DIA/FDA Oligonucleotide-Based Therapeutics Conference

The rapid advancement of RNA-targeted therapeutics has led to a growing number of approved oligonucleotide medicines and an expanding body of long-term safety data. This session will review key learnings from the clinical and post-marketing safety experience across approved oligonucleotide therapies. A particular focus will be on the emerging experience with olezarsen and the Phase 3 development programs in both familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG) populations. Speakers will examine how safety profiles evolve as programs move from early development into larger and longer-duration studies, highlighting insights gained from clinical outcome trials, extension studies, and real-world exposure databases. The session will also explore the role of cardiovascular outcome studies and other large-scale clinical programs in defining the long-term benefit-risk profile of oligonucleotide medicines. Through case-based discussions, participants will review how extended exposure datasets have clarified important safety questions, identified potential class effects, and refined approaches to laboratory monitoring and patient management. As a focused example, the conference will discuss the experience with transthyretin (TTR) lowering therapies, examining the accumulated clinical and post-marketing evidence, and the practical implications for monitoring and need for vitamin A supplementation. Bringing together experts in clinical development, pharmacovigilance, and regulatory science, this session will highlight the monumental progress achieved in the oligonucleotide field and provide a contemporary perspective on the safety experience that is shaping the next generation of RNA-targeted medicines. Participants will gain practical insights into long-term safety evaluation, risk mitigation, and the translation of accumulating evidence into clinical practice.