Drug intelligence / Profile preview

LNP-siVRMA

Development stage
Preclinical
Lead developer
Universidad de Navarra
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, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous, Intramuscular, Subcutaneous, Oral, Inhalation, Intratumoral, Intracerebral, Intrauterine
01

Overview

LNP-siVRMA is a therapeutic agent comprising lipid nanoparticles (LNPs) encapsulating a small interfering RNA (siRNA) specifically designed to target and inhibit the expression of VRMA. VRMA is an N6-methyladenosine (m6A) regulatory gene found to be essential for the survival of Multiple Myeloma (MM) cells. By inhibiting VRMA, LNP-siVRMA aims to reduce global m6A levels, leading to G1 cell cycle arrest, reduced NF-κB signaling, and impaired mitochondrial respiration in MM cells, thereby suppressing tumor growth. This epitranscriptomic-based strategy represents a promising approach for treating MM.

02

Targets

VIRMA (Virilizer-like methyltransferase-associated protein)

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