| Size | Price | Stock | Qty |
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| 1mg |
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| 500mg | |||
| 1g | |||
| Other Sizes |
| Targets |
Inotersen targets transthyretin (TTR) mRNA. It is an antisense oligonucleotide that binds to TTR mRNA and causes its degradation through RNase H-mediated cleavage. This results in a reduction of serum TTR protein and TTR protein deposits in tissues. By reducing TTR production, Inotersen reduces the accumulation of amyloid fibrils in patients with hereditary TTR-mediated amyloidosis.
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| ln Vitro |
In vitro, Inotersen is an antisense oligonucleotide that inhibits hepatic production of transthyretin (TTR). It binds to TTR mRNA and causes its degradation. The compound reduces TTR protein levels in cell-based models.
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| ln Vivo |
In vivo, Inotersen is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. It reduces serum TTR protein and TTR protein deposits in tissues. The compound slows the progression of neuropathy and improves quality of life in affected patients.
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| Enzyme Assay |
The in vitro assay for Inotersen involves measuring its ability to reduce TTR mRNA and protein levels. Cells expressing TTR are treated with Inotersen, and TTR mRNA is quantified by RT-qPCR. TTR protein levels are measured by ELISA or Western blotting.
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| Cell Assay |
In vivo, Inotersen is administered subcutaneously. It is an antisense oligonucleotide that is taken up by hepatocytes. The compound reduces serum TTR protein levels in a dose-dependent manner. Clinical studies have demonstrated its efficacy in patients with hereditary TTR-mediated amyloidosis.
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| Animal Protocol |
In vivo animal studies for Inotersen are not detailed in the available sources. The compound is an approved drug for hereditary TTR-mediated amyloidosis. Preclinical studies would have been conducted in animal models of TTR amyloidosis. Clinical trials have been conducted in patients with this condition.
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| ADME/Pharmacokinetics |
Inotersen is administered subcutaneously. As an antisense oligonucleotide, it has a molecular weight of approximately 7,000 g/mol. The compound is taken up by hepatocytes and has a prolonged half-life due to its resistance to nuclease degradation. Specific PK parameters are available in the clinical literature.
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| Toxicity/Toxicokinetics |
Inotersen has a boxed warning for thrombocytopenia and glomerulonephritis. Patients receiving Inotersen require regular monitoring of platelet counts and renal function. Other common side effects include injection site reactions, nausea, and headache.
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| References | |
| Additional Infomation |
Inotersen is an FDA-approved drug for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. It is marketed under the brand name Tegsedi. The compound is a transthyretin-directed antisense oligonucleotide that reduces TTR protein production. It is also available from chemical suppliers for research purposes.
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| Molecular Formula |
C230H318N69O121P19S19
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|---|---|
| Molecular Weight |
7183.11210107803
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| CAS # |
1492984-65-2
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| Related CAS # |
Inotersen sodium;1432726-13-0
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| Appearance |
Typically exists as solid at room temperature
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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 |
| 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) |
May dissolve in DMSO (in most cases), if not, try other solvents such as H2O, Ethanol, or DMF with a minute amount of products to avoid loss of samples
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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.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 0.1392 mL | 0.6961 mL | 1.3922 mL | |
| 5 mM | 0.0278 mL | 0.1392 mL | 0.2784 mL | |
| 10 mM | 0.0139 mL | 0.0696 mL | 0.1392 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
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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