DAY 1 (MAY 23, 2024)

 

09:30 – 10:15    Registration open

 

10:15 – 10:30     Welcome and official opening  by Dr. Atze Bergsma (Erasmus MC, The Netherlands)

 

10:30 – 11:15     International Keynote Lecture: Dr. Shalini Andersson, AstraZeneca, Sweden

                           Developing Next Generation Oligonucleotide Therapeutics

                           Chair: Atze Bergsma

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11:15 – 12:15     Session 1 (chair: Najoua El Boujnouni)

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11:15 – 11:30     Emilio D.R.O. Harris-Mostert (Erasmus The Netherlands)    

                           In silico guided target sequence selection for RNase H1-dependent antisense oligonucleotides 

11:30 – 11:45     Laurie M.C. Kerkhof (LUMC, the Netherlands)

                           Exploring the role of 5’ UTR-mediated regulation of ataxin-1 protein expression using CRISPR                                 and splice-modulating antisense oligonucleotides 

11:45 – 12:00     Manon Bouckaert (Ghent University, Belgium)

                           Evaluation of a novel antisense oligonucleotide therapy targeting a 5’UTR mutation in the                                       RDH12 gene 

12:00 – 12:15     Sponsor pitches

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12:15 – 13:30     Lunch

 

13:30 – 15:00     Session 2 (chair: Ronald Buijsen)

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13:30 – 14:00     Prof. Inge Zuhorn (UMCG, the Netherlands)

                           Mechanisms of action in non-viral gene delivery

14:00 – 14:15     Bianca Zardetto (LUMC, the Netherlands)

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

14:15 – 14:30     Edwin van Oosten (Radboudumc, the Netherlands)

                           Personalized antisense oligonucleotide screening for a Stargardt disease patient carrying the                                 deep-intronic c.859-506G>C ABCA4 variant using human in vitro models

14:30 – 14:45     Lotte Schoen (LUMC, the Netherlands)

                           Evaluation of RNA trans-splicing (SMaRT) as therapeutic strategy for Spinocerebellar ataxia                                     type 1 (SCA1) 

14:45 – 15:00     Rosan Lechner (Erasmus, the Netherlands)    

                           Responsibly accelerating pre-clinical development of antisense oligonucleotides intended for                                personalized use in rare diseases

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15:00 – 16:30     Poster Session 1 (odd):

15:00 – 15:15     Selected Poster Pitches (odd), chair: Alex Garanto

                           Pitches by: 

                           Michael Kurczy (Poster #1), Kyra Swildens (Poster #3), Mink Neeleman (Poster #5),

                           Aline Mack (Poster #7), Janina Lösch (Poster #9), Tiberiu Stan (Poster #11)

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

 

16:30 – 18:00     Session 3 (chair: Jeroen Bremer)

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16:30 – 17:00     Prof. Raymond Schiffelers (UMCU, the Netherlands)

                           Nanospresso-NL: local manufacture of nucleic acid medicines

17:00 – 17:15     Joyce M. Hoek (UU, the Netherlands)

                           The feasibility and desirability of compounding as an alternative medicine-to-patient pathway                                 for personalized medicines in the Netherlands

17:15 – 17:30     Clemens Lochmann (University of Tübingen, Germany)

                           Comprehensive, scalable development of patient-customized splice modulation ASOs for                                       ataxia telangiectasia 

17:30 – 17:45     Kim Rodenburg (Radboudumc, the Netherlands)

                           Whole genome sequencing reveals causative deep-intronic PCDH15 and de novo USH2A                                       variants in patients with Usher syndrome, amenable to AON-mediated rescue  

17:45 – 18:00     Franciscus C. Vermeer (UMCG, the Netherlands)

                           ASO-mediated Exon skipping in intact human skin pointing towards a treatment for RDEB 

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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, pubquiz and drinks

 

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DAY 2 (MAY 24, 2024)

 

09:30 – 10:15     National Keynote Lecture: Prof. Rob Colin, Radboudumc, the Netherlands

                           Splicing modulation therapy for inherited retinal diseases

                           Chair: Alex Garanto

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10:15 – 11:45     Poster Session 2 (even):

10:15 – 10:30     Selected Poster Pitches (even), chair: Alex Garanto

                           Pitches by:

                           Kim Wijnant (Poster #2), Bas J.B. Voesenek (Poster #4), Amber Paijmans (Poster #6), 

                           Myron Tuasela (Poster #8), Redmar R van den Berg (Poster #10), Cristina Gontan (Poster #12)

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

 

11:45 – 13:15     Session 4: Dutch Center for RNA Therapeutics (DCRT) (chair: Prof. Ype Elgersma)

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11:45 – 12:00     DCRT update by Prof. Annemieke Aartsma-Rus (LUMC, the Netherlands)

12:00 – 12:15      Floor Nieuwenhuijse (Radboudumc, the Netherlands)

                           Exploring exon skipping therapy for an ultrarare CEP290 variant in a unique LCA patient

12:15 – 12:45     Prof. Matthis Synofzik (University of Tübingen, Germany)

                           A scalable platform approach for preclinical and clinical development of patient-customized                                   ASOs for brain diseases: the example of Ataxia Telangiectasia

12:45 – 13:15     Prof. Bart van de Warrenburg (Radboudumc, the Netherlands)

                           Trial-readiness in spinocerebellar ataxias: on the tortoise and the hare

 

13:15 – 14:15     Lunch

 

14:00 – 15:30     Session 5 (Chair: Anne-Fleur Schneider)

14:00 – 14:15     Linde Bouwman (LUMC, the Netherlands)

                           Allele-specific silencing of ATXN7 to treat Spinocerebellar Ataxia Type 7

14:15 – 14:30     Inês Fial (MRC Nucleic Acid Therapy Accelerator (NATA), United Kingdom)

                           Characterising antibodies targeting antisense oligonucleotide (ASO) modifications for                                             quantification of intracellular trafficking and in vivo biodistribution 

14:30 – 14:45     Remko Goossens (LUMC, the Netherlands)

                           Functional in vitro modelling of Duchenne antisense oligonucleotide therapies using 3D muscle                             bundles 

14:45 – 15:00     Juliette Kamp (Erasmus MC, the Netherlands)

                           Investigating the therapeutic potential of SYNGAP1 splice-switching oligonucleotides 

15:00 – 15:30     Niels van Hoorn (SMA patient organization, the Netherlands)

                           Experiencing the approval process of Spinraza: a patient perspective

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15:30 – 16:00     Award Ceremony and closing remarks

 

16:00 – 17:00     Networking borrel

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POSTER LIST:

Poster #1: Michael Kurczy  (AstraZeneca, Sweden)

                 Reveling the intracellular concentration of nucleic-acid based therapeutics using NanoSIMS  

Poster #2: Kim Wijnant (Radboudumc, the Netherlands)

                  Identification of targetable neurodevelopmental disorders for antisense oligonucleotide therapy

Poster #3: Kyra Swildens (Erasmus MC, the Netherlands)

                  The role of RNF12 in X-linked intellectual disability syndrome TOKAS 

Poster #4: Bas J.B. Voesenek (LUMC, the Netherlands)

                  Unravelling disease pathology for a novel app variant in an hiPSC-derived model using a multiscale                      pipeline for 3D image analysis 

Poster #5: Mink Neeleman (Radboudumc, the Netherlands)

                  Identification of new therapeutic targets for congenital disorders of glycosylation

Poster #6: Amber Paijmans (LUMC, the Netherlands)

                  A gapmer screen in SCA1 patient-derived cells

Poster #7: Aline Mack (University of Tübingen, Germany)

                  SKIP-AT: Towards a comprehensive, systematic platform approach for targeted exon skipping of ATM

Poster #8: Myron Tuasela (Erasmus MC, the Netherlands)

                  Current practices in the preclinical development of N-of-1 and EMA approved antisense                                        oligonucleotides

Poster #9: Janina Lösch (LUMC, the Netherlands)

                  Evaluation of gapmer ASO candidates targeting a heterozygous ACTL6B variant in neurons                                    transdifferentiated from patient fibroblasts 

Poster #10: Redmar R van den Berg (LUMC, the Netherlands)

                    ExonViz – An application to visualize transcripts and variants

Poster #11: Tiberiu Loredan Stan (LUMC, the Netherlands)

                    Validation lab: allowing standardized in vitro and in vivo experiments for candidate treatments for                          Duchenne muscular dystrophy

Poster #12: Cristina Gontan (Erasmus MC, the Netherlands)

                    Generation of a KANSL1 Dual Reporter Human iPSC Line for ASO Screening in Koolen-de Vries                            Syndrome Therapy 

Poster #13: Daniëlle Swinkels (Radboudumc, the Netherlands)

                    Exploring the Role of Lipids in Retinal Degeneration: Insights & Therapeutic Prospects  

Poster #14: Ilse Bijl (LUMC, the Netherlands)

                    Improving AON mediated exon skipping efficacy with antioxidant NAC treatment in mdx mice  

Poster #15: Iris W.M. Ensink (LUMC, the Netherlands)

                    Development of an individualized splice-modulating antisense oligonucleotide therapy for patients                      suffering from NDUFAF6-associated Leigh Syndrome 

Poster #16: Irene Flavia Ferro (Klinikum rechts der Isar - Helmholtz Munich, Germany)

                    De novo variation in the α-subunit of the voltage-gated sodium channel Nav1.2

Poster #17: Mariana Guimarães Ramos (LUMC, the Netherlands)

                    Selection of a lead antisense oligonucleotide in hiPSC cell lines for Spinocerebellar Ataxia 3 

Poster #18: Marlen C. Lauffer (LUMC, the Netherlands)

                    Recommendations for the identification of genetic variants eligible for individualized antisense                              oligonucleotide treatments 

Poster #19: Jody van Offenbeek (Charles River Laboratories, the Netherlands)

                    Development of an in vitro panel for neurotoxicity assessment of drug discovery candidates 

Poster #20: Marleen J. Luimes (LUMC, the Netherlands)

                    Evaluating muscle homing peptides for improved uptake of antisense oligonucleotide therapy for                        Duchenne muscular dystrophy 

Poster #21: Jack Morgan (LUMC, the Netherlands)

                    Antisense oligonucleotide-mediated exon skipping treatments for neurodevelopmental disorders                        associated with tandem-repeat domain missense variants in ANKRD17 

Poster #22: Tirsa van Westering (Charles River Laboratories, the Netherlands)

                    The drug discovery journey for a patient with Dup15q Syndrome 

Poster #23: Bas Röttgering (LUMC, the Netherlands)

                    ASO-induced exon 10 skipping of ATXN3: assessing the functionality of the truncated ΔpolyQ                                Ataxin-3 protein isoform 

Poster #24: Anouk Spruit (LUMC, the Netherlands)

                    Platform to screen for ASO-mediated neurotoxicity in human control-derived iPSC neuronal                                    cultures 

Poster #25: Nick van Dullemen (LUMC, the Netherlands)

                    Using a midi-gene assay for the development of an allele-specific gapmer antisense

                    oligonucleotide targeting a heterozygous ACTL6B variant 

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