UCLA receives $7.3 million grant to build state-of-the-art facility for developing gene, cell therapies

UCLA has received a $7.3 million grant from the National Institutes of Health to build a state-of-the-art facility in which to produce gene and cell therapies aimed at treating a host of illnesses and conditions.

The new 13,000-square-foot facility, to be constructed in UCLA’s Center for the Health Sciences, will provide a highly regulated environment with features such as systems to manage air flow and filtering, laboratory spaces and bioreactors. The new facility is expected to be ready for use in 2023.

“This grant provides critical funds to build a facility that will enable the development of a new generation of cellular therapies for cancer and other deadly diseases,” said Dr. Antoni Ribas, a UCLA professor of medicine and director of the Parker Institute for Cancer Immunotherapy Center at UCLA.

The new facility will be built according to U.S. Food and Drug Administration good manufacturing practices, a set of guidelines intended to ensure that facilities producing products for human use are built to maximize safety and effectiveness, and to reduce the risk for contamination.

It will replace a facility in UCLA’s Factor Building that UCLA scientists currently use for similar research. But that space, which was put together by combining existing research laboratories, lacks the capacity to process certain cells and handle other bioengineered products, and it cannot accommodate the growing number of UCLA scientists pursuing research on gene and cell therapies, said Dr. Stephen Smale, vice dean for research at the David Geffen School of Medicine at UCLA and principal investigator of the NIH grant.

“The new facility will be larger, so it will be able to support more projects simultaneously, and its design  will allow a smooth flow of products into and out of the facility,” Smale said. “The larger number of rooms is really important because even when a single therapy is being tested, cells from each patient need to be processed in their own room.”

Dr. Eric Esrailian, chief of the UCLA Vatche and Tamar Manoukian Division of Digestive Diseases, is helping to lead the expansion of UCLA’s immunology and immunotherapy efforts. “It will be a cornerstone for UCLA’s commitments to building on existing strengths in the areas of immunology and immunotherapy and expanding toward the creation of a transformational institute in these fields,” he said.

Despite the shortcomings of the current space, UCLA researchers have still produced groundbreaking work in it. These include tumor-targeting therapies developed by Ribas, Dr. Donald Kohn, Dr. Linda Liau, and other UCLA researchers.

Ribas, Kohn and Liau are also members of the UCLA Jonsson Comprehensive Cancer Center and the UCLA Broad Stem Cell Research Center. Kohn is a distinguished professor of microbiology, immunology and molecular genetics and Liau is chair of UCLA’s department of neurosurgery.

Kohn, who also developed a cure for “bubble baby syndrome,” said he will welcome the new facility because of its increased capacity for researchers to pursue treatments and cures that could significantly improve the health and quality of life of so many people. For instance, it will have the capacity to produce large batches of viral vectors — microbes that make it possible to introduce potentially curative genes into cells — for gene therapy studies.

“This new facility will allow the innovative cell and gene therapies pioneered at UCLA to be available to a wider number of patients and accelerate the development of novel cures,” said Kohn, whose work has also led to an experimental stem cell gene therapy for sickle cell disease that is showing promising early results in clinical trials.

Liau, a neuro-oncologist, said the new facility will enable researchers to create personalized vaccines and cell therapies for a much larger number of patients.

“In the current facility, we are only able to enroll one patient at a time in our cell therapy trials, so many eligible patients have had to be turned away,” Liau said. “With greater capacity to manufacture gene and cell therapy products that meet FDA good manufacturing practice standards, this new UCLA facility will really allow us to further innovate and accelerate our translational research toward a cure for brain cancer.”