This project aims to develop light-scattering microscopy methods for rapid, multi-attribute characterization of gene therapy particles at the single particle level. Emerging gene therapy treatments use gene delivery particles, such as viral vectors and non-viral carriers, to cure intractable diseases. But, because these small (20 - 200 nm) and complex nano-bioparticles are difficult to produce and accurately characterize, manufactured particles suffer significant heterogeneity issues which hinder treatment efficacy and safety. To speed treatment development and improve efficacy and safety we are developing light-scatter-based optical microscopes to provide physical measurements (e.g. mass, size, concentration, composition) of individual gene delivery particles.
Overall, Gene therapy is rapidly growing field with $13B market and 27.8% predicted annual growth in 2024. NIST is developing approaches for advanced characterization of critical quality attributes – See Gene Delivery Systems for broader program goals
We are assembling a multidisciplinary, collaborative team of researchers with expertise across five critical project areas and are actively seeking postdoctoral candidates.
Contact us at gtp [at] nist.gov (Gene Therapy Particles)
Optics and Microscopy Instrumentation
Design and build novel laser-based microscope systems for quantitative measurements of light scatter from individual bio-nanoparticles.
Employ advanced image analysis techniques to interpret data, enabling precise measurements of particle loading, size, and concentration.
Develop upstream and downstream processes to produce and purify viral vectors in a bioreactor.
Utilize conventional and state of the art measurement methods to analyze viral vector titer and purity through the manufacturing process. Design orthogonal measurement assays to compare novel light scattering measurements with current methods.
Collaborate with project team to integrate microfluidic and optical measurements with on-line bioreactor measurements.