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2024-04-01

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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项与 Exagamglogene Autotemcel 相关的文献（医药）

2025-05-01·PHARMACOECONOMICS

Innovative Payment Models for Sickle-Cell Disease Gene Therapies in Medicaid: Leveraging Real-World Data and Insights from CMMI’s Gene Therapy Access Model

Article

作者: Nair, Kavita ; Leonard, Jim ; DiStefano, Michael J ; Wright, Garth C ; Zemplenyi, Antal ; McQueen, R Brett ; Anderson, Kelly E

OBJECTIVE:

This study aims to evaluate the financial implications of implementing various payment models, including outcome-based agreements (OBAs), volume-based rebates, and guaranteed rebates, for the newly approved gene therapies, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), in the treatment of sickle-cell disease (SCD) from the perspective of Colorado Medicaid. The analysis specifically examines the cost of standard of care (SoC) for severe SCD, the impact of different eligibility criteria based on vaso-occlusive events (VOEs), and the potential financial impacts associated with rebate structures.

METHODS:

Data from the Colorado Department of Health Care Policy & Financing (HCPF) database was used to estimate the annual costs for Medicaid-enrolled patients with severe SCD from 2018 to 2023. Patients were selected based on various eligibility criteria, including the number of VOEs, acute chest syndrome events, and stroke diagnoses. Three-state Markov models (SCD, stable, and dead) were constructed to compare the costs of SoC and gene therapies. The durability of gene therapy effectiveness and the financial impact of OBAs, volume-based rebates, and guaranteed rebates were evaluated over a 6-year contract period, with scenarios reflecting different VOE criteria and treatment durability.

RESULTS:

The average annual SoC cost for severe SCD patients (N = 138) was US$45,941 (SD US$59,653), with higher costs associated with more frequent VOEs. Gene therapies exa-cel and lovo-cel, with one-off list prices of US$2.2 million and US$3.1 million, respectively, exhibited high upfront costs, resulting in a negative cumulative balance averaging - US$2.11 million for exa-cel and - US$3.00 million for lovo-cel per patient over 6 years compared with SoC. Outcome-based rebates could potentially save Medicaid approximately US$260K (uncertainty interval 88K-772K) per patient on average for exa-cel and US$367K (uncertainty interval 122K-1111K) for lovo-cel after they pay the full up-front cost. Volume-based and guaranteed rebates also offered potential savings but varied in impact based on contract duration and effectiveness of gene therapy.

CONCLUSIONS:

The study highlights critical considerations for Medicaid in negotiating OBAs for SCD gene therapies. Achieving budget neutrality over 6 years is unlikely due to low SoC costs. However, payment models can enhance value-based spending by linking high therapy costs and potential rebates to the health gains these treatments may offer. OBAs offer offsets contingent on therapy effectiveness durability and contract terms (such as length and price), while varying eligibility criteria impact budgets and outcomes. Medicaid real-world data is crucial for navigating complexities in defining eligible populations and structuring OBAs.

2025-05-01·ADVANCES IN THERAPY

Impact of Different Definitions of Vaso-Occlusion on Efficacy Assessments in Sickle Cell Disease Clinical Trials

Review

作者: Xuan, Fengjuan ; Imren, Suzan ; Eckrich, Michael J ; Li, Nanxin ; Locatelli, Franco ; Grupp, Stephan A ; Frangoul, Haydar

INTRODUCTION:

Patients with sickle cell disease (SCD) experience recurrent, severe pain events due to vaso-occlusion. Eliminating these acute pain events is a key outcome in SCD clinical trials; however, the definition of a vaso-occlusive crisis (VOC) or a vaso-occlusive event (VOE) has not been consistently applied, hampering comparisons of treatment efficacy between different therapeutic approaches. We have examined the degree to which differing definitions of vaso-occlusion in clinical trial endpoints impact efficacy outcomes.

METHODS:

Descriptions of clinical endpoints related to vaso-occlusion and pain events were reviewed from trials of exagamglogene autotemcel (exa-cel), lovotibeglogene autotemcel (lovo-cel), renizgamglogene autogedtemcel (reni-cel), hydroxyurea, L-glutamine, voxelotor, and crizanlizumab. Patient-level data from the published exa-cel Phase 3 pivotal trial (CLIMB SCD-121; data cut 14 Jun 2023) was used to evaluate efficacy outcomes based on differing endpoint definitions of vaso-occlusion.

RESULTS:

In the seven clinical trials reviewed, definitions of vaso-occlusion and/or pain events varied by care setting, duration of care, treatments used, and associated complications, with the frequency and duration of medical facility visits for acute pain events being most dissimilar between trials. Definitions of severe VOCs (exa-cel), VOC (voxelotor), and sickle cell-related pain crises (SCPCs; crizanlizumab and L-glutamine) included pain events requiring a medical facility visit of any duration, whereas the definition of painful crises (hydroxyurea) required a medical facility visit of > 4 h and the definition of severe vaso-occlusive events (VOEs; lovo-cel and reni-cel) required a hospital or emergency room (ER) observation unit visit lasting ≥ 24 h or ≥ 2 visits to a day unit or ER over a 72-h period. Based on the definition of severe VOCs, 29/30 patients [96.7%; 95% confidence interval (CI): 82.8, 99.9] in the CLIMB SCD-121 trial were considered free from severe VOCs for ≥ 12 consecutive months, whereas when the severe VOEs definition was applied to the same data, all patients (30/30; 100.0%; 95% CI: 88.4, 100.0) were considered free from severe VOEs for ≥ 12 consecutive months.

CONCLUSION:

Differences exist in definitions of vaso-occlusion and pain events used in SCD clinical trials. Severe VOCs (exa-cel), VOC (voxelotor), and SCPCs (crizanlizumab and L-glutamine) were more broadly inclusive than severe VOEs (lovo-cel and reni-cel) or painful crisis (hydroxyurea). Clinically, these differences resulted in differing numbers of patients being considered free from vaso-occlusion pain events, underscoring the challenge in comparing frequencies of pain events across SCD clinical trials.

2025-01-01·CYTOTHERAPY

Sickle cell disease gene therapy drug expenses and reimbursement: a litmus test for commercial pricing strategy and patient access for curative therapies

Article

作者: Galipeau, Jacques ; Hume, Ashley ; Grilley, Bambi J

Durable gene therapies newly approved by the Food and Drug Administration for sickle cell disease offer the promise of cure with subsequent improvement in quality of life through avoidance of repeated hospitalizations and worsening of disabilities in these patients. In the United States, following Food and Drug Administration approval, lovotibeglogene autotemcel (Lyfgenia, bluebird bio, Inc, Sommerville, MA, USA) and exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals Inc, Boston, MA, USA) were launched in 2024 for treatment of patients 12 years and older with frequent vaso-occlusive crises. Here we will discuss the first treatment with lovotibeglogene as a test case serving to highlight the multiple drug reimbursement pathways in the United States for cell and gene products for orphan disorders and their impact on patient adoption and access.

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项与 Exagamglogene Autotemcel 相关的新闻（医药）

2025-05-12

·BioPharmaDive

Innovating a healthier tomorrow: Entering a new era of medicine

The number of registered US clinical trials has almost tripled in the last decade, from over 180,000 in 2015 to nearly 520,000 today. Improvements in our understanding of the human genome, technological tools like artificial intelligence, and global investments in scientific research have all contributed to this exciting new era of medicine. But how are these exciting discoveries being translated into real world benefits? In this article, well explore the progress being made on the cutting-edge of science today and how Danaher is supporting this growth. Together, these breakthroughs model how simple ideas can serve as catalysts for incredible innovation and allow us to envision new possibilities. Messenger RNA (mRNA) technology is best known for the foundational role it played in developing COVID-19 vaccines. In fact, Katalin Kariko and Drew Weissman were awarded the Nobel Prize for their work on mRNA vaccines. However, the potential for mRNA-based medicines extends far beyond COVID-19 and could advance treatment for an astonishing array of diseases. mRNA therapies are currently being explored to treat cancers, vaccinate against infectious diseases, and even cure inherited genetic conditions. However, one major challenge facing the industry is ensuring the reliable manufacturing of high-quality mRNA molecules for an affordable price. Overcoming these challenges requires a comprehensive mRNA production ecosystem that houses solutions and capabilities under one roof. By providing access to a diverse set of businesses, Danaher is enabling their partners to imagine new frontiers for mRNA across a range of sizes and scales. Artificial Intelligence (AI) and machine learning applications are another frequently discussed advancement that is already being implemented in approximately 5-10% of pathology labs. Pathologists are frequently tasked with studying images (such as those obtained from tissue biopsies) to help guide treatment plans or diagnose patients. Recently, the technology to scan, store, view, and share high-quality digital images of pathology slides has enabled pathologists to consult on patient cases remotely. This allows more equitable access to high-quality diagnostic care and can result in quicker turnaround times. Even more exciting, AI and machine learning applications can help filter out healthy tissue samples from these databases, ensuring that the valuable time of expert pathologists is spend analyzing the samples of patients who can benefit most from their expertise. Implementing these digital pathology practices is in the early phase, but Danaher believes large-scale adoption is possible and will improve the healthcare experience for both patients and clinicians.Another exciting technology prepared to dramatically improve healthcare is CRISPR. The discovery of CRISPR revolutionized genetics and made gene editing a modern-day reality. CRISPR is yet another Nobel Prize-winning discovery, made in 2012 by Jennifer Doudna and Emmanuelle Charpentier. In 2023, the FDA approved a sickle cell treatment called Casgevy, which was the first CRISPR-based therapy in the US. There are many other CRISPR-based therapies in development, and this gene editing technology may particularly benefit people with rare diseases. However, current scientific and regulatory models are ill-suited to assess these therapies in a timely manner. Organizations such as the Innovative Genomics Institute and Danaher are working to address these issues and help realize the full promise of CRISPRs precise gene editing ability.In addition to clinical advancement, Danaher is supporting new research projects that will greatly enhance our understanding of health. For example, a global non-profit called the Human Immunome Project (HIP) is working to decode and model the immune system to improve health outcomes worldwide. This organization is seeking to build a comprehensive immunological dataset that will capture the diversity and complexity of this vital system. The immune system plays a central role in human health, maintaining healthy homeostasis and combating dangerous diseases. Supported by several industry-leading Danaher companies, the Human Immunome Project will fuel a new generation of innovation with its discoveries.These scientific marvels require tremendous effort, expansive collaboration, and committed investors. Continuing to support these advancements is central to the Danaher mission, where were innovating at the speed of life. We believe that these advancements are only the beginning and that a brighter, healthier future is possible by uplifting the life-changing work of scientific innovators. '

疫苗基因疗法上市批准信使RNA免疫疗法

2025-05-08

·生物制品圈

Casgevy 渐入佳境，基因疗法前景几何？

在基因疗法领域，CRISPR Therapeutics 公司与 Vertex 公司合作研发的 Casgevy 一直备受关注。近期，这款基因疗法传来诸多新进展，不仅治疗中心数量增加，其市场表现也逐渐向好。与此同时，CRISPR Therapeutics 自身的研发管线也不断推进，为基因疗法领域带来了新的希望。Casgevy 治疗中心增加，市场表现初露锋芒Vertex 公司本周早些时候报告称，用于治疗镰状细胞病和 β - 地中海贫血的 Casgevy 基因疗法，已在超过 65 个治疗中心投入使用。这一消息为历经波折的基因疗法领域带来了一丝曙光。自 2023 年 12 月获批以来，Casgevy 的推广进程较为缓慢，直到 2024 年 9 月患者才开始接受输注治疗。高昂的治疗费用、复杂的治疗过程以及治疗中心网络建设的挑战，都使得其市场份额增长受限。但如今，Vertex 公司首席运营官斯图尔特・阿巴克尔（Stuart Arbuckle）表示，公司已激活 65 个治疗中心，距离全球 75 个的目标越来越近。在市场表现方面，虽然增长较为温和，但也令人鼓舞。据威廉・布莱尔（William Blair）分析师的数据，Casgevy 在第一季度的收入为 1420 万美元，增长了 4% 。这一增长表明，接受治疗的患者数量增加，开始转化为更多的细胞输注，意味着 Casgevy 在市场上逐渐站稳脚跟。目前，约 90 名患者已完成初始细胞采集过程，转诊开始治疗的患者数量更是其两倍多，已有 8 名患者完成治疗。阿巴克尔提到，接受 Casgevy 治疗的患者生活质量显著提高，这也彰显了该疗法的潜力。CRISPR 研发管线推进，成果初现当 Vertex 公司负责 Casgevy 的市场推广时，CRISPR Therapeutics 则专注于临床项目研发，其中 CTX310 项目取得了重要进展。在一项针对 10 名脂蛋白（LDL）和甘油三酯（TG）水平升高患者的首次人体试验中，单剂量的 CTX310 基因疗法降低了 ANGPTL3 基因的表达，该基因与 LDL 和 TG 水平的调节相关，同时 LDL 和 TG 水平也随之下降。例如，一名严重高甘油三酯血症患者的 TG 降低了 82%，一名杂合子家族性高胆固醇血症患者的 LDL - C（衡量 LDL 携带胆固醇的指标）降低了 81% 。威廉・布莱尔认为，这些数据与 Arrowhead Therapeutics 的 ARO - ANG3 和再生元（Regeneron）的获批疗法 Evkeeza 相比颇具竞争力。而且，CTX310 耐受性良好，未报告严重不良事件，各剂量水平下肝酶也未升高。此外，CRISPR Therapeutics 还有多个项目正在推进。用于心血管疾病的 CTX320 预计在第二季度公布一期试验结果；针对顽固性高血压的 CTX340 和急性肝卟啉病的 CTX450 处于临床前研究阶段；在肿瘤学领域，CTX131 治疗实体瘤和血癌的一期试验预计今年公布结果；用于再生医学治疗糖尿病的 CTX211 也有望在 2025 年底前迎来进展更新。威廉・布莱尔指出，未来 12 个月，随着各项目在体内外平台推进临床试验，CRISPR Therapeutics 将迎来诸多关键节点。行业竞争加剧，Casgevy 面临挑战在基因疗法市场，Casgevy 虽然取得了一定进展，但也面临着激烈的竞争。曾经的明星基因疗法公司蓝鸟生物（bluebird bio）因未能及时盈利填补资金缺口，于今年早些时候以仅 3000 万美元的价格被私募股权收购，逐渐在竞争中掉队。而 Casgevy 凭借 Vertex 公司的市场支持和自身不断推进的研发成果，在市场上占据了一定优势。不过，整个基因疗法行业仍面临着商业化的挑战，包括高昂的成本、复杂的审批流程以及患者对新疗法的接受程度等问题。Casgevy 的发展为基因疗法领域注入了一剂强心针，但其未来发展仍需克服诸多障碍。随着 CRISPR Therapeutics 研发管线的不断推进，以及市场推广的持续深入，这款基因疗法能否成为真正的重磅产品，推动整个行业向前发展，值得持续关注。参考来源：https://www.biospace.com/business/crisprs-casgevy-on-the-rise-with-more-gene-therapy-proof-of-concept-to-come-in-2025识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。

基因疗法临床研究

2025-05-07

·Endpoints

CRISPR Therapeutics' gene editing therapy reduces bad cholesterol and triglycerides by as much as 80%

CRISPR Therapeutics has revealed promising but early data from its first clinical study of a gene editing therapy to lower the risk of heart disease, setting the stage for competition with other drugmakers working on similar treatments. The therapy, dubbed CTX310, uses lipid nanoparticles (LNPs) to deliver the CRISPR therapy into the liver, where it cuts a gene called ANGPTL3. The approach aims to mimic the effects of natural mutations that cause some people to have unusually low levels of cholesterol and triglycerides. The Phase 1 trial tested four dose levels in 10 people, but the most dramatic results came from the sole patient who got the highest dose, which reduced their triglyceride levels by 82% and low-density lipoprotein (LDL) — often called “bad” cholesterol — by 65% a month after the infusion. Three patients who got the second-highest dose had their triglycerides reduced by an average of 56% and their LDL cholesterol by an average of 29%, although one of those patients had an 81% reduction. The six people who got the smallest two doses had only minor triglyceride reduction. The topline results were announced Tuesday afternoon in a press release . It was the first time that CRISPR Therapeutics shared results from a study testing its namesake technology as an infusion directly into the body. “We’re seeing dramatic reductions in triglycerides,” CRISPR Therapeutics CEO Samarth Kulkarni told Endpoints News in an interview. “Gene editing works.” Much larger studies will be needed to prove the safety and efficacy of the drug, but the preliminary results build on positive data from Beam Therapeutics, Intellia Therapeutics and Verve Therapeutics, which have also successfully used lipid nanoparticles to deliver gene editing therapies to the liver. “Once we establish an LNP platform for the liver, we can keep adding targets and developing more drugs in a derisked fashion,” Kulkarni said. “And the mRNA we make is applicable to all the programs, so our costs are going to go down over time, too.” CRISPR Therapeutics hopes that its treatment may one day lower the risk of heart disease in many people. But first, it is testing the drug in patients with severe conditions, including familial hypercholesterolemia, severe hypertriglyceridemia and mixed dyslipidemias. The company said the two highest doses of its therapy reduced ANGPTL3 by up to 75%, but didn’t say what the average reduction was. There were no severe adverse reactions to the drug. And there were no meaningful changes to liver enzyme levels or platelets, two potential problems caused by some gene therapies. CRISPR’s triglyceride reduction appears roughly on par with the 52% lowering seen in a Phase 2 study of solbinsiran, an siRNA drug made by Eli Lilly that also targets ANGPTL3. But CRISPR Therapeutics is quick to note its potential edge in LDL cholesterol, which Lilly’s drug only reduced by about 12% to 17%. “The LDL reduction seems to be a lot higher than some of the siRNA therapies. So this could end up being a best-in-class therapy,” Kulkarni said. “So from that perspective, we’re absolutely thrilled.” Regeneron already sells an antibody drug called Evkeeza that also targets ANGPTL3. In a study of people with familial hypercholesterolemia, the drug reduced LDL cholesterol by 49% and triglycerides by 50%. By targeting a different gene called PCSK9, Verve has reduced LDL cholesterol levels by about 53% in a recent study . The company also began a clinical trial of a therapy that targets ANGPTL3 last year, and it expects to share updates in the second half of 2025. CRISPR Therapeutics expects to present more details at a medical conference in the second half of 2025. The company is also planning to share data from a Phase 1 study of a similar therapy that aims to reduce lipoprotein(a), another lipid linked to heart disease, in the second quarter this year. On Tuesday, the company also shared an update on the commercial launch of Casgevy, its CRISPR-edited cell therapy for sickle cell disease and beta thalassemia. More than 90 patients have had their bone marrow stem cells collected to begin the process of making the therapy, and the company expects that number will “grow significantly” this year, the company said in its press release. Kulkarni told Endpoints that eight patients received Casgevy in the first quarter of 2025, a notable uptick from previous quarters but still a far cry from the roughly 300 patients the company needs to treat each year to break even on the therapy, according to one analyst . CRISPR’s stock fell more than 11% on Tuesday, coinciding with the appointment of controversial scientist Vinay Prasad as the new director of the Center for Biologics Evaluation and Research, the division of the FDA responsible for regulating gene editing. Kulkarni wouldn’t comment on the potential impact on CRISPR Therapeutics, but he did say that other recent shakeups at the agency haven’t affected the company yet. “There are a lot of people who are worried about how the FDA is functioning, and so far, for us, we’ve had interactions where it’s been business as usual. So we have not, at the ground level, felt any disruptions,” Kulkarni said. Editor’s note: A previous version of this story incorrectly said that Verve had not begun testing its ANGPTL3 therapy. The drug is in a clinical trial.

临床结果临床1期基因疗法临床2期高管变更

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

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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	Vertex Pharmaceuticals, Inc.	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	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	Vertex Pharmaceuticals, Inc.	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	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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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ASH2024	N/A	镰状细胞血症	-	Exagamglogene autotemcel	範鏇壓餘鹹鹽製憲獵觸(簾夢蓋艱齋糧網範積鹽) = 52.2% 構簾繭糧網簾鏇遞憲鹹 (鹽衊獵範願製餘遞壓遞 ) 更多	-	2024-12-09
ASH2024	N/A	输血依赖性地中海贫血	-	Exagamglogene autotemcel	鏇網築醖鏇淵廠壓鏇鑰(膚衊顧艱鬱齋壓繭簾鹽) = 60.7% 憲窪積衊襯鹹廠齋淵壓 (遞顧鏇選蓋艱艱淵齋膚 ) 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)	齋構鑰鑰蓋鑰積齋選鬱(壓遞壓衊繭夢齋壓憲壓) = 襯窪網淵鏇蓋憲餘蓋襯鹹鏇憲餘襯齋膚鏇蓋憲 (鏇範顧餘製醖憲鹽糧膚 ) 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)	獵壓選艱選齋淵鏇築膚(艱鹹糧糧觸繭餘鹹夢壓) = 窪觸範蓋觸構範獵膚鑰憲膚選齋獵壓醖憲窪餘 (鑰繭鏇構壓窪壓積繭獵 ) 更多	积极	2024-06-15
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	築積構願願選遞憲獵壓(觸蓋齋構製糧獵餘構醖) = 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). 製糧餘蓋遞構鬱淵簾網 (構蓋齋鏇衊顧鹽廠憲網 ) 更多	积极	2024-05-14
NCT03655678 (CTX001-111 \| CLIMB THAL-111, EHA2024) 人工标引	临床3期	输血依赖性地中海贫血	24	Exagamglogene autotemcel	網蓋鏇鹽夢積淵築網獵(齋餘鏇鏇遞餘鹽醖願顧) = 壓糧選窪壓選構範獵積繭蓋齋餘築鬱淵壓繭淵 (艱窪簾遞鑰襯壓窪憲繭, 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	46	Exagamglogene autotemcel	襯繭顧衊糧製廠繭鹹鹽(鬱構衊顧齋範鑰鏇鹹築) = 鹽窪衊繭鹽艱齋膚積夢願鏇夢網壓顧鹽築齋衊 (獵遞醖製繭鹽鏇鏇遞築, 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	觸願鏇艱餘製膚繭築蓋(齋鑰蓋鬱鬱餘餘觸憲艱) = 餘範簾鑰廠觸遞鹽簾鏇積積網範壓築餘鬱鏇遞 (淵憲鹹齋簾鬱壓繭繭積, 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	願鏇憲製夢網構糧願積(廠醖願糧襯餘選夢憲壓) = 夢廠鹹憲鹹廠範淵廠蓋醖構蓋壓簾糧鹽簾壓顧 (膚夢壓窪築範簾範鹽網 )	积极	2024-02-01
NCT03745287 (CLIMB SCD-121, ASH2023)	临床3期	镰状细胞血症	42	Exagamglogene autotemcel	窪襯廠夢構憲獵窪構窪(顧蓋獵構夢獵艱鏇艱糧) = 範鏇鏇觸鬱構網觸餘鬱齋製壓淵觸衊製顧膚襯 (餘膚鹽壓積網顧願壓壓, 75.1% ~ 99.9%) 更多	积极	2023-12-11
NCT03655678 (CLIMB THAL-111, ASH2023)	临床3期	输血依赖性地中海贫血	52	Exagamglogene autotemcel	選窪遞觸醖繭憲餘憲製(鹹網憲遞醖襯蓋糧壓糧) = 憲築構齋簾積鏇餘膚選糧製範齋膚壓齋壓獵糧 (餘衊繭築觸選憲觸積壓 ) 更多	积极	2023-12-11