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sefaxersen
- Development stage
- Phase 3
- Lead developer
- Roche
- 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
- Subcutaneous, Intravitreal
01
Overview
Sefaxersen is an investigational antisense oligonucleotide (ASO) therapy designed to inhibit the production of complement factor B (CFB), a key protein in the alternative pathway of the complement system. By targeting CFB mRNA, sefaxersen reduces systemic levels of complement factor B and thereby decreases activity in the alternative complement pathway. This mechanism is intended to reduce inflammation and protect against tissue damage in diseases where overactivation of this pathway plays a role. Sefaxersen was initially developed by Ionis Pharmaceuticals and is currently being further developed by Roche for indications including primary IgA nephropathy (IgAN) and geographic atrophy secondary to age-related macular degeneration. The drug has reached Phase 3 clinical trials for IgA nephropathy[1][2][4][5][6].
- Other names
- Sefaxersen
02
Targets
CFB (Complement Factor B)
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