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Frederick National Laboratory for Cancer Research and the National Center for Advancing Translational Sciences Therapeutic Development Branch in the National Institutes of Health have launched the Gene Therapy Immunology and Standards Laboratory and Initiative, a national resource to aid the scientific community. The laboratory promotes standardization in methods for studying and reporting on gene therapies, an emerging field of medicine that treats certain diseases by modifying the mutations that cause them. Ligia Pinto, Ph.D., director of the Frederick National Laboratory’s Vaccine, Immunity, and Cancer Directorate, heads the program. The Gene Therapy Immunology and Standards Laboratory draws on the Frederick laboratory’s strong history of standardization experience. Pinto’s team previously spearheaded the International HPV Serology Standardization Initiative in 2017 and the Serological Sciences Network, which focused on COVID-19. “We have a team with a high level of expertise in immunology and standardization … and also the framework we established for standardization to support clinical trials in other areas. This laboratory [aims] to leverage that knowledge, those tools,” Pinto said. The laboratory will standardize aspects of the gene therapy development process to aid different therapies moving toward clinical trials. When researchers use assays, protocols, standard operating procedures, and validated technologies set to the same standards across different studies, the results become more easily comparable. “Then we all start talking the same ‘language,’” Pinto said. Once that happens, the findings become easier for scientists to reproduce and verify. Conclusions can be drawn with more confidence and reliability. Standardization is useful for “expediting public health decisions,” Pinto said. When reliable data is generated and easily compared, conclusions and decisions are easier to make, leading to a faster and smoother road to clinical trials and, eventually, available treatments. Gene therapies can be tailored toward specific illnesses, making them a prime option for rare diseases and precision medicine. However, because of the way some treatments—including some of those supported by the National Center for Advancing Translational Sciences—are delivered, via nonharmful viral vectors, it’s important to test patients for pre-existing immunity to the treatments to ensure they can safely participate in a clinical trial. This is one area where the laboratory is making a difference. Researchers use blood sera sampling, an area of Pinto and team’s ’s expertise, to detect neutralizing antibodies against the vectors that could compromise the effectiveness of the therapy or increase the risk of adverse immune responses. Because of the importance of having accurate and precise data before testing treatments, Pinto explains, it’s essential that strong, specific, sensitive, and reproducible methods are developed to test sera for these antibody responses. In addition, they are working to develop assays to test how T cells, a type of immune cell, respond to gene therapies. This information can clarify how immune system responses might influence the therapy’s effectiveness. The National Center for Advancing Translational Sciences supports gene therapy development through its work on the Platform Vector Gene Therapy (PaVe-GT) and MMA-101 projects, collaborations within the National Institutes of Health that are using vectors to deliver gene therapies for rare genetic diseases. Standardizing the assays needed to perform these studies will enable researchers to compare results across them and compare the efficacy of different therapies. Future plans to share the process and standardization resources also align with PaVe-GT’s goal of disseminating information to the rare disease and gene therapy communities and can help other researchers develop quality data. For the National Center for Advancing Translational Sciences, PaVe-GT is a way to expedite the pathway toward clinical trials for gene therapies for various rare diseases. The Gene Therapy Immunology and Standards Laboratory and Initiative is another way to hasten the development of high-quality data by creating a framework of standardization that can be applied across many gene therapy studies, making it easier to draw meaning from them. Leveraging the expertise of both groups, as well as that of other stakeholders, will be the key to accelerating what’s possible in gene therapy studies. “It takes a village,” said Pinto. “It takes time, and it takes involvement from many stakeholders. But it’s very important to develop standardization tools and have rich datasets to support and expedite those trials.”