Before an individualized genetic treatment can reach a patient, it must go through preclinical development, a process that encompasses therapeutic design, laboratory screening, proof-of-concept testing, and early safety evaluation. Over the past several years, this process has yielded numerous successful treatments for patients using n-of-few modalities, including ASOs, base editing, with additional approaches in development. As more academic groups and companies have entered this space, interest in N1C’s Preclinical Design Workgroup has grown, bringing together researchers who share assays, protocols, and outcomes through presentations and open discussion. Expanding this collective knowledge base is essential to making individualized genetic treatments faster, safer, and accessible to more patients.

The Preclinical Design Workgroup provides information and support for the design and testing of individualized therapeutic modalities with a focus on efficacy and safety evaluation. We publish guidelines on therapeutic design and manufacturing for research purposes, and organize online meetings where researchers share experiences with tools and assays relevant to individualized development.