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GalNAc-modified siTff2
- 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
- Subcutaneous
01
Overview
GalNAc-modified siTff2 is a chemically synthesized small interfering RNA (siRNA) conjugated to a trivalent N-acetylgalactosamine (GalNAc) ligand designed for targeted delivery to hepatocytes via the asialoglycoprotein receptor (ASGPR), which is primarily expressed on liver cells. This drug exploits the RNA interference (RNAi) pathway, where the siRNA guides the RNA-induced silencing complex (RISC) to degrade messenger RNA (mRNA) for the gene Tff2, reducing its expression. GalNAc conjugation substantially increases the potency, hepatic specificity, stability (via chemical modifications such as 2′-O-methyl, 2′-fluoro, and phosphorothioate substitutions), and duration of effect of the siRNA, while allowing for infrequent subcutaneous dosing[1][2][3][4]. The modification encourages efficient cellular uptake into hepatocytes and rapid endosomal escape, making it a prominent platform for liver-targeted gene silencing therapies[3][4]. The primary indication is the investigation of the Tff2 target in liver-related diseases; since "siTff2" refers to an siRNA against Tff2, further specifics depend on the intended therapeutic or experimental context.
- Other names
- GalNAc-siTff2GalNAc-siTff-2GalNAc-siTff 2
02
Targets
ASGPR (Asialoglycoprotein Receptor 1)
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