Agenda

Welcome coffee.

Please enjoy breakfast at your hotel prior to arriving at the venue.

Co-chairs:

Alex Garanto, Ph.D., Radboud Univ. Medical Center

Audrius Kilikevicius, Ph.D., Eli Lilly and Company

9.00-9.25:

Rewriting Genetic Information in RNA using Oligonucleotide-directed Adenosine Deamination, Peter Beal, Ph.D., UC Davis

9.25-9.50:

Next-generation mRNA vaccines: New RNA modalities and delivery systems, Camilla Foged, Ph.D., University of Copenhagen

9.50-10.15:

Improving the Safety and Specificity of RNAi Therapeutics, Maja M. Janas de Angelis, Ph.D., DABT, Alnylam Pharmaceuticals

10.15-10.40:

Safety assessment of therapeutic oligonucleotides with focus on off-targets, Patrik Andersson, Ph.D., AstraZeneca

10.40-11.05:

Early clinical development of nucleic acid therapeutics, Art Krieg, MD, Zola Therapeutics

Workshop sponsored by :

11.15 - 13.00

Hosted Lunch (for all participant)

Mentorship Program Lunch (by invitation only)

Co-chairs: Sarah Allen, Ph.D. Candidate, UMass Chan Medical School

Emilio Harris-Mostert, Ph.D. Candidate, Erasmus MC

Session Keynote:

13.00-13.25:

Session Keynote: From Molecules to Meaning: An Academic Journey to ASO Therapies for Duchenne Muscular Dystrophy, Aurelie Goyenvalle, Ph.D., UMR-1179- University of Versailles, INSERM

13.25-13.40:

Accurate AI-Driven Prediction of Potent Therapeutic Fully Chemically Modified siRNAs, Kathryn R. Monopoli, UMass Chan Medical School

13.40-13.55:

Ligand Valency, Placement and Positioning Do Matter: Programmable Surface Modification of Rectangular DNA Origami Modulates Its Biological Activity, Natalia Navarro, IQAC-CSIC, CIBER-BBN, Barcelona, Spain

13.55-14.10:

Unraveling and controlling late-onset neurotoxicity of CNS-targeted antisense oligonucleotides through strategic chemical modifications, Takayuki Kuroda MD, NucleoTIDE and PepTIDE Drug Discovery Center, Institute of Science Tokyo

14.10-14.25:

Fatty acid mediated uptake system enables improved oligo delivery to muscle and brain, Argimiro Mayoral-Olmos, ARTHEx Biotech

14.25-14.40:

Immunoengineering of light-inducible RNA agonists enables spatiotemporal control of RIG-I activation for cancer immunotherapy, Sandra A. Lewash, University Hospital Bonn

Chair:

Richard Geary, Ph.D., Ionis Pharmaceuticals & President, OTS

15.30 - 15.45 Welcome and Opening Remarks

15.45 - 16.45 Keynote Presentation:

The Remarkable Versatility of Splice-Switching ASOs

Adrian R. Krainer, Ph.D., St. Giles Foundation Professor, Cancer Center Program Co-Leader

Co-Chairs:

Eva-Maria Manz, Ph.D., ETH Zürich

Jathavan Asohan, Ph.D. Candidate, McGill University

Panel discussion on diverse career paths in academia and industry.

Panellists:

Reka Haraszti, Ph.D., Universitätsklinikum Tübingen

Keith Gagnon, Ph.D., Wake Forest University

Graham Parker, Ph.D., Executive Editor, Mary Ann Liebert Inc.

Marc Lemaitre, Ph.D., Ph.D., ML_Consult Llc

Krystal Johnson, Ph.D, Alnylam Pharmaceuticals

Annabelle Biscans, Ph.D., AstraZeneca

Open to Students and Post-Docs.

16.45 - 19.30

Welcome Reception with Exhibitors (no host Poster Viewing)

Early Career Scientist Social Meet-up

(RSVP in the registration form - for students only)

Location: EXTRA Bar

Klauzál u. 15, 1072 Budapest | https://extrabudapest.com/en

sponsored by :