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RG7800 tetrahydrochloride

RG7800 tetrahydrochloride
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RG7800 tetrahydrochloride

Catalog No. T78046
RG7800 hydrochloride is an orally active modulator of SMN2 splicing, exhibiting EC50 values of 23 nM for SMN2 splicing and 87 nM for SMN protein production. This compound holds promise as a therapeutic agent for spinal muscular atrophy.
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Pack SizePriceAvailabilityQuantity
2 mg$425 days
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Product Introduction

Bioactivity
Description
RG7800 hydrochloride is an orally active modulator of SMN2 splicing, exhibiting EC50 values of 23 nM for SMN2 splicing and 87 nM for SMN protein production. This compound holds promise as a therapeutic agent for spinal muscular atrophy.
In vitro
RG7800 hydrochloride is an orally active SMN2 splicing modulator that exhibits half-maximal effective concentrations (EC50s) of 23 nM for SMN2 splicing and 87 nM for SMN protein production. It holds the distinction of being the inaugural small molecule SMN2 splicing modifier to progress into human clinical trials [1].
In vivo
RG7800 exhibits a favorable metabolism and pharmacokinetic (DMPK) profile in both rats and cynomolgus monkeys, paired with good oral bioavailability. Administered at doses of 1, 3, and 10 mg/kg orally, RG7800 dose-dependently increases SMN protein levels in both the central nervous system and peripheral tissues of Δ7 mice [1].
AliasRO6885247 tetrahydrochloride
Chemical Properties
Molecular Weight562.36
FormulaC24H32Cl4N6O
Cas No.
Storage & Solubility Information
StoragePowder: -20°C for 3 years | In solvent: -80°C for 1 year

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