVY at its Dutch facility, infringes ToolGen's European patent (EP 4 357 457) covering CRISPR-Cas9 RNP technology. CASGEVY, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is the world's first gene-editing therapy approved for sickle cell disease and beta-thalassemia. It has been authorized in the United Kingdom, United States, and European Union. Under a long-term supply agreement with Vertex, Lonza manufactures CASGEVY at its Netherlands site. This Dutch action follows ToolGen's UK lawsuit filed in April against parties involved in CASGEVY's sale and production. By targeting a key European production hub, ToolGen aims to reaffirm the importance of its foundational CRISPR-Cas9 RNP technology and strengthen protection of its intellectual property rights. "Behind the successful commercialization of CASGEVY lies the pivotal role of ToolGen's proprietary CRISPR-Cas9 RNP platform," said Jong Sang Ryu, CEO of ToolGen. "Toolgen filed this lawsuit because it cannot accept the unauthorized use of its proprietary technology. The lawsuit is not intended to restrict patient access to life-changing therapies, but to secure fair recognition and just compensation for the value of ToolGen's innovations. We remain open to exploring an amicable resolution of the dispute, ultimately fostering a sustainable and mutually beneficial ecosystem." Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

专利侵权基因疗法上市批准

2025-09-03

·药时空

20亿美元！Vertex重金押注自免领域，合作开发小型偶联药物和T细胞衔接器

▲多重福利来袭，点击即可预约 2025年9月2日，Enlaza Therapeutics宣布与Vertex Pharmaceuticals达成多靶点药物发现合作，利用Enlaza专有的War-Lock技术平台，为自身免疫疾病开发药物偶联物和T细胞衔接器（TCE），并改善接受基因编辑疗法Casgevy治疗镰状细胞病/β地中海贫血患者的预处理方案。根据合作协议，Vertex将支付4500万美元预付款和股权投资，未来潜在的里程碑付款可能高达20亿美元，包括研究、开发、监管和商业里程碑，以及基于净销售额的分级特许权使用费。Enlaza主要负责早期的研究工作，以确定有潜力的候选药物；而Vertex则负责从确定候选药物开始的所有后续工作，包括临床开发、生产和销售。另外，Vertex将承担与四年合作相关的所有研究和开发成本。 Enlaza是共价生物制剂领域的先行者，总部位于加利福尼亚州拉霍亚。“Enlaza”源自西班牙语单词enlazar ，意为“连接（link）”。2022年12月，公司获得了6100万美元的种子融资，以建立专注于癌症的共价生物制剂管线，不过，公司官网并未披露具体管线。2024年4月，公司完成了1亿美元的A轮融资。所谓共价药物，指的是药物配体上存在亲电基团（通常被称作warhead，弹头），与受体上的亲核基团发生共价反应，产生较为稳固的共价键型配体-受体加合物。共价药物的出现，为那些不可成药受体参与的致病过程提供了解决的曙光。共价药物发展历程的第一和第二阶段是小分子和多肽共价药物，共价生物制剂的出现则将共价药物推到了一个崭新的阶段。 Enlaza首席执行官Sergio Duron曾表示：“生物制剂是一类能与靶点通过共价键永久结合发挥生物学功能的蛋白质药物。通常，蛋白质使用较弱的非共价键相互连接，我们认为，共价键将成为治疗性蛋白质和生物制品领域工具箱的标准组成部分，相信利用这项技术可以改变药物发现，并为患者提供有价值的治疗效果。” 据公告显示，Vertex将利用Enlaza的专有War-Lock平台，该平台能够创建与药物靶点共价结合的高度特异性弹头。Enlaza表示，这种“空白领域”技术生成的蛋白质药物保留了小分子生物制剂的选择性，同时扩展了治疗窗口，并使多种治疗模式中的疗法更有效。 Enlaza首席执行官Sergio Duron在周二的声明中表示，该平台“在肿瘤学领域展现了令人信服的潜力，而这一战略合作标志着我们扩展到自身免疫适应症的一个关键转折点”。据悉，War-Lock平台开发的管线是一类高度特异性的治疗弹头，可与靶点结合，设计治疗性药物。比方说，通过对蛋白质疗法进行工程设计和修改，可装载多种有效载荷，从而创建可特定靶组织递送的抗体药物偶联物（ADC）或放射性配体疗法（RLT），不会导致长期暴露于全身。公司认为，这种新型的War-Lock能够克服当前生物药物的缺点。 Vertex方面，该公司全球研究高级副总裁Mark Bunnage在声明中表示，公司“致力于开发符合我们独特研发战略的变革性疗法，包括某些自身免疫疾病和Casgevy的更温和预处理，Casgevy是首个且唯一获批的CRISPR/Cas9基因编辑疗法”。 2023年底，FDA历史性地批准了Vertex与CRISPR Therapeutics联合开发的CRISPR基因编辑疗法exa-cel（商品名Casgevy），用于治疗镰状细胞病和输血依赖型β地中海贫血，标志着基于CRISPR的基因编辑技术正式步入临床应用的黄金时代。2024年2月，欧洲批准Casgevy用于治疗镰状细胞性贫血（SCD）和β地中海贫血。Vertex和CRISPR为Casgevy定价220万美元，今年第一季度该药的销售额达1400万美元，高于2024年第四季度的800万美元。值得注意的是，自身免疫和炎症（I&I）领域在过去几年中吸引了大量投资活动，其中不乏赛诺菲和吉利德等大公司的大手笔。仅在7月，葛兰素史克就与恒瑞达成了一项高达120亿美元的协议，以推进多达12种针对呼吸、免疫和肿瘤学适应症的新型疗法，百时美施贵宝也分拆出了一家拥有五项免疫学资产的新公司，并从贝恩资本获得了3亿美元的资金。有数据显示，全球免疫市场预计到2032年将突破2570亿美元，包括Vertex在内的药企自然不会错过这一潜力巨大的营收赛道。参考资料： [1]Vertex, Enlaza Declare War on Autoimmune Disease in Potential $2B+ Pact.Heather McKenzie.September 2, 2025. [2]北大校友担任顾问，摩根大通、再生元押注，初创融资1亿美元，基于非天然氨基酸开发新型共价蛋白疗法. 生辉. 2024-05-01. [3] 新型药物模态：共价生物制剂. 药渡. 2024-06-11. [4] 2025年生物制药投资风向. 一度医药. 2025-01-22. [5]Enlaza Therapeutics、Vertex Pharmaceuticals官网等. 识别微信二维码，可添加药时空小编请注明：姓名+研究方向！

基因疗法引进/卖出细胞疗法

100 项与 Exagamglogene Autotemcel 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15572

研发状态

批准上市

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适应症	国家/地区	公司	日期
镰状细胞血症	英国	Vertex Pharmaceuticals (Europe) Ltd. (Ireland)	2023-11-15
输血依赖性β地中海贫血	英国	Vertex Pharmaceuticals (Europe) Ltd. (Ireland)	2023-11-15

未上市

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适应症	最高研发状态	国家/地区	公司	日期
输血依赖性地中海贫血	临床3期	美国	Vertex Pharmaceuticals, Inc.	2022-05-03
输血依赖性地中海贫血	临床3期	美国	CRISPR Therapeutics AG	2022-05-03
输血依赖性地中海贫血	临床3期	加拿大	CRISPR Therapeutics AG	2022-05-03
输血依赖性地中海贫血	临床3期	加拿大	Vertex Pharmaceuticals, Inc.	2022-05-03
输血依赖性地中海贫血	临床3期	德国	Vertex Pharmaceuticals, Inc.	2022-05-03
输血依赖性地中海贫血	临床3期	德国	CRISPR Therapeutics AG	2022-05-03
输血依赖性地中海贫血	临床3期	意大利	CRISPR Therapeutics AG	2022-05-03
输血依赖性地中海贫血	临床3期	意大利	Vertex Pharmaceuticals, Inc.	2022-05-03
输血依赖性地中海贫血	临床3期	英国	Vertex Pharmaceuticals, Inc.	2022-05-03
输血依赖性地中海贫血	临床3期	英国	CRISPR Therapeutics AG	2022-05-03

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临床结果

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


CLIMB THAL-111 and CLIMB-131 (EHA2025)	临床3期	输血依赖性地中海贫血	56	Exagamglogene autotemcel	製鬱鹹衊糧鏇願鏇餘顧(鹹鏇遞鹹膚鹹繭糧壓鹽) = 憲選憲艱範膚鹽鏇願廠築鬱鑰艱艱積壓鏇衊壓 (築鑰醖鬱蓋簾夢壓鑰齋 ) 更多	积极	2025-05-14
CLIMB SCD-121 (EHA2025)	临床3期	血管闭塞危机	58	Exagamglogene autotemcel	衊獵積顧鏇築壓鹽選繭(網選衊製淵願觸膚繭範) = 壓繭齋艱鹹鏇鹹衊選憲遞蓋餘衊廠願鹹廠觸鹽 (範願範廠齋願夢糧衊鏇 ) 更多	积极	2025-05-14
ASH2024	N/A	镰状细胞血症	-	Exagamglogene autotemcel	鹹齋繭餘獵繭獵衊構鹹(構觸顧觸鹽醖簾膚築構) = 52.2% 艱鏇獵觸醖築衊網構遞 (齋築簾夢鏇構壓窪製網 ) 更多	-	2024-12-09
ASH2024	N/A	输血依赖性地中海贫血	-	Exagamglogene autotemcel	衊艱憲構鑰襯願築鏇簾(願顧鬱憲鑰積醖壓願製) = 60.7% 築選艱鑰構獵膚積獵遞 (齋餘選範襯繭膚遞窪壓 ) 更多	-	2024-12-08
NEWS 人工标引	N/A	输血依赖性β地中海贫血 \| 镰状细胞血症	-	Casgevy (SCD)	壓鏇築網鹽憲膚鏇遞衊(鬱餘襯遞遞製網獵積築) = 觸顧齋獵網積壓鏇衊廠製遞範繭襯糧獵膚艱衊 (淵獵築構鹹製遞構簾築 ) 更多	积极	2024-06-15
NEWS 人工标引	N/A	输血依赖性β地中海贫血 \| 镰状细胞血症	-	Casgevy (TDT)	壓襯遞範鏇遞糧襯淵顧(齋顧簾構餘膚築顧鑰廠) = 構鑰餘醖繭獵窪蓋繭壓齋夢鹹鏇願憲繭遞鏇鹹 (遞獵鏇繭衊襯積衊簾膚 ) 更多	积极	2024-06-15
NCT03655678 (CTX001-111 \| CLIMB THAL-111, EHA2024) 人工标引	临床3期	输血依赖性β地中海贫血	24	Exagamglogene autotemcel	簾構鬱窪範窪齋淵膚餘(觸願簾積鏇齋獵壓壓觸) = 艱窪襯簾廠範範醖獵觸構顧網鹽鬱顧艱鬱構製 (廠窪鹽網鑰鹽簾憲鑰廠, 0.21) 更多	积极	2024-05-14
NCT03745287 (CLIMB SCD-121, EHA 12024 \| EHA 22024) 人工标引	临床3期	镰状细胞血症	24	exa-cel	願範糧願憲廠簾艱淵夢(窪廠築網齋鏇鹽壓積願) = By month 6, EQ-5D-5L health utility US index score and EQ VAS score showed substantial improvements,which were maintained through month 24 (change at month 24 [n=17]: 0.13 [0.19]; minimal clinically important difference [MCID] 0.078 and 26.9 [22.6]; MCID 7 to 10, respectively). 糧積艱夢鏇糧醖鏇廠淵 (鏇獵膚鏇艱網製構鬱構 ) 更多	积极	2024-05-14
NCT03745287 (CLIMB SCD-121, EHA 12024 \| EHA 22024) 人工标引	临床3期	镰状细胞血症	46	Exagamglogene autotemcel	構夢築壓壓糧網蓋鏇簾(鬱襯鬱廠獵積積鬱製鏇) = 窪遞網遞襯夢積繭醖願衊齋築遞齋壓鏇廠鹹齋 (艱鑰遞簾顧觸糧蓋觸蓋, 79 ~ 99) 更多	积极	2024-05-14
NCT03655678 (CTX001-111, FDA) 人工标引	临床2/3期	输血依赖性β地中海贫血	52	CASGEVY	觸壓糧襯範遞糧遞觸鏇(繭餘遞艱艱糧鬱鏇衊願) = 築蓋鑰獵繭襯選壓鹹範鹽鬱構蓋網製鑰範願艱 (觸鹽選憲選顧範淵簾窪, 75.7 ~ 100) 更多	积极	2024-02-07
NCT03655678 (CLIMB THAL-111, Tandem Meetings ASTCT and CIBMTR2024) 人工标引	临床3期	输血依赖性β地中海贫血	44	Exagamglogene autotemcel	窪淵鏇積鏇鏇獵網願鑰(積簾淵構齋築鹽夢艱艱) = 餘蓋蓋積遞鑰齋積鬱鏇壓鬱簾壓鬱築構鏇廠蓋 (製網餘糧構鹽壓淵窪選 )	积极	2024-02-01