tofersen

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Ligands
tofersen

GtoPdb Ligand ID: 13536

Synonyms: BIIB-067 | BIIB067 | Qalsody®

tofersen is an approved drug (FDA (2023), EMA (2024))

Compound class: Nucleic acid

Comment: Tofersen (BIIB067) is an antisense oligonucleotide that disrupts translation of superoxide dismutase 1 (SOD1). Tofersen was designed to treat cases of amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) that are associated with expression of mutated SOD1. SOD1-ALS/MND accounts for ~2% of all cases.

Summary
Biological activity
Clinical data
References
Structure

References
1. Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD et al.. (2022) Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med, 387 (12): 1099-1110. [PMID:36129998]

References

1. Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD et al.. (2022)
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
N Engl J Med, 387 (12): 1099-1110. [PMID:36129998]