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

Name: Tofersen sodium
Brand: MedChemExpress
SKU: HY-132580A
Rating: 5.0 (2 reviews)

Art. -Nr.: HY-132580A Reinheit: 97.55%: Data Sheet
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Tofersen (BIIB067) sodium is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC₅₀ of 320 pM. Tofersen sodium mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen sodium 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 sodium can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis.

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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), sodium salt

Tofersen sodium Chemische Struktur

CAS. Nr. : 1898254-60-8

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1 mg	Auf Lager	Estimated Time of Arrival: December 31
5 mg	Auf Lager	Estimated Time of Arrival: December 31
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Based on 2 publication(s) in Google Scholar

Other Forms of Tofersen sodium:

Tofersen scrambled control In-stock
Tofersen In-stock
Tofersen-d₂₇ Angebot einholen

2 Publications Citing Use of MCE Tofersen sodium

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Beschreibung

IC₅₀ & Target^[2]

SOD mRNA

0.32 nM (IC₅₀)

In Vitro

Tofersen (72 h) sodium potently silences SOD1 mRNA in human HeLa cells with an IC₅₀ of ~0.32 nM^[2].
Tofersen sodium-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 sodium-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.

In Vivo

Tofersen (42 nmol; intracerebroventricular injection; single dose) sodium extends median survival of SOD1^G93A 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) sodium 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) sodium 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) sodium to the CSF of SOD1^G93A 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) sodium extends survival and improves muscle response function in transgenic rodent models of SOD1-mediated ALS^[8].
Tofersen (Intrathecal) sodium 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.

Molekulargewicht

7128 (free acid)

Formel

C₂₃₀H₂₉₈N₇₂Na₁₉O₁₂₃P₁₉S₁₅

CAS. Nr.

1898254-60-8

Appearance

Solid

Color

White to light yellow

SMILES

[Tofersen (sodium)]

Versand

Room temperature in continental US; may vary elsewhere.

Speicherung

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

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

Reinheit & Dokumentation

Purity: 97.55%

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Data Sheet (287 KB) SDS (254 KB)

COA (245 KB) RP-HPLC (233 KB) MS (92 KB)

Handling Instructions (2242 KB)

Verweise

[1]. Steffke C, et al. Targeted Proteomics upon Treatment with Tofersen Identifies Novel Response Markers for Superoxide Dismutase 1-Linked Amyotrophic Lateral Sclerosis. Ann Neurol. 2025;98(6):1318-1334.

[2]. Weiss A, et al. RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice. Mol Ther. 2025;33(8):3917-3938.

[3]. Miller TM, et al. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022;387(12):1099-1110.

[4]. Miller TM, et al. Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis. JAMA Neurol. 2026 Feb 1;83(2):115-125.

[5]. Ediriweera GR, et al. Lipid nanoparticles and transcranial focused ultrasound enhance the delivery of SOD1 antisense oligonucleotides to the murine brain for ALS therapy. J Control Release. 2025;378:221-235.

[6]. Miller TM, et al. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol. 2013 May;12(5):435-42.

[7]. Duan C, et al. Intrathecal administration of a novel siRNA modality extends survival and improves motor function in the SOD1G93A ALS mouse model. Mol Ther Nucleic Acids. 2024;35(1):102147. Published 2024 Feb 15.

[8]. 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. [Content Brief]

[9]. Wiesenfarth M, et al. Effects of tofersen treatment in patients with SOD1-ALS in a "real-world" setting - a 12-month multicenter cohort study from the German early access program. EClinicalMedicine. 2024;69:102495. Published 2024 Feb 15.

Tofersen sodium Related Classifications

Neurological Disease

Molaritätsrechner
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Die Formel zur Berechnung von Molaritäten

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

Mass		Concentration		Volume		Molecular Weight *
	=		×		×

The dilution calculator equation

Konzentration _{(Stammlösung)} × Volumen _{(Stammlösung)} = Konzentration _{(Ziellösung)} × Volumen _{(Ziellösung)}

Diese Gleichung wird häufig abgekürzt als: C₁V₁ = C₂V₂

Concentration _(start)	×	Volume _(start)	=	Concentration _(final)	×	Volume _(final)
	×		=		×
C1		V1		C2		V2

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

Kundenbewertung

Other Forms of Tofersen sodium:

Tofersen sodium Related Antibodies

2 Publications Citing Use of MCE Tofersen sodium

Spezialitätsempfehlungen

•Related Small Molecules:

•Related Proteins:

Biologische Aktivität

Reinheit & Dokumentation

Verweise

Kundenbewertung

Tofersen sodium Related Antibodies

Tofersen sodium Related Classifications

Molaritätsrechner

Verdünnungsrechner

Die Formel zur Berechnung von Molaritäten

The dilution calculator equation

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Ihre kürzlich angesehenen Produkte:

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