1. Cell Cycle/DNA Damage
  2. DNA/RNA Synthesis
  3. Tofersen

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).

The free form of the compound is prone to instability, it is advisable to consider the stable salt form (Tofersen sodium) that retains the same biological activity.

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

Size Price Stock
1 mg USD 144 Ask For Quote & Lead Time
5 mg USD 403 Ask For Quote & Lead Time
10 mg USD 645 Ask For Quote & Lead Time
25 mg USD 1290 Ask For Quote & Lead Time
50 mg USD 2064 Ask For Quote & Lead Time
100 mg USD 3302 Ask For Quote & Lead Time

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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].

In Vitro

Tofersen targets the SOD1 mRNA using antisense oligonucleotides (ASOs) that bind to the SOD1 mRNA by Watson-Crick base pairing. Tofersen activates RNase H1 that destroys the targeted RNA[2].

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

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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Volume (start)

V1

=
Concentration (final)

C2

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Volume (final)

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

Animal weight
(per animal)

g

Dosing volume
(per animal)

μL

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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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.

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Tofersen
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