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Program

WEDNESDAY, SEPTEMBER 25, 2024

08:40 - 10:20 RNA Systems Biology

Chair: Prof. Maria Carmo-Fonseca, Portugal

Opening Remarks

08:40 - 08:50

Prof. Maria Carmo-Fonseca, Institute for Molecular Medicine (IMM), University of Lisbon, Portugal

Keynote Lecture: Finding where to target RNA for therapeutic interventions

08:50 - 09:30

Prof. Yoseph Barash, University of Pennsylvania, Philadelphia, PA, USA

09:35 – 09:55

Prof. Manuel Irimia, Centre for Genomic Regulation, Barcelona, Spain

10:00 – 10:20

Dr. Katharina Höfer, Max Planck Institute for Terrestrial Microbiology, Marburg, Germany

10:50 - 11:35 RNA Systems Biology (cont.)

Chair: Prof. Maria Carmo-Fonseca, Portugal

10:50 – 11:10

Prof. Mary A. O’Connell, Central European Institute of Technology (CEITEC), Brno, Czech Republic

11:15 – 11:35

Dr. Martin Akerman, Envisagenics Inc. New York, USA

Poster Teasers

11:40 – 12:00

Board No.

1

Exploring circRNAs in cancer: Nanopore sequencing for MHC presentation insights
Tsipora Saadoun, Hebrew University of Jerusalem, Israel

2

Glycolytic metabolism plays a leading role in cancer. How to fight this reprogramming?
Dr. Giulia Toti, Sapienza University of Rome, Italy

3

Dissecting the influence of cargo in the performance of polymeric nanoformulations for mRNA delivery
Filipe Bras, University of Coimbra, Portugal

4

Development of exon editors of ABCA4 RNA in human retinal explants and non-human primate retina for the treatment of Stargardt disease
Dr. David Mauger, Ascidian Therapeutics, USA

5

Investigating the neuroprotective potential of RBM3-inducing antisense-oligonucleotides in preclinical models of neurodegenerative disorders
Dr. Tom Haltenhof, Free University of Berlin, Germany

6

A blind retrospective analysis of a novel predictive marker to immune checkpointinhibition in lung cancer, calculated from histopathological slides through inferred transcriptomics
Omer Tirosh, Pangea Biomed, Tel Aviv, Israel

13:00 - 14:10 RNA Systems Biology (cont.)

Chair: Prof. Maria Carmo-Fonseca, Portugal

The interplay of transcription and splicing

13:00 – 13:20

Prof. Maria Carmo-Fonseca, Institute for Molecular Medicine (IMM), University of Lisbon, Portugal

miRNA-based logic circuits encoded on self-amplifying RNA for highly specific cancer cell classification

13:25 - 13:45

Prof. Ron Weiss, Massachusetts Institute of Technology, USA

Development of FTO inhibitors for mRNA stabilization to treat AML cancer

13:50 - 14:10

Dr. Amir MorRNAble, Ness Ziona, Israel

14:15 - 15:25 RNA Therapeutics Development

Chair: Prof. Rotem Karni, Israel

Opening Remarks

14:15-14:20

Prof. Rotem Karni, The Institute for Medical Research Israel-CanadaHebrew University-Hadassah Medical School, Israel

Keynote Lecture: Antisense therapy for diffuse midline glioma

14:20 - 15:00

Prof. Adrian R. Krainer, Cold Spring Harbor Laboratory, New York, USA

15:05 - 15:25

Prof. Annemieke Aartsma-RusLeiden University Medical Center, The Netherlands

16:00 - 17:10 RNA Therapeutics Development (cont.)

Chair: Prof. Rotem Karni, Israel

REM-422, a small molecule that reprograms RNA processing to target MYB-driven hematological and solid cancers

16:00 - 16:20

Dr. Silvia Buonamici, Remix Therapeutics, Watertown, MA, USA

Oral splicing-regulation therapy for genetic diseases and cancer

16:25 - 16:45

Prof. Masatoshi Hagiwara, MD & PhD, Professor & Chairman, Graduate School of Medicine, Department of Drug Discovery Medicine, Kyoto University, Japan

Neutrophil-specific mRNA delivery - shooting at a moving target​

16:50 - 17:10

Prof. Zvika Granot, Faculty of Medicine, Hebrew University, Jerusalem, Israel

Panel Discussion: Transforming Science into Practical Solutions: Insights from the CanceRNA Consortium

17:15 - 18:45

This panel will delve into the critical business and commercialization strategies necessary to translate the CancerRNA Consortium’s research findings into tangible medical solutions and products. We will explore the primary barriers and challenges associated with commercializing RNA-based therapeutics, with a focus on innovations in cancer treatment. Additionally, the discussion will highlight how partners within the CancerRNA Consortium are addressing these challenges to advance effective new anti-cancer therapies.

RNA-based therapeutics – commercialization strategies and challenges
Tamar Raz, CEO, Hadasit Medical Research Services and Development, Jerusalem, Israel

Maximizing research impact – Leveraging CANCERNA Scientific Results in Tech Transfer
Diana Domínguez,
New Ventures Manager, Technology and Business Development Office, Centre for Genomic Regulation (CRG), Barcelona, Spain

IP on RNA Therapeutics
Miguel Duarte,
Technology Transfer Officer, Institute of Molecular Medicine, University of Lisbon, Portugal

Bridging academia and industry for advanced therapies: RNAble/FutuRx accelerator case study
Dr. Amir Mor
, RNAble, Ness Ziona, Israel

From bench to product – considerations to advance a molecule into commercial development
Simon Geissler, Global CMC Development, Merck Healthcare, Germany

THURSDAY, SEPTEMBER 26, 2024

08:50 - 09:35 RNA Therapeutics Development (cont.)

Chair: Prof. Rotem Karni, Israel

Splice-switching antisense oligonucleotide drug discovery for rare neurodegenerative diseases

08:50 - 09:10

Prof. Michelle L. Hastings, University of Michigan, Ann Arbor, MI, USA

Modulating mRNA stability for genetic diseases and cancer therapy

09:15 - 09:35

Prof. Rotem Karni, The Institute for Medical Research Israel-Canada, Hebrew University-Hadassah Medical School, Israel

09:40 - 10:25 RNA Splicing in Cancer

Chairs: Prof. Juan Valcarcel, Spain; Prof. Michal Lotem, Israel

Opening Remarks

09:40 - 09:45

Prof. Juan Valcarcel, Centre for Genomic Regulation, Barcelona, Spain

Keynote Lecture: Autism-like phenotype caused by a neuroanal-microexon mis-splicing impairing kinetic stability of ribonucleoprotein condensates and regulation of mRNA translation in neurons

09:45 - 10:25

Dr. Raul Mendez, Barcelona Institute for Research in Biomedicine, Barcelona, Spain

10:50 - 12:20 RNA Splicing in Cancer (cont.)

Chairs: Prof. Juan Valcarcel, Spain; Prof. Michal Lotem, Israel

Building specificity of anti-tumor splicing inhibitors targeting SF3B1

10:50 – 11:10

Prof. Juan Valcarcel, Centre for Genomic Regulation, Barcelona, Spain

11:15 – 11:35

Prof. Florian Heyd, Free University of Berlin, Germany

Alternative splicing of immune receptors as a regulatory mechanism

11:40 – 12:00

Prof. Michal Lotem, Hadassah Hebrew University Medical Center, Jerusalem, Israel

12:00 – 12:20

Prof. Niels Schaft, University of Erlangen, Erlangen, Germany

12:20 - 12:50 Oral Presentations 1

Chair: Prof. Juan Valcarcel, Spain

RPL3L-specific ASOs as a therapy for heart disease: Dr. Ivan Milenkovic, Centre for Genomic Regulation, Barcelona, Spain

12:20 – 12:25

Inhibition of nonsense-mediated mRNA decay may improve stop codon read-through therapy for Duchenne muscular dystrophy: Yuval Cohen, Hebrew University of Jerusalem, Israel

12:25 – 12:30

Antisense oligonucleotides, an innovative RNA therapeutic for lung adenocarcinomas: Dr. Martina Guerrero, Centre for Genomic Regulation, Barcelona, Spain

12:30 – 12:35

Evaluating LNP- encapsulated decoy oligonucleotides for the treatment of spliceosomal mutant myeloid blood cancers: Carla Azar, Hebrew-University-Hadassah Medical School, Jerusalem, Israel

12:35 – 12:40

Summary

12:40 – 12:50
13:50 - 14:20
Chair: Prof. Rotem Karni, Israel

Enhancing anti-tumor immune response by modulating splicing to generate neoantigens: Anurag Prabhu, Hebrew University of Jerusalem, Israel

13:50 - 13:55

Targeted polymeric nanoparticles for personalized mRNA-based immunotherapy of cancer: Dr. Jan Doerrie, Universitatsklinikum Erlangen, Germany

13:55 - 14:00

RNA modifications - Design principals and biological functions: Dr. Aldema Sas-Chen, Tel Aviv University, Israel

14:00 - 14:05

A novel imaging-based R-loop screening platform: Dr. Robert M. Martin, Institute for Molecular Medicine (IMM), University of Lisbon, Portugal

14:05 - 14:10

Summary

14:10 - 14:20

Oral Presentations

Summary

12:40 – 12:50

RNA Splicing in Cancer (cont.)

14:20 -15:10

Chairs: Prof. Juan Valcarcel, Spain; Prof. Michal Lotem, Israel
14:20 – 14:40

Prof. Evelien Smits, University of Antwerp, Belgium

A-to-I RNA editing in cancer

14:40– 15:10

Prof. Erez Levanon, Bar Ilan University, Ramat Gan, Israel

Friday, September 27, 2024

CanceRNA Internal Meeting | RNA therapeutics

09:00 - 16:00
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