Posted by Michael Wonder on 29 Jul 2018
Ultragenyx announces first patient dosed in Phase 1/2 study of DTX401, a gene therapy for the treatment of glycogen storage disease Type Ia; FDA grants fast track designation to gene therapy program
26 July 2018 - Ultragenyx today announced that the first patient has been dosed in the Phase 1/2 study of DTX401, an adeno-associated virus vector based gene therapy for the treatment of patients with glycogen storage disease Type Ia.
Data from the three-patient first dose cohort are expected in the second half of 2018.
The U.S. FDA has also granted fast track designation to DTX401 for the treatment of glycogen storage disease Type Ia.
