Drug intelligence / Profile preview

MGN-4893

Development stage
Unknown
Lead developer
Viridian Therapeutics
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

MGN-4893 is an investigational microRNA-based therapeutic that acts as an inhibitor of microRNA-451. It was developed for the treatment of polycythemia vera, a rare myeloproliferative disorder characterized by excessive production of blood cells and platelets. MicroRNA-451 plays a crucial role in regulating blood cell development, and its inhibition is thought to modulate disease processes in polycythemia vera. MGN-4893 received FDA orphan drug designation in 2011 for this indication but has not advanced beyond preclinical or early discovery stages as of the latest updates. The drug was originally developed by miRagen Therapeutics (now Viridian Therapeutics), which focuses on innovative RNA-based therapies[1][2][6].

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