We are a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases. Our Enhanced Delivery Oligonucleotide, or 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.

We were founded in 2018 with technology spun out from the University of Oxford and the Medical Research Council of United Kingdom Research and Innovation to further develop and commercialize this novel peptide delivery approach. This technology was created and refined over a decade by Michael Gait, Ph.D. and Professor Matthew Wood, M.D., Ph.D. We have exclusively licensed the patents, patent applications and know-how associated with this technology.

Our EDO peptides are engineered to optimize tissue penetration, cellular uptake and nuclear delivery, and in preclinical studies we have observed their ability to transport oligonucleotides into a broad range of target tissues, including smooth, skeletal, and cardiac muscle and the central nervous system, or CNS. 

We are currently in clinical-stage development, with our lead product candidate, PGN-EDO51, having entered the clinic in the second quarter of 2022. We are developing PGN-EDO51 to treat individuals with Duchenne muscular dystrophy, or DMD, whose mutations are amenable to an exon 51-skipping therapeutic approach. An exon is a segment of a gene that – together with other exons – contains the code that is translated into a protein. Exon skipping is a therapeutic modality that enables mutations in the gene to be bypassed, thereby repairing this code and enabling production of a truncated, yet functional version of the target protein. Following the review of our preclinical dataset by Health Canada and subsequent authorization of our Clinical Trial Application, or CTA, we initiated a Phase 1 clinical trial of PGN-EDO51 in healthy normal volunteers, or HNV, and we anticipate receiving topline data from this trial by the end of 2022.

We are also developing PGN-EDODM1 for the treatment of myotonic dystrophy type 1, or DM1, for which we anticipate submitting an investigational new drug, or IND, application in the first half of 2023, and PGN-EDO53 for the treatment of DMD patients whose mutations are amenable to an exon 53-skipping therapeutic approach, for which we anticipate reporting exon skipping data in NHPs in the second half of 2022. Alongside these therapeutic candidates, we have initiated research efforts on EDO therapeutics for further DMD exon-skipping populations, including exon 45- and exon 44-skipping amenable patients, and for additional indications, including neuromuscular diseases and neurologic disorders. We anticipate advancing additional programs into CTA- and IND-enabling studies in 2024.

(Note: PepGen, Inc. slightly upsized its IPO at pricing on May 5, 2022, by increasing the number of shares to 9.0 million, up from 7.2 million shares in the prospectus, and pricing the IPO at $12 – $1 below the bottom of its $13-to-$15 price range – to raise $108.0 million. The IPO raised $7.2 million more than the estimated proceeds of $100.8 million (assuming $14 mid-point pricing) under the terms in its S-1/A filing (the prospectus). PepGen, Inc. filed its IPO terms in an S-1/A dated May 2, 2022. The S-1 was filed on April 15, 2022.)