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.

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.

NCT05356195

/ Active, not recruiting临床3期

A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia

This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

开始日期2022-05-03

申办/合作机构

Vertex Pharmaceuticals, Inc.

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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）

2026-12-31·JOURNAL OF MEDICAL ECONOMICS

Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with sickle cell disease with recurrent vaso-occlusive crises in the United States

Article

作者: Xie, Yanwen ; Gargano, Michael ; Lopez, Andrea ; Udeze, Chuka ; Ogunsile, Foluso Joy ; Jeyakumar, Sushanth ; Li, Nanxin ; Yang, Hongbo

OBJECTIVE:

Exagamglogene autotemcel (exa-cel) is a one-time nonviral gene-edited therapy approved in the United States (US) for treatment of patients aged ≥12 years with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs). Standard of care (SOC) for SCD includes symptomatic care, hydroxyurea and/or red blood cell transfusions. This study estimated the long-term clinical outcomes and cost-effectiveness of exa-cel relative to SOC among patients with SCD with recurrent VOCs.

METHODS:

A Markov model was used to compare the expected lifetime costs and clinical outcomes of patients with SCD with recurrent VOCs treated with exa-cel versus SOC from the US payer and societal perspectives. The model structure is based on disease severity, characterized by VOC frequency, which impacts the risk of developing SCD-related complications and mortality. The model incorporated data from the phase 3 pivotal CLIMB SCD-121 trial alongside published literature. Model outcomes included number of VOCs and other acute complications, proportion of patients developing chronic complications, life years (LYs), quality-adjusted LYs (QALYs), costs, and incremental cost-effectiveness ratios (ICERs).

RESULTS:

Over a lifetime horizon, exa-cel was projected to improve survival by 30.8 years (mean age of death, exa-cel: 74.5 vs. SOC: 43.6), reduce the number of VOC events by 77 (7 vs. 84), and reduce undiscounted disease-related costs by $3.34 M ($0.55 M vs. $3.89 M) compared to treatment with SOC. Patients treated with exa-cel also were less likely to experience acute complications or develop chronic complications compared to SOC. The ICER per discounted QALY for exa-cel versus SOC was $16,800 from the payer perspective; exa-cel was dominant (less costly, more effective than SOC) from the societal perspective.

CONCLUSIONS:

Compared to SOC, exa-cel was projected to considerably reduce the number of VOCs, improve survival, and reduce disease-related costs in patients with SCD. Exa-cel was projected to be a cost-effective treatment option.

2026-03-01·TRENDS IN BIOTECHNOLOGY

Third-generation novel technologies for gene editing

Review

作者: Gupta, Pushpendra K ; Kumar, Sourabh

Gene editing technologies have revolutionized the field of biotechnology. CRISPR-Cas methods using RNA-guided enzymes are the most used gene editing tools and have produced gene-edited crops (rice, wheat, corn, etc.) and human therapeutics (Casgevy, approved for commercial use; Vertex Pharmaceuticals). However, these systems have some limitations, including the requirement of a protospacer adjacent motif sequence, generation of undesirable double-strand breaks (DSBs), and the inability to edit long genomic segments. Some of these limitations were partially addressed by the development of second-generation editors, including base editors (BEs) and prime editors (PEs). Third-generation gene editing technologies such as seekRNA and bridgeRNA can overcome most of these limitations and are the subject of this review.

2025-12-23·Blood Advances

Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel

Article

作者: Liu, Tina ; Sharma, Akshay ; Kuo, Kevin H. M. ; Li, Nanxin ; Liem, Robert I. ; Hobbs, William ; Eckrich, Michael J. ; Locatelli, Franco ; Smith, Wally ; Bhatia, Monica ; Grupp, Stephan A. ; Kohli, Puja ; Wall, Donna ; Rubin, Jaime ; Mapara, Markus Y. ; Molinari, Lyndsay ; Dedeken, Laurence ; Frangoul, Haydar ; Imren, Suzan

Abstract:

Severe sickle cell disease (SCD), an inherited blood disorder characterized by recurrent vaso-occlusive crises (VOCs), significantly impairs health-related quality of life (HRQoL). Exagamglogene autotemcel (exa-cel), a one-time, ex vivo CRISPR/Cas9 gene-edited cell therapy, eliminated VOCs in 97% of participants with severe SCD in the phase 3 CLIMB SCD-121 trial. Here, we describe changes in patient-reported outcomes (PROs) in 30 adults and 12 adolescents with ≥16 months follow-up. Adult PROs include EuroQol Quality of Life Scale–5 dimensions–5 levels of severity (EQ-5D-5L), Functional Assessment of Cancer Therapy Bone Marrow Transplant (FACT-BMT), Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me), and 11-point pain Numerical Rating Scale (NRS). In adolescents, EuroQol Quality of Life Scale–5 dimensions–youth (EQ-5D-Y), Pediatric Quality of Life Inventory (PedsQL), and pain NRS were used. Adult EQ-5D-5L US and UK healthy utility index and EQ visual analog scale (VAS) scores, which were lower than general population norm at baseline, substantially improved by month 6 after exa-cel infusion and were sustained through month 36, with similar improvements in FACT-General (FACT-G) total and bone marrow transplant subscale. All FACT-G (physical, social/family, emotional, functional well-being) and ASCQ-Me (emotional, social functioning, stiffness, sleep impact) subscales showed clinically meaningful improvement, including ASCQ-Me pain subscales (impact, episode frequency, severity), with substantial decreases in pain episode frequency by month 6. Mean pain NRS score decreased by month 6 and was sustained. Consistent with adults, adolescents had improvements in mean EQ-5D-Y VAS score, PedsQL score, and pain NRS. Exa-cel led to broad and clinically meaningful HRQoL benefits in adults and adolescents with SCD. The CLIMB SCD-121 and CLIMB-131 trials were registered at www.clinicaltrials.gov as #NCT03745287 and #NCT04208529, respectively.

1,105

项与 Exagamglogene Autotemcel 相关的新闻（医药）

2026-03-26

·Bio药研新境

顶刊医药精读系列之第三篇（基因编辑）：（1）体内递送四层壁垒：从肝靶向到全身治疗的产业突围

原创日更｜顶刊医药精读系列之第三篇（基因编辑）：（1）体内递送四层壁垒：从肝靶向到全身治疗的产业突围 Nature Biotechnology 综述《Targeted delivery of genome editors in vivo》超详细解读。想象一下，CRISPR（全称成簇规律间隔短回文重复序列）递送系统如同运送一台精密仪器。CRISPR 本是细菌抵御病毒的天然防御机制，经改造后成为能精准修正 DNA‘错别字’的‘分子手术刀’。然而现实中，它常被肝脏‘误收’而非送达目标细胞。这不仅大幅降低药效，更可能引发严重副作用。这正是基因编辑当前面临的最大瓶颈：我们已手握高效的‘分子手术刀’，却尚未构建出精准的‘物流系统’——能将这把手术刀安全、准确地投递到每一个需要修复的细胞中。 Doudna 实验室（CRISPR 技术发明人 Jennifer Doudna 的团队）这篇综述，本质上是一份 "分子快递系统" 的设计蓝图。下面我们将用物流快递的视角，带大家拆解这份蓝图。一、百年梦想：从 “一颗砷剂” 到 “四层安检” 的精准进化 1. 历史渊源：不只是科幻 1908 年，德国科学家保罗・埃尔利希提出 “魔法子弹” 概念时，梅毒是令人闻风丧胆的绝症。当时的主流疗法是汞制剂，那种 “杀敌一千，自损八百” 的毒性让患者在治愈前往往先被药物摧毁。埃尔利希怀揣着一个大胆的梦想：能否发明一种像长了眼睛的子弹一样的药物？他做到了。他合成的洒尔佛散（Salvarsan），虽然含有剧毒的砷元素，却成为了人类历史上第一种 “魔法子弹”。它利用细菌与人体细胞在生化结构上的巨大差异（就像在人群中找一个穿着外星制服的入侵者），成功实现了对病原体的 “精准打击”，证明了靶向治疗在理论上是可行的。 2. 为什么今天更难了？然而，一个世纪后的今天，当我们手握能改写生命密码的基因编辑技术（如 CRISPR）时，却发现实现真正的 “魔法子弹” 比埃尔利希时代难了无数倍。・敌人变了：当年对付的是外来细菌，现在要修正的是自身基因。患病细胞和健康细胞的基因组 99.9% 完全相同，要在数万亿个几乎一模一样的细胞中只修改坏掉的那一个，好比要在不惊醒任何人的情况下，给人群中唯一那个只换了不同颜色领带的人悄悄换件衣服。・容错率变了：当年的药物误伤或许只是轻微不适，而基因编辑一旦出错就是永久性修改 DNA，可能导致癌症或遗传给后代。这种不可逆性要求我们的精准度必须接近极致。・战场变了：人体不是一个简单的容器，而是一座拥有 37 万亿细胞的 “超级城市”。这里有复杂的高速公路（血管）、严密的围墙（血脑屏障）、还有时刻警惕的保安部队（免疫系统）。巨大的基因编辑工具（大分子复合物）想要穿过这些防线，比当年的小分子砷剂困难得多。 3. 现代版解决方案：四层 “智能安检系统” 既然无法像当年那样依靠单一的 “化学差异” 一击必中，科学家们便构建了一套更精密的防御体系。最新综述《Targeted delivery of genome editors in vivo》提出了基因编辑递送的 “四层筛选屏障” 模型。这就像是一套层层叠加的机场安检系统，不再指望一道关卡就能百分百安全，而是通过多重验证，让误差率呈指数级下降：第一层：航线选择（给药途径）・任务：决定包裹送到哪个城区。・逻辑：是通过静脉注射直达肝脏？还是视网膜下注射直达眼睛？从物理路径上先排除大部分无关区域。第二层：门牌识别（分子靶向）・任务：决定包裹送到哪栋楼。・逻辑：给药物装上 “导航仪”（如抗体、GalNAc），让它只结合特定细胞表面的受体，确保只有目标细胞（如肝细胞）更易 “开门收货”。第三层：身份验证（表达调控）・任务：决定包裹只有特定住户能签收并使用。・逻辑：即使药物误入了错误的细胞，利用组织特异性启动子或 miRNA 调控元件：通过 "关闭开关" 策略（在脱靶细胞中利用 miRNA 降解编辑酶 mRNA）或 "开启开关" 策略（在靶细胞中解除抑制），控制编辑酶的表达。第四层：任务触发（功能效应 / 活性调控）・任务：决定只有坏掉的机器才被维修。・逻辑：这是最后一道防线。利用靶基因只在病变细胞中表达的特性，或者通过光 / 热 / 小分子诱导激活编辑器，确保在特定条件下，编辑酶活性受控。核心洞察：更精准的靶向递送可能需要多屏障叠加的系统工程。通过 “选对城区→找对楼栋→验明正身→按需启动” 的四重叠加，我们正逐步逼近埃尔利希百年前许下的终极承诺：只治愈疾病，不伤害生命。接下来，让我们逐一拆解这四道精妙绝伦的 “安检关卡”，看看科学家是如何在人体这座超级城市中，完成这场高难度的 “精准快递” 任务的。二、第一层：航线选择 —— 给药途径的 "地理围栏" 既然人体是一座复杂的 “超级城市”，那么如何将基因编辑这个 “精密包裹” 送到目的地？目前的策略主要分为两大流派：静脉注射（全城广播）与局部注射（精准同城配送）。它们各有千秋，也各有软肋。 1. 静脉注射：全城广播，但肝脏是主要清除器官原理：把 CRISPR 装进脂质纳米颗粒（LNP）—— 一种由脂肪分子构成的微小泡泡。这就像给 CRISPR 穿了一件 “脂肪防护衣”，既能保护它不被血液中的酶分解，又能帮它穿过细胞膜。这项技术与新冠 mRNA 疫苗的递送原理完全一致。药物通过静脉输注进入血液，随循环流遍全身。为什么像 “全城广播”？血液流经所有器官，理论上药物能到达任何角落。但现实很骨感：肝脏是体内的 “快递分拣中心”。它的核心功能就是过滤血液中的外来物质。结果，大部分静脉注射 LNP 都会被肝脏截留。成功案例（肝脏疾病的主场）正因为肝脏易 “截胡”，它反而成了治疗肝脏疾病的天然靶点。・NTLA-2001（治疗转甲状腺素蛋白淀粉样变性）：突变基因在肝脏产生 “垃圾蛋白” 沉积全身。该疗法通过静脉注射，28 天内让患者体内异常蛋白水平降低 87%—— 相当于直接撕毁了肝脏里 “生产垃圾的错误图纸”。・婴儿 CPS1 基因治疗：一名 7 月龄婴儿因基因突变无法代谢氨，面临脑损伤风险。两次静脉输注后，相当于给肝脏 “重装了代谢系统”，婴儿恢复健康。・VERVE-101（治疗高胆固醇血症）：靶向肝脏里的 PCSK9 基因，大幅降低 “坏胆固醇”，减少心血管风险。庞大管线：目前还有遗传性血管水肿、α1 - 抗胰蛋白酶缺乏症、乙肝、HIV 等十多种疾病正在利用这一策略进行临床试验。核心局限：对于非肝脏疾病，静脉注射就像往全城发传单却只想让图书馆的人看到 —— 大部分传单被分拣站（肝脏）截留，能到达心脏、肌肉或大脑的寥寥无几。效率极低，且可能让肝脏细胞误收药物引发副作用。 2. 局部注射：精准同城配送，绕过 “中央分拣” 对于眼睛、耳朵、大脑等 “偏远地区”，或者为了避免肝脏的截留，科学家选择了直接送货上门。这绕过了血液循环这个 “中央分拣系统”，实现了宏观上的组织靶向。 A. 眼睛：免疫特权区的 “微创手术” 眼睛是身体的 “瑞士”—— 免疫特权区（免疫系统攻击阈值高），且有严密的血 - 眼屏障。静脉给药很难进入，但这恰恰保护了眼睛免受全身免疫反应的影响。 BRILLIANCE 临床试验（治疗莱伯先天性黑朦）：・操作：视网膜下注射腺相关病毒（AAV，一种安全的 “微型快递车”），直接将药物注入光感受器细胞居住的 “小区” 间隙。・效果：就像把维修工直接送到故障设备旁。14 名患者中 6 人视力改善，且血液中几乎检测不到病毒，说明药物被牢牢锁在眼内，安全性极高。其他前沿探索：・视网膜色素变性：用 AAV8 携带 Cas9 修复基因。・斯塔加特病：用 AAV9 携带碱基编辑器（像 “修字笔” 一样只改单个碱基，不切断 DNA）。・黄斑变性：用 AAV 携带 Cas13（专门编辑 RNA 的工具）。多样化路径：除了视网膜下注射，还有玻璃体内注射（送到 “楼道” 治前层疾病）和前房内注射（送到 “家门口” 治青光眼）。 B. 耳朵：耳蜗内的 “微观杂技” 耳蜗是深处的骨性迷宫，内部的毛细胞一旦受损不可再生。案例：通过超细针头，在显微镜引导下进行耳蜗内注射，将装载微型 dCas13X 编辑器的 AAV9 载体送入，纠正导致听力损失的基因突变。难点：这就像通过钥匙孔给手表换电池，操作空间极小，稍有不慎就会损坏珍贵的听力 “零件”。 C. 大脑：穿越 “铜墙铁壁” 的替代方案血脑屏障（BBB）是大脑的 “防弹玻璃”，阻挡了 99% 的大分子药物。策略：既然穿不过去，就直接 “空投”。案例：通过手术进行脑室内注射，将携带高保真 Cas13Y 的 AAV 载体注入充满脑脊液的脑室。药液像 “水流” 一样随脑脊液扩散至整个脑组织，接触神经元。这相当于把药物倒进 “大脑的蓄水池”，让它自然灌溉需要修复的 “植物”。 D. 其他局部给药：肠道、泌尿与骨髓・口服工程化噬菌体：口服携带 CRISPR 的噬菌体，它在肠道内变身 “智能抗生素”，只杀致病大肠杆菌，不伤有益菌群。就像派 “细菌杀手” 清理 “坏蛋”，不打扰 “好居民”。・尿道内注射（ELIMINATE 试验）：直接注入泌尿系统，精准打击尿路感染细菌，避免抗生素耐药性。・肌内注射：直接注入肌肉治疗杜氏肌营养不良。相当于把 “维修工” 派到肌肉现场，但扩散范围仅限几毫米。・骨髓内注射：通过穿刺注入骨髓腔，靶向造血干细胞（血细胞的 “母细胞”），治疗镰状细胞贫血等血液病。 3. 深度洞察：为什么 “到了组织” 不等于 “到了细胞”？读到这里，读者可能会产生一个疑问：既然药物已经通过注射精准到达了肌肉、骨髓或眼睛，为什么文中还说存在 “无法细胞特异性” 的局限？难道这不就是靶向吗？这里必须厘清两个极易混淆的概念：“组织靶向” 与 “细胞特异性”。组织靶向（宏观层面）上述的局部注射（如肌内注射、视网膜下注射），成功解决了宏观定位问题。它们确保药物主要分布在目标器官内，避免了药物在肝脏等其他器官的浪费。这就像快递员成功把包裹送到了 “肌肉小区” 或 “视网膜大厦”。细胞特异性（微观层面）然而，一个器官内部是一个复杂的 “微型社区”。以肌肉为例，里面不仅住着需要治疗的肌细胞，还混杂着起支撑作用的成纤维细胞、血管里的内皮细胞和巡逻的免疫细胞。问题出在 “最后一米”：当你把药液注射到肌肉组织时，药液会弥漫在细胞间隙中。此时，周围所有类型的细胞都浸泡在药液里。・如果药物载体没有装备专门的 “门牌识别器”（特异性配体），它就会被周围所有具备吞噬能力的细胞（包括不需要治疗的成纤维细胞）随机 “吃掉”。・如果药物进入细胞后，没有 “智能开关”（组织特异性启动子），那么即使在错误的细胞里，基因编辑器也会开始工作，切割 DNA。后果：・疗效稀释：大量药物被非目标细胞（如成纤维细胞）浪费性摄取，导致真正需要治疗的肌细胞药量不足。・安全隐患：在非目标细胞中发生意外的基因编辑（脱靶效应），可能导致细胞功能异常甚至癌变。结论：局部注射只是实现了 “物理上的接近”，却尚未自动解决 “生物学上的精准”。要真正提升效果，必须在 “送对城区” 的基础上，进一步给药物装上 “门牌识别器” 和 “智能开关”。下期预告下一期，我们继续解读这篇文献综述，敬请期待！参考文献提示：本文基于Nature Biotechnology 综述《Targeted delivery of genome editors in vivo》的基因编辑递送机制研究，结合ASGCT、ISSCR及欧洲基因与细胞治疗学会（ESGCT）2024-2025年会议摘要，以及FDA《人类基因治疗产品指导原则》、EMA《基因治疗药物质量管理指南》进行通俗解读。注：本文仅为学术科普，不构成医疗建议。所涉在研基因编辑药物临床进度以企业最新披露为准，已获批或附条件批准产品（如CASGEVY、LYFGENIA等）适应症以说明书及监管机构最新批复为准。基因编辑治疗存在脱靶编辑、免疫原性及长期安全性未知风险，具体治疗方案请咨询具备基因治疗资质的专科医生。原创日更｜关注小编 --获取生物制药行业的前沿动态-- --感谢点赞、关注、转发-- 点击上方关注小编

2026-03-25

·PRNewswire

Complement Therapeutics Announces First Patient Dosed with CTx001 in the Phase I/II Opti-GAIN Study for Geographic Atrophy Secondary to AMD

First patient dosed in Opti-GAIN, a first-in-human Phase I/II clinical trial of CTx001 in Geographic Atrophy secondary to AMD CTx001 is an investigational AAV-based gene therapy designed to deliver mini-CR1, a potent modulator of multiple pathways of the complement system Opti-GAIN is supported by Pre-GAIN, an ongoing natural history study designed to provide short-term GA progression insights and support the development of novel endpoints MUNICH, March 25, 2026 /PRNewswire/ -- Complement Therapeutics GmbH (CTx), a clinical-stage biotechnology company developing next-generation therapeutics for complement-mediated diseases, today announced that the first patient has been dosed in Opti-GAIN, the Company's first-in-human Phase I/II clinical trial of CTx001 in patients with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). Opti-GAIN is a multi-centre Phase I/II study evaluating the safety, tolerability and preliminary efficacy of CTx001 administered via a single subretinal injection. Part 1 is an open-label, dose-escalation study across three dose cohorts, followed by a dose-expansion phase in Part 2. The Opti-GAIN study is being conducted by the company's UK subsidiary. "Geographic Atrophy remains an area of significant unmet need, and there is a clear need to evaluate novel approaches for patients facing progressive vision loss," said Dr Arshad M. Khanani, the trial's Chief Investigator and Director of Clinical Research at Sierra Eye Associates, Reno, Nevada, USA. "I am pleased to be involved in this first-in-human study and to have administered CTx001 to the first patient in Opti-GAIN. I look forward to working with Complement Therapeutics to deepen our understanding of this promising one-time investigational gene therapy approach in patients with GA secondary to AMD." CTx001 is an investigational AAV2-based gene therapy designed to transduce retinal cells with a construct encoding mini-CR1, a truncated and secreted form of Complement Receptor 1 that can modulate both the alternative and classical complement pathways. Subretinal delivery of CTx001 enables local retinal production of mini-CR1, whilst the small size of mini-CR1 may support penetration across Bruch's membrane resulting in broad ocular biodistribution, including the choriocapillaris. Consequently, CTx001 builds on clinically validated complement biology in GA whilst aiming for a potentially best in class profile due to broad ocular biodistribution, strong potency and modulation of multiple complement pathways combined with extended durability. The Opti-GAIN study is being advanced alongside Pre-GAIN, the Company's ongoing natural history study in GA currently enrolling in both the United States and United Kingdom. Together, these studies are intended to support patient selection and the evaluation of novel structural and functional endpoints, including ellipsoid zone (EZ) and focal Optical Coherence Tomography (OCT)-based microperimetry. Opti-GAIN is among the first studies to prospectively evaluate focal OCT-based microperimetry, an approach that has the potential to advance how the treatment effect is assessed in Geographic Atrophy. "Dosing the first patient in Opti-GAIN is an important milestone for Complement Therapeutics and for the advancement of CTx001 in Geographic Atrophy," said Dr Rafiq Hasan, Chief Executive Officer of Complement Therapeutics. "We believe the combination of a differentiated asset and an integrated clinical development strategy sets CTx001 apart. By advancing Opti-GAIN alongside Pre-GAIN, we are generating natural history and interventional data to better inform patient selection, endpoint strategy and future clinical development." Dr Muhammad Ali Memon, Chief Medical Officer of Complement Therapeutics, added: "The precise surgical dosing strategy, adaptive immunomodulatory regimen and well-characterised patient population, from Complement's non-interventional i-GAIN and Pre-GAIN studies, provides a strong platform for CTx001 to demonstrate a therapeutic effect within Opti-GAIN". More information about Complement's clinical studies at ClinicalTrials.gov, including Opti-GAIN (NCT07392255) and Pre-GAIN (NCT07144137). Logo - SOURCE Complement 21% more press release views with Request a Demo

基因疗法临床研究

2026-03-24

·PRNewswire

Anti-Aging Protein Research Takes a Step Forward with Cell Cloning

Issued on behalf of Avaí Bio, Inc. VANCOUVER, BC, March 24, 2026 /PRNewswire/ -- Equity-Insider.com — A protein called α-Klotho circulating in the bloodstream protects the brain, heart, kidneys, and the immune system. Peer-reviewed research has linked higher Klotho levels to reduced risk of Alzheimer's, cardiovascular disease, and certain cancers. Mayo Clinic research connects declining Klotho to arterial stiffness and vascular calcification. The challenge is that the body cuts production of this protein by approximately 50% after the age of 40. The molecule that guards against the deadliest age-related diseases starts to decline just as the risk for these conditions starts to rises. Market projections highlight the scale of the issue: Alzheimer's alone is projected to reach $32.8 billion by 2033, cardiovascular disease remains the leading global cause of death, and kidney disease affects 850 million people worldwide. The global cell therapy market has surpassed $8.2 billion in 2026. The broader cell and gene therapy sector is forecast to surge from $10.4 billion to more than $45 billion by 2035, with more than 40 FDA-approved products now on the market. Regenerative medicine alone is projected to reach $578 billion by 2033. The science of reversing biological decline is no longer theoretical, it is an industry. And the companies building the cellular foundations for these therapies are at the forefront of market attention. Vertex Pharmaceuticals (NASDAQ: VRTX) demonstrated what that looks like at scale. The company's gene-edited cell therapy Casgevy — developed with CRISPR Therapeutics (NASDAQ: CRSP) — became the world's first approved CRISPR-based treatment, now available for sickle cell disease and transfusion-dependent beta-thalassemia. Vertex expects to file regulatory submissions for the 5–11 age group in the first half of 2026. CRISPR Therapeutics, meanwhile, reported Phase 1 data showing its in vivo gene-editing therapy CTX310 achieved mean reductions of 73% in ANGPTL3, 55% in triglycerides, and 49% in LDL cholesterol after a single intravenous infusion — a one-dose cardiovascular intervention that could reshape metabolic disease treatment. Altimmune (NASDAQ: ALT) is advancing pemvidutide, a GLP-1/glucagon dual receptor agonist for metabolic disease that targets the $65 billion metabolic syndrome opportunity through hormonal regulation of fat metabolism. Arrowhead Pharmaceuticals (NASDAQ: ARWR) dosed the first patients in a Phase 1/2a trial of ARO-DIMER-PA, the first clinical candidate designed to silence two genes simultaneously for atherosclerotic cardiovascular disease. These are platform-level interventions pulling institutional capital into longevity and regenerative medicine at unprecedented speed. But before any cell therapy can reach patients, it needs a starting point. And that's the step one this company just completed. Avaí Bio (OTCQB: AVAI) recently announced a critical early-stage milestone alongside joint venture partner Austrianova: beginning the creation of a Master Cell Bank (MCB) of genetically modified cells that overexpress the α-Klotho protein. An MCB is the process of taking a single genetically engineered cell and cloning it into tens of millions of identical copies, creating a standardized, GMP-compliant bank of cellular starting material. It's the essential foundation from which all future working cell banks and therapy development will proceed — the step that ensures consistency, quality, and scalability before any therapeutic product can be developed. "We are excited to enter the first step in the production phase of α-Klotho producing cells as part of our commitment to deliver safe, effective treatments for aging associated diseases," said Chris Winter, CEO of Avaí Bio. The cells banked in the MCB will be used in conjunction with Austrianova's proprietary Cell-in-a-Box® encapsulation technology, which protects therapeutic cells inside a biocompatible shell to allow continuous protein secretion without triggering immune rejection. The technology is backed by over 50 peer-reviewed publications and decades of development. Avaí Bio's dual-program approach targets both the Klothonova anti-aging platform and the Insulinova diabetes program, each leveraging this same encapsulation technology. The company participated in the 15th European Pancreas and Islet Transplantation Association Symposium in January 2026, where Dr. Eva Maria Lilli Brandtner evaluated advanced cells for potential application in diabetes therapy. The protein that protects against the most serious age-related diseases is steadily disappearing from the bloodstream. The science to potentially restore it exists. And the company that just created the standardized cellular foundation to advance that science — Avaí Bio (OTCQB: AVAI) — is doing so in a market where every platform capable of developing cell-based therapeutics is being re-evaluated. For more information on Avaí Bio (OTCQB: AVAI) and its Klothonova and Insulinova programs, visit Equity-Insider.com Read this and more news for Avaí Bio at: Equity-Insider.com Article Source: CONTACT: EQUITY INSIDER [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. Equity Insider is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Avaí Bio, Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares of Avaí Bio, Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Avaí Bio, Inc. which were purchased in the open market. MIQ reserves the right to buy and sell, and will buy and sell shares of Avaí Bio, Inc. at any time thereafter without any further notice. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material disseminated by MIQ has been approved by the above mentioned company; this is a paid advertisement, and we own shares of the mentioned company that we will sell, and we also reserve the right to buy shares of the company in the open market, or through other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment. SOURCES: Straits Research, Global Cell Therapy Market 2026 — Precedence Research, Cell and Gene Therapy Market Forecast — Grand View Research, CAR T-Cell Therapy Market — Astute Analytica, Regenerative Medicine Market 2025–2033 — Logo: 21% more press release views with Request a Demo

临床1期细胞疗法基因疗法临床结果

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
β地中海贫血	临床3期	美国	Vertex Pharmaceuticals, Inc.	2018-09-07
β地中海贫血	临床3期	加拿大	Vertex Pharmaceuticals, Inc.	2018-09-07
β地中海贫血	临床3期	德国	Vertex Pharmaceuticals, Inc.	2018-09-07
β地中海贫血	临床3期	英国	Vertex Pharmaceuticals, Inc.	2018-09-07
输血依赖性地中海贫血	临床3期	美国	Vertex Pharmaceuticals, Inc.	2018-09-07
输血依赖性地中海贫血	临床3期	加拿大	Vertex Pharmaceuticals, Inc.	2018-09-07
输血依赖性地中海贫血	临床3期	德国	Vertex Pharmaceuticals, Inc.	2018-09-07
输血依赖性地中海贫血	临床3期	英国	Vertex Pharmaceuticals, Inc.	2018-09-07

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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


NCT03655678 (CLIMB-111, Tandem Meetings ASTCT and CIBMTR2026)	临床3期	输血依赖性地中海贫血 \| 镰状细胞血症	102	Exagamglogene autotemcel (Transfusion-dependent β-thalassemia)	餘築廠願艱鏇鏇積願簾(鑰艱膚齋艱鹹遞蓋網簾) = There were no malignancies through follow-up in CLIMB-131. 鹹鑰膚糧顧窪鬱蓋築製 (鹹鹽糧窪積構鑰願餘夢 )	积极	2026-02-04
				Exagamglogene autotemcel (Sickle Cell Disease)
NCT03655678 (CLIMB THAL-111, Tandem Meetings ASTCT and CIBMTR2026)	临床3期	输血依赖性地中海贫血 \| 镰状细胞血症 CRISPR-Cas9 gene edited	102	Exagamglogene autotemcel + Exagamglogene autotemcel (Transfusion-dependent β-thalassemia)	齋鹽獵蓋觸獵構鏇夢製(壓襯願糧憲糧糧窪鏇齋) = 獵鹽鬱遞鏇鏇襯鹽鹹觸廠鑰糧遞糧觸鬱範糧窪 (艱糧廠醖觸構淵衊鑰鬱 ) 更多	积极	2026-02-04
				Exagamglogene autotemcel + Exagamglogene autotemcel (Sickle Cell Disease)	齋鹽獵蓋觸獵構鏇夢製(壓襯願糧憲糧糧窪鏇齋) = 壓蓋簾顧鹽築齋遞鑰鹽廠鑰糧遞糧觸鬱範糧窪 (艱糧廠醖觸構淵衊鑰鬱 ) 更多
NCT03745287 \| NCT04208529 (CLIMB-131, ASH2025 \| Pubmed) 人工标引	临床3期	镰状细胞血症	45	Exagamglogene autotemcel (CLIMB SCD‑121)	鬱衊積鏇鹽網艱餘網鑰(膚齋遞網製艱廠淵蓋鹽) = 簾鹽遞衊鹹獵鬱鏇襯築淵襯觸壓積淵醖淵淵繭 (簾網遞築範選鏇鹹夢顧, 78.8 ~ 97.5) 更多	积极	2025-12-06
				Exagamglogene autotemcel (CLIMB SCD‑121 + CLIMB-131 combined (LTFU))	鬱衊積鏇鹽網艱餘網鑰(膚齋遞網製艱廠淵蓋鹽) = 膚蓋餘觸壓簾網製簾憲淵襯觸壓積淵醖淵淵繭 (簾網遞築範選鏇鹹夢顧 ) 更多
NCT05356195 \| NCT05329649 (CLIMB SCD-151, ASH2025 \| Company_Website) 人工标引	临床3期	输血依赖性地中海贫血 \| 镰状细胞血症	23	Exagamglogene autotemcel (transfusion-dependent β-thalassemia (CLIMB THAL-141))	簾製鏇憲襯築遞衊簾襯(餘齋構醖簾構衊製醖襯) = 窪蓋觸築蓋衊壓衊鏇製夢鏇壓鹽淵獵鹽醖壓壓 (夢壓餘糧獵獵齋願餘壓 ) 更多	积极	2025-12-06
				Exagamglogene autotemcel (sickle cell disease (CLIMB SCD-151))	艱繭積顧膚鏇鏇齋夢襯(製簾衊壓構醖膚繭製糧) = 夢願鹹蓋夢衊範蓋積襯獵壓積壓鹽願膚鬱襯鏇 (製積淵鏇願鏇衊餘膚觸 ) 更多
NCT03655678 (CLIMB-111, ASH2025)	临床3期	输血依赖性地中海贫血	56	Exagamglogene autotemcel	餘淵簾壓鑰糧壓齋遞壓(範網淵淵窪簾夢積選願) = 壓憲築構獵鹹糧衊顧簾鹽膚糧簾遞簾顧壓艱鹹 (衊蓋構蓋廠蓋窪蓋積壓 ) 更多	积极	2025-12-06
CLIMB SCD-121 (EHA2025)	临床3期	血管闭塞危机	58	Exagamglogene autotemcel	壓積觸膚範膚鹽憲築壓(膚窪顧簾膚願觸顧廠廠) = 蓋鬱壓夢淵齋網鑰餘壓蓋襯鬱遞繭獵襯鏇簾夢 (壓觸餘廠窪簾膚製觸鑰 ) 更多	积极	2025-05-14
CLIMB THAL-111 and CLIMB-131 (EHA2025)	临床3期	输血依赖性地中海贫血	56	Exagamglogene autotemcel	選鹹艱製觸製餘願壓獵(製選觸餘鏇鬱廠淵範鹽) = 齋鹽選鏇廠築醖襯鏇膚糧衊繭製餘鏇獵鑰糧鏇 (獵襯選顧鏇襯觸鹽繭獵 ) 更多	积极	2025-05-14
ASH2024	N/A	镰状细胞血症	-	Exagamglogene autotemcel	壓鏇觸製簾艱構顧築鹹(構廠鬱遞遞遞膚廠艱壓) = 52.2% 淵顧艱淵築範襯鏇憲遞 (壓糧鹽製獵鏇衊鏇憲鹽 ) 更多	-	2024-12-09
ASH2024	N/A	输血依赖性地中海贫血	-	Exagamglogene autotemcel	網積選範鬱艱網憲網鹽(願繭網觸範淵壓製繭壓) = 60.7% 觸鏇壓醖淵鬱壓願繭壓 (糧鑰鏇構齋觸衊鏇繭鑰 ) 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)	願壓顧選顧淵繭鑰夢構(獵膚願淵齋範鏇築簾艱) = 範鏇壓鑰淵鏇積糧衊廠淵鹹觸糧醖築餘選範觸 (鏇製窪選願鹹餘膚繭蓋 ) 更多	积极	2024-06-15
				Casgevy (TDT)	艱鹹齋廠選醖範鏇範鏇(窪築選蓋鹹窪簾壓壓範) = 鏇鹽壓醖製醖壓窪糧蓋壓膚窪鏇夢築顧築鏇積 (壓糧獵簾廠夢淵醖鬱遞 ) 更多