Drug intelligence / Profile preview

EDI-M420

Development stage
Preclinical
Lead developer
Empire Discovery Institute
Modality
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, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

EDI-M420 is a preclinical-stage antisense oligonucleotide (ASO) therapeutic developed by the Empire Discovery Institute (EDI). It is designed to modulate mitochondrial energy metabolism by targeting specific natural RNA sequences. The drug is being investigated for the treatment of lung and cardiovascular diseases, leveraging a platform focused on addressing mitochondrial dysfunction. As an ASO, EDI-M420 likely functions by binding to its target RNA to either trigger degradation or modulate splicing/translation, thereby restoring or enhancing mitochondrial bioenergetics in affected tissues.

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