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cofirasersen
- Development stage
- Phase 1
- Lead developer
- Ionis Pharmaceuticals
- Modality
- 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
- Administration
- Inhalation
01
Overview
Cofirasersen (ION-827359) is an investigational Generation 2.5 antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for the treatment of chronic obstructive pulmonary disease (COPD) and chronic bronchitis. Administered via oral inhalation, the drug is designed to target and reduce the expression of the epithelial sodium channel (ENaC) in the airways. By inhibiting ENaC, cofirasersen aims to decrease sodium and water reabsorption from the airway surface liquid, thereby improving hydration of the mucus and facilitating its clearance. A Phase 2a study evaluating its efficacy on lung function (FEV1) was terminated in 2021 due to a strategic business decision by the developer.
- Other names
- cofirasersen
02
Targets
SCNN1B (ENaC beta subunit)Sodium channel epithelial 1 subunit alpha (SCNN1A) mRNASCNN1G (ENaC gamma subunit)
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