BOSTON--(BUSINESS WIRE)--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 that the Company’s data will be presented in two oral presentations at the 30th Annual International Congress of the World Muscle Society (WMS) being held October 7-12, 2025, in Vienna, Austria. One of the presentations will include previously reported positive FREEDOM-DM1 Phase 1 clinical data.
Title: Results from 15 mg/kg single dose PGN-EDODM1 cohort of FREEDOM-DM1- a phase 1 study in people with myotonic dystrophy type 1 (DM1) (#230)
Session: Clinical Trial Updates – Hall D
Date & Time: Saturday, October 11th at 8:30-8:45am CET
Presenter: Hanns Lochmüller, MD, PhD, FAAN, Children's Hospital of Eastern Ontario Research Institute, Ottawa, Canada
Title: Dystrophin results from the 10 mg/kg cohort of CONNECT1-EDO51 phase 2 study of PGN-EDO51 in people with Duchenne amenable to exon 51 skipping (#190)
Session: Clinical Trial Updates – Hall D
Date & Time: Saturday, October 11th at 7:30-7:45am CET
Presenter: Hugh McMillan, MD, Children's Hospital of Eastern Ontario, Ottawa, Canada
Following the conference, the presentations presented at the 30th Annual Congress of the WMS will be available on PepGen’s website under Scientific Publications.
About PGN-EDODM1
PGN-EDODM1, PepGen's investigational candidate in development for the treatment of myotonic dystrophy type 1 (DM1), utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 addresses the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the dystrophia myotonica protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, and disrupting the binding between the CUG repeat expansion and MBNL1. PepGen believes this innovative therapeutic approach may have considerable advantages over oligonucleotide modalities that rely on knockdown or degradation of the DMPK transcripts as it will allow the DMPK transcripts to continue to perform their normal function within the cell, while also liberating MBNL1 to correct downstream mis-splicing events. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1.
About PepGen
PepGen Inc. is a clinical-stage biotechnology company developing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, the Company is generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases.
For more information, please visit PepGen.com. Follow PepGen on LinkedIn and X.
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