San Diego-based Novoron Bioscience, Inc. has received a USD 1.25 million NIH R44 SBIR grant from the National Institute on Aging to develop a human iPSC-derived brain organoid platform for screening small molecules that block tau propagation in Alzheimer's disease and related tauopathies.
The award supports a high-throughput screening system built on cortical organoids derived from human induced pluripotent stem cells, with wild-type and LRP1 knockout lines used to validate the platform's sensitivity to changes in tau propagation. LRP1 (low-density lipoprotein receptor-related protein 1) has established roles in tau uptake and transsynaptic spread, making it a mechanistically grounded control point for compound screening. The consortium — comprising Novoron alongside BrainStorm Therapeutics and Defined Bioscience — aims to identify small molecules that disrupt tau-LRP1 interactions, addressing a target class with no approved pharmacological agents. The team previously used a comparable organoid approach in Rett syndrome research to identify donepezil (Aricept), which is now entering a Department of Defense-funded Phase II trial, lending translational credibility to the platform strategy.
The grant reflects sustained NIH interest in human-relevant preclinical models as alternatives to animal systems that have historically failed to predict clinical outcomes in neurodegeneration. Organoid-based drug discovery has demonstrated utility in cystic fibrosis and colorectal cancer, and the Novoron award signals expanding application of the approach to tau biology specifically.
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