NIH Reporter #5R01NS136487-02

Research Solicitation: Microglia Replacement Therapy Using Engineered HSCs for Leukodystrophies

This opportunity is a research solicitation from the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), focused on developing microglia replacement therapy for leukodystrophies using engineered hematopoietic stem cells (HSCs). - Government Buyer: - National Institutes of Health (NIH) - National Institute of Neurological Disorders and Stroke (NINDS) - Research Location: - University of Pennsylvania, Philadelphia - Products/Services Requested: - Research services to develop immune cell therapy for Krabbe disease - Use of engineered HSCs for microglia replacement - Gene therapy development and validation - Preclinical testing in mouse and nonhuman primate models - Unique/Notable Requirements: - Emphasis on clinical feasibility and safety - Use of large animal (nonhuman primate) models for translational research - No specific commercial products, part numbers, or OEMs identified - No specific OEMs or vendors are named in the solicitation - The opportunity is for research services, not for the procurement of goods or equipment

Description

This project aims to develop a clinically feasible immune cell therapy for Krabbe disease using engineered hematopoietic stem cells. It involves microglia replacement, gene therapy, and the use of large animal models. The research will validate the approach in mouse models and demonstrate safety and translatability in nonhuman primates. The project is funded by the National Institute of Neurological Disorders and Stroke (NINDS) and involves collaboration between the Bennett and Kiem labs at the University of Pennsylvania.

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