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Tofersen

CHEMBL3833346 Phase 4 Approuvé Oligonucleotide
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

Tofersen is an antisense oligonucleotide (ASO) that binds to SOD1 mRNA through Watson-Crick base pairing, recruiting RNase H-mediated degradation of the mRNA and reducing production of mutant SOD1 protein, which causes toxic gain-of-function neurodegeneration in SOD1-associated ALS. It is administered intrathecally and is approved for the treatment of adults with ALS associated with a mutation in the SOD1 gene, representing the first ALS therapy targeting a specific genetic cause. By reducing SOD1 protein levels in cerebrospinal fluid and plasma, it slows functional decline in SOD1-ALS.

Mécanisme d'action

Binds to complementary mRNA sequences through Watson-Crick base pairing, modulating gene expression by promoting mRNA degradation via RNase H or altering pre-mRNA splicing.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

Mécanisme

Binds to complementary mRNA sequences through Watson-Crick base pairing, modulating gene expression by promoting mRNA degradation via RNase H or altering pre-mRNA splicing.

HBD / HBA

- / -

No targets recorded

Target interaction data is not yet available for this drug.

No interactions recorded

Drug interaction data is not yet available for this compound.

No side effects recorded

Side effect data is not yet available for this drug.

Foire aux questions

Tofersen is an antisense oligonucleotide (ASO) that binds to SOD1 mRNA through Watson-Crick base pairing, recruiting RNase H-mediated degradation of the mRNA and reducing production of mutant SOD1 protein, which causes toxic gain-of-function neurodegeneration in SOD1-associated ALS. It is administered intrathecally and is approved for the treatment of adults with ALS associated with a mutation in the SOD1 gene, representing the first ALS therapy targeting a specific genetic cause. By reducing SOD1 protein levels in cerebrospinal fluid and plasma, it …

Binds to complementary mRNA sequences through Watson-Crick base pairing, modulating gene expression by promoting mRNA degradation via RNase H or altering pre-mRNA splicing.

Yes, Tofersen is an approved drug. It has reached clinical phase 4. It is classified as a Oligonucleotide.

{# References & Data Sources section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

References & Data Sources

  • ChEMBL — European Bioinformatics Institute (EBI). CHEMBL3833346. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.

Avertissement médical

This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.

Data sources: ChEMBL, PubChem, DailyMed.