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1mg | ||
5mg | ||
10mg | ||
Other Sizes |
ln Vitro |
Tofersen targets the SOD1 mRNA by binding to it through Watson-Crick base pairing with antisense oligonucleotides (ASOs). In order to degrade the targeted RNA, tofersen activates RNase H1[2].
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References |
[1]. Miller T, et, al. Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2020 Jul 9;383(2):109-119.
[2]. McCampbell A, et, al. Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models. J Clin Invest. 2018 Aug 1;128(8):3558-3567. |
Molecular Weight |
7128.00
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CAS # |
2088232-70-4
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Appearance |
Powder
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Synonyms |
BIIB067; BIIB 067; Qalsody; BIIB-067; ISIS SOD1Rx; ISIS-SOD1Rx; ISIS SOD1Rx; Tofersen
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HS Tariff Code |
2934.99.9001
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Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment (e.g. under nitrogen), avoid exposure to moisture. |
Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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Solubility (In Vitro) |
H2O : ~100 mg/mL (~14.0 mM)
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Solubility (In Vivo) |
Solubility in Formulation 1: 100 mg/mL (14.03 mM) in PBS (add these co-solvents sequentially from left to right, and one by one), clear solution; with sonication.
 (Please use freshly prepared in vivo formulations for optimal results.) |
Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
1 mM | 0.1403 mL | 0.7015 mL | 1.4029 mL | |
5 mM | 0.0281 mL | 0.1403 mL | 0.2806 mL | |
10 mM | 0.0140 mL | 0.0701 mL | 0.1403 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration: mg/mL;
Method for preparing DMSO stock solution: mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation::Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O,mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
NCT Number | Recruitment | interventions | Conditions | Sponsor/Collaborators | Start Date | Phases |
NCT03070119 | Active Recruiting |
Drug: Tofersen | ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation |
Biogen | March 8, 2017 | Phase 3 |
NCT02623699 | Completed | Drug: Tofersen Drug: Placebo |
Amyotrophic Lateral Sclerosis | Biogen | January 20, 2016 | Phase 3 |
NCT03764488 | Completed | Drug: 99mTc-MAG3-BIIB067 Drug: Tofersen |
Healthy Volunteers | Biogen | December 20, 2018 | Phase 1 |