Leading International Consortium on Gene Therapy Outcomes

Sonali Chaudhury, MD, Head of Stem Cell Transplantation and Cellular Therapies at the Center for Cancer and Blood Disorders, is leading an international consortium focused on gene therapy outcomes. 

The Real World Experience Consortium for Commercial Gene Therapy Products in Non-Malignant Disorders (ReGenT) is comprised of more than 55 pediatric and adult qualified/approved treatment centers in North America, Europe and Asia that provide FDA-approved ex-vivo genetically modified hematopoietic stem cells to patients with nonmalignant blood disorders. 

The consortium’s mission is to foster collaboration by creating a multi-institutional forum that will guide strategic planning, create peer-based learning platform and approaches and harmonize treatment and data collection across gene therapy centers to establish standardized practices and describe clinically relevant outcomes using commercial products.

The consortium is planning to conduct clinically relevant projects that will potentially improve prediction of outcomes that impact patients and families at every step of the process. Dr. Chaudhury and her colleagues are currently investigating apheresis collection and product manufactured. In the real-world setting, a significantly higher proportion of patients, especially with sickle cell disease, need more collection cycles with alternate mobilization strategies than previously described in the clinical trials. This project will help improve standards of apheresis collection and potentially develop predictive models to guide alternate strategies.

CAR T-Cell Therapy Insights from Lurie Children’s-Hosted Symposium

Lurie Children’s proudly hosted the 2025 INSPIRED Symposium, which attracted 350 physicians in the pediatric CAR T-cell therapy field. Experts from 16 countries and 84 pediatric institutions met to strengthen collaboration and improve future outcomes for patients receiving this cutting-edge treatment. More than 40 speakers, including Kevin McNerney, MD, from the Center for Cancer and Blood Disorders, spotlighted cutting-edge work in leukemias, solid tumors and autoimmune diseases, with emphasis on current best practices and unmet needs.

View videos of symposium presentations

Pivotal CAR T-Cell Trials for Pediatric Autoimmune Conditions

Hannah Lust, MD, from the Stem Cell Transplant Program at the Center for Cancer and Blood Disorders, and Pooja Patel, DO, from Rheumatology, are spearheading a groundbreaking collaboration that brings advanced cell-based therapies — traditionally used in oncology — to transform care for pediatric and young adult patients with severe autoimmune conditions. This interdisciplinary effort leverages Lurie Children’s expertise in cellular therapy to address complex, refractory rheumatologic diseases that have been challenging to manage with existing therapies.

The program launched two actively enrolling clinical trials utilizing CAR T-cell therapy. CAR T-cells are engineered to target cells that are the source of pathogenic antibodies in rheumatologic diseases. Eliminating these cells decreases inflammation and related symptoms. The RESET-Myositis trial targets idiopathic inflammatory myositis in patients as young as 6 years old. The AUTOGRAPH – SLE/LN study is focused on refractory systemic lupus erythematosus and lupus nephritis currently in patients aged 18 and older.

These pivotal trials represent a major step forward in precision medicine. Drs. Lust and Patel are combining the diagnostic precision of rheumatology with the technical capabilities of cellular therapy, offering new avenues of hope and medication-free cure, ultimately to improve patient quality of life.