The company, PepGen, has decided to stop developing PGN-EDO51. The treatment was being tested for Duchenne muscular dystrophy in people who might benefit from exon 51 skipping. This means all testing of the treatment will now come to an end.
Development of Duchenne muscular dystrophy treatment PGN-EDO51 stopped
29 May 2025
Researchers designed PGN-EDO51 as an experimental genetic therapy to help the body produce dystrophin, a protein that supports muscle strength, by skipping exon 51 of the dystrophin gene. People with Duchenne muscular dystrophy (DMD) do not produce enough dystrophin, leading to muscle weakness.
Unfortunately, data from the CONNECT1-EDO51 clinical trial suggested the treatment didn’t lead to the production of enough dystrophin to offer a meaningful benefit. As a result, the research team decided to stop further testing, to avoid exposing people to a treatment that is unlikely to help.
PepGen also planned to start another clinical trial in the UK, called CONNECT2-EDO51. This trial will no longer go ahead.
While we’re disappointed PGN-EDO51 will not move forward, researchers could use the insights from this study to help guide the development of future treatments for DMD.
If you or your family member has been involved in the study, we recommend you contact the study site for any further information. PepGen has also released a letter to the community.
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