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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
- 10:20 - 10:50 Break
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
- 12:00 – 13:00 Lunch Break
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 Mor, RNAble, 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-Canada, Hebrew 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-Rus, Leiden University Medical Center, The Netherlands
- 15:30 - 16:00 Coffee Break
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
- 10:30 - 10:50 Coffee Break
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
- 12:50 – 13:50 Lunch Break
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
- 15:20 – 15:50 | Break
