Podium Presentation

Aliri was recently one of 10 labs to participate in the Oligonucleotide Ring Trial, in which industry experts joined together to evaluate the effectiveness of LC-MS, LBA, and qPCR when quantifying the concentrations of oligonucleotide in biological samples. This first-of-its-kind study aimed to give clarity to drug developers about the methodology best suited for future development programs.  Specifically, the Ring Trial focused on three types of oligonucleotides, an ASO (Fomivirsen), a GalNAc-siRNA (Lumasiran), and a PMO (Viltolarsen).

Troy Voelker, Sr. Lab Director at Aliri and Chair of the AAPS Oligonucleotide Discussion Group, led the LC-MS method development of the PMO (Viltolarsen), which was analyzed using three mass spectrometry platforms: a QExactive, a time-of-flight (TOF), and a triple quadrupole instrument. In this presentation, he reveals exciting data that proves hybridization LC-MS superior to LBA in achieving lower detection limits and higher specificity.

Over the past two decades, omics technologies have steadily expanded from discovery research into translational and clinical development, offering unprecedented insights into biology, disease mechanisms, and therapeutic response. Advances in genomics, transcriptomics, proteomics, metabolomics, and, more recently, spatial and multi-omics platforms have created powerful opportunities to identify predictive biomarkers, refine patient stratification, and accelerate drug development. The added value of omics lies in their ability to capture complex, system-level biology that traditional single-analyte assays cannot address, thereby bridging the gap between exploratory research and precision medicine. 

Yet this rapid progress has also highlighted a critical challenge: how to validate omics data in a way that is scientifically rigorous but also practical. Current analytical validation paradigms were largely developed for conventional assays such as ELISA or qPCR, which measure one or a handful of targets at a time. Applying the same frameworks directly to high-dimensional omics assays often results in processes that are overly burdensome, expensive, and poorly aligned with the dynamic nature of omics platforms. 

Moving forward, the field needs more efficient and adaptive validation processes, aligned with the specific purpose of each omics application. This involves applying full rigor when data will inform regulatory submissions or clinical decisions, while using streamlined, fit-for-purpose approaches for exploratory research and mechanistic studies. 

In this presentation, Aliri R&D Director and Spatial-Omics Expert, Corinne Ramos, Ph.D., illustrates these challenges and opportunities through a spatial multi-omics use case, where paired patient biopsies were profiled with spatial transcriptomics and proteomics to uncover mechanistic insights, identify predictive biomarkers, and generate regulatory-ready evidence for clinical development. 

Download the presentation to learn more.