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|Cat. No. :||CS-0044495|
|CAS No. :|
|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) 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. 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.
Protocol: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.Mice
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.
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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