Paving a Path for the Clinic – Preclinical Characterization of Nanomedicine

M. Scully
Frederick National Laboratory for Cancer Research,
United States

Keywords: nanomedicine, cancer


Nanomedicines are complex formulations and the collective properties that guide the nanomedicine’s safety and efficacy profile may not be fully captured with standard characterization techniques. Still, nano-based drug delivery candidates must meet the same standards for quality, safety and efficacy as small molecule drugs in the regulatory pathway. The Nanotechnology Characterization Lab (NCL) was developed in partnership with the National Cancer Institute (NCI), National Institute of Standards and Technology (NIST) and the Food and Drug Administration (FDA) to address the characterization needs for nano-based therapies and diagnostics. We perform vital preclinical studies on promising nanomedicine candidates to facilitate their clinical translation. Nanomedicine candidates submitted from researchers and organizations worldwide are subjected to an analytical cascade that characterizes nanoparticles’ physical attributes, in vitro biological properties, and in vivo compatibility. The full spectrum of testing allows NCL to identify physicochemical properties that affect biological outcomes. Leveraging over a decade of nanotechnology characterization, NCL also develops methods that can evaluate the clinical potential of early-stage nanomedicine, support regulatory decision-making, and address the growing needs of nanomedicine developers. In this talk, I will focus on the need for appropriate characterization of nanomedicines and introduce advanced characterization techniques designed to address regulatory questions. Armed with this analytical toolkit, nanomedicine developers are better equipped to overcome characterization challenges associated with drug and gene delivery. When adequately addressed early in the development stage, characterization of nanomedicine can prevent costly and time-consuming redesign later in the process. Funded by NCI Contract No. HHSN261200800001E.