| Size | Price | Stock | Qty |
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| 5mg |
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| 10mg |
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| 50mg |
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| Other Sizes |
| Targets |
EC1.5x: 23 nM (SMN2 splicing), 87 nM (SMN protein)[1]
RG7800 targets the SMN2 gene splicing machinery. As an SMN2 splicing modulator, it increases the inclusion of exon 7 in the SMN2 transcript, leading to increased production of full-length, functional SMN protein. The tetrahydrochloride salt is the standard pharmaceutical form. The EC1.5x values are 23 nM for SMN2 splicing and 87 nM for SMN protein production. |
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| ln Vitro |
In human clinical trials, RG7800 hydrochloride is the first small chemical SMN2 splicing modifier. It is an oral active SMN2 splicing modulator with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein[1].
In vitro, RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein, respectively. It increases exon 7 inclusion in SMN2 pre-mRNA, resulting in elevated levels of functional SMN protein. This activity has been demonstrated in cell-based assays using fibroblasts from SMA patients. |
| ln Vivo |
In both rats and cynomolgus monkeys, RG7800 exhibits a favorable agent metabolism and pharmacokinetic (DMPK) profile with good oral bioavailability. RG7800 (1, 3, 10 mg/kg, po) increases the level of SMN protein in the brain and peripheral tissue of Δ7 mice in a dose-dependent manner[1].
In vivo, RG7800 is orally bioavailable and has been shown to increase SMN protein levels in animal models of SMA. It has been evaluated in clinical trials as a potential disease-modifying therapy for spinal muscular atrophy. The compound is the first small molecule SMN2 splicing modifier to enter human clinical trials. |
| Enzyme Assay |
A cell-free assay for RG7800 is not typical; its activity is assessed in cell-based splicing assays. To assess binding to the SMN2 pre-mRNA, an RNA binding assay using a fluorescently labeled SMN2 exon 7-intron 7 RNA probe and purified splicing factors (e.g., hnRNPs) can be performed. The compound is incubated with the RNA-protein complex, and the shift in fluorescence polarization or mobility is measured. However, standard functional assays are cell-based.
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| Cell Assay |
SMN2-luciferase reporter cells (e.g., HEK293 cells stably transfected with an SMN2 exon 7-luciferase minigene) are seeded in 96-well plates. Cells are treated with RG7800 tetrahydrochloride at varying concentrations (0.1-1000 nM) for 24-48 h. Luciferase activity is measured. Alternatively, SMA patient fibroblasts are treated with the compound, and SMN protein levels are quantified by ELISA or Western blot. The EC1.5x for SMN protein induction is 87 nM.
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| Animal Protocol |
RG7800 tetrahydrochloride has been studied in mouse models of SMA. A standard protocol: neonatal SMNdelta7 mice (a severe SMA model) are treated with RG7800 (1-30 mg/kg) via oral gavage starting from postnatal day 2 (P2) to P21. Body weight, survival, and motor function (e.g., righting reflex, grip strength) are assessed. SMN protein levels in spinal cord and muscle tissues are measured by Western blot and immunofluorescence. The compound is expected to increase SMN protein and improve survival.
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| ADME/Pharmacokinetics |
RG7800 tetrahydrochloride is an orally active small molecule with a molecular weight of 514.01 (free base). The tetrahydrochloride salt form enhances solubility. The compound exhibits high oral bioavailability. Specific PK parameters (t½, Cmax, AUC, F) have been reported in clinical trials: RG7800 is rapidly absorbed with Tmax of 1-4 hours and t½ of 10-20 hours. The compound is metabolized by CYP450 enzymes.
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| Toxicity/Toxicokinetics |
In clinical trials, RG7800 was generally well-tolerated. The most common adverse effects were mild to moderate, including headache, nausea, and fatigue. However, one clinical trial was halted due to retinal findings in long-term animal studies (non-human primates). Formal toxicology studies have shown effects on the retina at high doses. The compound should be handled with caution.
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| References | |
| Additional Infomation |
RG7800 tetrahydrochloride is a research-grade compound and has been investigated in clinical trials for SMA. It is the first small molecule SMN2 splicing modifier to enter human clinical trials. However, as of 2026, it has not received regulatory approval (clinical development has been discontinued or repurposed). This product is for research use only.
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| Molecular Formula |
C24H32CL4N6O
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|---|---|
| Molecular Weight |
562.36
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| Related CAS # |
RG7800;1449598-06-4
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| Appearance |
Light yellow to 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 :≥ 113.5 mg/mL (~201.83 mM)
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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 | 1.7782 mL | 8.8911 mL | 17.7822 mL | |
| 5 mM | 0.3556 mL | 1.7782 mL | 3.5564 mL | |
| 10 mM | 0.1778 mL | 0.8891 mL | 1.7782 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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