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SLN548
- Development stage
- Preclinical
- Lead developer
- Silence Therapeutics
- 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, Intravenous
01
Overview
SLN548 is a short interfering RNA (siRNA) therapeutic developed by Silence Therapeutics. It is designed to target and silence genes involved in the alternative pathway of the complement system, with the aim of treating disorders driven by dysregulation of this pathway. The drug leverages Silence's proprietary mRNAi GOLD platform for liver-targeted delivery, enabling precise gene silencing in diseases with a genetic basis. Its primary indication under development is for disorders of complement activation, which are immune system diseases often associated with rare or hematological conditions[1][3][9].
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