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| 1mg |
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| Targets |
Remlarsen is a miRNA class ligand that targets the microRNA-29b (miR-29b) pathway. As a synthetic mimic, it binds to its natural target mRNA sequences, thereby "agonist" or restoring the activity of miR-29b. miR-29b is a known master regulator of fibrosis, as it represses the expression of multiple extracellular matrix (ECM) genes, including collagens (COL1A1, COL1A2, COL3A1). By replenishing miR-29b activity, Remlarsen counteracts the fibrotic process.
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| ln Vitro |
Remlarsen (MRG-201) has the ability to control the expression of PD biomarkers in the skin[1].
In vitro, Remlarsen is active in regulating pharmacodynamic (PD) biomarker expression in skin cells. The miR-29b mimic represses the expression of multiple extracellular matrix (ECM) genes and fibroplasia. By inhibiting the production of collagen and other ECM components, it directly reduces the fibrotic activity of fibroblasts, which are the primary effector cells responsible for scar formation. |
| ln Vivo |
In vivo, Remlarsen has demonstrated the potential to prevent the formation of fibrotic scars or cutaneous fibrosis. The compound is active in regulating PD biomarker expression in the skin, indicating successful target engagement and biological effect. Intradermal injection of Remlarsen into skin leads to a reduction in collagen and other ECM gene expression, providing proof-of-concept for its use as an anti-fibrotic agent for dermal conditions.
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| Enzyme Assay |
For a cell-free system, the binding of Remlarsen to its target mRNAs is not typically measured in a direct enzyme assay. Instead, its activity is assessed using a luciferase reporter assay. A vector containing the 3'-UTR of a target gene (e.g., a collagen gene) with the miR-29b binding site, fused to a luciferase reporter gene, is transfected into cells. A decrease in luciferase signal upon treatment with Remlarsen indicates functional binding and activity of the miRNA mimic.
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| Cell Assay |
In cellular assays, primary human dermal fibroblasts are cultured and treated with Remlarsen (e.g., via transfection) for 24-72 hours. After treatment, total RNA is extracted, and the expression of fibrosis-related genes such as COL1A1, COL3A1, and other ECM genes is measured by quantitative real-time PCR (qRT-PCR) to confirm target engagement and efficacy. Protein levels of collagen are measured by Western blotting or ELISA.
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| Animal Protocol |
Remlarsen is typically administered via intradermal injection in animal models, such as mice, to evaluate its anti-fibrotic efficacy. For example, in a mouse model of cutaneous fibrosis, the compound is injected into the skin either prophylactically or therapeutically. At the end of the study, the skin tissue is harvested, and the levels of collagen and other ECM components are measured by histology (e.g., Masson's Trichrome staining) and biochemical assays to assess the extent of fibrosis.
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| ADME/Pharmacokinetics |
Pharmacokinetic (PK) data for Remlarsen is typical for a synthetic oligonucleotide. As a nucleic acid, its bioavailability is low when administered systemically, and it is primarily designed for local administration (e.g., intradermal) to the target organ. It is metabolized by ubiquitous nucleases and is rapidly cleared from circulation, with a half-life that is short unless chemically modified. Its distribution is primarily to tissues.
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| Toxicity/Toxicokinetics |
The literature provided does not include detailed toxicology data. However, as a miR-29b mimic, its safety profile is an important aspect of its preclinical and clinical development. The primary concerns for oligonucleotide therapeutics include potential off-target effects (binding to unintended mRNA sequences) and immune stimulation (via toll-like receptors). Extensive preclinical toxicology studies are required to assess these risks.
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| References |
[1]. Corrie L Gallant-Behm, et al. A MicroRNA-29 Mimic (Remlarsen) Represses Extracellular Matrix Expression and Fibroplasia in the Skin. J Invest Dermatol. 2019 May;139(5):1073-1081.
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| Additional Infomation |
Remlarsen is one of the first miRNA mimics to enter clinical development for an anti-fibrotic indication. It has completed a Phase 1 clinical trial (NCT02603224) in healthy volunteers to assess its safety, tolerability, and pharmacokinetics. The compound represents a novel class of therapeutics targeting the epigenetic regulation of fibrosis. The research highlights the potential of using miRNA mimics as a platform technology for treating diseases characterized by excessive scarring. The active compound is a double-stranded RNA (AS: 7961.8; SS: 8097.7) with a molecular weight of 16059.5. Remlarsen is a microRNA-29b mimic, acts a miR-29b agonist, and has been tested in Phase I clinical trials for cutaneous fibrosis. Remlarsen is not yet approved for any clinical use. A Phase I study is completed, but further development is ongoing or suspended. It has been assigned the DrugBank ID DB19131.
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| Molecular Weight |
16059.5 (AS: 7961.8; SS: 8097.7)
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| CAS # |
1848266-71-6
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| Appearance |
White to off-white 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: 100 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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