Drug intelligence / Profile preview

siCOL1A1

Development stage
Unknown
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

siCOL1A1 is a **small interfering RNA (siRNA)** therapeutic agent designed to specifically silence the expression of the **COL1A1 gene**, which encodes the alpha-1 chain of type I collagen. Type I collagen is the predominant structural protein in the extracellular matrix and is significantly upregulated in fibrotic diseases and various cancers. The therapeutic mechanism involves RNA interference-mediated degradation of COL1A1 mRNA, leading to reduced collagen production. In **liver fibrosis**, siCOL1A1 has been formulated in **lipid nanoparticles (LNP-siCol1a1)** for targeted delivery to hepatic stellate cells and other nonparenchymal liver cells. This approach has demonstrated up to 90% suppression of procollagen α1(I) expression and 40-60% reduction in hepatic collagen deposition in preclinical models of both parenchymal and biliary liver fibrosis. In **cancer research**, particularly hepatocellular carcinoma (HCC) and colorectal cancer (CRC), siCOL1A1 has shown efficacy in suppressing tumor cell migration, invasion, and metastatic potential by inhibiting epithelial-to-mesenchymal transition (EMT) and reducing cancer stem cell characteristics. The knockdown of COL1A1 leads to decreased expression of EMT markers like Slug and vimentin, increased E-cadherin expression, and reduced stemness markers including SOX2, OCT4, and CD133.

Other names
LNP-siCol1a1LNP-siCol-1a1LNP-siCol 1a1siCol1a1siCol-1a1siCol 1a1
02

Targets

COL1A1 (Collagen type I alpha 1 chain)

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