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


Cat. No.: HY-101792 Purity: 99.86%
Handling Instructions

RG7800 a small molecule SMN2 splicing modifier to enter human clinical trials to treat spinal muscular atrophy.

For research use only. We do not sell to patients.
RG7800 Chemical Structure

RG7800 Chemical Structure

CAS No. : 1449598-06-4

Size Price Stock Quantity
Free Sample (0.5-1 mg)   Apply now  
10 mM * 1 mL in DMSO USD 132 In-stock
5 mg USD 120 In-stock
10 mg USD 180 In-stock
50 mg USD 540 In-stock
100 mg USD 900 In-stock
200 mg   Get quote  
500 mg   Get quote  

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Customer Review

  • Biological Activity

  • Protocol

  • Technical Information

  • Purity & Documentation

  • References


RG7800 a small molecule SMN2 splicing modifier to enter human clinical trials to treat spinal muscular atrophy.

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].

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].

Preparing Stock Solutions
Concentration Volume 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
10 mM 0.2401 mL 1.2004 mL 2.4008 mL
Please refer to the solubility information to select the appropriate solvent.
Animal Administration

RG7800 is prepared in DMSO.

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.

Molecular Weight








Powder -20°C 3 years
  4°C 2 years
In solvent -80°C 6 months
  -20°C 1 month

Room temperature in continental US; may vary elsewhere

Solvent & Solubility

10 mM in DMSO

* "<1 mg/mL" means slightly soluble or insoluble. "≥" means soluble, but saturation unknown.

Purity: 99.86%

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