About
Changing the course

Our Mission

PepGen is advancing the next generation of oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases.

Our 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. These have long promised to transform healthcare with their ability to specifically target RNA sequences that cause disease, and yet the efficacious delivery of oligonucleotide therapeutics has lagged and remains a major challenge. Our proprietary EDO platform has shown the potential to treat such genetic disorders by optimizing the safe and efficient delivery of therapeutic oligonucleotide cargos into affected tissues and cells, thereby addressing the key challenge that has impeded the clinical translation and validation of this class of molecules. We have leveraged these characteristics to develop a robust pipeline of potentially world-leading therapeutics for the treatment of serious genetic diseases.

PepGen is headquartered in Boston, Massachusetts.

Our Technology

Our EDO platform is the result of a decade of work by pioneering researchers and biotech leaders. The 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. Furthermore, the high levels of pharmacological activity observed in preclinical studies support our belief that our EDO platform technology has the potential to deliver therapeutic agents to the cell nucleus.

We are advancing our two lead programs towards the clinic: one for DMD patients treatable with an exon 51 skipping oligonucleotide and the other targeting myotonic dystrophy type 1 (DM1), both rare genetic diseases with a high unmet medical need. We are also developing three further therapeutic candidates for other DMD patient populations. While our current pipeline focuses on neuromuscular diseases, we believe the broad applicability of our delivery platform should enable us to create breakthrough therapeutics targeting a wide variety of serious diseases.

Meet the Team

Executive Team

James McArthur, PhD

President and Chief Executive Officer

Noel Donnelly, MBA

Chief Financial Officer

Jaya Goyal, PhD

Executive Vice President Research and Preclinical Development

Sonia Bracegirdle, DPhil

Senior Vice President Strategy and Operations

Niels Svenstrup, PhD

Senior Vice President Chemistry, Manufacturing and Controls

Michelle Mellion, MD

Senior Vice President, Head of Clinical Development

Senior Leadership

Jennifer Cormier

Senior Vice President Clinical Operations

Jane Larkindale, DPhil

Vice President Clinical Science

Jeffrey Foy, PhD

Vice President, Toxicology

Isami Salcedo

Vice President of Program Management

Board of Directors

James McArthur, PhD

President and Chief Executive Officer

Laurie Keating, JD

Board Chair

Josh Resnick, MD, MBA

Non-Executive Director

Heidi Henson

Non-Executive Director

Chris Ashton, PhD

Non-Executive Director

Habib Joseph Dable

Non-Executive Director

Scientific Advisors

Matthew Wood, PhD, MD

Professor of Neuroscience, Oxford University

Brenda Wong, MD

Founding Director, UMass Duchenne Program

John Watts, PhD

Associate Professor, UMass Medical School

Charles Thornton, MD

Professor of Neurology & Neuroscience, U Rochester Medical Center

Beth McNally, MD

Director, Center for Genetic Medicine, Northwestern University

Art Krieg, MD

Former President of the Oligonucleotide Therapeutics Society

Michael Gait, PhD

Founder

John W. Day, MD, PhD

Professor of Neurology, Pediatrics (Genetics) and Pathology, Stanford University

Caroline Godfrey, PhD

Founder