From research to clinical manufacturing: advancing gene-edited HSC therapies through collaboration

Stephanie Cherqui is Professor in the Department of Pediatrics, Division of Genetics at the University of California San Diego, Director of the UC San Diego Gene Therapy Initiative, and Ben and Wanda Hildyard Endowed Chair for Mitochondrial and Metabolic Diseases. Her research focused on developing hematopoietic stem cell gene therapy for metabolic and neurological disorders, with a strong track record of translating fundamental discoveries into clinical applications. Among her most significant achievements is the development of the first-in-human autologous hematopoietic stem cell gene therapy for cystinosis, an inherited metabolic disorder. Dr Cherqui has leveraged these discoveries to expand her therapeutic approach to multiple disorders such as Friedreich’s ataxia, mucopolysaccharidosis type IIIC, and Alzheimer’s disease. She is the Chair of the Cystinosis Stem Cell and Gene Therapy Consortium, the Chair of the Scientific Review Board of the Cystinosis Research Foundation, and a member of the ASGCT Gene and Cell Therapy of Genetic and Metabolic Diseases committee.

Dr Kaufman is a Professor in Department of Medicine, Divisions of Regenerative Medicine and BMT. At UCSD, he does clinical work in hematology/BMT and serves as Scientific/Medical Director of the UCSD Advanced Cell Therapy Laboratory (ACTL) that provides GMP cell manufacturing to translate new cell-based therapies to clinical trials. Research in the Kaufman lab uses human pluripotent stem cells to understand the development of blood and immune cells. Recent studies have focused on the ability to use human iPSC-derived natural killer (NK) cells to kill diverse types of human cancer cells. These studies have used cellular engineering to enhance the anti-tumor activity of the iPSC-derived NK cells by various strategies. This work has now been translated into clinical trials for treatment of relapsed/refractory cancers- both hematologic malignancies and solid tumors. Additional studies demonstrate efficient production of macrophages from human iPSCs. These cells can be used for treatment of non-malignant diseases or engineered to mediate anti-tumor activity. His group has also developed novel targeted virus-like particles for in vivo engineering of immune cells to express CARs that mediate anti-tumor activity.

Namritha Ravinder is Director of R&D for Cell and Gene Therapy Platforms at Thermo Fisher Scientific. Since joining the company in 2008, she has led R&D efforts spanning cell engineering, gene delivery, genome editing and modulation, viral vector production, and CRISPR technologies. In her current role, Namritha leads R&D efforts focused on workflow automation and the development of closed, automated platforms for cell and gene therapy manufacturing, spanning cell processing, gene delivery and cell engineering, and formulation and fill-finish. She holds a doctorate in Biotechnology from the University of Alabama in Huntsville and completed a postdoctoral fellowship in HIV virology at Children’s Hospital Los Angeles.