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RG7800 tetrahydrochloride

Catalog No. T78046
Alias RO6885247 tetrahydrochloride

RG7800 hydrochloride is an orally active SMN2 splicing modulator with EC50 values of 23 nM for splicing and 87 nM for SMN protein production, holding promise as a therapeutic agent for [spinal muscular atrophy].

RG7800 tetrahydrochloride

RG7800 tetrahydrochloride

Catalog No. T78046Alias RO6885247 tetrahydrochloride
RG7800 hydrochloride is an orally active SMN2 splicing modulator with EC50 values of 23 nM for splicing and 87 nM for SMN protein production, holding promise as a therapeutic agent for [spinal muscular atrophy].
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2 mg$425 days
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Product Introduction

Bioactivity
Description
RG7800 hydrochloride is an orally active SMN2 splicing modulator with EC50 values of 23 nM for splicing and 87 nM for SMN protein production, holding promise as a therapeutic agent for [spinal muscular atrophy].
In vitro
RG7800 hydrochloride is an orally active SMN2 splicing modulator with EC50s of 23 nM for SMN2 splicing and 87 nM for SMN protein production. It is the first small molecule SMN2 splicing modifier to advance 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
Storage & Solubility Information
StorageShipping with blue ice.

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