State-of-the-art GMP facility for the development of lifesaving gene and cell therapies - Summary UCLA began investigations into cell and gene therapies in the early 1990’s and was one of the first academic medical centers to establish a Good Manufacturing Practices (GMP) facility to provide a highly regulated environment for producing novel therapeutics for clinical research subjects. Over the past 25 years, the UCLA Human Gene and Cell Therapy Facility has supported more than 20 clinical trials directed against cancer (e.g. melanoma, sarcoma, glioblastoma multiforme, non-small cell lung cancer), HIV/AIDS, Sickle Cell Disease, Severe Combined Immune Deficiency (SCID), other inherited blood cell disorders, and Age-Related Macular Degeneration. The SCID therapy has been successfully used for an apparent cure of more than 40 pediatric patients with ADA SCID, a previously fatal childhood disease. Other trials, including a trial of a dendritic cell therapy for glioblastoma and engineered T cell/dendritic cell trials for melanoma and other malignancies, have provided additional promising results. With UCLA’s own successes and the recent worldwide successes with CAR-T cell therapies and other gene therapies, a dramatic expansion of cell and gene therapy trials is occurring. Unfortunately, UCLA’s current GMP facility was not purpose built, but was constructed by combining several research laboratories. It lacks several key features of a GMP facility and has insufficient capacity to handle the current and future load of cell product and bioengineered product manufacturing that will derive from multiple therapies in the development pipeline. Thus, a new GMP facility is essential to support the many advances in translational therapies that are created across the UCLA campus, primarily in the David Geffen School of Medicine (DGSOM), but also in the College of Letters and Science, the Broad Stem Cell Center, the Jonsson Comprehensive Cancer Center, and the School of Engineering. The construction plan for a new GMP facility was developed on the basis of our own experiences and those of others, by a committee established by the DGSOM Dean’s Office that interviewed local stakeholders, external leaders in the establishment and operation of GMPs, bioengineers, and architects. The Facility will be located in a large, 13,000 sq. ft. space in UCLA’s Center for Health Sciences (CHS). The proposed state-of-the-art facility is designed with essential features, such as unidirectional flow, appropriate air handling systems with terminal HEPA filters and air pressure controllers for individual rooms, adequate support spaces, including a centralized quality testing laboratory and cleanrooms for bioengineering projects to accommodate larger equipment, including bioreactors and 3-D printers. Given our established expertise in manufacturing a variety of cellular therapy products for a wide range of therapeutic indications, the experienced support staff needed to run a successful GMP facility and the strong commitment from the UCLA leadership, construction of the proposed new Human Gene and Cell Therapy Facility will allow UCLA to continue at the forefront in the development and clinical evaluation of novel therapeutics for many severe medical conditions.
