Drug intelligence / Profile preview

antisense oligonucleotides targeting PVT1

Development stage
Preclinical
Lead developer
Ionis Pharmaceuticals
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

Antisense oligonucleotides (ASOs) targeting the long non-coding RNA (lncRNA) PVT1 are experimental therapeutic agents designed to silence the PVT1 oncogene. PVT1 is frequently overexpressed in various cancers, including gastric adenocarcinoma, esophageal adenocarcinoma, and acute myeloid leukemia (AML). These ASOs, which include LNA GapmeR variants, function by binding to PVT1 transcripts (both linear and circular isoforms) and inducing RNase H-dependent degradation. Preclinical research, notably involving Ionis Pharmaceuticals and MD Anderson Cancer Center, has shown that PVT1 silencing can inhibit leukemia cell growth, induce apoptosis, and sensitize tumor cells to DNA damage response (DDR) inhibitors like ATM or ATR inhibitors.

Other names
PVT1 ASOsPVT-1 ASOsPVT 1 ASOsantisense LNA GapmeR variants targeting PVT1
02

Targets

PVT1 (Plasmacytoma variant translocation 1)

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