UCLA has received a $2 million grant from the California Institute for Regenerative Medicine to increase the capacity of its gene and cell therapy manufacturing facility and workforce.
Established in 1993 – one of the first university-owned facilities of its kind in the U.S. – the UCLA Human Gene and Cell Therapy Facility has supported more than 25 clinical trials aimed at treating a range of cancers, HIV/AIDS, sickle cell disease, severe combined immune deficiency, other inherited blood cell disorders and blinding eye conditions. Thanks to the success of these trials and advances in the fields of stem cell research and regenerative medicine, including CAR-T cell therapies and gene editing, the number of clinical trials of gene and cell therapies at UCLA and beyond has increased dramatically.
Like all pharmaceuticals, gene and cell therapies must be produced under conditions that meet rigorous U.S. Food and Drug Administration safety standards – known as Good Manufacturing Practices, or GMPs, before they can be tested in humans. GMPs include regulations that govern the manufacturing processes, facilities, equipment, documentation and personnel involved in the production therapeutics to ensure their safety, quality and efficacy.
Current GMP infrastructure nationwide is not sufficient to support the rapidly growing clinical pipeline of gene and cell therapies. In recognition of this, a number of funding agencies are focusing on solving bottlenecks to expand capacity. In 2020, UCLA received a $7.3 million grant from the National Institutes of Health to build a new, state-of-the-art facility that will enable the development and manufacturing of these therapies on a larger scale. The new facility will significantly expand the university’s capacity to support clinical manufacturing.
The new funding from CIRM will complement the NIH grant by building the infrastructure necessary to attract, train and retain qualified staff capable of manufacturing products for a wide range of cell and gene therapy trials.
“This grant will enable us to hire essential manufacturing staff and create a strong foundation to efficiently scale this team to meet increasing demand in the future,” said Dr. Dawn Ward, medical director of the UCLA Human Gene and Cell Facility and associate clinical professor of pathology and laboratory medicine at the David Geffen School of Medicine.
To attract this critical workforce, Ward and her team will begin by building an outreach program to raise awareness of career opportunities in GMP facilities among a variety of audiences, with a particular focus on reaching groups that are typically underrepresented in scientific careers.
This includes students enrolled in CIRM-supported training programs such as COMPASS, a two-year regenerative medicine educational experience for UCLA undergraduates, and the CSUN-UCLA Stem Cell Scientist Training Program, which provides Cal State Northridge undergraduates with internships in UCLA research labs.
Additional grant workforce development and retention activities include creating mentoring programs for leadership positions, providing career development opportunities and partnering with industry to build paid training programs for people in technical positions.
The new funding will also be used to develop systems to help investigators and their teams overcome additional roadblocks to producing gene and cell therapies. This includes implementing an electronic quality management system, expanding analytical capabilities and standardizing processes to increase efficiency and reduce risk.
The grant awarded to UCLA was one of five infrastructure grants announced Wednesday by CIRM with the overall objective of establishing a statewide manufacturing network to speed up and de-risk pathways to commercializing cell and gene therapies, advance industry standards and build a diverse, highly skilled manufacturing workforce in California.
UCLA’s Human Gene and Cell Therapy Facility is a collaboration of the David Geffen School of Medicine, the Human Gene and Cell Therapy Program, the Jonsson Comprehensive Cancer Center and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.