1st Dutch Antisense Therapeutics Symposium
June 3rd 2022, Leiden
9:00 - Arrival, Registration and Coffee
9:55 - Welcome and opening of the symposium
10:00 National keynote lecture
Annemieke Aartsma-Rus (LUMC): A brief history of ASO therapy development: Developments in the current millennium.
SESSION 1
10:45 Roland Brock (Radboudumc) - Peptide-based delivery strategies for antisense oligonucleotides
11:15 Peter van den Akker (UMCG) - Exon-skipping therapy for dystrophic epidermolysis bullosa
11:45 Julie Rutten (LUMC) - Skipping NOTCH3 exons to prevent protein aggregation in CADASIL patient arteries
12:15 Marlen Lauffer (LUMC) - Dutch Center for RNA Therapeutics
12:30 Lunch
SESSION 2
13:15 Early Career Investigator Presentations
13:15 Yvonne K. Jongejan (LUMC) - Small interfering RNAs for allele-selective silencing of murine von Willebrand factor
13:30 Jurriën Prins (LUMC) - A novel antisense strategy to inhibit BK polyomavirus replication
13:45 Irene Vázquez-Domínguez (Radboudumc) - Deciphering the efficacy and safety of different antisense oligonucleotide chemical modifications
in a retinal context
14:00 International Keynote Lecture:
Bruno Godinho (Atalanta Therapeutics) - Delivery of stabilized siRNA scaffolds to the central nervous system
14:45 Marjon Pasmooij (Medicines Evaluation Board) - Antisense to medicine: a regulator’s perspective
15:15 Poster Pitches
Edward Geeurickx (Biogazelle) - Identification of potent and specific antisense oligonucleotides for target gene knockdown or splice modulation
using a high-throughput RT-qPCR based cellular screening platform
Dyah Karjosukarso (Radboudumc) - Towards clinical application of antisense oligonucleotides for the treatment of Stargardt disease
Elsa Kuijper (LUMC) - Therapeutic effect of antisense oligonucleotide treatment in YAC128 Huntington mice
Hilde Smeenk (ErasmusMC) - Using antisense oligonucleotides to reactivate paternal UBE3A in Angelman syndrome patient-derived neurons
Nuria Suárez Herrera (Radboudumc) - Combined AON-U7snRNA as splicing modulation therapy to correct multiple deep-intronic ABCA4 variants
Alper Yavas (LUMC) - Modulation of IGF1 splicing towards mechano growth factor isoform by antisense oligonucleotide-mediated exon inclusion
15:30 POSTER SESSION with Coffee and Cookies
SESSION 3
16:45 Early Career Investigator Presentations
16:45 Claudia Milazzo (Erasmus MC) - ASO treatment rescues UBE3A expression and multiple phenotypes of an Angelman Syndrome mouse model
17:00 Tom Metz (LUMC) - Biodistribution of radioactively labelled AONs after ICV and IT injection in mice
17:15 Janine Reurink (Radboudumc) - WGS identifies deep-intronic variants in USH2A that are amenable for AON-based splice correction therapy
17:30 Elizabeth Vroom (Duchenne Parent Project Netherlands) - The role of patient organizations in antisense oligonucleotide drug development
18:00 Awards, closing remarks
18:10 Drinks and Networking
19:30 Dinner (not included, pre-registration required)
EVENT SPONSORED BY:
