Within the last decade, novel cell and gene immunotherapies have been rapidly translated into clinical practice, where they have improved cancer outcomes and become a therapy-of-choice for adults with myeloma and lymphoma. While there is an approved immunotherapy product available to treat pediatric acute lymphocytic leukemia (ALL), there are no other licensed products for children and adolescents, in part due to lack of commercial interest. Therefore, pediatric clinical trials are conducted mainly by academic investigators. Manufacturing of cell and gene therapy products by for-profit companies is prohibitively expensive for academic investigators. The Good Manufacturing Practices (GMP) Fac...

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Within the last decade, novel cell and gene immunotherapies have been rapidly translated into clinical practice, where they have improved cancer outcomes and become a therapy-of-choice for adults with myeloma and lymphoma. While there is an approved immunotherapy product available to treat pediatric acute lymphocytic leukemia (ALL), there are no other licensed products for children and adolescents, in part due to lack of commercial interest. Therefore, pediatric clinical trials are conducted mainly by academic investigators. Manufacturing of cell and gene therapy products by for-profit companies is prohibitively expensive for academic investigators. The Good Manufacturing Practices (GMP) Facility at Baylor College of Medicine meets the needs of academic investigators by providing viral vectors and cells at cost. The facility received a "Children's Access to Regenerative Medicine in Texas" (CARMIT) Core Facility Award from CPRIT to support clinical trials for pediatric patients in Texas. During the last five years of the award, more than 130 Texas children received novel cell and gene therapies manufactured by our GMP facility to treat blood and solid cancers. We hope to continue closing the gap in inequitable health care delivery and improving outcomes in children by: (1) providing novel, cutting-edge autologous cell therapy products, (2) manufacturing off-the-shelf cell banks from healthy donors that are available for immediate treatment, (3) implementing the manufacture of new vectors to genetically modify cells and shorten manufacturing times, and (4) developing new methods to modify genes in cell therapy products, making them safer and more effective. We propose to continue producing novel cell and gene therapies that support academic investigators and allow a larger pool of pediatric patients to access these therapies, ultimately ensuring that our GMP facility remains among the most advanced in the USA and worldwide.

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