Egaptivon pegol(Egaptivon pegol) - 药物靶点：vWF_专利_临床

概要

基本信息

药物类型

DNA aptamer

别名

Egaptivon pegol (USAN)

靶点

vWF

作用机制

vWF抑制剂(血管性血友病因子抑制剂)

治疗领域

免疫系统疾病神经系统疾病心血管疾病+ [3]

在研适应症-

非在研适应症

脑梗塞颈动脉狭窄颅内栓塞+ [3]

原研机构

Archemix Corp.

在研机构-

非在研机构

Archemix Corp.

最高研发阶段终止临床2期

首次获批日期-

最高研发阶段(中国)-

特殊审评-

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结构

使用我们的RNA技术数据为新药研发加速。

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序列信息

Sequence Code 271898487

来源: *****

关联

11

项与 Egaptivon pegol 相关的临床试验

EUCTR2008-008532-82-AT / Not yet recruitingN/AIIT

A Study of the Pharmacodynamics, Pharmacokinetics, and Safety & Efficacy of ARC1779 Injection in Patients with von Willebrand Disease Type 2B - ARC1779 for VWD-2B

开始日期2009-02-19

申办/合作机构-

NCT00742612 / Terminated临床2期

A Study of the Effect of ARC1779 Injection on Cerebral Microembolism in Patients Undergoing Carotid Endarterectomy

The purpose of this study is to determine, in patients undergoing carotid endarterectomy, the effect of ARC1779 Injection on the number of microembolic signals detected by transcranial Doppler immediately after surgery. This study will also evaluate the safety of ARC1779 Injection with respect to bleeding risk in patients in the peri-operative (during surgery) period.

开始日期2009-02-01

申办/合作机构

Archemix Corp.

[+1]

EUCTR2008-001872-54-IT / Not yet recruitingN/A

A Randomized, Double-blind, Placebocontrolled, Clinical Outcome Study of ARC1779 Injection in Patients with Thrombotic Microangiopathy - ND

开始日期2009-01-28

申办/合作机构

Archemix Corp.

100 项与 Egaptivon pegol 相关的临床结果

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100 项与 Egaptivon pegol 相关的转化医学

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100 项与 Egaptivon pegol 相关的专利（医药）

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23

项与 Egaptivon pegol 相关的文献（医药）

2024-07-01·Seminars in Thrombosis and Hemostasis

Progress in von Willebrand Disease Treatment: Evolution towards Newer Therapies

Review

作者: Jilma, Bernd ; Moser, Miriam M. ; Schoergenhofer, Christian

Abstractvon Willebrand disease (VWD) is a very heterogenous disease, resulting in different phenotypes and different degrees of bleeding severity. Established therapies (i.e., desmopressin, antifibrinolytic agents, hormone therapy for heavy menstrual bleeding, and von Willebrand factor [VWF] concentrates) may work in some subtypes, but not in all patients. In recent years, progress has been made in improving the diagnosis of VWD subtypes, allowing for more specific therapy. The impact of VWD on women's daily lives has also come to the fore in recent years, with hormone therapy, tranexamic acid, or recombinant VWF as treatment options. New treatment approaches, including the replacement of lacking factor VIII (FVIII) function, may work in those subgroups affected by severe FVIII deficiency. Reducing the clearance of VWF is an alternative treatment pathway; for example, rondaptivon pegol is a VWFA1 domain-binding aptamer which not only improves plasma VWF/FVIII levels, but also corrects platelet counts in thrombocytopenic type 2B VWD patients. These approaches are currently in clinical development, which will be the focus of this review. In addition, half-life extension methods are also important for the improvement of patients' quality of life. Targeting specific mutations may further lead to personalized treatments in the future. Finally, a few randomized controlled trials, although relatively small, have been published in recent years, aiming to achieve a higher level of evidence in future guidelines.

2022-01-01·[Rinsho ketsueki] The Japanese journal of clinical hematology

[Development of a novel aptamer blocking the interaction between the VWF A1 domain and platelet GP Ib for the treatment of arterial thrombosis].

Article

作者: Harada, Kaori ; Matsumoto, Masanori

A DNA aptamer to the von Willebrand factor (VWF) A1 domain, TAGX-0004, inhibits the binding of VWF to platelets. Nucleic acid aptamers are single-stranded DNA or RNA molecule forming three-dimensional structures that are capable of specifically binding to proteins and are expected to contribute to alternative medicine. ARC1779, an aptamer targeting the VWF A1 domain, had been evaluated in a phase II clinical trial of patients with acquired thrombotic thrombocytopenic purpura (aTTP); however, its development was terminated. Caplacizumab, an anti-VWF A1 domain nanobody, is now increasingly employed as first-line therapy for the treatment of aTTP in Western countries. However, there have been reports regarding adverse bleeding events and the high cost of the treatment. In this study, the inhibitory effects of TAGX-0004 were compared with those of ARC1779 and caplacizumab on in vitro platelet aggregation and thrombus formation. TAGX-0004 had an excellent high affinity to the VWF A1 domain and superior efficacy, such as its potent inhibitory activity in in vitro platelet aggregation and thrombus formation. Therefore, it can potentially overcome the problems associated with caplacizumab and can be developed as a promising drug not only for aTTP treatment but also for the treatment of the various VWF-mediated thrombotic disorders.

2021-02-01·Biochemistry (Moscow)4区 · 生物学

The Use of Fluorescently Labeled ARC1779 Aptamer for Assessing the Effect of H2O2 on von Willebrand Factor Exocytosis

4区 · 生物学

Article

作者: Goncharov, Nikolay V ; Tsitrina, Aleksandra A ; Avdonin, Pavel V ; Avdonin, Piotr P ; Trufanov, Sergey K ; Rybakova, Elena Yu

AbstractHere, we propose a new approach for quantitative estimation of von Willebrand factor (vWF) exposed on the surface of endothelial cells (ECs) using the ARC1779 aptamer that interacts with the vWF A1 domain. To visualize complex formation between vWF and the aptamer, the latter was conjugated with the Cy5 fluorescent label. Cultured human umbilical vein endothelial cells (HUVEC) were stained with the ARC1779-Cy5 conjugate and imaged with a fluorescence microscope. The images were analyzed with the CellProfiler software. vWF released from the Weibel–Palade bodies was observed as bright dot-like structures of round and irregular shape, the number of which increased several times after HUVEC exposure to histamine or thrombin. Staining with ARC1779-Cy5 also revealed long filamentous vWF structures on the surface of activated HUVEC. vWF secretion by ECs is activated by the second messengers cAMP and Ca2+. There is evidence that hydrogen peroxide also acts as a second messenger in ECs. In addition, exogenous H2O2formed in leukocytes can enter ECs. The aim of our study was to determine the effect of H2O2on the vWF exposure at the surface of HUVEC using the proposed method. It was shown that hydrogen peroxide at concentration 100 µM, which is lower than the cytotoxicity threshold of H2O2for cultured HUVEC, increased several times the number of dot-like structures and total amount of vWF exposed on plasma membrane of HUVEC, which suggest that H2O2acts as a mediator that activates exocytosis of Weibel–Palade bodies and vWF secretion in the vascular endothelium during inflammation and upon elevated generation of endogenous reactive oxygen species in ECs.

100 项与 Egaptivon pegol 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D09892	-	-	DB05202

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
脑梗塞	临床2期	美国	Archemix Corp.	2009-02-01
脑梗塞	临床2期	英国	Archemix Corp.	2009-02-01
颈动脉狭窄	临床2期	英国	Archemix Corp.	2009-02-01
颈动脉狭窄	临床2期	美国	Archemix Corp.	2009-02-01
颅内栓塞	临床2期	美国	Archemix Corp.	2009-02-01
颅内栓塞	临床2期	英国	Archemix Corp.	2009-02-01
血栓性血小板减少性紫癜	临床2期	奥地利	Archemix Corp.	2008-01-01
2型血管性血友病	临床2期	奥地利	Archemix Corp.	2008-01-01
2型血管性血友病	临床2期	奥地利	Archemix Corp.	2008-01-01
血栓形成	临床2期	美国	Archemix Corp.	2006-12-01

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临床结果

适应症

分期

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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


NCT00742612 (Pubmed \| Cochrane Database Syst Rev)	临床1期	-	-	ARC1779	淵壓選衊繭膚衊襯築壓(鬱觸艱淵選願觸醖鏇築) = 繭遞簾衊鑰繭選積鏇範鹹齋觸獵鬱廠築簾構窪 (範積夢簾觸艱構願襯顧 )	-	2011-08-01
NCT00742612 (Pubmed \| Cochrane Database Syst Rev)	临床1期	-	-	Placebo	淵壓選衊繭膚衊襯築壓(鬱觸艱淵選願觸醖鏇築) = 壓鬱壓遞艱觸範壓鑰餘鹹齋觸獵鬱廠築簾構窪 (範積夢簾觸艱構願襯顧 )	-	2011-08-01
NCT00432770 (Pubmed \| J Am Coll Cardiol)	临床1期	-	47	ARC1779	獵餘積夢窪顧廠鹹夢窪(窪糧蓋餘積膚壓選築鑰) = 蓋選鬱鑰膚鹽壓築選醖憲鹽艱齋簾鏇膚鏇夢壓 (憲淵醖願獵觸淵糧範艱 ) 更多	-	2007-12-04