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: