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Substance Name: Tofersen [USAN]
RN: 2088232-70-4
UNII: 2NU6F9601K

Note

  • Antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis.

Molecular Formula

  • C230-H317-N72-O123-P19-S15
 

Classification Code

  • Treatment of Amyotrophic Lateral Sclerosis
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Names and Synonyms

Name of Substance

  • Tofersen [USAN]

Synonyms

  • All-P-ambo-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'->5')-2'-O-(2-methoxyethyl)adenylyl-(3'->5')-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'->5')-2'-O-(2-methoxyethyl)guanylyl-(3'->5')-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'->5')-P-thiothymidylyl-(3'->5')-2'-deoxy-P-thioadenylyl-(3'->5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'->5')-2'-deoxy-P-thioadenylyl-(3'->5')-P-thiothymidylyl-(3'->5')-P-thiothymidylyl-(3'->5')-P-thiothymidylyl-(3'->5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'->5')-P-thiothymidylyl-(3'->5')-2'-deoxy-P-thioadenylyl-(3'->5')-2'-O-(2-methoxyethyl)-5-methylcytidylyl-(3'->5')-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'->5')-2'-O-(2-methoxyethyl)guanylyl-(3'->5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'->5')-2'-O-(2-methoxyethyl)-5-methyluridine
  • BIIB067
  • Tofersen
  • UNII-2NU6F9601K

Systematic 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)

Registry Numbers

CAS Registry Number

  • 2088232-70-4

FDA UNII

  • 2NU6F9601K

System Generated Number

  • 2088232704