Drug intelligence / Profile preview

asidna

Development stage
Phase 2
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, Intratumoral
01

Overview

AsiDNA is a first-in-class signal-interfering DNA (siDNA) molecule designed to disrupt the DNA damage response (DDR) in cancer cells. It consists of a short, cholesterol-conjugated, double-stranded DNA oligonucleotide that mimics genomic double-strand breaks. By acting as a decoy, AsiDNA binds to and hyperactivates key DNA repair proteins, including DNA-dependent protein kinase (DNA-PK) and poly(ADP-ribose) polymerase (PARP), effectively sequestering them away from actual sites of DNA damage caused by radiotherapy or chemotherapy. This mechanism prevents the repair of lethal DNA lesions, leading to selective tumor cell death while sparing healthy cells. Developed by Onxeo, AsiDNA is being evaluated in clinical trials for various solid tumors, including breast cancer and ovarian cancer, particularly in combination with DNA-damaging agents or PARP inhibitors to overcome resistance.

Brand names
AsiDNA
Other names
DbaitCo-DbaitsiDNA
02

Targets

DNA-PK (DNA-dependent protein kinase)ATM (Ataxia telangiectasia mutated protein)

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