DAY 1 (FRIDAY June 3rd)

 

9:30 Arrival, Registration and Coffee

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9:55 Welcome

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10:00 National keynote lecture:

Annemieke Aartsma-Rus (LUMC) 

A brief history of ASO therapy development: Developments in the current millennium

 

SESSION 1

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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 - 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

 

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