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PepGen voluntarily pauses enrollment in the CONNECT2-EDO51 Phase 2 study in UK

March 4, 2025

PepGen has made the voluntary decision to temporarily pause enrollment in the CONNECT2-EDO51 Phase 2 study previously active in the United Kingdom (UK) which was already on hold in the United States (US) studying their investigational product PGN-EDO51, a medicine which is being tested as a treatment for Duchenne muscular dystrophy (DMD). One of the most common causes of DMD is a change or a missing or extra part of the dystrophin gene, called exons. Some medicines, like PGN-EDO51, are medicines that help make to skip the part of the gene that does not work properly. PGN-ED051 is an exon skipping medicine that targets exon 51.

PepGen plans to focus on efforts of acquiring and understanding the data from the 10 mg/kg cohort of PGN-EDO51, which is being tested in a Phase 2 clinical trial in Canada called CONNECT1. In the CONNECT1 trial, the safety and effectiveness of multiple ascending doses (MAD) of PGN-EDO51 are being tested, starting with 5mg/kg. To date, all people in the 5mg/kg dose group have been treated and those who have been or will be treated with a higher 10mg/kg dose have been enrolled. Some have already been dosed and are now in the long-term study of the treatment.

Are you or your child participating in the CONNECT1 trial? Contact us to be personally updated when additional information is available at [email protected]

Read the full press release from PepGen below.


Want to know more about Duchenne Research?

Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment:

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