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

RG7800 

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  
5 mg USD 80 In-stock
Stock in the United States
Estimated Time of Arrival: December 31
10 mg USD 110 In-stock
Stock in Sweden
Estimated Time of Arrival: December 31
50 mg USD 280 In-stock
Stock in Sweden
Estimated Time of Arrival: December 31
100 mg USD 450 In-stock
Stock in Sweden
Estimated Time of Arrival: December 31
200 mg   Get quote  
500 mg   Get quote  

* Please select Quantity before adding items.

Customer Review

Other Forms of RG7800:

  • Biological Activity

  • Protocol

  • Technical Information

  • Purity & Documentation

  • References

Description

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].

Solvent & Solubility
In Vitro: 

DMSO : 1.4 mg/mL (3.36 mM; Need ultrasonic and warming)

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
10 mM 0.2401 mL 1.2004 mL 2.4008 mL
*Please refer to the solubility information to select the appropriate solvent.
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
Molecular Weight

416.52

Formula

C₂₄H₂₈N₆O

CAS No.

1449598-06-4

SMILES

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

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

Room temperature in continental US; may vary elsewhere

Purity: 99.86%

  • Molarity Calculator

  • Dilution Calculator

The molarity calculator equation

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

Mass   Concentration   Volume   Molecular Weight *
= × ×

The dilution calculator equation

Concentration (start) × Volume (start) = Concentration (final) × Volume (final)

This equation is commonly abbreviated as: C1V1 = C2V2

Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
× = ×
C1   V1   C2   V2

Inquiry Online

Your information is safe with us. * Required Fields.

Product name

 

Salutation

Applicant name *

 

Email address *

Phone number *

 

Organization name *

Country or Region *

 

Requested quantity *

Remarks

Bulk Inquiry

Inquiry Information

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

RG7800

Cat. No.: HY-101792