RG7800 hydrochloride is an orally activeSMN2splicing modulator, withEC_1.5xs of 23 nM and 87 nM forSMN2splicing andSMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.

EC1.5x: 23 nM (SMN2splicing), 87 nM (SMN protein)^[1]

RG7800 hydrochloride is an orally activeSMN2splicing modulator, with EC_1.5xs of 23 nM and 87 nM forSMN2splicing and SMN protein; and is the first small moleculeSMN2splicing modifier to enter human clinical trials^[1].

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].

Solid

C₂₄H₂₈N₆O.xHCl

Room temperature in continental US; may vary elsewhere.

4°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

H₂O : ≥ 113.5 mg/mL

*"≥" means soluble, but saturation unknown.