Ongoing studies with Umoja Biopharma, HDT Bio, and the Allen Institute
Nonhuman primate studies are crucial in biomedical research, particularly developing new therapies. The pigtail macaque monkeys at the Washington National Primate Research Center share a closer genetic and physiological resemblance to humans than other animal models. This similarity allows researchers to predict better how a therapy might behave in humans, offering valuable insights into potential efficacy and safety.
Such is the case with Umoja Biopharma’s VivoVec platform for chimeric antigen receptor T (CAR-T) cell therapy. In this in vivo study, the researchers observed the activation and expansion of CAR-T cells in the monkeys and their impact on target cells. The researchers, supported by the animal care and research staff at WaNPRC, compared the CAR-T cell expansion and persistence in monkeys with data from previous studies on ex vivo CAR-T therapies in similar primate models. This provided valuable insights into the platform’s potential advantages.
Overall, this public/private partnership is a critical bridge between preclinical research and clinical translation, providing essential data on the safety, efficacy, and feasibility of new therapies like Umoja’s VivoVec platform for CAR-T cell therapy.
A separate project led by HDT Bio Formulations Director, Dr. Amit Khandar, aims to assess the effectiveness of their LION™ delivery technology and self-replicating RNA (repRNA) vaccine platform against HIV-1. The focus is on inducing broadly neutralizing antibodies (bNAbs), crucial for targeting the heavily masked envelope glycoprotein of the virus. In the R33 phase, the collaboration with UW’s Fuller Laboratory and the WaNPRC will evaluate immunogenicity and efficacy in nonhuman primates (NHPs), pivotal steps before potential clinical trials. This phase will provide essential data on protection against a SHIV challenge and guide decisions regarding human trials. Safety assessments, especially concerning mRNA-based vaccines, are emphasized, drawing from mouse models and NHPs to address concerns ensuring confidence in the vaccine’s safety profile. The ultimate goal is to advance toward clinical trials, leveraging promising results from NHP studies to inform future steps in vaccine development.
“Ultimately, it might be that a combination of nucleic acid platforms will be needed to achieve the “holy grail” that is efficacy in humans. The combination of safety, immunogenicity, and efficacy in nonhuman primates will support the decision to enter clinical trials. If the data is promising, we have the manufacturing infrastructure to work with existing HIV clinical trial networks and enter phase I evaluation. – Dr. Amit Khandhar, HDT Bio
Scientists at the Allen Institute celebrate their global collaboration in mapping the hundreds of billions of cells in rodents, macaque monkeys, and human brains. They are mapping them by their type and function as part of the BRAIN Initiative® Cell Atlas Network, or BICAN, with financial support from NIH’s B.R.A.I.N. Initiative, or Brain Research Through Advancing Innovative Neurotechnologies® Initiative.
The WaNPRC, by providing the Allen Institute’s NHP models, is helping to bridge the knowledge gap between rodent models and humans.
“The Allen Institute has made amazing progress characterizing the cell types that compose the mouse brain and connections among them. Extending these efforts to primates is a critical next step on the way to clinical application. This is being achieved via the symbiotic relationship they have with the WaNPRC.” – Dr. Gregory Horwitz, WaNPRC Neuroscience unit chief
This brain atlas lays the groundwork for mapping the entire mammalian brain and enhancing comprehension of puzzling brain disorders, such as those affecting the neurons responsible for motor function, like amyotrophic lateral sclerosis (ALS).