Assessing Lentiviral Cell Therapy for Cystinosis: Stephanie Cherqui, PhD


The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD

"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.

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
Related Videos
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
M. Peter Marinkovich, MD, on Bringing RDEB Treatment to the Local Level
Caspian Oliai, MD, MS, the medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center
Frederick “Eric” Arnold, PhD
Genovefa (Zenia) Papanicolaou, MD, an infectious diseases specialist at Memorial Sloan Kettering Cancer Center
Jeffrey Chamberlain, PhD, on Exciting New Research at MDA 2024
Alan Beggs, PhD, on Challenges in Therapeutic Development for Rare Diseases
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
PJ Brooks, PhD
John DiPersio, MD, PhD, the director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine
© 2024 MJH Life Sciences

All rights reserved.