# PepGen PepGen is an Asset Manager based in Boston, United States. ## Overview - **Organization type:** Asset Manager - **Headquarters:** Boston, United States - **Region:** North America - **Address:** Boston, MA, United States - **Founded:** 2018 - **Assets under management:** Undisclosed - **Website:** pepgen.com - **LinkedIn:** https://www.linkedin.com/company/pepgentx ## Regulatory record - **Reports private funds:** No ## About PepGen launched in 2018 with foundational science from Matthew Wood's lab at the University of Oxford, where his team advanced the understanding of how conjugating peptides to therapeutic oligonucleotides could improve their escape from cellular endosomes. James McArthur, a veteran of rare-disease drug development, joined early to translate the platform into a pipeline of candidates targeting severe neuromuscular diseases, starting with Duchenne muscular dystrophy (DMD). The company operates from Boston, Massachusetts, with research roots in Oxford, UK. The company's strategy centers on its Enhanced Delivery Oligonucleotide (EDO) platform, a technology that links cell-penetrating peptides to oligonucleotide therapies to increase delivery into muscle tissue. Its lead program, PGN-EDO51, targets DMD patients amenable to exon 51 skipping and entered the Phase 2 CONNECT1-EDO51 trial in early 2024. A second DMD candidate, PGN-EDO53 for exon 53 skipping, is in preclinical development, while PGN-EDODM1 aims to treat myotonic dystrophy type 1 (DM1) by targeting the toxic CUG repeat RNA. In June 2023, the company reported initial positive clinical data from its Phase 1 trial showing PGN-EDO51 produced mean exon skipping levels exceeding two percent in muscle biopsies (per PepGen, June 2023). The platform has attracted equity investment from a syndicate of life-science specialists including RA Capital Management, Boxer Capital, and Samsara BioCapital. In 2022, PepGen raised an initial public offering on Nasdaq, pricing shares at $12 to fund its clinical development programs through key data readouts. As of the end of 2024, the company reported a cash runway into 2026 to support ongoing Phase 2 trials for both DMD and DM1 (per PepGen, November 2024). The firm operates no disclosed adjacent philanthropic or multi-family vehicles. PepGen's architecture is notable for being a publicly traded biotechnology company whose entire pipeline depends on a single, internally developed delivery technology licensed from an academic institution, rather than an in-licensed compound or external partnership model. CEO James McArthur's prior experience includes leading Rare Disease Research at Biogen, where he oversaw the approval of Spinraza, the first approved therapy for spinal muscular atrophy — a background that directly informs PepGen's clinical-pathway focus on genetically defined neuromuscular conditions with high unmet need. ## Sectors - Biotechnology - Healthcare Services ## Offices - Oxford, United Kingdom ## People - James McArthur — President and CEO - Matthew Wood — Co-founder and Scientific Advisor ## Questions ### How does PepGen's Enhanced Delivery Oligonucleotide platform differ from standard oligonucleotide therapies? 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. ### What clinical data has PepGen disclosed for its Duchenne muscular dystrophy program? 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. ### Who leads PepGen's scientific and corporate strategy? 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. ### What other disease targets does PepGen pursue beyond Duchenne muscular dystrophy? 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. ### What is the structure of PepGen's Oxford University relationship? 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. ### Is PepGen exploring partnerships with larger pharmaceutical companies? 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. ## Related profiles - [AMC Robotics Corp](https://altss.com/profile/amc-robotics-corp) - [Giant Mining Corp.](https://altss.com/profile/giant-mining-corp) --- Last updated: 2026-06-03T20:00:00.000Z Canonical page: https://altss.com/profile/pepgen-inc Maintained by Altss — https://altss.com — methodology: https://altss.com/methodology