2023 Presentations:

1: Determining Criticality of Impurities in Oligonucleotide Fragments for Liquid Phase Synthesis
Presented by Xiao Z., Biogen
Fragments containing tetra/pentamers were used in convergent liquid-phase synthesis of oligonucleotide. Understanding of the impurities in the fragments is crucial for synthesis optimization, purification development, and setting an appropriate control strategy. A method was developed to classify critical and non-critical impurities in the fragments using HR-MS based on their reactivity with coupling reagents. This information was then utilized to prioritize and identify the critical impurities.

2: Use of SIM LC-MS Analysis for Mono PO Impurity Quantitation in Oligonucleotides
Presented by Ashley M., Nitto Denko Avecia
A common modification in oligonucleotides is the replacement of oxygen with sulfur in the internucleotide linkages to enhance oligonucleotide target specificity. Incomplete oxidative thiolation can lead to mono phosphodiester impurity in the final oligonucleotide. The diastereomers created can lead to peak broadening and coelution of the impurity with the FLP. LC-MS analysis using Selected Ion Monitoring (SIM) can be used to quantitate this mono phosphodiester impurity.

3: The Identification of Dysregulated Genes by Whole Transcriptome Analysis of the SCA7 Mouse Model
Presented by Yongmoo K., Seoul National University Hospital
Spinocerebellar ataxia (SCA) is a group of genetic, degenerative disease which has no proven cure. In this study, we have conducted epigenetics and transcriptomics analysis of SCA7 mice model and revealed genetic affiliations of some mRNA expressions and DNA methylations with other factors. Transcriptome analysis of SCA7 mouse will provide clues for the pathomechanism of SCA7 and suggest possible therapeutic targets.

4: Bioanalytical Evaluation of Single-Stranded DNA for Gene Therapy Applications: A Reference Standard for Quality Control Workflow
Presented by Zhixiang L., PerkinElmer Health Science, Inc.
Rapid growth in the use of single-stranded DNA (ssDNA) in biotherapeutics underscores the importance of quality control to ensure the integrity and purity of ssDNA-based payloads. This report provides a reliable, higher throughput reference standard, and bioanalytical screening solution for ssDNA samples. The novel ssDNA ladder ranges from 1kb to 7kb, can be used to evaluate critical quality attributes (CQAs) such as relative titer quantification, purity, and integrity of ssDNA from rAAV preparations.




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2024 Conference Programs

Oligo Discovery & Delivery