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PepGen Announces Positive Preclinical Data for PGN-EDO53, PGN-EDO45 and PGN-EDO44, Three Novel Duchenne Muscular Dystrophy Candidates
- In non-human primates (NHPs), high levels of exon 53 skipping were observed after a single dose of PGN-EDO53 – almost 7-times higher than those observed for
About this update from Pepgen Inc.
[{"type":"text","content":"- In non-human primates (NHPs), high levels of exon 53 skipping were observed after a single dose of PGN-EDO53 – almost 7-times higher than those observed for a comparator peptide-phosphorodiamidate morpholino oligonucleotide (PPMO) conjugate molecule used as a positive control - PGN-EDO45 development candidate demonstrated high levels of exon skipping in wild-type human myoblasts in vitro, and outperformed a comparator PPMO used as a positive control at every dose level - - High, dose-dependent exon skipping levels were observed for PGN-EDO44 in an in vitro assay – - This data supports the potential of PepGen’s Enhanced Delivery Oligonucleotide platform to potentially transform the treatment of DMD - BOSTON, Nov. 14, 2022 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced NHP exon skipping data for PGN-EDO53, PepGen’s product candidate in development for the treatment of Duchenne muscular dystrophy (DMD) patients whose mutations are amenable to an exon 53 skipping approach. In addition, PepGen announced in vitro exon skipping data for PGN-EDO45 and PGN-EDO44, PepGen’s product candidates in development for the treatment of DMD patients amenable to exon 45 and exon 44 skipping approaches, respectively. Together with PGN-EDO51, PepGen’s DMD pipeline candidates, if approved, could treat approximately 50% of patients whose disease is amenable to an exon skipping approach. All of PepGen’s DMD therapeutic candidates utilize the same Enhanced Delivery Oligonucleotide (EDO) peptide as the company’s clinical stage PGN-EDO51 program, which has recently reported out positive Phase 1 data in healthy volunteers. In this trial, PGN-EDO51 exhibited the highest levels of oligonucleotide delivery and exon skipping in a clinical study following a single dose when compared to publicly available data for other exon 51 skipping approaches. “PepGen is driven by deep commitment to the DMD patient community, and we are very pleased to provide a comprehensive update on our DMD pipeline that supports the potential expansion of our EDO technology to additional patients who are amenable to an exon skipping approach. We are particularly encouraged by the ...