PepGen

The EDO platform conjugates cell-penetrating peptides to therapeutic oligonucleotides, designed to significantly improve escape from endosomes after cellular uptake and increase concentration in muscle tissue. Standard oligonucleotide therapies often achieve limited tissue distribution because the molecules remain trapped in endosomes, reducing the proportion that reaches the nucleus to modify RNA splicing. PepGen has demonstrated proof of concept in clinical trials, with the lead DMD candidate PGN-EDO51 producing exon-skipping levels above what has been reported for existing commercially approved exon 51-skipping therapies.

In June 2023, PepGen reported interim Phase 1 data for PGN-EDO51 showing mean exon 51 skipping levels above two percent in bicep muscle biopsies at the highest dose level of 10 mg/kg, measured 28 days after a single intravenous dose (per PepGen, June 2023). No drug-related serious adverse events were observed in the Phase 1 healthy-volunteer study. These results informed the dosing regimen for the Phase 2 CONNECT1-EDO51 trial in DMD patients, which completed enrollment in March 2025.

James McArthur serves as President and CEO. He previously led Rare Disease Research at Biogen, where he oversaw the development and FDA approval of Spinraza for spinal muscular atrophy. Matthew Wood, the scientific co-founder, is a professor of neuroscience at the University of Oxford whose laboratory performed the foundational oligonucleotide-delivery research that PepGen licensed to build its platform.

PepGen's second clinical-stage program is PGN-EDODM1 for myotonic dystrophy type 1, a progressive muscle-wasting disease for which no disease-modifying therapies are approved. The DM1 candidate entered a Phase 1 clinical trial in 2023 and advanced into a Phase 2 study in 2024. PepGen also has a preclinical Duchenne candidate, PGN-EDO53, targeting the less common exon 53-skipping-amenable DMD population.

PepGen completed an initial public offering on Nasdaq in May 2022 at $12 per share, raising gross proceeds of approximately $108 million. Its pre-IPO investor syndicate included biotech-focused funds such as RA Capital Management and Samsara BioCapital. As of its third-quarter 2024 financial results, the company reported cash and equivalents projected to fund operations into 2026 (per PepGen, November 2024).

PepGen licensed the foundational EDO platform intellectual property from Oxford University Innovation, the university's technology-transfer office. Scientific co-founder Matthew Wood continues as an advisor to the company while maintaining his academic research program at Oxford. The company maintains a research presence in Oxford to remain connected to the originating academic laboratory.

As of the latest public disclosures, PepGen is advancing its clinical programs independently and has not announced a collaboration or licensing partnership with a large pharmaceutical company for its lead candidates. The company has stated it retains worldwide commercial rights to its DMD and DM1 programs.