WARNING: This product is for research use only, not for human or veterinary use.
Hodoodo CAT#: H333101
CAS#: 2088232-70-4
Description: Tofersen, also known as BIIB067; ISIS-SOD1Rx, is an antisense oligonucleotide that mediates RNase H-dependent degradation of superoxide dismutase 1 (SOD1) mRNA to reduce the synthesis of SOD1 protein. Tofersen was approved for treatment of amyotrophic lateral sclerosis (ALS).
Hodoodo Cat#: H333101
Name: Tofersen
CAS#: 2088232-70-4
Chemical Formula:
Exact Mass: 0.00
Molecular Weight: 0.000
Elemental Analysis:
Synonym: BIIB067; BIIB-067; BIIB 067; ISIS-SOD1Rx; ISIS SOD1Rx; ISIS SOD1Rx; Tofersen; Nucleic Acid Sequence Length: 20: 6 a, 5 c, 3 g, 5 t, 1 u;
IUPAC/Chemical Name: DNA, d([2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-sp-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]rA-sp-T-sp-A-sp-m5C-sp-A-sp-T-sp-T-sp-T-sp-m5C-sp-T-sp-A-sp-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-sp-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]m5rU)
InChi Key:
InChi Code:
SMILES Code:
Appearance: To be determined
Purity: >98% (or refer to the Certificate of Analysis)
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.
Storage Condition: Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).
Solubility: To be determined
Shelf Life: >2 years if stored properly
Drug Formulation: To be determined
Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).
HS Tariff Code: 2934.99.9001
More Info:
| Biological target: | |
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The following data is based on the product molecular weight 0.00 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.
| Concentration / Solvent Volume / Mass | 1 mg | 5 mg | 10 mg |
|---|---|---|---|
| 1 mM | 1.15 mL | 5.76 mL | 11.51 mL |
| 5 mM | 0.23 mL | 1.15 mL | 2.3 mL |
| 10 mM | 0.12 mL | 0.58 mL | 1.15 mL |
| 50 mM | 0.02 mL | 0.12 mL | 0.23 mL |
| Formulation protocol: | |
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| In vivo protocol: |
1: Meyer T, Schumann P, Weydt P, Petri S, Koc Y, Spittel S, Bernsen S, Günther R, Weishaupt JH, Dreger M, Kolzarek F, Kettemann D, Norden J, Boentert M, Vidovic M, Meisel C, Münch C, Maier A, Körtvélyessy P. Neurofilament light-chain response during therapy with antisense oligonucleotide tofersen in SOD1-related ALS: Treatment experience in clinical practice. Muscle Nerve. 2023 Mar 16. doi: 10.1002/mus.27818. Epub ahead of print. PMID: 36928619.
2: Imbimbo BP, Triaca V, Imbimbo C, Nisticò R. Investigational treatments for neurodegenerative diseases caused by inheritance of gene mutations: lessons from recent clinical trials. Neural Regen Res. 2023 Aug;18(8):1679-1683. doi: 10.4103/1673-5374.363185. PMID: 36751779.
3: Jiang J, Wang Y, Deng M. New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022. Front Pharmacol. 2022 Nov 28;13:1054006. doi: 10.3389/fphar.2022.1054006. PMID: 36518658; PMCID: PMC9742490.
4: Sparasci D, Castelli C, Staedler C, Gobbi C, Ripellino P. Inclusions in macrophages of the cerebrospinal fluid during treatment with Tofersen. Muscle Nerve. 2023 Feb;67(2):E3-E5. doi: 10.1002/mus.27763. Epub 2022 Dec 16. PMID: 36477882.
5: Benatar M, Wuu J, Andersen PM, Bucelli RC, Andrews JA, Otto M, Farahany NA, Harrington EA, Chen W, Mitchell AA, Ferguson T, Chew S, Gedney L, Oakley S, Heo J, Chary S, Fanning L, Graham D, Sun P, Liu Y, Wong J, Fradette S. Correction to: Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study. Neurotherapeutics. 2022 Sep;19(5):1686. doi: 10.1007/s13311-022-01286-9. Erratum for: Neurotherapeutics. 2022 Jul;19(4):1248-1258. PMID: 36175782; PMCID: PMC9606151.
6: Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S; VALOR and OLE Working Group. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705. PMID: 36129998.
7: Dorst J, Genge A. Clinical studies in amyotrophic lateral sclerosis. Curr Opin Neurol. 2022 Oct 1;35(5):686-692. doi: 10.1097/WCO.0000000000001099. Epub 2022 Aug 4. PMID: 35942672.
8: Abati E, Manini A, Comi GP, Corti S. Inhibition of myostatin and related signaling pathways for the treatment of muscle atrophy in motor neuron diseases. Cell Mol Life Sci. 2022 Jun 21;79(7):374. doi: 10.1007/s00018-022-04408-w. PMID: 35727341; PMCID: PMC9213329.
9: Suzuki N, Nishiyama A, Warita H, Aoki M. Genetics of amyotrophic lateral sclerosis: seeking therapeutic targets in the era of gene therapy. J Hum Genet. 2023 Mar;68(3):131-152. doi: 10.1038/s10038-022-01055-8. Epub 2022 Jun 13. PMID: 35691950; PMCID: PMC9968660.
10: Benatar M, Wuu J, Andersen PM, Bucelli RC, Andrews JA, Otto M, Farahany NA, Harrington EA, Chen W, Mitchell AA, Ferguson T, Chew S, Gedney L, Oakley S, Heo J, Chary S, Fanning L, Graham D, Sun P, Liu Y, Wong J, Fradette S. Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study. Neurotherapeutics. 2022 Jul;19(4):1248-1258. doi: 10.1007/s13311-022-01237-4. Epub 2022 May 18. Erratum in: Neurotherapeutics. 2022 Sep 29;: PMID: 35585374; PMCID: PMC9587202.
11: Temp AGM, Naumann M, Hermann A, Glaß H. Applied Bayesian Approaches for Research in Motor Neuron Disease. Front Neurol. 2022 Mar 24;13:796777. doi: 10.3389/fneur.2022.796777. PMID: 35401404; PMCID: PMC8987707.
12: Mullard A. ALS antisense drug falters in phase III. Nat Rev Drug Discov. 2021 Dec;20(12):883-885. doi: 10.1038/d41573-021-00181-w. PMID: 34716445.
13: Ito D. Promise of Nucleic Acid Therapeutics for Amyotrophic Lateral Sclerosis. Ann Neurol. 2022 Jan;91(1):13-20. doi: 10.1002/ana.26259. Epub 2021 Nov 20. PMID: 34704267.
14: Monine M, Norris D, Wang Y, Nestorov I. A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration. J Pharmacokinet Pharmacodyn. 2021 Oct;48(5):639-654. doi: 10.1007/s10928-021-09761-0. Epub 2021 May 15. PMID: 33991294.
15: Hardiman O. Major advances in amyotrophic lateral sclerosis in 2020. Lancet Neurol. 2021 Jan;20(1):14-15. doi: 10.1016/S1474-4422(20)30447-6. PMID: 33340474.
16: Chen JJ. Overview of current and emerging therapies for amytrophic lateral sclerosis. Am J Manag Care. 2020 Aug;26(9 Suppl):S191-S197. doi: 10.37765/ajmc.2020.88483. PMID: 32840332.
17: Abbasi J. In Familial ALS, Antisense Agent Reduces Disease-Related Proteins. JAMA. 2020 Aug 11;324(6):539. doi: 10.1001/jama.2020.14032. PMID: 32780127.
18: Miller T, Cudkowicz M, Shaw PJ, Andersen PM, Atassi N, Bucelli RC, Genge A, Glass J, Ladha S, Ludolph AL, Maragakis NJ, McDermott CJ, Pestronk A, Ravits J, Salachas F, Trudell R, Van Damme P, Zinman L, Bennett CF, Lane R, Sandrock A, Runz H, Graham D, Houshyar H, McCampbell A, Nestorov I, Chang I, McNeill M, Fanning L, Fradette S, Ferguson TA. Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2020 Jul 9;383(2):109-119. doi: 10.1056/NEJMoa2003715. PMID: 32640130.
