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tofersen
- Development stage
- Approved
- Lead developer
- Biogen
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
Tofersen is an antisense oligonucleotide designed to treat adults with amyotrophic lateral sclerosis (ALS) who have a confirmed mutation in the superoxide dismutase 1 (SOD1) gene. It works by binding to SOD1 mRNA, leading to its degradation via RNase H-mediated mechanisms and thereby reducing the synthesis of toxic SOD1 protein. This reduction in mutant SOD1 protein is intended to slow disease progression in this specific genetic subset of ALS patients. Tofersen is administered intrathecally and was developed as a targeted therapy for SOD1-ALS, representing the first FDA-approved treatment for this form of ALS[1][3][5][6][8].
- Brand names
- Qalsody
- Other names
- tofersenISIS 333611ISIS333611ISIS-333611BIIB067BIIB-067BIIB 067IONIS SOD1RxISIS-SOD1RxISIS-SOD-1RxISIS-SOD 1Rx
02
Targets
SOD (Manganese Superoxide Dismutase)
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