DAY 1 (JUNE 12, 2025)

 

09:30 – 10:30    Registration open

 

10:30 – 10:45     Welcome and official opening  by Dr. Jeroen Bremer (UMC Groningen, The Netherlands)

 

10:45 – 11:30     International Keynote Lecture: Dr. Anna Perdrix Rosell, Sixfold Bioscience, UK

                           Beyond the Liver: Advancing Computational and Chemical Strategies for Oligo Delivery

                           Chair: Jeroen Bremer​

​

11:30 – 12:15     Session 1 (Chair: Ronald Buijsen)

​

11:30 – 11:45     Tom Baladi (AstraZeneca, Sweden)

                           Engineering small molecules endosomal escape enhancers for improving oligonucleotide                                         therapeutic window 

11:45 – 12:00     Rosan Lechner (Erasmus MC, Rotterdam, The Netherlands)

                           Retrospective assessment of the yield of genetic testing in unexplained epilepsy to determine                                 potential therapeutic impact of personalized antisense oligonucleotides

12:00 – 12:15     Sponsor pitches

​​

12:15 - 12:20.    Group Picture

​​

12:20– 13:30     Lunch

 

13:30 – 15:00     Session 2 (Chair: Anne-Fleur Schneider)

​

13:30 – 14:00     Dr. Erin McConnell (Carleton University, Canada)

                           Using aptamers as a therapeutic tool in cell and animal models of Parkinson's diseases

14:00 – 14:15     Olivia Lewis (Utrecht University, Utrecht, The Netherlands)

                           Decoding RNA Regulation: Challenges and Opportunities for RNA-Based Therapies in Europe

14:15 – 14:30     Laurie Kerkhof (LUMC, Leiden, The Netherlands)

                           5’UTR-mediated regulation of ataxin-1 protein expression using antisense oligonucleotides and                               CRISPR-Cas9 gene editing

14:30 – 14:45     Eline van de Ven (Radboudumc, Nijmegen, The Netherlands)

                           Preclinical development of an AON-based approach to restore sialic acid metabolism in

                           NANS-CDG

14:45 – 15:00     Frits Deiman (UMCG, Groningen, The Netherlands)

                           Phospholamban antisense therapy reduces protein aggregation and enhances calcium and                                     contractile kinetics in PLN-R14del induced pluripotent stem cell derived cardiomyocytes

​

15:00 – 16:30     Poster Session 1 (odd), Chair: Alex Garanto

15:00 – 15:15     Selected Poster Pitches (odd)

                           Pitches by Poster #1, Poster #3, Poster #5, Poster #7, Poster #9, Poster #11, Poster #13 and

                           Poster #15

​

15:15 – 16:30     Poster walk with coffee and tea

 

16:30 – 18:00     Session 3 (Chair: Najoua El Boujnouni)

​

16:30 – 17:00     Andrea Gargano (University of Amsterdam, The Netherlands)

                           Ion-pairing hydrophilic interaction chromatography: a powerful separation technique for     

                           impurity profiling of therapeutic phosphorothioated oligonucleotides.

17:00 – 17:15     Redmar van den Berg (LUMC, Leiden, The Netherlands)

                           Genetic Therapy Generator Toolkit

17:15 – 17:30     Emilio Harris-Mostert (Erasmus MC, Rotterdam, The Netherlands)

                           Using the multi-electrode array to predict the acute neurotoxicity of antisense oligonucleotides

17:30 – 17:45     Ingrid de Vries (Sapreme Technologies, The Netherlands)

                           Utilizing Sapreme’s endosomal escape enhancer platform as oligonucleotide selection tool and                               for enhanced therapeutic delivery

​​

17:45 – 17:50     Closing of day 1 by Jeroen Bremer

 

19:00 – 23:00     Networking event (not included in the meeting registration costs)

19:00 – 19:30     Walk in and networking: Bax (Friesestraatweg 201, 2a, 9743 AC Groningen)

19:30 – 23:00     Dinner, social activity and drinks

 

​

DAY 2 (JUNE 13, 2025)

 

09:00 – 09:45     National Keynote Lecture: Dr. Marjon Pasmooij, Utrecht University, The Netherlands

                           Navigating two worlds: what a dual career in research and regulation has taught me

                           Chair: Alex Garanto

​

09:45 – 11:15     Poster Session 2 (even), Chair: Alex Garanto

09:45 – 10:00     Selected Poster Pitches (even)

                           Pitches by Poster #2, Poster #4, Poster #6, Poster #8, Poster #10, Poster #12 and Poster #14

​

10:00 – 11:15     Poster walk with coffee and tea

​

11:15 – 12:45     Session 4: Dutch Center for RNA Therapeutics (DCRT) (Chair: Bianca Zardetto)

​

11:15 – 11:45     Hélène Tran (Servier, France) 

                           From bench to bedside: unlocking the potential of antisense oligonucleotides research to

                           clinical applications in rare brain disorders

11:45 – 12:00     Seppe Hermans (​Technische Universität Ilmenau, Germany)

                           Tailoring antisense oligonucleotide manufacturing to N-of-1 needs

12:00 – 12:15     Edwin van Oosten (Radboudumc, Nijmegen, The Netherlands)

                           Towards personalized antisense oligonucleotide treatment for Stargardt disease: targeting an                                 ultrarare deep-intronic ABCA4 variant using human in vitro models

12:15 – 12:45     Roxana Redis (Charles River, the Netherlands)

                           Strategic approaches for ultrarare diseases

 

12:45 – 13:45     Lunch

 

13:45 – 15:15     Session 5 (Chair: Atze Bergsma)

13:45 – 14:00     Eva Niggl (Erasmus MC, Rotterdam, The Netherlands)

                          Targeting Rheb with antisense oligonucleotides delays epilepsy onset and progression in two                                  Tuberous Sclerosis Complex mouse models and iPSC-derived neurons

14:00 – 14:15     Tiberiu Stan (LUMC, Leiden, The Netherlands)

                           Efficient exon skipping and dystrophin restoration after a single in vivo dose of antibody                                           oligonucleotide conjugate (AOC) in the mdx mouse model for Duchenne muscular dystrophy

14:15 – 14:30     Imke Schuurmans (Radboudumc, Nijmegen, The Netherlands)

                           Targeting AASS improves neurotoxicity and mitochondrial function in patient-derived astrocyte                               models for pyridoxine dependent epilepsy

14:30 – 14:45     Ilaria Brentari (University of Trento, Italy)

                           Optimizing RNA therapies for dementia using hiPSC-derived FTDP-17 neuronal model

14:45 – 15:15     Stefan Bos (SMA patient organization, the Netherlands)

                           Experiencing the approval process of Spinraza: a patient perspective

​

15:15 – 15:30     Award Ceremony and closing remarks (Jeroen Bremer)

 

15:30 – 16:30     Networking borrel

​

​​

Poster list:

Poster #1 - Raúl Andrés Santamaría (LUMC)

Development of a safety pipeline for n-of-1 ASOs in rats

​

Poster #2 - Marlen Lauffer (LUMC)

Making the right choice: Which ASO approach is most suitable for my project?

 

Poster #3 - Kim Wijnant (Radboudumc)

Integrating long- and short-read sequencing to identify targetable unproductive splicing for gene upregulation in neurodevelopmental disorders

​

Poster #4 - Manoe Janssen (Radboudumc)

Therapy Accelerator at Radboudumc: Accelerating Therapy development for Rare Diseases 

​

Poster #5 - Gijs-Jan Scholten (LUMC)

Allele-specific antisense oligonucleotides to treat Spinocerebellar Ataxia Type 1 (SCA1)

 

Poster #6 - Okke Faber (LUMC)

TReXome DB – A database for identifying exon skipping targets in protein tandem repeat domains

 

Poster #7 - Bram Jaasma (UMCG)

Sequencing and expression of human monoclonal IgG for conjugated ASO delivery

​

Poster #8 - Rindert Venema (UMCG)

Isolation and cultivation of a human monoclonal IgG for conjugated ASO delivery

​

Poster #9 - Jack Morgan (LUMC)

Antisense oligonucleotide-mediated exon skipping for missense variants within protein tandem-repeat domains: Use case ANKRD17

​

Poster #10 - Noortje Groenendijk (LUMC)

Development of splice-switching antisense oligonucleotides targeting a deep intronic variant in NDUFAF6-associated Leigh syndrome

​

Poster #11 - Ludvík Hejl (Radboudumc)

Development of splice-altering AONs for an intronic ATP6AP1-CDG variant

​

Poster #12 - Nadine Maas (Erasmus MC)

Developing antisense oligonucleotides for an N=1 case of UNC13A

​

Poster #13 - Iqra Ahsan (Hertie-Institute for Clinical Brain Research, University of Tübingen)

Development and Efficacy Testing of exon-skipping Antisense Oligonucleotide (ASO): a systematic platform approach for Ataxia-Telangiectasia

​

Poster #14 - Anouk Spruit (LUMC)

Neurotoxicity platform development of human iPSC-derived neuronal cultures

 

Poster #15 - Tracy Wüsthoff (LUMC)

Designing allele-specific gapmer antisense oligonucleotides to silence mutant ACTL6B transcripts using a midi-gene system

​

Poster #16 - Antonia Kritsima (University of Amsterdam)

Characterization of therapeutic oligonucleotides by liquid chromatography hyphenated to mass spectrometry

​

Poster #17 - Susanne Kuiper (Dutch Medicines Evaluation Board)

Making sense of antisense: Clinical pharmacology insights for oligonucleotide-based therapeutics

 

Poster #18 - Clara Stock (Dutch Medicines Evaluation Board)

Oligonucleotide-based therapeutics: Hard to handle? - Evaluation of Genotoxicity testing from a regulatory perspective

​

Poster #19 Dana Vervloet (Erasmus MC)

Development of an in vitro luminescence-based tool for ASO screening

​

Poster #20 - Ilse Wallaard (Erasmus MC)

Development of a Cost-Effective ASO Delivery System in Patient-Derived Neural Progenitor Cells and Beyond

 

Poster #21 - Rudiger Kaspera (ICON)

Insights into pharmacologically active metabolites of Antisense oligonucleotides and small interfering RNAs

 

Poster #22 - Anne-Fleur Schneider (LUMC)

Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse

 

Poster #23 - Bianca Zardetto (LUMC)

Exon inclusion strategy in a patient-derived oligodendrocyte cell model for PLP1-associated hypomyelination of early myelinating structures

​

Poster #24 - Liselotte Timp (LUMC)

3D tissue engineered skeletal muscle to model dystrophinopathies and determine the efficacy of exon skip therapies 

​

Poster #25 - Willeke van Roon-Mom (LUMC)

Off-target effects of splice-switching antisense oligonucleotides in neuronally differentiated human induced pluripotent stem cells

​

Poster #26 - Boris Shneyer (Maastricht University)

Splicing modulation therapy for LAMA2-MD/MDC1a patients carrying the c.5562+5G>C founder mutation

 

Poster #27 - Kim van der Gouw (UMCG)

Improving the efficiency of antisense oligonucleotide-mediated exon skipping of COL7A1 to treat recessive dystrophic epidermolysis bullosa

​

Poster #28 - Soham Mandal (Axolabs GmbH)

Comparison of bioanalytical assays to assess pharmacokinetics and biodistribution of ASOs– PNA-HPLC Assay and LC-MS/MS

​

Poster #29 - Ingrid Kolen (University & University Hospital Heidelberg)

Personalized ASO therapy for ALS-associated KIF5A mutations around exon 27: a multi-strategy approach

​

Poster #30 - Bas Rottgering (VICO therapeutics)

ASO-induced exon 10 skipping of Ataxin-3: assessing the functionality of the truncated ΔpolyQ Ataxin-3 protein isoform

​

Poster #31 – Svetlana Pasteuning (Optics11 Life)

Advancing Tissue-Engineered Muscle Models: High-Throughput and Automation of the Cuore Smartlid Platform

​​​

​