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Fbn1 siRNA
- Development stage
- Preclinical
- Lead developer
- Engitix 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
- Intravenous
01
Overview
Fbn1 siRNA is a lipid nanoparticle (LNP)-formulated small interfering RNA (siRNA) designed to target and knock down the expression of fibrillin-1 (FBN1). Developed under a collaboration between Takeda and Engitix, Fbn1 siRNA is being investigated for the treatment of liver fibrosis associated with metabolic dysfunction-associated steatohepatitis (MASH) [1355]. Fibrillin-1 is an extracellular matrix (ECM) glycoprotein that acts as a driver of fibrogenesis and pathological elastic fiber accumulation [1355]. By silencing FBN1 expression, the therapeutic candidate aims to reduce collagen and elastin deposition, thereby preventing or reversing liver fibrosis [1355].
- Other names
- FBN1 siRNAFBN-1 siRNAFBN 1 siRNAfibrillin-1 siRNAfibrillin1 siRNAfibrillin 1 siRNA
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