RG7800 is a small molecule SMN2 splicing modifier. RG7800 has the potential to be used in spinal muscular atrophy. RG7800 corrects selective splicing of the human SMN2 gene in the brain of a transgenic SMA model mouse, leading to an increase in SMN protein in the brain.
| Molecular Weight | 416.52 |
| Formula | C24H28N6O |
| CAS Number | 1449598-06-4 |
| Solubility (25°C) | DMSO：1.4 mg/mL |
| Storage | 4-8°C |
| Species | Mouse | Rat | Rabbit | Guinea pig | Hamster | Dog |
| Weight (kg) | 0.02 | 0.15 | 1.8 | 0.4 | 0.08 | 10 |
| Body Surface Area (m2) | 0.007 | 0.025 | 0.15 | 0.05 | 0.02 | 0.5 |
| Km factor | 3 | 6 | 12 | 8 | 5 | 20 |
| Animal A (mg/kg) = Animal B (mg/kg) multiplied by | Animal B Km |
| Animal A Km |
For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.
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