Familial Hemophagocytic Lymphohistiocytosis (FHL) research hub

MUNC13.4 - A phase I/II Open Label non randomized study, monocentric, single arm, evaluating Safety and Efficacy of Gene Therapy of FHL 3 caused by mutations in the human UNC13D gene by transplantation of a single dose of autologous CD34+ cells transduced ex vivo with the UNC13D LV vector expressing the UNC13D cDNA

This study is testing a new gene therapy for a rare immune condition called Familial Hemophagocytic Lymphohistiocytosis (FHL), specifically type 3. It uses a patient's own modified cells to fix the underlying problem. Doctors are checking if it's safe and if it helps people get better.