UX810

UX810 is an investigational **AAV-based gene therapy** designed for the treatment of Duchenne muscular dystrophy. It delivers a **microdystrophin gene** to patients’ cells using an adeno-associated virus (AAV) vector, with the goal of restoring dystrophin protein function that is otherwise deficient or absent in individuals with Duchenne. The therapy is being developed collaboratively by **Ultragenyx Pharmaceutical** and **Solid Biosciences**. The primary mechanism is gene transfer to enable **dystrophin protein replacement** in muscle tissue, aiming to address the underlying cause of the disease at the genetic level. Current development status is **preclinical**.[1][13][15]

Other names

AAV based microdystrophin gene therapy (Ultragenyx/Solid Biosciences)