Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one time through a venous catheter inserted into a patient's peripheral limb vein. A tapering course of prophylactic...
Gene Therapy
ABO-102 Gene Therapy | Type IIIA | Phase I-II | Ultragenyx
Page reviewed by: Dr. Cara O'Neill, FAAP Page last updated: July 29, 2022 For web accessibility options: Click/tap the floating blue icon on the right. The ABO-102 Gene Therapy uses a self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene...
LYS-SAF302 Gene Therapy | Type IIIA | Phase II-III | Lysogene
Page reviewed by: Dr. Cara O'Neill, FAAP Page last updated: November 21, 2022 For web accessibility options: Click/tap the floating blue icon on the right. Update: Nov. 21, 2022 Lysogene recently issued a press released regarding their Type A gene therapy study. In...