
2nd Dutch Antisense Therapeutics Symposium
Provisional program
DAY 1 (THURSDAY June 1st)
9:45 – 10:15 Registration Open
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10:15 – 10:30 Welcome and official opening
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10:30 – 11:15 International Keynote Lecture 1: Aurélie Goyenvalle, Univ. of Versailles, France
Splice switching approaches for the treatment of neuromuscular diseases
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11:15 – 12:30 Session 1:
11:15 – 11:45 Enrico Mastrobattista (Utrecht University)
Synthetic nanocarriers for local and systemic delivery of nucleic acid therapeutics
11:45 – 12:00 Selected Abstract 1
12:00 – 12: 15 Selected Abstract 2
12:15 – 12:30 Sponsor pitches
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12:30 – 13:30 Lunch
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13:30 – 15:00 Session 2:
13:30 – 14:00 Monica Aguila (ProQR Therapeutics)
Exploring the therapeutic potential of new RNA editing technologies leveraging ADAR endogenous machinery
14:00 – 14:15 Selected Abstract 3
14:15 – 14:30 Selected Abstract 4
14:30 – 14:45 Selected Abstract 5
14:45 – 15:00 Selected Abstract 6
15:00 – 15:15 Selected Poster Pitches Session 1
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15:15 – 16:30 Poster Session 1 with coffee and tea
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16:30 – 18:00 Session 3:
16:30 – 17:00 Erik de Vrieze (Radboudumc)
Preclinical development of allele-specific antisense therapies for dominantly-inherited hearing loss disorders
17:00 – 17:15 Selected Abstract 7
17:15 – 17:30 Selected Abstract 8
17:30 – 17:45 Selected Abstract 9
17:45 – 18:10 Virginia Arechavala-Gomeza (Biocruces Bizkaia Health Research Institute, Spain)
Delivery Networking is key in the development of nucleic acid therapeutics
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18:10 Closing of day 1
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19:00 Networking drinks, dinner and pubquiz (not incl. in the registration costs)
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DAY 2 (FRIday June 2nd)
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9:00 – 9:45 International Keynote Lecture 2: Timothy Yu, Boston Children’s Hospital, USA
Pathways for patient-centered interventional genomic medicine
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9:45 – 11:15 Session 4: Dutch Center for RNA Therapeutics (DCRT)
Chair: Willeke van Roon-Mom (LUMC)
9:45 – 10:00 Marlen Lauffer (LUMC)
Building a framework for the identification and selection of cases for personalized ASO treatments
10:00 – 10:15 Ype Elgersma (ErasmusMC)
ASO treatment for Angelman Syndrome: Promises and threats
10:15 – 10:30 Rob Collin (Radboudumc)
Splicing modulation therapy for inherited retinal diseases: the example of Stargardt disease
10:30 – 11:00 ASO development and Regulatory Aspects - Ask the experts! (Q&A)
Valentina Cordo (European Medicines Agency), Marlen Lauffer (LUMC), Ype Elgersma (ErasmusMC), Rob Collin (Radboudumc).
Panel moderated by Annemieke Aartsma-Rus (LUMC)
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11:00 – 11:15 Selected Poster Pitches Session 2
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11:15 – 12:45 Poster Session 2 with coffee and tea
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12:45 – 13:45 Lunch
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13:45 – 15:30 Session 5:
13:45– 14:15 Nicole Datson (VICO Therapeutics)
VO659, an allele-preferential CAG repeat-targeting ASO with therapeutic potential for
patients with Huntington’s disease and spinocerebellar ataxia types 1 and 3
14:15 – 14:30 Selected Abstract 10
14:30 – 14:45 Selected Abstract 11
14:45 – 15:00 Selected Abstract 12
15:00 – 15:30 Ronald Jansen (BeatBatten!)
Batten disease: a patient perspective
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15:30 – 15:45 Award ceremony
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15:45 – 16:00 Closing remarks
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16:00 – 17:30 Networking drinks
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EVENT SPONSORED BY:
