Testing Centers for Evaluation of Somatic Cell Genome Editing Approaches for Translational Applications (U42 Clinical Trial Not Allowed)

About this grant

This Notice of Funding Opportunity (NOFO) invites applicants to support Testing Centers for Evaluation of Somatic Cell Genome Editing Approaches for Translational Applications. Each center will provide resources and testing services to the growing community, developing new genome editing technologies and conducting preclinical testing based on these technologies for therapeutics for human diseases. The testing centers will work with NIH-funded investigators to assess the efficacy and safety of genome editing and delivery technologies, determine genome editing thresholds for specific diseases associated with minimal off-target effects, and ascertain feasibility parameters in model systems. This NOFO is especially interested in the application of new approach methodologies (NAMs) to complement traditional in vivo models for the purposes described in this announcement. Research Initiative Details The Testing Centers will establish in vivo and in vitro research models applicable to human health and disease, including NAMs and human-biology-based models. The Testing Centers will develop assays and Standard Operating Procedures (SOPs) for evaluating new genome editing technologies. Data generated by the Centers will help determine whether a treatment strategy using a specific set of tools is effective, has an acceptable safety profile, or requires significant improvement in editing efficacy and specificity. Although each Testing Center will work on a limited number of focused projects, the program and pipeline(s) developed are expected to support multifaceted research activities, build core model systems that can be adapted to accommodate a broad spectrum of diseases, and provide fee-for-service resources to the wider biomedical community on a local, regional, and national basis. The goal of projects supported by this NOFO is to provide research resources that facilitate optimization and enhance scientific rigor, transparency, and experimental reproducibility in biomedical research.