"We still don’t fundamentally know how therapeutics get into cells. Solving that is now in human trials for Duchenne." - Milken Global 2025

Entrada Therapeutics’ CEO on a platform that carries therapeutics into cells and directs them where they need to go: functional cures for Duchenne muscular dystrophy now in clinical trials in Europe, a myotonic dystrophy partnership with Vertex, how rare-disease trials find patients through advocacy groups, and why newborn screening is the key to treating early.