Currently, cystine depleting therapy using cysteamine is used to treat cystinosis, however, this is a very difficult, non-curative treatment regimen associated with numerous side effects. Stephanie Cherqui, PhD, University of California, San Diego, La Jolla, CA, discusses the updated results of an ongoing Phase I/II trial (NCT03897361) investigating the efficacy of CTNS-RD-04, a novel autologous hematopoietic stem and progenitor cell (HSPC)-based gene therapy, in patients with cystinosis. In the first three male patients treated, CTNS-RD-04 was found to decrease cystine crystal formation, decrease cystine content in white blood cells and improve photophobia. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.