OM #04

We showed in the mouse model of Cystinosis that bone marrow stem cells, especially the hematopoietic stem cells (HSCs), holds great promise to treat Cystinosis. Because the use of allogeneic (foreign) HSCs is very risky, our goal is to develop an autologous HSC transplantation for Cystinosis, i.e. the use of the patients’ own stem cells, that will be gene-corrected to introduce a functional CTNS gene using a lentiviral vector (safe version of an HIV-derived vector). The overall objective of this project is to complete the definitive preclinical and manufacturing studies, and to submit an Investigational New Drug (IND) application to support a Phase 1/2 clinical trial, which will be a first-in-human autologous stem cell and gene therapy treatment strategy for Cystinosis. The clinical trial will be conducted at the University of California, San Diego (UCSD)