Drug intelligence / Profile preview

GP-asPNA

Development stage
Phase 1
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
01

Overview

GP-asPNA is a specific construct of antisense peptide nucleic acid (asPNA), a class of synthetic oligomers where the natural sugar-phosphate backbone of DNA is replaced by a pseudopeptide polymer. This unique structure provides high binding affinity and excellent stability, making GP-asPNA resistant to nucleases and proteases found in biological systems. Its mechanism of action involves interfering with gene expression by hybridizing to specific RNA sequences via Watson-Crick base pairing. This binding obstructs the translation process by physically blocking ribosomes from accessing messenger RNA (mRNA), thereby reducing the synthesis of target proteins. This antisense effect is highly sequence-specific, offering a significant advantage over traditional small molecule inhibitors by reducing the likelihood of off-target effects. GP-asPNA is being explored for diverse therapeutic applications, including novel antibacterial agents (e.g., against Methicillin-resistant Staphylococcus aureus), antiviral drugs, and potential uses in gene-editing, RNA modulation, and oncology as anticancer agents.

Other names
antisense peptide nucleic acid constructasPNA construct

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