OPT Congress 2023 Plenary Session Program
MONDAY, MARCH 13
3:50 pm Plenary Chairperson's Remarks
Dmitry Samarsky, PhD, CTO, Sirnaomics
3:55 pm PLENARY PRESENTATION:
Biological Studies with Thiomorpholino Oligonucleotides
Marvin Caruthers, PhD, Distinguished Professor, University of Colorado
Currently underway are more than 20 biological studies focused on the use of thiomorpholino oligonucleotides (TMOs) as a therapeutic drug for the treatment of various genetic diseases. These collaborations are being carried out with cells in culture
and several have progressed to studies in mouse models. Without exception, TMOs are more active than any other analogue tested and, in one case, a TMO is active under conditions where the 2'-MOE analogue is toxic to mice in the same treatment group.
4:25 pm PLENARY PRESENTATION:
Advances in CRISPR Genome Editing for Therapeutics Application
Rubina Parmar, PhD, Vice President, Chemistry & Delivery Sciences, Intellia Therapeutics, Inc.
4:55 pm PLENARY PRESENTATION:
Recent Developments in Oligo Conjugates
Arthur Levin, PhD, CSO, Avidity Biosciences
The promise of oligonucleotide therapeutics is to use Watson-Crick-Franklin base-pairing rules to design drugs directly and rationally based on genomic
information. Until recently, that promise has remained elusive because of cell barriers to oligonucleotide uptake. Receptor-mediated uptake through bioconjugation oligonucleotides has changed that. Avidity’s AOC technology uses monoclonal antibodies
to cell surface proteins that are internalized in order to facilitate the functional delivery of oligonucleotide therapeutics into a broad range of cell and tissue types.
TUESDAY, MARCH 14
3:15 pm Plenary Chairperson's Remarks
Jonathan Watts, PhD, Associate Professor, RNA Therapeutics Institute, University of Massachusetts Chan Medical School
3:20 pm PLENARY PRESENTATION:
Nucleic Acid Delivery Systems for RNA Therapy and Genome Editing
Daniel Anderson, PhD, Professor, Chemical Engineering, Massachusetts Institute of Technology
Here we describe our work developing nanoformulations for RNA therapy and genome editing. Libraries
of degradable polymers and lipid-like materials have been synthesized, formulated, and screened for their ability to deliver RNA payloads inside cells. These nanoformulations facilitate in vivo delivery to a range of tissues and can enable
targeted gene suppression with siRNA, gene expression with mRNA, or even permanent genetic editing using the CRISPR/Cas9 system.
3:50 pm PLENARY PRESENTATION:
Advances in Chemistry Made RNAi Therapeutics Possible
Mano Manoharan, PhD, Distinguished Scientist & Senior Vice President, Innovation Chemistry, Alnylam Pharmaceuticals
For siRNAs, chemical modifications are necessary to regulate metabolic
stability, potency (through effects on the interaction with the Ago2 enzyme and the targeted mRNA strand), and safety (impacted by metabolites and on-target specificity). We have evaluated numerous chemical modifications beyond the standard 2’-O-methyl,
2’-fluoro, and phosphorothioate linkages. These include backbone chiral phosphorothioates, glycol nucleic acids, altriol nucleic acids, gem 2′-deoxy-2′-α-F-2′-β-C-methyl, 5’-morpholino, and amino-oxy click
chemistry (AOCC) mediated conjugates. Furthermore, novel spatial architectures like circular siRNAs have also been evaluated. This presentation will summarize how chemistry has made possible the currently exciting world of RNAi therapeutics.
4:20 pm PLENARY PRESENTATION:
Development of mRNA-Based Vaccines
Steve Pascolo, PhD, Senior Scientist, University Hospital of Zurich; Founder & CEO, Miescher Pharma GmbH
The safety and efficacy of mRNA-based vaccines was evidenced during the COVID-19
pandemic: less than one year after the publication of the sequence of SARS-CoV-2, mRNA vaccines against COVID-19 were approved. More mRNA vaccines (against infectious diseases and cancer) are in clinical developments and are expected to be approved
in the coming years. New mRNA formats (circular, replicating) and formulations (lipoplexes and polyplexes) are also being tested to further improve mRNA vaccines. I will present the past, present and future of mRNA vaccines.