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C5 siRNA (Argo Biopharma)
- Development stage
- Preclinical
- Lead developer
- Shanghai Argo Biopharmaceutical
- 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
- Subcutaneous
01
Overview
C5 siRNA (Argo Biopharma) is an investigational small interfering RNA (siRNA) therapeutic being developed by Shanghai Argo Biopharmaceutical for the treatment of complement-mediated rare diseases. Utilizing the company's proprietary RADS (RNAi-based Advanced Delivery System) platform, the drug is designed to target and silence the mRNA encoding complement component 5 (C5) in the liver. By reducing the hepatic production and subsequent systemic levels of the C5 protein, the therapy aims to inhibit the terminal complement pathway, which is a key driver in various rare autoimmune and inflammatory conditions such as paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and generalized myasthenia gravis (gMG). The program is currently in the discovery and IND-enabling stages of development.
- Other names
- Complement component 5 siRNA
02
Targets
C5
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