RG7800 是一种 SMN2 拼接修饰剂，有用于治疗脊髓性肌萎缩的研究潜力。

RG7800 has the potential for spinal muscular atrophy treatment. RG7800 is an SMN2 splicing modifier.

RG7800 is shown to promote the inclusion of exon 7 in SMN2 mRNA, generating full-length mRNA in vitro using fibroblasts from an SMA type I patient. RG7800 increases the SMN protein level by induction of alternative splicing of the SMN2 mRNA.

RG7800 displays favorable drug metabolism and pharmacokinetic profile in the rat and in cynomolgus monkey with good oral bioavailability. RG7800 dose-dependently corrects SMN2 splicing by including exon 7 to create FL mRNA, showing that RG7800 corrects alternative splicing of the human SMN2 gene in the brain of transgenic SMA model mice, leading to an increase of the SMN protein in the brain. The treatment of RG7800 displays a clear dose-dependent increase in SMN protein levels in the SMA mouse model. Mice treated with RG7800 demonstrate a dose-dependent increase in survival beginning at the low dose (0.3/1 mg/kg). Approximately 80?90% survive beyond PND50/PND60 with profound body weight gain when the study is terminated, in the middle and high dose groups (1/3 and 3/10 mg/kg, respectively) .

1449598-06-4

C24H28N6O

416.52

RO6885247

DMSO:1.4 mg/mL (3.36 mM),Need ultrasonic and warming
Ethanol:2.5 mg/mL (6.00 mM),Need ultrasonic
H2O:1.25 mg/mL (3.00 mM),ultrasonic and adjust pH to 3 with HCl
Powder: -20°C for 3 years
In solvent: -80°C for 2 years