| Size | Price | Stock | Qty |
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| 1mg |
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| Other Sizes |
| Targets |
BT200 targets the A1 domain of von Willebrand Factor (VWF), a large multimeric glycoprotein that plays a central role in hemostasis by mediating platelet adhesion and aggregation at sites of vascular injury [3L22-L23]. By binding to the A1 domain, BT200 blocks the interaction between VWF and the platelet receptor glycoprotein Ibalpha (GPIb) [14L2-L4]. This prevents the formation of the primary hemostatic plug and arterial thrombosis. BT200 also has a second, novel mechanism: it decreases the clearance of VWF and its carrier protein factor VIII (FVIII) from the bloodstream, thereby increasing their plasma levels [3L22-L24]. This dual mechanism is unique.
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| ln Vitro |
In vitro, BT200 has been shown to be a potent inhibitor of VWF-GPIb binding [14L2-L4]. This is typically assessed using surface plasmon resonance (SPR) or ELISA-based binding assays. The aptamer binds to the VWF A1 domain with high affinity, effectively blocking the interaction between VWF and the GPIb receptor. It is a high-purity, pegylated nucleic acid used in assay protocols to study thrombosis.
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| ln Vivo |
In vivo, BT200 has demonstrated efficacy in preventing arterial thrombosis in preclinical models [14L2-L4]. In a healthy volunteer clinical trial, it was shown to increase plasma levels of VWF/FVIII [3L17-L18]. Furthermore, in a study on type 2B von Willebrand disease, rondoraptivon pegol (BT200) increased platelet counts and VWF/FVIII levels, indicating a therapeutic effect beyond simply inhibiting platelet aggregation [3L20-L24]. This in vivo activity supports its research in hemophilia and VWD.
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| Enzyme Assay |
BT200's binding to the VWF A1 domain is typically assessed using non-cellular techniques such as Surface Plasmon Resonance (SPR). In an SPR assay, the VWF A1 domain or full-length VWF is immobilized on a sensor chip. A solution containing varying concentrations of BT200 is flowed over the chip. The association and dissociation rates are monitored in real-time to calculate the equilibrium dissociation constant (KD). Alternatively, an ELISA-based competition assay can be used.
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| Cell Assay |
For cell-based assays, BT200's activity is assessed by its ability to inhibit VWF-mediated platelet aggregation. Platelet-rich plasma (PRP) is isolated from whole blood. The PRP is pre-incubated with BT200, then aggregation is induced by adding ristocetin (which promotes VWF-GPIb binding) or botrocetin (which specifically activates VWF). The degree of platelet aggregation is measured by light transmission aggregometry (LTA). BT200's effect on platelet aggregation is quantified and a dose-response curve is generated.
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| Animal Protocol |
In vivo animal studies are performed to evaluate the anti-thrombotic efficacy of BT200. A commonly used model is the ferric chloride-induced arterial thrombosis model in mice. In this model, the carotid artery is injured by applying a patch saturated with FeCl3. BT200 is administered intravenously prior to injury. The time to occlusion (the time it takes for a stable thrombus to completely block the artery) is measured by Doppler flow probe. BT200-treated animals typically show a significantly prolonged time to occlusion or complete prevention of occlusion compared to vehicle controls.
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| ADME/Pharmacokinetics |
Pharmacokinetic (PK) data for BT200 in humans and healthy volunteers is available [3L17-L18]. As a pegylated aptamer, it has a longer half-life than its non-pegylated parent, BT100, due to reduced renal clearance. The PEGylation reduces nuclease degradation, increases the hydrodynamic radius, and decreases immunogenicity, resulting in a significantly extended circulation time. Its primary route of elimination is renal and metabolic.
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| Toxicity/Toxicokinetics |
Comprehensive toxicological data for BT200 are not detailed in standard product literature, but it has been evaluated in human trials [3L17-L18]. In a Phase 1 trial in healthy volunteers, it was reported to be safe and well-tolerated. In clinical trials for hemophilia A and type 2B VWD, BT200 also showed a favorable safety profile. As a research-use compound, standard safety precautions for handling aptamers should be followed.
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| References | |
| Additional Infomation |
BT200 (rondaptivon pegol, rondoraptivon pegol) is a pegylated nucleic acid aptamer, derived from BT100, that functions as a VWF inhibitor [14L11-L13]. It is a high-purity (≥98%) compound provided as an off-white to light yellow solid powder [14L2][14L16]. It is water-soluble (≥50 mg/mL) [14L22]. The powder should be stored at -20degC for up to 3 years or at 4degC for 2 years [14L17]. The compound is for research use only and not for human therapy.
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| Appearance |
Off-white to light yellow solid powder
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment, avoid exposure to moisture. |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
H2O :≥ 50 mg/mL
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| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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