Gene Therapy

ABO-101 Gene Therapy | Type IIIB | Phase I-II | Abeona

Page last updated: Feb. 23, 2024 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Summary Information Study Title: “Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.hNAGLU for...

UX111 Gene Therapy | Type IIIA | Phase I-II-III | Ultragenyx

Page last updated: Feb. 16, 2024 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Summary Information The UX111 (formerly ABO-102) Gene Therapy uses a self-complementary adeno-associated virus...

LYS-SAF302 Gene Therapy | Type IIIA | Phase II-III | Lysogene

Page last updated: February 19, 2024 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Summary Study: “Open-label, Single-arm, Multi-center Study of Intracerebral Administration of Adeno-associated...