Senior Scientist Roche Pharma Research and Early Development, Switzerland
Abstract: Antisense therapies have the potential of being transformative treatments for several diseases with no alternative therapeutic options. In this talk, we will focus on the potential of antisense oligonucleotides (ASOs) as therapeutic agents able to control gene expression of specific target mRNAs. In particular, we are using computational strategies, including bioinformatics and machine learning, to design libraries of ASOs with the goal of maximizing their properties in terms of potency, specificity and safety. In addition, the use of high-throughput screening platforms determines the selection of the top performing ASOs that will undergo further drug development phases.