DAY 1 (JUNE 11, 2026)

 

09:00 – 10:00     Registration open

 

10:00 – 10:15     Welcome and official opening

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10:15 – 11:00     International Keynote Lecture: Prof. Jonathan Watts, UMass Chan Medical School, USA

                           Innovations in Antisense Neurotherapeutics: Dendrons, Duplexes and Divalent ions

 

11:00 – 12:00     Session 1 (Chair: tbd)

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11:00 – 11:15     Ingrid Kolen (University of Heidelberg, Germany)

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

                           strategy approach       

11:15 – 11:30     Clemens Lochmann (University of Tübingen, Germany)

                           Efficacy of ASO-mediated splice restauration of exonic cryptic splice donor-gain variants in ATM

                           is largely determined by the variant’s missense effect

11:30 – 11:45     Bas Röttgering (Vico Therapeutics / LUMC, the Netherlands)

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

                           ataxin-3 protein isoform

11:45 – 12:00     Sponsor pitches

12:00 – 12:05     Group Picture

 

12:05 – 13:30     Lunch

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13:30 – 15:00     Session 2 (Chair: tbd)

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13:30 – 14:00     Dr. Frauke Coppieters (Ghent University Hospital, Belgium)

                           Antisense oligonucleotides for rare disorders

14:00 – 14:15     Aleksandra Khatova (BTU Cottbus-Senftenberg, Germany)

                           Gene expression dynamics limit efficacy of ASOs in kinetic simulations 

14:15 – 14:30     Christiaan Stavast (Erasmus MC, the Netherlands)

                           A novel approach to inhibit TSH-receptor signaling as a potential treatment for Graves’ disease

14:30 – 14:45     Hossein D Banadaki (Radboudumc, the Netherlands)

                           Structure-driven and haplotype-informed antisense oligonucleotide targeting of dominant     

                           PRPH2 pathogenic variant

14:45 – 15:00     Myrthe de Graaf (UMCG, the Netherlands)

                           PLN-ASO therapy enhances calcium handling in human myocardial slices: a step towards                                          targeted heart failure therapy  

 

15:00 – 16:30     Poster Session 1

15:00 – 15:15     Selected Poster Pitches

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

                         

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

 

16:30 – 18:00     Session 3 (Chair: tbd)

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16:30 – 17:00     Dr. Kris Siezen (MEB, Utrecht, the Netherlands)

                           Oligonucleotide-based Therapeutics Through the Regulator’s Lens

17:00 – 17:15     Thierry Roob (Erasmus MC, the Netherlands)

                           ERASOR: A platform to design and select antisense oligonucleotides for personalized treatment 

                           of rare diseases 

17:15 – 17:30     Jamie Hyde (LUMC, the Netherlands)

                           Tiled Amplicon Phasing (TAP): A cost-effective method for phasing entire genes to identify

                           targets for allele-specific therapeutics

17:30 – 17:45     Ludvík Hejl (Radboudumc, the Netherlands)

                           Development of splice-switching AONs for a deep-intronic ATP6AP1-CDG variant and associated

                           challenges in AON evaluation  

17:45 – 18:00     Anne Schneider (LUMC, the Netherlands)

                           Combinatorial treatment of anti-oxidant NAC and antisense oligonucleotides can improve

                           treatment effect in mdx mice by improved dystrophin distribution and reduced pathology 

 

18:00 – 18:05     Closing of day 1

 

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

19:00 – 19:30     Walk in and networking

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

 

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 ​​​​​DAY 2 (JUNE 12, 2026)

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09:30 – 10:15     National Keynote Lecture: Prof. Willeke van Roon-Mom, LUMC, the Netherlands

                           RNA therapy development for PolyQ diseases

 

10:15 – 11:45     Poster Session 2 

10:15 – 10:30     Selected Poster Pitches 

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

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10:30 – 11:45     Poster walk with coffee and tea

 

11:45 – 13:15     Session 4 - Dutch Center for RNA Therapeutics (DCRT) (Chair: tbd)

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11:45 – 12:25     DCRT keynote speaker: Prof. Neil Shneider, Columbia University, USA

                           Silencing FUS and Other Rare Genetic Forms of ALS

12:25 – 12:40     Xiaohua Zhou (Phylim, the Netherlands)

                           Targeting a KCNA2 Loss-of-Function Variant with Gapmer ASOs: Toward Personalized Therapy

                           for Kv1.2 Channelopathies

12:40 – 12:55     Seppe Hermans (BianoGMP, Germany)

                           Scalable platform technology for rapid, high-quality manufacturing of neurological N-of-1

                           splice-switching antisense oligonucleotides 

12:55 – 13:15     Prof. Rob Collin (Radboudumc, the Netherlands)

                           Development and implementation of ASO-based therapeutic intervention for ultrarare variants

 

13:15 – 14:15     Lunch

 

14:15 – 15:45     Session 5 (Chair: tbd)

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14:15 – 14:30     Dana Vervloet (Erasmus MC, the Netherlands)

                           Development of a luminescence-based screening assay for allele-selective gapmer ASOs

14:30 – 14:45     Gijs-Jan Scholten (LUMC, the Netherlands)

                           Population genomics–based assessment of haploinsufficiency to inform safe downregulation in 

                           dominant neurodegenerative diseases

14:45 – 15:00     Ewout Groen (UMC Utrecht, the Netherlands)

                           Predictors and post-translational control of SMN2 splice–modifying treatment response in 

                           patient- derived fibroblasts

15:00 – 15:15     Tiberiu Loredan Stan (LUMC, the Netherlands)

                           Sustained muscle function improvement after a single in vivo dose of antibody oligonucleotide                               conjugate (AOC) in the mdx mouse model for Duchenne muscular dystrophy 

15:15 – 15:45     Maria Kortekaas (VSOP, Utrecht, the Netherlands)

                           Title tbd

 

15:45 – 16:00     Award Ceremony and closing remarks

 

16:00 – 17:00     Networking and drinks​​​​​

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Poster list:

 

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Poster #1

Aaron Dangel (University of Tübingen, Germany)

Targeting splice loss mutations by patient-customized ASOs: development and validation of an ASO targeting strategy by example of Ataxia telangiectasia

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Poster #2

Aidan Oubrie (Radboudumc, the Netherlands)

Antisense oligonucleotide detection in placental models to evaluate maternal-fetal exposure to Inotersen   

 

Poster #3

Lianne Beuk (Erasmus MC, the Netherlands)

A Preclinical Exploration of the Potential of Antisense Oligonucleotide Therapy in mTORopathies

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Poster #4

Emilio Harris-Mostert (Erasmus MC, the Netherlands)

Consensus framework to assess eligibility of genes and hereditary diseases for genetic therapy development 

 

Poster #5

Esmee Moen (CBG-MEB, the Netherlands)

Assessing the dose selection of oligonucleotides

 

Poster #6

Jessie Hendricks (Radboudumc, the Netherlands)

Antisense oligonucleotide-mediated splice correction for an ultrarare synonymous variant in CHM that causes the retinal disease choroideremia 

 

Poster #7

David Schiffelers (Radboudumc, the Netherlands)

Combining iPSC-derived Retinal Models and Untargeted Lipidomics to Identify Targets for Pathway Modulation in Sjögren-Larsson Syndrome  

 

Poster #8

Jip van den Hooff (Erasmus MC, the Netherlands)

Designing a broadly applicable ASO therapy for SYNGAP1 disorders 

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Poster #9

Myrthe van Tol (LUMC, the Netherlands)

SNP-based Antisense Oligonucleotides for Allele-specific silencing of ATXN1 in Spinocerebellar Ataxia type 1  

 

Poster #10

Seyedarash Ayatollahi (BTU Cottbus-Senftenberg, Germany)

ASOkai: A Modular Open-Source Framework for Systematic ASO Design and Evaluation 

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Poster #11

Rindert Venema (UMCG, the Netherlands)

Antibody-drug conjugation strategy for targeted systemic delivery of oligonucleotides to the skin

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Poster #12

Qianjun Ma (Erasmus MC, the Netherlands)

Mechanistic Insights into RNase H1 Cleavage to Enable Rational Allele-Selective Antisense Oligonucleotide Designs 

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Poster #13

Baiju Parikh (Oligo Factory, the Netherlands)

The N=1 Collaborative ASO Control Panel for Toxicity Studies: A Community-Distributed Reference Set 

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Poster #14

Clara Stock (CBG-MEB, the Netherlands)

The importance of dosing and exposure considerations for developmental and reproductive toxicity testing of oligonucleotide-based therapeutics - a regulatory perspective 

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Poster #15

Daria Zhuravleva (Uppsala University, Sweden)

Application of continuous-categorical IRT modelling to enhance statistical power in N-of-1 trials: simulation-based evaluation 

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Poster #16

Filippo Furlan (Radboudumc, the Netherlands)

Joining EFFecT Doctoral Network to investigate therapies and novel possible disease mechanisms in PMM2-CDG

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Poster #17

Kim van der Gouw (UMCG, the Netherlands)

Oligo-walk to identify potent antisense oligonucleotides for exon skipping of COL7A1 

 

Poster #18

Nadine Maas (Erasmus MC, the Netherlands)

Development of an N-of-1 therapeutic antisense oligonucleotide for a unique UNC13A variant causing a neurodevelopmental disorder 

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Poster #19

Rachna Vikram (LUMC, the Netherlands)

Spliceosome‐mediated RNA trans-splicing to correct a novel pathogenic mutation in the APP intracellular domain implicated in ischemic stroke

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Poster #20

Roan Hofstede (Leiden University of Applied Sciences, the Netherlands)

Development of a safety pipeline for individualised ASOs in rats

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Poster #21

Silvana Jirka (LUMC, the Netherlands)

A 3D-skeletal Muscle Model for Evaluating Antisense Therapeutics in FSHD

 

Poster #22

Tania Martianez Canales (Charles River Laboratories, the Netherlands)

HSPB8 targeting and modulation – a multimodality approach 

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Poster #23

Tirsa van Westering (Charles River Laboratories, the Netherlands)

Therapeutics development for a patient with a KCNQ3/ Kv7.3 disorder using an iPSC-based platform measuring neural network activity  

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