The PaVe-GT pilot project is a collaborative effort that applies NCATS’ translational science model by bringing experts from multiple Institutes and Centers together to work as a single team. These collaborators will work together to test a translational science hypothesis: Can researchers significantly increase the efficiency of gene therapy trial startup by using the same capsid and manufacturing methods for different AAV gene therapies, thereby minimizing redundancies in the preclinical development of gene therapies for extremely rare diseases?
National Center for Advancing Translational Sciences
NCATS is applying its collaborative translational science model to transform understanding, diagnosis and treatment of rare diseases. NCATS’ fundamental approach is to shift from considering each rare disease in isolation to identifying and developing treatments for rare diseases based on their commonalities. NCATS invests resources and expertise at the points where research is most difficult and therefore often abandoned. Translation is a team sport. The NCATS Division of Preclinical Innovation (DPI), Office of Rare Diseases Research (ORDR), and Office of Strategic Alliances (OSA) work together to achieve PaVe-GT objectives.
National Human Genome Research Institute (NHGRI)
The NHGRI Division of Intramural Research develops and implements technology to understand, diagnose and treat genomic and genetic diseases. The NHGRI Organic Acid Research Section, led by Dr. Charles Venditti, focuses on the genetics, pathophysiology and treatment of the hereditary methylmalonic acidemias and disorders of intracellular cobalamin metabolism.
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Dr. Forbes D. Porter, M.D., Ph.D. is the Clinical Director for NICHD and serves as the Clinical Director for NCATS.
National Institute of Neurological Disorders and Stroke (NINDS)
Investigators in the NINDS intramural program conduct research in the basic, translational and clinical neurosciences. The NINDS Neuromuscular and Neurogenetic Disorders of Childhood Section, led by Dr. Carsten Bonneman, focuses on early-onset neuromuscular disorders of childhood, on their genetic and molecular pathogenesis and the pathways involved, and on developing preclinical translational models to lead to actual clinical trials.
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