Advancing Therapeutics Through Measurement Science

Summary

As part of NIST Flow Cytometry Consortium Working Group 4 Gene Delivery Systems activities, NIST held the Metrology for Advanced Therapeutics Workshop on September 5, 2025.  This workshop followed the meetings of Consultative Committee for Amount of Substance: Metrology in Chemistry and Biology (CCQM) Working Groups on Cell, Nucleic Acid, and Protein Analysis.  Goals included planning and implementation of interlaboratory comparisons contributing to the development of new standards.

Workshop participants recognized the strong need for fit-for-purpose reference materials, from highest-order SI-traceable standards to in-house reference materials and control materials, as well as robust bridging methods, and traceability schemes.  In addition, workshop participants agreed that by sharing knowledge, harmonizing standards, and fostering partnerships the community can accelerate the development of better measurements and technologies and ultimately support the safe and effective delivery of advanced therapies.

Description

Advanced therapies, such as immunotherapy and gene delivery systems, are transforming the therapeutic landscape by providing curative treatments for previously untreatable diseases.  In particular, emerging gene delivery systems, such as viral vectors (e.g., adeno-associated, viruses, lentiviruses, etc.) and non-viral platforms (e.g., lipid nanoparticles, polymeric nanoparticles, etc.), represent critical components of gene- and RNA-based therapies including in vivo and ex vivo gene therapies, gene editing, and mRNA-based vaccines and therapeutics.

Unlike traditional small-molecule drugs, gene delivery systems and other related advanced therapeutics are structurally and functionally complex and are often highly sensitive to variations in composition, dose, purity and biological activity.  Successful development and clinical application require advanced measurement capabilities to ensure consistency, safety, and efficacy.  Challenges such as particle characterization, cargo integrity, transfection efficiency, and immunogenicity highlight the urgent needs for improved standards, reference materials, and validated analytical methods.

This collaborative workshop brought together experts from global national metrology institutes (NMIs), industry, and academia to examine the pivotal role of measurement science in advancing gene delivery technologies.  It provided a forum to share insights, identify critical measurement needs, and lay foundations for a coordinated metrological framework that supports innovation while ensuring product quality and regulatory confidence.

Workshop organizers

Jamie Almeida, Megan Cleveland, Ed Kwee, and Sheng Lin-Gibson

Additional information

https://www.ibbr.umd.edu/groups/metrology-for-advanced-therapeutics-workshop 

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