Gene therapy provides the hope of actual cures. These therapies still have many challenges to overcome before they will become widely developed therapeutic options.
Oligonucleotide (OGN) therapy is increasingly recognized as an important therapeutic modality on the promise of curing diseases. However, unlike most biotherapeutics, the development of OGN drugs often requires their determination with high sensitivity and specificity in multiple and complex biologic matrices such as blood and tissues in order to measure their exposure and distribution for understating PK, PD and safety.
At Frontage Laboratories, we have developed highly sensitive and specific quantitative methods for determination of OGN in plasma and tissues using hybridization immunoassay and LC-MS techniques. In this webinar, we will present the method development, validation and the comparison of these techniques for the quantification of OGN as well as a case study with antisense oligonucleotides.