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Tofersen

Cat. No. M21583
Tofersen Structure
Synonym:

BIIB067; ISIS-SOD1Rx

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Biological Activity

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.

Chemical Information
Molecular Weight 7128.00
CAS Number 2088232-70-4
Solubility (25°C) Water ≥ 60 mg/mL
Storage -20°C, dry, sealed
Conversion of different model animals based on BSA (PMID: 27057123)
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.

References

[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

[3] Michael Benatar, et al. Neurotherapeutics. Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study

[4] Daisuke Ito. Ann Neurol. Promise of Nucleic Acid Therapeutics for Amyotrophic Lateral Sclerosis

[5] Timothy Miller, et al. N Engl J Med. Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

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Keywords: Tofersen, BIIB067; ISIS-SOD1Rx supplier, DNA/RNA Synthesis, inhibitors, activators


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