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TGFβ siRNA
- Development stage
- Preclinical
- Lead developer
- University of Minnesota
- 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, Topical
01
Overview
TGFβ siRNA (transforming growth factor-beta small interfering RNA) is a target-specific RNA interference (RNAi) therapeutic modality designed to silence the expression of transforming growth factor-beta (TGFβ), a key pro-fibrotic cytokine. By degrading TGFβ mRNA, the siRNA prevents the translation and secretion of TGFβ protein, thereby inhibiting downstream signaling pathways that drive extracellular matrix deposition, fibroblast activation, and tissue fibrosis. In preclinical research, TGFβ siRNA has been formulated in various delivery vehicles, such as mannosylated albumin nanoparticles (MANPs) to target CD206+ pro-fibrotic macrophages, or polypeptide nanoparticles, to treat conditions like pulmonary fibrosis, chronic graft-versus-host disease (cGVHD) with bronchiolitis obliterans (BO), sclerodermatous GVHD, and liver fibrosis.
- Other names
- TGF-beta siRNATGF-beta1 siRNATGF-beta-1 siRNATGF-beta 1 siRNATGFβ1 siRNAtransforming growth factor beta siRNAtransforming growth factor-beta small interfering RNA
02
Targets
TGFB1 (Transforming growth factor Beta-1 proprotein)TGFBR1
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