PepGen is a biotechnology company working to develop innovate treatments for myotonic dystrophy. This week, the company announced interim results from its Phase 2 clinical study FREEDOM2-DM1. This study is evaluating PepGen’s investigational therapy, PGN-EDODM1, in people with myotonic dystrophy type 1 (DM1). The main objectives at this stage of the study are to assess the safety and tolerability of the treatment.
PGN-EDODM1 is designed to target the underlying cause of DM1 by preventing toxic RNA from disrupting normal cell function.
Interim results have shown that this treatment was generally well tolerated, with improvements in RNA mis-splicing and early trends towards improvement across multiple functional outcome measures.
DM1 is a genetic muscle wasting condition that can affect many parts of the body, including muscle strength, movement, heart function and breathing. Currently, there are no approved treatments that target the underlying cause of DM1.
PGN-EDODM1 remains an experimental treatment and further research is needed to determine its effectiveness.