2. Immunology/Inflammation
  3. SOD
  4. Tofersen

Tofersen  (Synonyms: BIIB067; ISIS-SOD1Rx; ISIS 333611)

Cat. No.: HY-132580 Purity: 96.53%
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Tofersen (BIIB067) is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC50 of 320 pM. Tofersen mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15, and serum neurofilament light chain, neurofilament heavy chain. Tofersen can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis.

For research use only. We do not sell to patients.

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-Tsp-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)

Tofersen Chemical Structure

CAS No. : 2088232-70-4

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Based on 2 publication(s) in Google Scholar

Other Forms of Tofersen:

Tofersen Related Antibodies

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Description

Tofersen (BIIB067) is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC50 of 320 pM. Tofersen mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15, and serum neurofilament light chain, neurofilament heavy chain. Tofersen can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis[1][2][3][4][5][6][7][8][9].

IC50 & Target[2]

SOD1 mRNA

0.32 nM (IC50)

In Vitro

Tofersen (72 h) potently silences SOD1 mRNA in human HeLa cells with an IC50 of ~0.32 nM[2].
Tofersen-loaded Ca2+P lipid nanoparticles (72 h) reduce SOD1 protein levels by >3.3-fold in HEK293T cells after 72 h of incubation[5].
Tofersen-loaded Ca2+P lipid nanoparticles (0.78-50 nM; 1-48 h) exhibit dose- and time-dependent uptake into NSC-34 mouse motor neuron-like cells, are non-cytotoxic at concentrations up to 25 nM after 48 h, and undergo pH-dependent release of Tofersen within the cytoplasm[5].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Tofersen Related Antibodies
In Vivo

Tofersen (42 nmol; intracerebroventricular injection; single dose) extends median survival of SOD1G93A ALS mice to 143 days (pre-symptomatic) or 139 days (symptomatic) and reduces cortical and cerebellar SOD1 mRNA by 40%-60%[2].
Tofersen (0.25 mg/kg; i.v.; single dose) combined with optimized FUS and microbubbles delivers 5-10% of the administered dose to mouse brain tissue, achieving >3.5-fold higher brain uptake than without FUS[5].
Tofersen (0.5 mg/kg; i.v.; once weekly; 9 weeks) combined with FUS reduces SOD1 expression in targeted mouse brain cortex regions and preserves spinal cord motor neuron count without inducing neuroinflammation in G93A-SOD1 ALS mice[5].
Delivery of Tofersen (ISIS 333611) to the CSF of SOD1G93A rats distributes to the brain and spinal cord, reduces spinal cord SOD1 mRNA and protein levels, and prolongs survival in this ALS model[6].
Tofersen (Intrathecal) extends survival and improves muscle response function in transgenic rodent models of SOD1-mediated ALS[8].
Tofersen (Intrathecal) lowers SOD1 protein concentrations in non-human primates[8].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: B6SJL-Tg(SOD1*G93A)1Gur/J (equal numbers of male and female, pre-symptomatic 6 weeks old or symptomatic 12 weeks old)[2]
Dosage: 42 nmol (~300 μg)
Administration: intracerebroventricular injection; single dose
Result: Extended median survival to 143 days (pre-symptomatic cohort) and 139 days (symptomatic cohort).
Reduced SOD1 mRNA by 40%-60% in the cortex and cerebellum in both treatment cohorts.
Animal Model: B6.Cg Sod1G93A (G93A-SOD1) transgenic (9-week-old, male and female, ALS model)[6]
Dosage: 0.5 mg/kg
Administration: i.v.; once weekly; 9 weeks
Result: Significantly reduced SOD1 fluorescence intensity in the FUS-exposed cerebral cortex compared to FUS-only or Tofersen-only control groups.
Showed no significant difference in SOD1 levels in non-FUS-exposed cerebellar regions.
Increased spinal cord motor neuron count significantly compared to both control groups.
Detected no significant increases in microglial (IBA1+ cell count) or astrocytic (GFAP fold change) activity in FUS-exposed brain regions, indicating no treatment-related neuroinflammation.
Clinical Trial
Molecular Weight

7128.00

CAS No.
Appearance

Solid

Color

Off-white to light yellow

Sequence

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-Tsp-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)
SMILES

[Tofersen]
Shipping

Room temperature in continental US; may vary elsewhere.
Storage

-20°C, stored under nitrogen, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)

Solvent & Solubility
In Vitro: 

H2O : 100 mg/mL (14.03 mM; Need ultrasonic)

Preparing
Stock Solutions
Concentration Solvent Mass 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
View the Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.

  • Molarity Calculator

  • Dilution Calculator

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

Mass
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Concentration
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Volume
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Molecular Weight *

Concentration (start) × Volume (start) = Concentration (final) × Volume (final)

This equation is commonly abbreviated as: C1V1 = C2V2

Concentration (start)

C1

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V1

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Concentration (final)

C2

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V2

In Vivo:

For the following dissolution methods, please prepare the working solution directly. It is recommended to prepare fresh solutions and use them promptly within a short period of time.
The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.

  • Protocol 1

    Add each solvent one by one:  PBS

    Solubility: 100 mg/mL (14.03 mM); Clear solution; Need ultrasonic

In Vivo Dissolution Calculator
Please enter the basic information of animal experiments:

Dosage

mg/kg

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(per animal)

g

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(per animal)

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Number of animals

Recommended: Prepare an additional quantity of animals to account for potential losses during experiments.
Calculation results:
Working solution concentration: mg/mL
This product has good water solubility, please refer to the measured solubility data in water/PBS/Saline for details.
The concentration of the stock solution you require exceeds the measured solubility. The following solution is for reference only.If necessary, please contact MedChemExpress (MCE).
Purity & Documentation
References

Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

Optional Solvent Concentration Solvent Mass 1 mg 5 mg 10 mg 25 mg
H2O 1 mM 0.1403 mL 0.7015 mL 1.4029 mL 3.5073 mL
5 mM 0.0281 mL 0.1403 mL 0.2806 mL 0.7015 mL
10 mM 0.0140 mL 0.0701 mL 0.1403 mL 0.3507 mL

* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.

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Tofersen Related Classifications

Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

Tofersen2088232-70-4BIIB067 ISIS-SOD1Rx ISIS 333611BIIB 067BIIB-067ISIS333611ISIS 333611ISIS-333611SODSuperoxide Dismutaseserumamyloid-beta 1-40cerebrospinal fluidSOD1 proteinneurofilament heavy chainHEK293T cellsHeLa cellsamyloid-beta 1-42neurofilament light chainSOD1 mRNAInhibitorinhibitorinhibit

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