| Size | Price | Stock | Qty |
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| 1mg |
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| 5mg |
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| 10mg |
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| Other Sizes |
| Targets |
The target is FUS mRNA, specifically the sixth intron of the FUS transcript. Ulefnersen binds by Watson–Crick pairing and recruits RNase H to cleave the FUS transcript, thereby reducing FUS protein synthesis in the brain and spinal cord. Lowering FUS protein is proposed to mitigate toxic gain-of-function mechanisms and motor neuron degeneration.
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| ln Vitro |
In patient-derived cells and disease-relevant models, ulefnersen reduces FUS mRNA and protein levels in a dose-dependent manner, decreases cytoplasmic FUS mislocalization, and attenuates stress granule–related toxicity. In ALS-FUS mouse models, postnatal administration reduces FUS protein in brain and spinal cord and delays motor neuron degeneration.
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| ln Vivo |
In ALS-FUS mouse models, central nervous system administration of ulefnersen lowers FUS protein in target tissues and slows motor function decline. Early-phase clinical studies in FUS-ALS patients have evaluated intrathecal dosing, with biomarker and safety readouts supporting target engagement and the rationale for further development.
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| Enzyme Assay |
Binding and silencing are assessed in vitro using synthetic FUS transcript fragments and recombinant RNase H; cleavage products are resolved by gel electrophoresis or capillary electrophoresis, and the concentration dependence of cleavage is used to confirm sequence-specific antisense activity.
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| Cell Assay |
Cultured patient fibroblasts or induced motor neurons are transfected with ulefnersen using lipid or electroporation; FUS mRNA is quantified by RT-qPCR, FUS protein by immunoblotting or immunofluorescence, and stress granule formation or neuronal survival is assessed after 48–72 h.
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| Animal Protocol |
ALS-FUS model mice receive ulefnersen by intracerebroventricular or intrathecal injection; brain and spinal cord tissues are collected at defined intervals, and FUS mRNA, FUS protein, and motor neuron counts are measured, with plasma and cerebrospinal fluid drug levels monitored to relate exposure to pharmacodynamic effect.
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| References |
| Molecular Formula |
C230H321N68O126P19S13
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|---|---|
| Molecular Weight |
7059.72
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| CAS # |
2589926-25-8
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| Related CAS # |
Ulefnersen sodium; 2589926-27-0
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| Sequence |
DNA, d([2′-O-(2-methoxyethyl)]rG-sp-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-G-sp-T-sp-m5C-sp-A-sp-m5C-sp-m5C-sp-T-sp-T-sp-T-sp-m5C-sp-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rA-sp-[2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]m5rC)
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| Appearance |
White to off-white solid powder
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| SMILES |
[Ulefnersen]
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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) |
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 | 0.1416 mL | 0.7082 mL | 1.4165 mL | |
| 5 mM | 0.0283 mL | 0.1416 mL | 0.2833 mL | |
| 10 mM | 0.0142 mL | 0.0708 mL | 0.1416 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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