April 4 - 8, 2024
Siracusa, Sicily

Chairs:   Steven F. Dowdy, UCSD School of Medicine, USA
  Laura Sepp-Lorenzino, Intellia Therapeutics, USA
  Matt Stanton, Generation Bio, USA



24 conference program - Delivery Of Nucleic Acid Therapeutics II: Biology, Engineering And Development, Steven F. Dowdy, Laura Sepp-Lorenzino, and Matt Stanton (Article)

Living in the world of Nucleic Acid Medicine, the Cinderella Molecules, Mano Manoharan (Abstract)

Setting the Delivery Stage, Steve Dowdy (Abstract)

TfR Ab conjugated oligos for muscle delivery, Art Levin (Abstract)

TfR-Fab conjugated oligos for muscle delivery, Oxana Beskrovnaya (Abstract)

CRISPR LNPs, Kristy Wood (Abstract)

Design and Evaluation of Novel ionizable Lipids for RNA Delivery, Pad Chivukula (Abstract)

Delivery of a First-in-Class miR-10b Antagomir to Treat Metastatic Cancer:The Long Winding Road from Pre-Clinical Development to Initial Clinical Experience, Zdravka Medarova (Abstract)

Splice-switching oligo conjugates for the treatment of Erythropoietic Protoporphyria, Phil Becker and Jonathan . Hall (Abstract)

LNP Endosomal Escape, Anders Wittrup (Abstract)

Harnessing endocytosis to improve the delivery of oligonucleotide-based drugs, Marino Zerial (Abstract)

ENDOSCAPE: Triterpene glycoside conjugates allow efficient endosomal escape of diverse targeted payloads, Guy Hermans (Abstract)

Predictive Ex Vivo Models of LNP Behavior, Dwight Morrow (Abstract)

GalAhead and PNP, double targeting the way forward!, Jim Weterings (Abstract)

Exploring cellular uptake and trafficking of lipid conjugated antisense oligonucleotides, Emma Kay, Priyanka Mangla, Michael J Munson, Laurent Knerr, Shalini Andersson, Anders Dahlén, and Annabelle Biscans (Abstract)

Deep learning-enhanced single particle tracking reveals intracellular delivery and escape of oligonucleotides, Nikos Hatzakis (Abstract)

Lipidated conjugates for CNS delivery, Anastasia Khvorova (Abstract)


Chemical evolution of artificial peptide carriers for nucleic acid delivery, Ernst Wagner (Abstract and Presentation)

Selective Organ Targeted (SORT) LNPs: focus on lung delivery and PCD program, Dan Siegwart (Abstract)

Nucleic acid immunity: the key to successful nucleic acid therapeutics, Gunther Hartmann (Abstract)


Designing Thermostable Hermes lipopolyplex nanoparticles for mRNA and synthetic hpDNA delivery, Amy Walker, Gözde Tezcan, Hassan Idris, Matthew Walker, and Ashish Dhir (Abstract and Presentation)

Visualization of lipid nanoparticle disintegration and localized endosomal membrane damage, Johanna M. Johansson, Hampus Du Rietz, Hampus Hedlund, Erik Oude . Blenke, Said Harun, Lennart . Lindfors, and Anders Wittrup (Abstract)


Molecular Nano-Motors (MNMs): A novel delivery modality for oligonucleotides, Hamutal Ben Dov and Hagit Grimberg (Abstract and Presentation)

Recent Discoveries in ExtraHepatic Delivery of RNA Cargoes, Pete Smith (Abstract)

Centyrin-targeted siRNA Conjugates for Extra-hepatic Delivery: ABX1100, a CD71 Centyrin-Gys1 siRNA Conjugate for Treatment of Pompe Disease, Sukumar Sakamuri (Abstract)

Engineering Exosomes to Enable and Create Improved Genetic Medicines, Tony De Fougerolles (Abstract)

LNP delivery, Guarav Sahay (Abstract)

In Vivo Delivery of RNA Gene Writers to the Liver and Beyond, William Salomon, Zsanett Jancso, Lorenzo Tozzi, Drishti Parwanda, Giulia Schiroli, Annie Chivatakarn, Rahul Nair, Michael T. Monte, Luciano Apponi, Laura Saunders, Han Gu, Rebecca L. Goldman, Apiwat Wangweerawong, Lei Liu, Rahul Palchaudhuri, Aaron Zannini, Thomas V. Colace, Gulen Tonga, Tiffany Morales, Christina Ferren, Alyssa Sanfilippo, Chelsea Habif, Gilbert Ndzeidze, Zhjjie Ma, Rachit Jain, David Didio, Jack a Chu, Pinky Htun, James B. Rottma, Jeff An, Carol Cai, Erin Spofford, Yu Cao, Fred Briones, William Querbes, Michael C. Holmes, Hari Pujar, Z. Jane Wang, Cecilia Cotta-Ramusino, Anne Bothmer, Jean-Antoine Ribeil, and John P. Manis (Abstract)

“I translate, therefore I deliver”: understanding LNP delivery capabilities on a granular scale to reveal therapeutic opportunities, Charlotte Dunne, Katrin Radloff, Daniel Tondera, Ansgar Santel, Jorg Kaufmann, Oliver Kiel, Volker Fehring, and Leonidas Gkionis (Abstract)

Just a Little Delivery Reality Check, Paul Burke (Abstract)

AAV delivery, Dirk Grimm (Abstract)

A Modular, Antibody-Based AAV Retargeting Platform For Efficient And Specific In Vivo Gene Delivery, Leah Sabin (Abstract)

Development of a human hematopoietic stem cell targeted platform through multiplexed targeting and de-targeting modifications of a common viral gene transfer vector for safe and effective editing of stem cell compartment in vivo., Dmitry Shayakhmetov, Jia Yao, Svetlana Atasheva, Nelson C. Di Paolo, Nicole Wagner, and Phoebe L. Stewart (Abstract)

DNA LNP delivery, Matt Stanton (Abstract)

Targeting mRNA LNPs to New Cell types, Stefaan De Koker (Abstract)

LNP Structure, Jay Kulkarni (Abstract)

The World of LNPs, Pieter Cullis (Abstract)

Oligophore and Semaphore for extrahepatic delivery of therapeutic RNA, Covadonga Paneda, Reda Juskeviciene, Martina Herrera-Jezowska, Nicole Grosse, Veronica Francardo, and Fabio Fais (Abstract)

AI-driven design of four-component lipid nanoparticles unlocks systemic mRNA delivery to skeletal and cardiac muscles, Ruilu Feng, Daniel Quevedo, Xiangxue Meng, Xue Wang, Jiani Lei, Lin Zhang, Feng Shi, Daniel Quevedo, Shaoli Liu, and Andong Liu (Abstract)

Using MD simulations to design more efficient lipid nanoparticles, Florian Mann, Adiran Garaizar, David Diaz-Oviedo, Nina Zablowsky, Sami Rissanen, Johannes Köbberling, Jiawei Sun, Christoph Steiger, Patrick Steigemann, and Katharina Meier (Abstract)

Safe and effective extra-hepatic delivery of DNA and mRNA using the non-viral fusogenix proteolipid vehicle platform, John Lewis, Arun Raturi, Ping Wee, Prakash Bhandari, Douglas Brown, Amirali Bukhari, and Roy Duncan (Abstract)

EndoPore: Targeted delivery of nucleic acid therapeutics using pore-forming proteins, Vineeta Tripathi, Nourhan A. M. Mahmoud, Hadeer K. S. Abdelrahman, and Simon Richardson (Abstract)

Investigating the effect of Lipid Bioconjugates on Gapmer ASO activity and toxicity, Gavin Garland, Veronica Dezi, Yunyue Wang, Ritwick Sawarkar, and Anne Willis (Abstract)

Dechipering endosomal escape of oligonucleotides from Lipid Nanoparticles by the single particle, Frank Schulz, Valia Margaritaki, Emily Winther Sørensen, and Nikos S. Hatzakis (Abstract)

Uptake of chemically modified antagomirs by lung relevant cell systems, Anna Rydzik, Julie Weidner, Werngard Czechtizky, Henric Olsson, Felix Gnerlich, Stefan Vollmer, Marion Garreau, Elisabeth Ax, Reetta Holmila, and Ewa Kolosionek (Abstract)

Syringable microcapsules for sustained, localized and controllable siRNA delivery, Yan Liu, Sean Bedingfield, Yang Wang, Rajesh Kulkarni, Lindsay Anne Wegiel, Byungkook Lee, and David Weitz (Abstract)

Innovative development of COVID-19 mRNA vaccine using the gold nanoparticle delivery platform, Jeehyeon Bae (Abstract)

Characterization of triterpene-mediated endosomal escape of cholesterol siRNA, Myriam Cerezo-Magaña (Abstract)

Exploring the Traut’s reagent as a versatile bifunctional linker for oligonucleotide conjugates, Daniele Addis and Dr. Soham Mandal (Abstract)