3rd Dutch Antisense Therapeutics Symposium
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 MC, 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), Tiberiu Stan (Poster #9), Janina Lösch (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 underlying early-onset visual impairment
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: Tiberiu Loredan Stan (LUMC, the Netherlands)
Validation lab: allowing standardized in vitro and in vivo experiments for candidate treatments for Duchenne muscular dystrophy
Poster #10: Redmar R van den Berg (LUMC, the Netherlands)
ExonViz – An application to visualize transcripts and variants
Poster #11: Janina Lösch (LUMC, the Netherlands)
Evaluation of gapmer ASO candidates targeting a heterozygous ACTL6B variant in neurons transdifferentiated from patient fibroblasts
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: 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
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: Daniëlle Swinkels (Radboudumc, the Netherlands)
Exploring the Role of Lipids in Retinal Degeneration: Insights & Therapeutic Prospects
Poster #26: Sebastian Sjöström (ETH Zürich, Switzerland)
Allele-selective ASO Reduces Aggregation in Kidney Renal Tubules in Mice with Severe ADTKD
Poster #27: Rossella Avagliano Trezza (Maastricht University, the Netherlands)
Antisense Oligonucleotide-Mediated Exon Skipping as Therapeutic Approach for a common LAMA2-MD mutation
Poster #28: 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
Poster #29: Olivia Lewis (UU, the Netherlands)
RNA Therapies Under a Regulatory Lens: Unravelling Classification Complexities in the EU
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