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Cat. No.: HY-101792

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RG7800

(Synonyms: RO6885247)

5 Cited Publications

Customer Review

Based on 5 publication(s) in Google Scholar

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.

Purity: 99.88%
CAS No.: 1449598-06-4
Formula: C₂₄H₂₈N₆O
Molecular Weight:416.52

Storage:

Powder -20°C, 3 years , 4°C, 2 years ; In solvent -80°C, 2 years , -20°C, 1 year

RG7800 Chemical Structure

CAS No.: 1449598-06-4

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Top Publications Citing

Use of MCE Products

Fluorescent Dye

Strong Fluorescence Signal / Multi-Disciplinary Applications / High Fluorescence Specificity

Biological Activity
- Description
- Cellular Effect
- In Vitro
- In Vivo
- Clinical Trial
Chemical Information
Publications (5)
Solvent & Solubility
- In Vitro
Protocol
Purity & Documentation
References
Help & FAQs
Classifications
Bioz

Biological Activity

Description

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

All DNA/RNA Synthesis Isoforms :

Cellular Effect

Cell Line	Type	Value	Description	References
NIH3T3	IC₅₀	>10 μM Compound: 2; RG7800	Dark toxicity in mouse 3T3 cells assessed as decrease in cell viability by neutral red uptake assay Dark toxicity in mouse 3T3 cells assessed as decrease in cell viability by neutral red uptake assay	[PMID: 30044619]
NIH3T3	IC₅₀	330 nM Compound: 2; RG7800	Phototoxicity in mouse 3T3 cells assessed as decrease in cell viability in presence of UV light by neutral red uptake assay Phototoxicity in mouse 3T3 cells assessed as decrease in cell viability in presence of UV light by neutral red uptake assay	[PMID: 30044619]

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

NCT Number	Sponsor	Condition	Start Date	Phase A549
NCT01329991	Plexxikon\|		2011-05	PHASE1

Chemical Information

CAS No. 1449598-06-4
Appearance Solid
Molecular Weight 416.52
Formula C₂₄H₂₈N₆O
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
Synonyms

RO6885247
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

Publications (5)

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Journal Impact Factor

Nature

Regulated control of gene therapies by drug-induced splicing. [Abstract]

2021 Aug;596(7871):291-295. PMID: 34321659
J Mol Med (Berl)

Combination of PARP inhibitor and temozolomide to suppress chordoma progression. [Abstract]

2019 Aug;97(8):1183-1193. PMID: 31201471
Life Sci Alliance

Drug screening with human SMN2 reporter identifies SMN protein stabilizers to correct SMA pathology. [Abstract]

2019 Mar 25;2(2):e201800268. PMID: 30910806
Patent

US20230340498A1.
bioRxiv

2020 Feb.

Solvent & Solubility

In Vitro:

1 M 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)

View the Complete Stock Solution Preparation Table

Preparing Stock Solutions

View the Complete Stock Solution Preparation Table

Concentration Solvent Mass

1 mg

5 mg

10 mg

Concentration

Solvent

Mass

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. 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.

Molarity Calculator

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

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Dilution Calculator

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Protocol

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].

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

Purity & Documentation

Select Batch:

Purity: 99.98%

Assay: 99.98%

ee.: 99.98%

Data Sheet (273 KB)
SDS (583 KB)
- English - EN (583 KB)
- Français - FR (583 KB)
- Deutsch - DE (583 KB)
- Norwegian - NO (583 KB)
- Español - ES (583 KB)
- Swedish - SV (583 KB)
- Italian - IT (583 KB)
- Korean - KR (583 KB)
- Portuguese - PT (583 KB)
COA (2659 KB)
Elemental Analysis Report (2659 KB)
HNMR (2659 KB)
CNMR (2659 KB)
RP-HPLC (2659 KB)
NP-HPLC (2659 KB)
MS (2659 KB)
LCMS (2659 KB)
IR (2659 KB)
GC (2659 KB)
OR (2659 KB)
SDS-PAGE (2659 KB)
SEC-HPLC (2659 KB)
Other Report (2659 KB)
Handling Instructions (2659 KB)

References

[1]. Ratni H, et al. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy. J Med Chem. 2016 Jul 14;59(13):6086-100. [Content Brief]

Help & FAQs

How should lyophilized recombinant proteins be reconstituted and stored?

1. Before opening the cap, centrifuge the vial at 13000 rpm for 20-30 seconds. This step will ensure that any lyophilized powder that may have adhered to the cap or walls is collected at the bottom of the vial, minimizing the risk of product loss.

Complete Stock Solution Preparation Table

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 / 1 M HCl	1 mM	2.4008 mL	12.0042 mL	24.0085 mL	60.0211 mL
Ethanol / 1 M HCl	5 mM	0.4802 mL	2.4008 mL	4.8017 mL	12.0042 mL
1 M 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

Keywords

100 mg

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Biological Activity
- Description
- In Vitro
- In Vivo
- Clinical Trial
Chemical Information
Publications (5)
Solvent & Solubility
- In Vitro
Protocol
Purity & Documentation
References

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RG7800

Cell Cycle/DNA Damage Compound Library

Clinical Compound Library

Anti-Aging Compound Library

Orally Active Compound Library

Targeted Diversity Library

Heterocyclic Compound Library

Bioactive Compound Library Max

MCE Bioactive Compound Library

Clinical Compound Library Plus

Bioactive Compound Library

High-Throughput Bioactive Compound Library

Publications Citing Use of MedChemExpress (MCE) RG7800

All DNA/RNA Synthesis Isoforms

Biological Activity

Chemical Information

Publications (5)

Solvent & Solubility

Protocol

Purity & Documentation

References

Help & FAQs

Complete Stock Solution Preparation Table

RG7800

Cell Cycle/DNA Damage Compound Library

Clinical Compound Library

Anti-Aging Compound Library

Orally Active Compound Library

Targeted Diversity Library

Heterocyclic Compound Library

Bioactive Compound Library Max

MCE Bioactive Compound Library

Clinical Compound Library Plus

Bioactive Compound Library

High-Throughput Bioactive Compound Library

Publications Citing Use of MedChemExpress (MCE) RG7800

All DNA/RNA Synthesis Isoforms

Biological Activity

Chemical Information

Publications (5)

Solvent & Solubility

Protocol

Purity & Documentation

References

Help & FAQs

Complete Stock Solution Preparation Table

RG7800 Related Classifications

HY-101792 Related Classifications