Drug intelligence / Profile preview

OX413

Development stage
Preclinical
Lead developer
Valerio Therapeutics
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

OX413 is a first-in-class decoy oligodeoxynucleotide developed by Onxeo using its proprietary PlatON platform. It is designed to target and hyperactivate poly [ADP-ribose] polymerase 1 (PARP1), acting as a decoy agonist. This hyperactivation leads to PARP trapping, rapid depletion of cellular NAD+ (metabolic exhaustion), and DNA repair abrogation, resulting in selective tumor cell death. Additionally, the accumulation of cytoplasmic chromatin fragments triggers the cGAS/STING pathway, promoting an innate and adaptive immune response. OX413 is being investigated preclinically for the treatment of various cancers, including breast, ovarian, and prostate cancers.

02

Targets

STING (Stimulator of interferon genes protein)Cyclic GMP–AMP synthase

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