RG7800 hydrochloride

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5mg In-stock USD 80.0
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  • Data Sheet

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Name: RG7800 hydrochloride; RO6885247 hydrochloride
Cat. No. : CS-0044495
CAS No. :    
Formula: C24H28N6O.xHCl
M. Wt. :
Solubility: H2O: ≥ 113.5 mg/mL


RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy. IC50 & Target: EC1.5x: 23 nM (SMN2 splicing), 87 nM (SMN protein)[1] In Vitro: RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; and is the first small molecule SMN2 splicing modifier to enter human clinical trials[1]. In Vivo: RG7800 is a favorable agent metabolism and pharmacokinetic (DMPK) profile in the rat and in cynomolgus monkey with good oral bioavailability. RG7800 (1, 3, 10 mg/kg, p.o.) dose-dependently elevates the SMN protein level in the brain and in peripheral tissue of Δ7 mice[1].


Animal Administration: RG7800 is dissloved in DMSO for i.p. administration. RG7800 is formulated in 0.5% HPMC and 0.1% Tween 80 for oral administration[1].[1]Mice[1]
Homozygous Δ7 mice are dosed ip with RG7800 or vehicle (100% DMSO, 2.5 mL/kg) once per day from PND3 through postnatal day 3 (PND23), and the dosing regimen is switched on PND24 to a 3-fold higher oral dose once daily in 0.5% HPMC and 0.1% Tween 80. Litters are randomized across groups. Body weight and survival are assessed daily. Survival analysis is done using GraphPad Prism (log-rank test), and a p <0.05 is considered as significant[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.

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