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RG7800

Catalog No. T12718L CAS 1449598-06-4

Synonyms: RO6885247

RG7800 (RO6885247) has the potential for spinal muscular atrophy treatment. RG7800 is an SMN2 splicing modifier.

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RG7800, CAS 1449598-06-4

Pack Size	Availability	Price/USD
2 mg	In stock	$ 39.00
5 mg	In stock	$ 64.00
10 mg	In stock	$ 88.00
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Purity: 99.9%

Biological Description

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Description	RG7800 (RO6885247) has the potential for spinal muscular atrophy treatment. RG7800 is an SMN2 splicing modifier.
In vitro	RG7800 has been demonstrated to facilitate the inclusion of exon 7 in SMN2 mRNA, thereby producing full-length mRNA in vitro from fibroblasts sourced from a patient with SMA type I. This compound enhances the level of SMN protein through the induction of alternative splicing in SMN2 mRNA.
In vivo	RG7800 displays favorable drug metabolism and pharmacokinetic profile in the rat and in cynomolgus monkey with good oral bioavailability. RG7800 dose-dependently corrects SMN2 splicing by including exon 7 to create FL mRNA, showing 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. The treatment of RG7800 displays a clear dose-dependent increase in SMN protein levels in the SMA mouse model. Mice treated with RG7800 demonstrate a dose-dependent increase in survival beginning at the low dose (0.3/1 mg/kg). Approximately 80?90% survive beyond PND50/PND60 with profound body weight gain when the study is terminated, in the middle and high dose groups (1/3 and 3/10 mg/kg, respectively) .

Synonyms	RO6885247
Molecular Weight	416.52
Formula	C24H28N6O
CAS No.	1449598-06-4

Storage

Powder: -20°C for 3 years | In solvent: -80°C for 1 year

Solubility Information

DMSO: 1.4 mg/mL (3.36 mM), Sonication and heating are recommended.

H2O: 1.25 mg/mL (3.00 mM), when pH is adjusted to 3 with HCl. Sonication is recommended.

Ethanol: 2.5 mg/mL (6.00 mM), Sonication is recommended.

References and Literature

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.

Related compound libraries

This product is contained In the following compound libraries：

Anti-Cancer Drug Library Drug Repurposing Compound Library Anti-Cancer Clinical Compound Library DNA Damage & Repair Compound Library Bioactive Compound Library NO PAINS Compound Library Pediatric Drug Library Clinical Compound Library Bioactive Compounds Library Max Anti-Cancer Compound Library

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Keywords

RG7800 1449598-06-4 Cell Cycle/Checkpoint DNA Damage/DNA Repair Others DNA/RNA Synthesis RO-6885247 Inhibitor RO 6885247 inhibit RO6885247 RG-7800 RG 7800 inhibitor

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