2025 Plenary Program

TUESDAY, MARCH 13

4:15 pm Plenary Chairperson's Remarks
Dmitry Samarsky, PhD, Former CTO, Sirnaomics

4:20 pm PLENARY PRESENTATION: siRNA Chemical Engineering
Anastasia Khvorova, PhD, Professor, RNA Therapeutic Institute, University of Massachusetts Medical School
The focus of our lab is to identify, characterize, and develop novel chemistries that promote simple, efficient, and non-toxic delivery of oligonucleotides and potent silencing of therapeutic targets in vivo. Some examples will be highlighted in this talk.

5:00 pm PLENARY PRESENTATION: TANGO: An RNA Splicing Approach to Upregulate Proteins
Edward Kaye, MD, CEO and Director, Stoke Therapeutics
Targeted Augmentation of Nuclear Gene Output (TANGO) is an RNA splicing approach that enables the upregulation of many proteins. Specifically designed Anti-sense Oligonucleotides (ASOs) splice out retained naturally occurring 'poison exons' or NMD exons from pre-mRNA, thus enabling an increase of full-length message and full-length protein. We are targeting autosomal dominant diseases which are missing 50% of an essential protein to correct the underlying genetic defect.

WEDNESDAY, MARCH 12

12:40 pm Plenary Chairperson's Remarks
Chandra Vargeese, PhD, CTO & Head, Platform Discovery Sciences, Wave Life Sciences

12:45 pm PLENARY PRESENTATION: Delivery with Bicycles and Camelids: Targeted Delivery of Oligonucleotide Drugs to Muscle and the Central Nervous System via the Transferrin Receptor
Eric Swayze, PhD, Executive Vice President, Research, Ionis Pharmaceuticals
Ligands for transferrin receptor can potentially provide solutions to the delivery of oligonucleotides to skeletal and cardiac muscle, as well as across the blood brain barrier. We have optimized oligonucleotide conjugates to TfR1 ligands including Bicycle peptides and camelid nanobodies to reduce the total dose of the administered drug. These constructs have achieved successful delivery to the target tissues, offering the potential for treatment of cardiovascular and neurological diseases.

1:25 pm PLENARY PRESENTATION: CRISPR Genome Editing for Therapeutic Applications: Advances in in vivo Editing
Ramsey Majzoub, PhD, Director, Intellia Therapeutics
At Intellia, we are advancing a full-spectrum genome editing company. We are deploying the industry’s broadest and deepest toolbox, including novel editing and delivery solutions, to harness the immense power of CRISPR-based technologies for in vivo and ex vivo therapeutic applications. In this presentation, we will share the advances in the therapeutic applications of CRISPR/Cas9 for in vivo genome editing.