Pepgen Inc. is forging ahead with tests of PGN-EDODM1 in other territories after the U.S. FDA placed a partial hold on the Freedom2-DM1 phase II trial, a multiple ascending-dose, randomized, placebo-controlled experiment in myotonic dystrophy type 1 (DM1).
Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant skeletal muscle disorder with a prevalence of approximately 1 in 8,000. The disorder is driven by aberrant expression of double homeodomain protein 4 (DUX4) within the D4Z4 macrosatellite array. Currently, effective treatments for FSHD are lacking. Strategies aimed at reducing DUX4 expression could hold promise as potential therapeutic approaches for FSHD.
Mainly expressed in the liver, microRNA‑122‑5p (miR‑122) exhibits increased circulating levels in the context of obesity. When elevated in the bloodstream, miR-122 can act on extrahepatic tissues, including vascular endothelial cells, where it contributes to endothelial dysfunction and may promote the development of diabetic vascular complications.
