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AsiDNA + carboplatin + paclitaxel

Development stage
Unknown
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, Covalent Small Molecules → Small Molecules, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Classical Binding Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Intravenous
01

Overview

AsiDNA + carboplatin + paclitaxel is a combination regimen investigated for the treatment of various solid tumors, particularly gliomas. **AsiDNA** is a first-in-class DNA repair inhibitor—specifically a short, double-stranded DNA molecule that mimics DNA double-strand breaks to act as a decoy, leading to hyperactivation and trapping of DNA-dependent protein kinase (DNA-PK) and PARP, thereby preventing the effective repair of DNA damage in tumor cells. This synthetic induction of false DNA damage signaling blocks several repair pathways (homologous recombination, non-homologous end joining, base excision repair), resulting in enhanced cytotoxicity when combined with DNA-damaging agents. **Carboplatin** is a platinum-based chemotherapeutic that induces DNA crosslinks, leading to cell cycle arrest and apoptosis via DNA damage. **Paclitaxel** is a chemotherapeutic agent that stabilizes microtubules, inhibiting their depolymerization and thereby blocking cell division during mitosis. The combination has shown synergistic anti-tumor activity in preclinical models (notably in glioma), including increased cell-cycle arrest, DNA damage, and apoptosis compared with monotherapy, as well as increased survival in animal models. Ongoing and recent early phase clinical trials (mainly phase I/II) are evaluating this combination for solid tumors, including recurrent glioblastoma[1][2][3][4][6].

Other names
AsiDNA + carboplatin + paclitaxel
02

Targets

PARP1 (Poly (adp-ribose) polymerase 1)TUBB (Tubulin (alpha and beta subunits))DNA-PK (DNA-dependent protein kinase)DNA

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