DTX101

DTX101 is an adeno-associated virus (AAV) gene therapy candidate developed for the treatment of hemophilia B. It utilizes the AAVrh10 serotype, a non-human primate-derived vector, to deliver a functional copy of the human coagulation Factor IX (FIX) gene to the liver. The AAVrh10 serotype was selected for its potential to bypass pre-existing immunity in the human population, which is a common challenge for other AAV-based therapies. Originally developed by Dimension Therapeutics, the program was acquired by Ultragenyx Pharmaceutical in 2017. The therapy is designed to provide long-term, stable expression of FIX following a single intravenous infusion, thereby reducing or eliminating the need for frequent prophylactic factor replacement therapy in patients with moderate-to-severe hemophilia B.