Antisense Oligonucleotides (ASOs) are short molecules comprised of ten to thirty nucleotides that bind to cellular RNAs via complementary base pairing to influence pre-messenger RNA (mRNA) splicing, mRNA stability, translation or RNA-protein interaction. This complementary binding forms the basis of ASO therapies whereby ASOs inhibit or restore the expression of target genes by diverse mechanisms, including degrading mRNA transcripts causing gene silencing/knockdown or altering the splicing of pre-mRNAs.

The widespread use of large-scale clinical genomic testing has exponentially increased the rate of new gene-disease relationships discovery for rare inherited disorders. Increasing use of automation and machine learning has also allowed for the discovery of individualized ASO therapies for these new diseases. However, there is still a considerable need for dissemination about best practices for triaging diseases, genes, and variants amenable to these individualized ASO therapies. There is also unfamiliarity around institutional implementation, regulatory and funding frameworks necessary for health care providers to offer these therapies to their patients.

The aim of this session is to spotlight the multifaceted implementation of individualized, investigational ASO therapies for the larger genomics community. The speakers for this session are experts on the development and implementation of individualized, investigational ASO therapies across the US and Europe. They will touch upon key insights and practical guidelines for implementing individualized ASO therapies, elucidating on prerequisites for disease to gene to variant amenable to these therapies and the institutional and funding frameworks for feasible access to custom ASO therapies across diverse health care systems and institutions. In addition, the session will provide attendees an overview from the FDA about the current landscape of the approval process. Overarchingly, the session will focus on what it takes to bring individualized ASOs from bench to bedside.