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PGN-EDO51
- Development stage
- Discontinued
- Lead developer
- PepGen
- Modality
- Small Molecules, Peptide-Drug Conjugates → Peptide Conjugates → Peptides, 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
PGN-EDO51 is an investigational antisense oligonucleotide therapy developed by PepGen for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. It utilizes Enhanced Delivery Oligonucleotide (EDO) technology, which conjugates a cell-penetrating peptide to the oligonucleotide to improve delivery and uptake into muscle cells. The drug is designed to bind pre-mRNA and modulate RNA splicing, specifically skipping exon 51 of the dystrophin gene transcript. This process can restore the reading frame in certain DMD mutations, enabling production of a shorter but functional dystrophin protein that may stabilize or improve muscle function. Clinical studies have shown dose-dependent increases in exon skipping and dystrophin production with a generally tolerable safety profile at clinically relevant doses[1][6][7][8].
- Brand names
- PGN-EDO51PGN-EDO-51PGN-EDO 51
- Other names
- PGN-EDO51PGN-EDO-51PGN-EDO 51PGN-ED051PGN-ED-051PGN-ED 051
02
Targets
DMD (Dystrophin)
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