Tofersen (BIIB067) is an antisense oligonucleotide that mediates RNase H-dependent degradation of superoxide dismutase 1 (SOD1) mRNA to reduce SOD1 protein synthesis. Tofersen can be used in amyotrophic lateral sclerosis (ALS) research.
| Molecular Weight | 7128.00 |
| CAS Number | 2088232-70-4 |
| Solubility (25°C) | Water ≥ 60 mg/mL |
| Storage | -20°C, dry, sealed |
| 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.
[1] Timothy M Miller, et al. N Engl J Med. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
[2] Johannes Dorst, et al. Curr Opin Neurol. Clinical studies in amyotrophic lateral sclerosis
[4] Daisuke Ito. Ann Neurol. Promise of Nucleic Acid Therapeutics for Amyotrophic Lateral Sclerosis
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