Oligonucleotide therapeutics safety considerations across drug development
As oligonucleotide therapeutics continue to advance, their safety assessment presents challenges that differ from traditional drug modalities. Discover how nonclinical safety studies, evolving regulatory expectations and real-world case examples can help you navigate these challenges with greater confidence.
Developing safe oligonucleotide therapeutics requires careful consideration of challenges that differ from those of traditional drug modalities. As antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) continue to transform therapy, understanding their unique nonclinical safety profile has become increasingly important.
This session explores species selection, tissue distribution, immune effects and class-specific toxicities, alongside current regulatory expectations and industry perspectives.
Through practical case examples, attendees will gain a deeper understanding of the challenges associated with oligonucleotide therapeutics and the considerations that support their safe and successful development.
What you’ll learn:
- Identify key safety considerations for oligonucleotide therapeutics, including mechanisms of action, pharmacokinetics, biodistribution and class-specific toxicities.
- Navigate key translational challenges when predicting clinical safety from nonclinical studies, including on-target, off-target and chemistry-driven toxicities.
- Understand evolving global regulatory expectations and the factors sponsors should consider when designing nonclinical safety programmes.
- Gain practical insights from real-world case examples to help inform safety assessment and development strategies.
- Ask our expert speakers your questions during the live Q&A.