Tofersen

• Description :

  Tofersen (BIIB067) is an antisense oligonucleotide that mediates RNase H-dependent degradation of superoxide dismutase 1 (SOD1) mRNA to reduce the synthesis of SOD1 protein. Tofersen can be used for the research of amyotrophic lateral sclerosis (ALS) [1].

• CAS Number :

  [2088232-70-4]

• Product Name Alternative :

  BIIB067; ISIS-SOD1Rx; ISIS 333611

• UNSPSC :

  12352208

• Target :

  DNA/RNA Synthesis

• Type :

  Oligonucleotides

• Related Pathways :

  Cell Cycle/DNA Damage

• Applications :

  Neuroscience-Neurodegeneration

• Field of Research :

  Neurological Disease

• Assay Protocol :

  https://www.medchemexpress.com/tofersen.html

• Purity :

  96.53

• Solubility :

  H2O : 100 mg/mL (ultrasonic)

• Smiles :

  [Tofersen]

• Molecular Formula :

  C230H317N72O123P19S15

• Molecular Weight :

  7128.00

• References & Citations :

  [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.

• Shipping Conditions :

  Blue Ice

• Storage Conditions :

  -20°C (Powder, stored under nitrogen, away from moisture)

• Scientific Category :

  Oligonucleotides

• Clinical Information :

  Launched