Description:

Alexander H. Stegh, PhD
Associate Professor
Departments of Neurology, Division of Neuro-oncology and
Medicine, Division of Hematology and Oncology
Feinberg School of Medicine
Northwestern University

Spherical nucleic acids (SNAs) represent one of the most promising and scalable platforms for gene regulation and immunomodulation. SNAs are composed of highly oriented nucleic acids spherically arranged around a nanoparticle core. Gene-regulatory SNAs effectively accumulate and pervasively infiltrate intracerebral brain tumors upon systemic intravenous delivery, reduce target expression, decrease tumor burden, and increase survival of brain tumor-bearing mice. Based upon these preclinical data, at Northwestern University, RNAi-based SNAs have completed early phase clinical testing in glioblastoma patients (clinical trial identifier: NCT03020017). I will discuss results from this clinical trial and describe efforts that leverage the unique 3D SNA architectures for activating innate DNA sensing pathways.