1. Cell Cycle/DNA Damage
  2. DNA/RNA Synthesis
  3. RG7800

RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.

For research use only. We do not sell to patients.

RG7800 Chemical Structure

RG7800 Chemical Structure

CAS No. : 1449598-06-4

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5 mg USD 80 In-stock
10 mg USD 110 In-stock
50 mg USD 280 In-stock
100 mg USD 450 In-stock
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Customer Review

Based on 5 publication(s) in Google Scholar

Other Forms of RG7800:

Top Publications Citing Use of Products
  • Biological Activity

  • Protocol

  • Purity & Documentation

  • References

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Description

RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.

In Vitro

RG7800 increases the SMN protein level via induction of alternative splicing of the SMN2 mRNA. RG7800 is shown to promote the inclusion of exon 7 in SMN2 mRNA, generating full-length mRNA in vitro using fibroblasts from an SMA type I patient[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

In Vivo

RG7800 shows favorable drug metabolism and pharmacokinetic profile in the rat and in cynomolgus monkey with good oral bioavailability. In SMA mouse model, treatment of RG7800 shows a clear dose dependent increase in SMN protein levels. Mice treated with RG7800 demonstrate a dose dependent increase in survival beginning at the low dose (0.3/1 mg/kg). In the middle and high dose groups (1/3 and 3/10 mg/kg, respectively), approximately 80−90% survive beyond PND50/PND60 with profound body weight gain when the study is terminated. RG7800 dose-dependently corrects SMN2 splicing by including exon 7 to create FL mRNA, suggesting that RG7800 corrects alternative splicing of the human SMN2 gene in the brain of transgenic SMA model mice, leading to an increase of the SMN protein in the brain[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Clinical Trial
Molecular Weight

416.52

Formula

C24H28N6O

CAS No.
Appearance

Solid

Color

Off-white to yellow

SMILES

O=C1C=C(C2=NN3C(C(CC)=NC(C)=C3)=C2)N=C4N1C=C(C5CCN(C)CC5)C=C4C

Shipping

Room temperature in continental US; may vary elsewhere.

Storage
Powder -20°C 3 years
4°C 2 years
In solvent -80°C 2 years
-20°C 1 year
Solvent & Solubility
In Vitro: 

1M HCl : 100 mg/mL (240.08 mM; Need ultrasonic)

Ethanol : 2.5 mg/mL (6.00 mM; Need ultrasonic)

H2O : 1.25 mg/mL (3.00 mM; ultrasonic and adjust pH to 3 with HCl)

H2O : < 0.1 mg/mL (insoluble)

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 2.4008 mL 12.0042 mL 24.0085 mL
5 mM 0.4802 mL 2.4008 mL 4.8017 mL
View the Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 2 years; -20°C, 1 year. When stored at -80°C, please use it within 2 years. When stored at -20°C, please use it within 1 year.

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Working solution concentration: mg/mL
The concentration of the stock solution you require exceeds the measured solubility. The following solution is for reference only.If necessary, please contact MedChemExpress (MCE).
Purity & Documentation

Purity: 99.86%

References
Animal Administration
[1]

Mice: Compounds (RG7800) are administered orally once daily (qd) for 10 days at three different doses (1, 3, and 10 mg/kg). One hour after the final dose, tissues are collected from the mice, and the level of the SMN protein is determined in the brain and quadriceps muscle[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

References

Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 2 years; -20°C, 1 year. When stored at -80°C, please use it within 2 years. When stored at -20°C, please use it within 1 year.

Optional Solvent Concentration Solvent Mass 1 mg 5 mg 10 mg 25 mg
H2O / Ethanol / 1M HCl 1 mM 2.4008 mL 12.0042 mL 24.0085 mL 60.0211 mL
Ethanol / 1M HCl 5 mM 0.4802 mL 2.4008 mL 4.8017 mL 12.0042 mL
1M HCl 10 mM 0.2401 mL 1.2004 mL 2.4008 mL 6.0021 mL
15 mM 0.1601 mL 0.8003 mL 1.6006 mL 4.0014 mL
20 mM 0.1200 mL 0.6002 mL 1.2004 mL 3.0011 mL
25 mM 0.0960 mL 0.4802 mL 0.9603 mL 2.4008 mL
30 mM 0.0800 mL 0.4001 mL 0.8003 mL 2.0007 mL
40 mM 0.0600 mL 0.3001 mL 0.6002 mL 1.5005 mL
50 mM 0.0480 mL 0.2401 mL 0.4802 mL 1.2004 mL
60 mM 0.0400 mL 0.2001 mL 0.4001 mL 1.0004 mL
80 mM 0.0300 mL 0.1501 mL 0.3001 mL 0.7503 mL
100 mM 0.0240 mL 0.1200 mL 0.2401 mL 0.6002 mL
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RG7800 Related Classifications

Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

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Product Name:
RG7800
Cat. No.:
HY-101792
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