Module232025

Cell Therapy for Pediatrics : Key Points

Applications : Used in regenerative medicine, immunotherapy, and cancer therapy via autologous/allogeneic cells delivered through injectables, bioscaffolds, or scaffold-free systems. Market Growth : Global cell therapy market growing at ~17% CAGR (2023 – 2030). Stem Cell Therapies : o PSCs, ASCs, and CSCs utilized for organ replication and cancer targeting. o Approved treatments: hematopoietic/mesenchymal stem cells for blood cancers; recent FDA approval includes Omisirge®. Challenges and Research : 202 pediatric stem cell studies mainly small, single-center trials; larger multi-center studies are needed. Non-Stem-Cell Therapies : Immune cells (e.g., T/NK cells), fibroblasts, hepatocytes, and islet cells serve therapeutic, preventive, or diagnostic roles.

Quality (CMC) Development Considerations

The Organisation for Professionals in Regulatory Affairs