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Synonyms: BT-200 | BT200
Compound class:
Nucleic acid
Comment: Rondaptivon pegol (BT200) belongs to the aptamer class of nucleic acid drugs/ligands. It binds to the A1 (platelet, heparin and collagen binding) domain of von Willebrand factor (vWF) [3]. Blocking interaction with receptors on platelets reduces platelet-mediated thrombogenesis [2]. Aptamer binding also prolongs the plasma half-life of factor VIII (FVIII) , which is mediated by interaction with the vWF D'/D3 domain, so offers potential in the treatment of FVIII deficiency hemophilia (hemophilia A) [1].
We have been unable to locate a full chemical SMILES for the ligand, or its HELM notation. |
| References |
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1. Ay C, Kovacevic KD, Kraemmer D, Schoergenhofer C, Gelbenegger G, Firbas C, Quehenberger P, Jilma-Stohlawetz P, Gilbert JC, Zhu S et al.. (2023)
The von Willebrand factor-binding aptamer rondaptivon pegol as a treatment for severe and nonsevere hemophilia A. Blood, 141 (10): 1147-1158. [PMID:36108308] |
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2. Kovacevic KD, Greisenegger S, Langer A, Gelbenegger G, Buchtele N, Pabinger I, Petroczi K, Zhu S, Gilbert JC, Jilma B. (2021)
The aptamer BT200 blocks von Willebrand factor and platelet function in blood of stroke patients. Sci Rep, 11 (1): 3092. [PMID:33542410] |
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3. Zhu S, Gilbert JC, Hatala P, Harvey W, Liang Z, Gao S, Kang D, Jilma B. (2020)
The development and characterization of a long acting anti-thrombotic von Willebrand factor (VWF) aptamer. J Thromb Haemost, 18 (5): 1113-1123. [PMID:32011054] |
