Drug intelligence / Profile preview

UBE2v1 siRNA

Development stage
Preclinical
Lead developer
University of Bari Aldo Moro
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
Intraperitoneal
01

Overview

UBE2v1 siRNA is a small interfering RNA (siRNA) therapeutic candidate designed to target and downregulate the expression of ubiquitin-conjugating enzyme E2 variant 1 (UBE2v1). Developed by researchers at the University of Bari Aldo Moro and the University of Rome Tor Vergata, this gene therapy approach utilizes lipid nanoparticles (LNPs) composed of ionizable cationic lipids, cholesterol, helper lipids, and PEG-lipids to deliver the siRNA. UBE2v1 is a key mediator of non-canonical lysine-63 (K63)-linked ubiquitination, which drives tubulointerstitial fibrosis and inflammatory signaling in renal tubular cells. By silencing UBE2v1, the therapy aims to counteract the progression of kidney fibrosis in diabetic kidney disease (DKD). Preclinical studies in streptozotocin-induced diabetic mice have demonstrated that intraperitoneal administration of UBE2v1 siRNA-loaded LNPs successfully downregulates renal UBE2v1 expression without inducing adverse inflammatory responses.

Other names
siUBE2v1siUBE-2v1siUBE 2v1UBE2v1-targeting siRNAUBE-2v1-targeting siRNAUBE 2v1-targeting siRNA
02

Targets

UBE2V1 (Ubiquitin-conjugating enzyme E2 V2)

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