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C3-silencing siRNA
- Development stage
- Unknown
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravitreal, Subcutaneous
01
Overview
C3-silencing siRNA refers to a class of small interfering RNA (siRNA) therapeutics designed to selectively target and degrade the mRNA of complement component 3 (C3), a central protein in the complement cascade. By inhibiting the hepatic or local production of C3, these RNA interference (RNAi) agents prevent the cleavage of C3 into C3a and C3b, thereby blocking all three pathways of complement activation (classical, alternative, and lectin). This therapeutic approach is being investigated for the treatment of various complement-mediated disorders, including glomerular kidney diseases (such as IgA nephropathy, C3 glomerulopathy, and membranous nephropathy), hematological conditions (such as paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome), and ocular diseases (such as geographic atrophy secondary to age-related macular degeneration).
- Other names
- C3 siRNAC-3 siRNAC 3 siRNAcomplement component 3 siRNAsmall interfering RNA targeting complement component 3
02
Targets
C3
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