Drug intelligence / Profile preview

gamma peptide nucleic acid

Development stage
Preclinical
Lead developer
University of Connecticut
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Peptides, 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, Intraperitoneal
01

Overview

Gamma peptide nucleic acid (γPNA) is a next-generation synthetic oligonucleotide platform characterized by a modification at the gamma-carbon of the peptide-like backbone. This modification pre-organizes the PNA into a right-handed helix, significantly increasing its binding affinity and sequence specificity for complementary DNA or RNA sequences compared to traditional PNAs. Developed primarily by researchers at the University of Connecticut and Yale University, γPNAs are being investigated as anti-gene therapies designed to target chromosomal DNA directly. By invading the genomic DNA of oncogenes like c-Myc, γPNAs inhibit transcription and subsequent protein expression. To overcome delivery challenges, these molecules are often conjugated to nuclear localization signals (NLS) for efficient nuclear uptake. Preclinical data suggests that γPNAs are effective in various cancer models, including lymphoma and triple-negative breast cancer, particularly when used in synergistic combinations with histone deacetylase inhibitors (HDACi) or other small molecule inhibitors.

Other names
gamma PNAγPNAanti-gene gamma peptide nucleic acid
02

Targets

MYC proto-oncogene promoter genomic DNAMYCN (N-myc proto-oncogene protein (MYCN) genomic DNA)

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