Assessing Lentiviral Cell Therapy for Cystinosis: Stephanie Cherqui, PhD

"For the stem cell manufacturing, the patients come to UCSD and they go through stem cell mobilization, and we take out their white blood cells during leukapheresis. The white blood cells is transported to the good manufacturing practice facility at UCLA where the CD44 hematopoietic cells are gene modified using a lentivirus vector.”

AVROBIO is developing CTNS-RD-04, an autologous, lentiviral, CD34+ cell therapy encoding for cystinosin for the potential treatment of cystinosis. Stephanie Cherqui, PhD, associate professor, University of California – San Diego, is developing the therapy in her lab and evaluating it for safety and efficacy in an ongoing phase 1/2 trial (NCT03897361).

Cherqui presented interim data from the first 3 patients treated in the trial at the 18th Annual WORLDSymposium, February 7-11, 2022, in San Diego, California. No treatment-related adverse events (AEs) or serious AEs have been reported. All patients exhibited a decrease in white blood cell cystine and are no longer taking oral cysteamine. Promising decreases in cystine were also noted in the cornea, skin, and kidneys.

CGTLive spoke with Cherqui to learn more about the trial, including trial protocols and CTNS-RD-04's mechanism of action.

REFERENCE

Cherqui S. Hematopoietic stem cell gene therapy for cystinosis: Updated results from a phase I/II clinical trial. Presented at: 18th Annual WORLDSymposium, February 7-11, 2022; San Diego, CA. Poster #46